Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

• Sponsor: PTC Therapeutics (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT01009294
• Collaborator: Genzyme, a Sanofi Company (Industry)
• Enrollment: 6
• Locations: 6
• Arm: 1
• Actual Duration (Months): 2.3
• Patient Per Site: 1
• Patients Per Site Per Month: 0.4

Study Details

Study Description

Brief Summary

Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a trial that enrolled boys with nonsense mutation DMD/BMD who have lost independent mobility due to the disease. This study evaluated the safety and tolerability of ataluren (PTC124) and also evaluated efficacy outcomes in this participant population.

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy; Becker Muscular Dystrophy	Drug: Ataluren; Drug: Chronic Corticosteroid Therapy	Phase 2

Detailed Description

It was planned that this Phase 2a, open-label, safety and efficacy study to be performed at 5 sites in the US and 1 site in the United Kingdom.

The study was to enroll ~30 boys with nonsense mutation DMD/BMD (nmDBMD) who have been nonambulatory for at least 1 year. Enrollment was to be stratified to ensure evaluation of ~15 participants who were receiving chronic corticosteroid therapy and of ~15 participants who were not receiving chronic corticosteroid therapy. Participants were to take ataluren 3 times per day (at breakfast, lunch, and dinner) for 48 weeks (~1 year). Study assessments were to be performed at clinic visits during screening, every 6 weeks for 2 visits and then every 12 weeks until the end of the study. Additional safety laboratory testing was to be required 4 times during the course of the study; this could have been performed at the investigational site, at an accredited local laboratory or clinic, or in the participant's home using a nursing service. When the blind for a similar study (PTC124-GD-007-DMD; NCT00592553) was revealed, the results indicated lack of efficacy for the high dose. Therefore, even though an independent data monitoring committee (DMC) agreed that both ataluren dose levels were well tolerated by the participants, the DMC recommended discontinuing ongoing studies with participants with nmDBMD receiving high-dose ataluren.

Study Design

• Study Type: Interventional
• Actual Enrollment: 6 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy
• Actual Study Start Date: Jan 13, 2010
• Actual Primary Completion Date: Mar 23, 2010
• Actual Study Completion Date: Mar 23, 2010

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Ataluren Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 milligrams/kilograms (mg/kg) in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.

Drug: Ataluren; Oral powder; Other Names: PTC124 ; Drug: Chronic Corticosteroid Therapy; Enrollment was stratified to ensure evaluation of approximately half of the participants were receiving chronic corticosteroid therapy and approximately half of participants were not receiving chronic corticosteroid therapy. Therefore, 3 out of 6 participants were receiving chronic corticosteriod therapy. For the participants receiving chronic corticosteriod therapy, a stable corticosteriod regimen was to be maintained during the study.

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Treatment Emergent Adverse Events (TEAEs) [Baseline up to Day 50]

   A TEAE is any untoward medical occurrence or undesirable event(s) experienced in a participant that begins or worsens following administration of the study drug or study treatment, whether or not considered related to the treatment by the Investigator. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), or persistent or significant disability/incapacity not related to nmDBMD. AEs included both SAEs and non-serious AEs. AEs were classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 (CTCAE) and coded using the Medical Dictionary for Regulatory Activities (MedDRA). A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.

Secondary Outcome Measures

1. Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test [Baseline and Week 6]

   Arm and hand function were assessed using the Jebsen test, a standardized clinical evaluation of tasks important to daily living. The test comprises of unilateral subtests performed with each hand (the dominant [DOM] hand and the non-DOM hand): moving and stacking light (250 grams) and heavy (500 grams) objects; picking up small, commonly encountered objects; stacking checkers; simulated feeding; simulated page turning; and writing. Participant performance of each task was timed. Longer time to complete the test indicates worse hand function.

2. Upper Limb Function as Measured by the Brooke Upper Extremity Functional Rating Scale [Baseline and Week 6]

   Upper extremity function was assessed using the Brooke Upper Extremity Functional Rating Scale, following standardized procedures. The Brooke Upper Extremity Functional Rating Scale graded arm and shoulder function from 1 to 6, with higher values indicating less function. A rating of "1" was used when the participant was able to abduct his arms in a full circle until they touch above his head, whereas a rating of "6" was used when the participant was unable to raise his hands to his mouth and had no useful function of hands.

3. Participant Activities of Daily Living as Assessed Using the Egen Klassifikation (EK) Scale [Baseline and Week 6]

   Activities of daily living after loss of ambulation were measured using the EK scale. The EK scale is an ordinal scale ranging from 0 to 30 points where 0 represents the highest level of independent function and 30 the lowest. The scale consists of 10 categories (each scored 0 to 3), involving different functional domains including 1) ability to use wheelchair, 2) ability to transfer from wheelchair, 3) ability to stand, 4) ability to balance in the wheelchair, 5) ability to move arms, 6) ability to use hands and arms when eating, 7) ability to turn in bed, 8) ability to cough, 9) ability to speak, and 10) physical well-being. The administration of the EK scale consisted of an interview of the participant to capture how he performs the tasks of daily life (as described by Categories 1 to 9) and how he perceives his wellbeing (as described by Category 10). The interviewer observed the participant and assigned the final score for the tasks that could be observed in the clinic.

4. Shoulder, Elbow, and Wrist Passive and Active Range of Motion as Measured by Goniometry [Baseline and Week 6]

   Goniometry was performed to test active and passive range-of motion (RoM) of the left (L) and right (R) shoulder, elbow, and wrist following standardized procedures. The observed angle for passive and active motion for each joint was measured in degrees. Greater degree of motion indicates better response.

5. Force Exerted During Elbow Flexion and Extension, Shoulder Abduction, Hand Grip, Key Grip, and Finger Pinch as Assessed by Upper Extremity Myometry [Baseline and Week 6]

   Upper extremity myometry was performed using a hand-held dynamometer following standardized procedures. The measured strength (peak force) was reported in Newtons. There are 0.22 pounds (lbs) in 1 Newton and approximately 10 Newton (9.80665 Newton) in 1 kilogram (kg). The threshold/range of the hand-held dynamometer is 0 to 500 Newtons. Bilateral assessments were done, and 3 measurements were recorded from each muscle group on each side, when possible. When the measurements were done in duplicate or triplicate, the best value was used. Greater value indicates better measurement.

6. Time to Complete Hand Fine Motor Coordination and Dexterity Tasks as Measured by 9-Hole Peg Test (9HPT) [Baseline and Week 6]

   Hand fine motor coordination and dexterity were assessed using the 9HPT using standardized procedures. The 9HPT is a unilateral test in which 9 pegs were placed in a board and then removed with the dominate and non-dominate hand within a 5-minute time limit. The amount of time required to put the pegs in the holes and remove them again with each hand was recorded. Each test was conducted twice per hand. Longer time to complete the test indicates worse hand fine motor coordination and dexterity.

7. Forced Vital Capacity (FVC) as Measured by Spirometry [Baseline and Week 6]

   Pulmonary function was assessed as FVC in participants by spirometry using a study-specific spirometer. Multiple tests were conducted, if needed.

8. Systolic and Diastolic Function as Measured by Echocardiography With Tissue Doppler [Week 24 and Week 48]

   Cardiac function was assessed by echocardiography, which included standard parameters (for example, ejection fraction, left ventricle diastolic and systolic dimensions), as well as parameters integrating Doppler flow analysis with imaging to evaluate perturbations in wall motion. A standardized data collection process harmonized data from all participating institutions and allowed for centralized review.

9. Heart Rate as Assessed by Radial Pulse [Baseline and Week 6]

   Heart rate was measured with the radial pulse. Following the Jebsen test, the participant rested for 5 minutes in a sitting position, and the heart rate for the last minute of this rest period was collected as the resting heart rate.

10. Verbal Memory and Attention as Assessed by the Digit Span Task [Baseline and Week 6]

    A series of digits (0-9) were presented to the participant in an auditory format only. The task had 2 parts: in the Forward Condition, the participant was requested to repeat back the digits in the order they were presented, and in the Backward Condition, he was requested to reverse the order of presentation.

11. HRQL as Measured by the PedsQL Inventory Generic Core Scale [Week 6]

    Health-related quality of life (HRQL) was measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Generic Core Scale. The generic core module comprised of 23 questions evaluating physical, emotional, social, and school functioning. Examples of items in each of the generic core module scales included: "It is hard for me to run"; "I feel sad or blue"; "I cannot do things that other kids my age can do;" and "It is hard to pay attention in class." Each of the generic core module items was scored on a 5-point response scale from 0 (never a problem) to 4 (almost always a problem). The appropriate age-specific version was completed. PedsQL Inventory Generic Core Scale data at Week 6 is presented.

12. HRQL as Measured by the PedsQL Multidimensional Fatigue Scale [Week 6]

    HRQL was measured by the PedsQL Multidimensional Fatigue Scale. The fatigue-specific module comprised of 18 questions evaluating general fatigue, sleep/rest fatigue, and cognitive fatigue. Fatigue-specific module obtains information relating to items such as: "I feel too tired to do things that I like to do"; "I spend a lot of time in bed"; and "I have trouble remembering more than one thing at a time." Each of the fatigue-specific module items was scored on a 5-point response scale from 0 (never a problem) to 4 (almost always a problem). The appropriate age-specific version was completed. PedsQL Multidimensional Fatigue Scale data at Week 6 is presented.

13. HRQL as Measured by the INQoL [Week 24 and Week 48]

    HRQL was measured by the Individualized Neuromuscular Quality of Life Questionnaire (INQoL). The INQoL consisted of 45 questions within 10 sections. Four of the sections evaluate key muscle disease symptoms (that is, weakness, locking [myotonia], pain, and fatigue), 5 sections evaluate the degree and importance of the impact of muscle disease on particular areas of life, and 1 section asks about the positive and negative effects of treatment. A higher score indicates greater symptom impact or worse HRQL, with a range of 0-7.

14. Muscle Fragility as Determined by Serum Creatine Kinase (CK) Levels [Baseline and Week 6]

    Blood samples collected for chemistry assays were used to quantify serum CK concentrations. Serum CK was assessed as a potential biomarker for muscle fragility, with a reduction in serum CK considered to be a positive outcome. The reference range was based on the age of the participant.

15. Gastrocnemius Muscle Dystrophin Expression as Determined by Immunofluoresence or by Western Blotting Techniques [Week 36]

    The gastrocnemius muscle was to be biopsied from 1 leg to assess for the production of dystrophin at Week 36. The production of dystrophin was to be measured by immunofluorescene staining of the sarcolemmal membrane or by Western blotting techniques with an antibody to the C-terminal portion of the dystrophin protein (excluding revertant fibers).

16. Study Drug Compliance [Baseline to Day 50]

    Study drug compliance was assessed by the participant daily diary and quantification of used and unused study drug. Compliance was assessed in terms of the amount of drug actually taken relative to the amount that should have been taken during the study.

17. Pharmacokinetics: Ataluren Plasma Exposure [0, 2, 3, 6, 8, 9, 12, 14, 15, and 24 hours after the morning dose]

    Blood for ataluren concentrations over a 24-hour period was to be collected on Days 2 and 3 of Week 6. Analysis of the blood samples was to be conducted using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC-MS/MS) method.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 7 Years and Older
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Diagnosis of DMD or BMD

• Presence of a nonsense mutation in the dystrophin gene

• Unable to ambulate independently for ≥1 year due to DMD/BMD

• Presence of sufficient shoulder and elbow function to perform study-related functional procedures (for example, 9-hole peg test)

• Adequate hepatic, renal, and adrenal function

• Ability to provide evaluable pretreatment echocardiogram and lung function assessments

• Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions

• Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if <18 years of age)

Exclusion Criteria:

• Initiation of systemic corticosteroid therapy within 6 months prior to start of study treatment or use of systemic aminoglycoside antibiotic within 3 months prior to start of study treatment

• Use of any intermittent systemic corticosteroid therapy regimen (for example, 10 days on followed by 10 days off, weekend dosing, every-other-day dosing); note that participants must have either been receiving a daily dosing regimen of prednisone, prednisolone, or deflazacort at the time of enrollment into the study or must have not been receiving any systemic corticosteroids

• Any change in treatment for congestive heart failure within 3 months prior to start of study treatment

• Ongoing warfarin or phenytoin therapy

• Prior therapy with ataluren

• Known hypersensitivity to any of the ingredients or excipients of ataluren (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127 [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab O Sil® M5P [colloidal silica], magnesium stearate).

• Exposure to another investigational drug within 2 months prior to start of study treatment

• History of major surgical procedure within 1 month prior to start of study treatment or expectation of major surgical procedure (for example, scoliosis surgery) during the 48-week treatment period of the study

• Ongoing immunosuppressive therapy (other than corticosteroids)

• Ongoing participation in any other clinical trial

• Requirement for daytime ventilator assistance

• Uncontrolled clinical symptoms and signs of congestive heart failure

• Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow up would be completed, or could impair the assessment of study results

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California-Davis	Davis	California	United States	95616
2	Children's Hospital of Boston	Boston	Massachusetts	United States	02115
3	University of Minnesota	Minneapolis	Minnesota	United States	55455
4	Washington University Medical School	Saint Louis	Missouri	United States	63110
5	Nationwide Children's Hospital	Columbus	Ohio	United States	43205
6	University of Newcastle	Newcastle upon Tyne		United Kingdom	NE1 3BZ

Sponsors and Collaborators

• PTC Therapeutics
• Genzyme, a Sanofi Company

Investigators

• Study Director: Leone Atkinson, MD, PhD, PTC Therapeutics

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Related Info

Publications

None provided.

• Responsible Party: PTC Therapeutics
• ClinicalTrials.gov Identifier: NCT01009294
• Other Study ID Numbers: PTC124-GD-008-DMD; 2009-013169-24
• First Posted: Nov 6, 2009
• Last Update Posted: Jul 29, 2020
• Last Verified: Jul 1, 2020

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by PTC Therapeutics: Duchenne muscular dystrophy; Becker muscular dystrophy; Nonsense mutation; Premature stop codon; DMD; BMD; Ataluren; PTC124
• Additional relevant MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne

Study Results

Participant Flow

Recruitment Details	A total of 11 participants with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) and were nonambulatory signed the informed consent form and were screened for eligibility. Six of these participants were enrolled at 2 sites. Three of the participants were receiving chronic corticosteroid therapy.
Pre-assignment Detail	When the Sponsor terminated the study, the participants were told to discontinue ataluren treatment, and to return all unused ataluren to the site for return to the Sponsor. Because of difficulty of traveling to the clinic for these nonambulatory participants, the planned final visits were not performed.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 milligrams/kilograms (mg/kg) in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Period Title: Overall Study
STARTED	6
Received at Least 1 Dose of Study Drug	6
COMPLETED	0
NOT COMPLETED	6

Baseline Characteristics

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Overall Participants	6
Age, Customized (Count of Participants)
12 to 17 years	3 50%
18 to 20 years	3 50%
Sex: Female, Male (Count of Participants)
Female	0 0%
Male	6 100%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Description	A TEAE is any untoward medical occurrence or undesirable event(s) experienced in a participant that begins or worsens following administration of the study drug or study treatment, whether or not considered related to the treatment by the Investigator. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), or persistent or significant disability/incapacity not related to nmDBMD. AEs included both SAEs and non-serious AEs. AEs were classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 (CTCAE) and coded using the Medical Dictionary for Regulatory Activities (MedDRA). A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Time Frame	Baseline up to Day 50

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
TEAEs	1 16.7%
Treatment Emergent SAEs	0 0%
AEs Related to Study Treatment	0 0%

2. Secondary Outcome

Title	Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test
Description	Arm and hand function were assessed using the Jebsen test, a standardized clinical evaluation of tasks important to daily living. The test comprises of unilateral subtests performed with each hand (the dominant [DOM] hand and the non-DOM hand): moving and stacking light (250 grams) and heavy (500 grams) objects; picking up small, commonly encountered objects; stacking checkers; simulated feeding; simulated page turning; and writing. Participant performance of each task was timed. Longer time to complete the test indicates worse hand function.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable upper limb function tasks data.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
Lifting Large Heavy Objects, DOM Hand, Baseline	11
Lifting Large Heavy Objects, DOM Hand, Week 6	10
Lifting Large Heavy Objects, Non-DOM Hand Baseline	11
Lifting Large Heavy Objects, Non-DOM Hand, Week 6	11
Lifting Large Light Objects, DOM Hand, Baseline	9
Lifting Large Light Objects, DOM Hand, Week 6	15
Lifting Large Light Objects, Non-DOM Hand Baseline	7
Lifting Large Light Objects, Non-DOM Hand, Week 6	12
Stacking Large Heavy Objects, DOM Hand, Baseline	118
Stacking Large Heavy Objects, DOM Hand, Week 6	45
Stacking Large Heavy Objects Non-DOM Hand Baseline	120
Stacking Large Heavy Objects, Non-DOM Hand, Week 6	92
Stacking Large Light Objects, DOM Hand, Baseline	69
Stacking Large Light Objects, DOM Hand, Week 6	32
Stacking Large Light Objects Non-DOM Hand Baseline	23
Stacking Large Light Objects, Non-DOM Hand, Week 6	69
Lifting Small Common Objects, DOM Hand, Baseline	16
Lifting Small Common Objects, DOM Hand, Week 6	19
Lifting Small Common Objects Non-DOM Hand Baseline	13
Lifting Small Common Objects, Non-DOM Hand, Week 6	15
Stacking Checkers, DOM Hand, Baseline	7
Stacking Checkers, DOM Hand, Week 6	7
Simulated Feeding, DOM Hand, Baseline	22
Simulated Feeding, DOM Hand, Week 6	40
Simulated Feeding, Non-DOM Hand, Baseline	38
Simulated Feeding, Non-DOM Hand, Week 6	34
Simulated Page Turning, DOM Hand, Baseline	12
Simulated Page Turning, DOM Hand, Week 6	15
Simulated Page Turning, Non-DOM Hand, Baseline	13
Simulated Page Turning, Non-DOM Hand, Week 6	12
Writing, DOM Hand, Baseline	22
Writing, DOM Hand, Week 6	66
Writing, Non-DOM Hand, Baseline	47
Writing, Non-DOM Hand, Week 6	78

3. Secondary Outcome

Title	Upper Limb Function as Measured by the Brooke Upper Extremity Functional Rating Scale
Description	Upper extremity function was assessed using the Brooke Upper Extremity Functional Rating Scale, following standardized procedures. The Brooke Upper Extremity Functional Rating Scale graded arm and shoulder function from 1 to 6, with higher values indicating less function. A rating of "1" was used when the participant was able to abduct his arms in a full circle until they touch above his head, whereas a rating of "6" was used when the participant was unable to raise his hands to his mouth and had no useful function of hands.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable upper limb function tasks data.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
Baseline	3
Week 6	3

4. Secondary Outcome

Title	Participant Activities of Daily Living as Assessed Using the Egen Klassifikation (EK) Scale
Description	Activities of daily living after loss of ambulation were measured using the EK scale. The EK scale is an ordinal scale ranging from 0 to 30 points where 0 represents the highest level of independent function and 30 the lowest. The scale consists of 10 categories (each scored 0 to 3), involving different functional domains including 1) ability to use wheelchair, 2) ability to transfer from wheelchair, 3) ability to stand, 4) ability to balance in the wheelchair, 5) ability to move arms, 6) ability to use hands and arms when eating, 7) ability to turn in bed, 8) ability to cough, 9) ability to speak, and 10) physical well-being. The administration of the EK scale consisted of an interview of the participant to capture how he performs the tasks of daily life (as described by Categories 1 to 9) and how he perceives his wellbeing (as described by Category 10). The interviewer observed the participant and assigned the final score for the tasks that could be observed in the clinic.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable EK Scale data.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
Ability to Use Wheelchair, Baseline	2
Ability to Use Wheelchair, Week 6	1
Ability to Transfer From Wheelchair, Baseline	2
Ability to Transfer From Wheelchair, Week 6	2
Ability to Stand, Baseline	3
Ability to Stand, Week 6	2
Ability to Balance in the Wheelchair, Baseline	0
Ability to Balance in the Wheelchair, Week 6	0
Ability to Move Arms, Baseline	2
Ability to Move Arms, Week 6	1
Ability to Use Hands/Arms When Eating, Baseline	2
Ability to Use Hands/Arms When Eating, Week 6	2
Ability to Turn in Bed, Baseline	1
Ability to Turn in Bed, Week 6	1
Ability to Cough, Baseline	0
Ability to Cough, Week 6	0
Ability to Speak, Baseline	0
Ability to Speak, Week 6	0
Physical Well-Being, Baseline	0
Physical Well-Being, Week 6	1

5. Secondary Outcome

Title	Shoulder, Elbow, and Wrist Passive and Active Range of Motion as Measured by Goniometry
Description	Goniometry was performed to test active and passive range-of motion (RoM) of the left (L) and right (R) shoulder, elbow, and wrist following standardized procedures. The observed angle for passive and active motion for each joint was measured in degrees. Greater degree of motion indicates better response.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable range of motion data.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
L Elbow Extension, Supine Passive RoM, Baseline	-10
L Elbow Extension, Supine Passive RoM, Week 6	-15
R Elbow Extension, Supine Passive RoM. Baseline	-13
R Elbow Extension, Supine Passive RoM, Week 6	-20
L Elbow Flexion, Sitting Active RoM, Baseline	115
L Elbow Flexion, Sitting Active RoM, Week 6	-10
R Elbow Flexion, Sitting Active RoM, Baseline	120
R Elbow Flexion, Sitting Active RoM, Week 6	70
L Elbow Flexion, Supine Passive RoM, Baseline	133
L Elbow Flexion, Supine Passive RoM, Week 6	138
R Elbow Flexion, Supine Passive RoM, Baseline	135
R Elbow Flexion, Supine Passive RoM, Week 6	135
L Shoulder Abduction, Sitting Active RoM, Baseline	18
L Shoulder Abduction, Sitting Active RoM, Week 6	0
R Shoulder Abduction, Sitting Active RoM, Baseline	20
R Shoulder Abduction, Sitting Active RoM, Week 6	5
L Shoulder Abduction, Supine Passive RoM, Baseline	170
L Shoulder Abduction, Supine Passive RoM, Week 6	175
R Shoulder Abduction, Supine Passive RoM, Baseline	180
R Shoulder Abduction, Supine Passive RoM, Week 6	175
L Shoulder Flexion, Sitting Active RoM, Baseline	10
L Shoulder Flexion, Sitting Active RoM, Week 6	10
R Shoulder Flexion, Sitting Active RoM, Baseline	10
R Shoulder Flexion, Sitting Active RoM, Week 6	0
L Shoulder Flexion, Supine Passive RoM, Baseline	165
L Shoulder Flexion, Supine Passive RoM, Week 6	170
R Shoulder Flexion, Supine Passive RoM, Baseline	170
R Shoulder Flexion, Supine Passive RoM, Week 6	175
L Wrist Extension, Sitting Active RoM, Baseline	68
L Wrist Extension, Sitting Active RoM, Week 6	63
R Wrist Extension, Sitting Active RoM, Baseline	65
R Wrist Extension, Sitting Active RoM, Week 6	73
L Wrist Extension, Sitting Passive RoM, Baseline	73
L Wrist Extension, Sitting Passive RoM, Week 6	73
R Wrist Extension, Sitting Passive RoM, Baseline	78
R Wrist Extension, Sitting Passive RoM, Week 6	78
L Wrist Flexion, Sitting Passive RoM, Baseline	75
L Wrist Flexion, Sitting Passive RoM, Week 6	75
R Wrist Flexion, Sitting Passive RoM, Baseline	80
R Wrist Flexion, Sitting Passive RoM, Week 6	68

6. Secondary Outcome

Title	Force Exerted During Elbow Flexion and Extension, Shoulder Abduction, Hand Grip, Key Grip, and Finger Pinch as Assessed by Upper Extremity Myometry
Description	Upper extremity myometry was performed using a hand-held dynamometer following standardized procedures. The measured strength (peak force) was reported in Newtons. There are 0.22 pounds (lbs) in 1 Newton and approximately 10 Newton (9.80665 Newton) in 1 kilogram (kg). The threshold/range of the hand-held dynamometer is 0 to 500 Newtons. Bilateral assessments were done, and 3 measurements were recorded from each muscle group on each side, when possible. When the measurements were done in duplicate or triplicate, the best value was used. Greater value indicates better measurement.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable upper extremity myometry data.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
Left Elbow Extension, Supine, Baseline	12
Left Elbow Extension, Supine, Week 6	16
Right Elbow Extension, Supine, Baseline	12
Right Elbow Extension, Supine, Week 6	15
Left Elbow Flexion, Supine, Baseline	7
Left Elbow Flexion, Supine, Week 6	5
Right Elbow Flexion, Supine, Baseline	7
Right Elbow Flexion, Supine, Week 6	9
Left Finger Pinch, Sitting, Baseline	10
Left Finger Pinch, Sitting, Week 6	6
Right Finger Pinch, Sitting, Baseline	10
Right Finger Pinch, Sitting, Week 6	7
Left Hand Grip, Sitting, Baseline	13
Left Hand Grip, Sitting, Week 6	13
Right Hand Grip, Sitting, Baseline	15
Right Hand Grip, Sitting, Week 6	23
Left Key Grip, Sitting, Baseline	13
Left Key Grip, Sitting, Week 6	15
Right Key Grip, Sitting, Baseline	11
Right Key Grip, Sitting, Week 6	18
Left Shoulder Abduction, Sitting, Baseline	10
Left Shoulder Abduction, Sitting, Week 6	15
Right Shoulder Abduction, Sitting, Baseline	12
Right Shoulder Abduction, Sitting, Week 6	14

7. Secondary Outcome

Title	Time to Complete Hand Fine Motor Coordination and Dexterity Tasks as Measured by 9-Hole Peg Test (9HPT)
Description	Hand fine motor coordination and dexterity were assessed using the 9HPT using standardized procedures. The 9HPT is a unilateral test in which 9 pegs were placed in a board and then removed with the dominate and non-dominate hand within a 5-minute time limit. The amount of time required to put the pegs in the holes and remove them again with each hand was recorded. Each test was conducted twice per hand. Longer time to complete the test indicates worse hand fine motor coordination and dexterity.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable 9HPT data.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
Dominant Hand, Baseline	37
Dominant Hand, Week 6	38
Non-Dominant Hand, Baseline	40
Non-Dominant Hand, Week 6	37

8. Secondary Outcome

Title	Forced Vital Capacity (FVC) as Measured by Spirometry
Description	Pulmonary function was assessed as FVC in participants by spirometry using a study-specific spirometer. Multiple tests were conducted, if needed.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable spirometry data. Data was not collected at Week 6 as no participants were evaluable at this timepoint.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
Baseline	1

9. Secondary Outcome

Title	Systolic and Diastolic Function as Measured by Echocardiography With Tissue Doppler
Description	Cardiac function was assessed by echocardiography, which included standard parameters (for example, ejection fraction, left ventricle diastolic and systolic dimensions), as well as parameters integrating Doppler flow analysis with imaging to evaluate perturbations in wall motion. A standardized data collection process harmonized data from all participating institutions and allowed for centralized review.
Time Frame	Week 24 and Week 48

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable echocardiography data. Since the study was terminated early, echocardiography data were not collected after the start of study drug administration.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	0

10. Secondary Outcome

Title	Heart Rate as Assessed by Radial Pulse
Description	Heart rate was measured with the radial pulse. Following the Jebsen test, the participant rested for 5 minutes in a sitting position, and the heart rate for the last minute of this rest period was collected as the resting heart rate.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable heart rate data.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
Baseline	88
Week 6	100

11. Secondary Outcome

Title	Verbal Memory and Attention as Assessed by the Digit Span Task
Description	A series of digits (0-9) were presented to the participant in an auditory format only. The task had 2 parts: in the Forward Condition, the participant was requested to repeat back the digits in the order they were presented, and in the Backward Condition, he was requested to reverse the order of presentation.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable verbal memory and attention data. The test was repeated until the participant had 0 correct responses, which was up to 7 times for the Forward Condition and up to 5 times for the Backward Condition at Baseline and at Week 6.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
Forward Condition, 1 Correct Response, Baseline	4
Forward Condition, 1 Correct Response, Week 6	1
Forward Condition, 2 Correct Responses, Baseline	19
Forward Condition, 2 Correct Responses, Week 6	4
Backward Condition, 1 Correct Response, Baseline	4
Backward Condition, 1 Correct Response, Week 6	2
Backward Condition, 2 Correct Responses, Baseline	6
Backward Condition, 2 Correct Responses, Week 6	0

12. Secondary Outcome

Title	HRQL as Measured by the PedsQL Inventory Generic Core Scale
Description	Health-related quality of life (HRQL) was measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Generic Core Scale. The generic core module comprised of 23 questions evaluating physical, emotional, social, and school functioning. Examples of items in each of the generic core module scales included: "It is hard for me to run"; "I feel sad or blue"; "I cannot do things that other kids my age can do;" and "It is hard to pay attention in class." Each of the generic core module items was scored on a 5-point response scale from 0 (never a problem) to 4 (almost always a problem). The appropriate age-specific version was completed. PedsQL Inventory Generic Core Scale data at Week 6 is presented.
Time Frame	Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable PedsQL Inventory Generic Core Scale data at Week 6.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	2
Participant-Reported, Health and Activities	4
Participant-Reported, Feelings	1
Participant-Reported, Getting along with Others	2
Participant-Reported, School	1
Parent-Reported, Physical Functioning	4
Parent-Reported, Emotional Functioning	0
Parent-Reported, Social Functioning	1
Parent-Reported, School Functioning	1

13. Secondary Outcome

Title	HRQL as Measured by the PedsQL Multidimensional Fatigue Scale
Description	HRQL was measured by the PedsQL Multidimensional Fatigue Scale. The fatigue-specific module comprised of 18 questions evaluating general fatigue, sleep/rest fatigue, and cognitive fatigue. Fatigue-specific module obtains information relating to items such as: "I feel too tired to do things that I like to do"; "I spend a lot of time in bed"; and "I have trouble remembering more than one thing at a time." Each of the fatigue-specific module items was scored on a 5-point response scale from 0 (never a problem) to 4 (almost always a problem). The appropriate age-specific version was completed. PedsQL Multidimensional Fatigue Scale data at Week 6 is presented.
Time Frame	Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable PedsQL Multidimensional Fatigue Scale data at Week 6.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	2
Participant-Reported, General Fatigue	1
Participant-Reported, Sleep/Rest Fatigue	1
Participant-Reported, Cognitive Fatigue	1
Parent-Reported, General Fatigue	1
Parent-Reported, Sleep/Rest Fatigue	0
Parent-Reported, Cognitive Fatigue	1

14. Secondary Outcome

Title	HRQL as Measured by the INQoL
Description	HRQL was measured by the Individualized Neuromuscular Quality of Life Questionnaire (INQoL). The INQoL consisted of 45 questions within 10 sections. Four of the sections evaluate key muscle disease symptoms (that is, weakness, locking [myotonia], pain, and fatigue), 5 sections evaluate the degree and importance of the impact of muscle disease on particular areas of life, and 1 section asks about the positive and negative effects of treatment. A higher score indicates greater symptom impact or worse HRQL, with a range of 0-7.
Time Frame	Week 24 and Week 48

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable INQoL data. Since the study was terminated early, INQoL data were not collected.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	0

15. Secondary Outcome

Title	Muscle Fragility as Determined by Serum Creatine Kinase (CK) Levels
Description	Blood samples collected for chemistry assays were used to quantify serum CK concentrations. Serum CK was assessed as a potential biomarker for muscle fragility, with a reduction in serum CK considered to be a positive outcome. The reference range was based on the age of the participant.
Time Frame	Baseline and Week 6

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable CK data. Data was not collected at Week 6 for CK Levels with the reference range of 18-363 UL as no participants were evaluable at this timepoint.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	6
CK Levels (Reference Range 18-198 U/L), Baseline	1217
CK Levels (Reference Range 18-198 U/L), Week 6	764
CK Levels (Reference Range 18-363 U/L), Baseline	2605
CK Levels (Reference Range 18-408 U/L), Baseline	2343
CK Levels (Reference Range 18-408 U/L), Week 6	2753

16. Secondary Outcome

Title	Gastrocnemius Muscle Dystrophin Expression as Determined by Immunofluoresence or by Western Blotting Techniques
Description	The gastrocnemius muscle was to be biopsied from 1 leg to assess for the production of dystrophin at Week 36. The production of dystrophin was to be measured by immunofluorescene staining of the sarcolemmal membrane or by Western blotting techniques with an antibody to the C-terminal portion of the dystrophin protein (excluding revertant fibers).
Time Frame	Week 36

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable dystrophin production data. Since the study was terminated early, gastrocnemius muscle dystrophin expression data were not collected after the start of study drug administration.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	0

17. Secondary Outcome

Title	Study Drug Compliance
Description	Study drug compliance was assessed by the participant daily diary and quantification of used and unused study drug. Compliance was assessed in terms of the amount of drug actually taken relative to the amount that should have been taken during the study.
Time Frame	Baseline to Day 50

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable study drug compliance data.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	4
Missed 0 Doses	1 16.7%
Missed 1 Dose	2 33.3%
Missed 2 Doses	0 0%
Missed 3 Doses	0 0%
Missed 4 Doses	1 16.7%
Missed >5 Doses	0 0%

18. Secondary Outcome

Title	Pharmacokinetics: Ataluren Plasma Exposure
Description	Blood for ataluren concentrations over a 24-hour period was to be collected on Days 2 and 3 of Week 6. Analysis of the blood samples was to be conducted using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC-MS/MS) method.
Time Frame	0, 2, 3, 6, 8, 9, 12, 14, 15, and 24 hours after the morning dose

Outcome Measure Data

Analysis Population Description

All enrolled participants who received at least 1 dose of study drug and with evaluable plasma data. Since the study was terminated early, steady state data were not collected at Week 6.

Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
Measure Participants	0

Adverse Events

Time Frame	Baseline up to Day 50
Arm/Group Title	Ataluren
Arm/Group Description	Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days.
All Cause Mortality
	Ataluren
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Ataluren
	Affected / at Risk (%)	# Events
Total	0/6 (0%)
Other (Not Including Serious) Adverse Events
	Ataluren
	Affected / at Risk (%)	# Events
Total	1/6 (16.7%)
Gastrointestinal disorders
Diarrhoea	1/6 (16.7%)
Infections and infestations
Nasopharyngitis	1/6 (16.7%)
Renal and urinary disorders
Dysuria	1/6 (16.7%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.