ZEPHYR: Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome

• Sponsor: Millendo Therapeutics SAS (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT03790865
• Collaborator: (none)
• Enrollment: 158
• Locations: 37
• Arms: 3
• Actual Duration (Months): 14
• Patients Per Site: 4.3
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).

Condition or Disease	Intervention/Treatment	Phase
Prader-Willi Syndrome; Hyperphagia	Drug: Livoletide; Drug: Placebo	Phase 2/Phase 3

Detailed Description

The protocol includes 2 consecutive parts:

1. The first part is a Phase 2b dose-response study consisting of a 3-month double-blind, placebo-controlled Core Period followed by a 9-month Extension Period.

2. The second part is a Phase 3 study consisting of a 6-month double-blind, placebo-controlled Core Period followed by a 6-month Extension Period. Phase 3 may be initiated following review of safety and efficacy results at the completion of the Phase 2b Core Period.

A total of approximately 50 patients per group (8 to 65 years of age) will be randomized (approximately 150 patients in total). In addition to this cohort of 150 patients, a separate cohort of patients 4 to 7 years of age will also be randomized.

Note: The 8-65 year old cohort has been fully enrolled. Enrollment into the 4-7 year age cohort remains ongoing.

Study Design

• Study Type: Interventional
• Actual Enrollment: 158 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase 2b/3 Study to Evaluate the Safety, Tolerability, and Effects of Livoletide (AZP-531), an Unacylated Ghrelin Analogue, on Food-related Behaviors in Patients With Prader-Willi Syndrome
• Actual Study Start Date: Mar 25, 2019
• Actual Primary Completion Date: Feb 26, 2020
• Actual Study Completion Date: May 25, 2020

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Low-Dose Livoletide Daily subcutaneous injection of ~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period.	Drug: Livoletide; Daily subcutaneous injection; Other Names: AZP-531
Experimental: High-Dose Livoletide Daily subcutaneous injection of ~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period.	Drug: Livoletide; Daily subcutaneous injection; Other Names: AZP-531
Placebo Comparator: Placebo Daily subcutaneous injection of 0.9% NaCl for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period.	Drug: Placebo; Daily subcutaneous injection

Outcome Measures

Primary Outcome Measures

1. Change in Hyperphagia and Food-related Behaviors (Hyperphagia Questionnaire for Clinical Trials; HQ-CT) [Baseline to month 3]

   Change from baseline to the end of the 3-month Core Period for HQ-CT total score. The HQ-CT score range is 0 to 36 where the higher score represents more severe abnormal food related behaviors.

Secondary Outcome Measures

1. Percentage Change From Baseline to Month 3 in Total Body Fat Mass in Patients Eight to 65 Years of Age Defined as Overweight/Obese [Baseline to month 3]

   Total body fat mass was assessed using dual energy X-ray absorptiometry (DXA) scan. DXAs were conducted at each local facility using standardized procedures and settings. Overweight/obese patients were defined as follows: patients ≥18 years of age: BMI ≥27 kg/m2 patients 4-17 years of age: BMI ≥90th percentile for the same age and sex

2. Change From Baseline to Month 3 in Waist Circumference in Patients Eight to 65 Years of Age Defined as Overweight/Obese [Baseline to month 3]

   The waist circumference was measured in fasting condition at the superior border of iliac crest, according to recommendations from the Anthropometry Procedures Manual of the National Health and Nutrition Examination Survey, Revised 2007. Overweight/obese patients were defined as follows: patients ≥18 years of age: BMI ≥27 kg/m2 patients 4-17 years of age: BMI ≥90th percentile for the same age and sex

3. Percentage Change From Baseline to Month 3 in Body Weight in Patients Eight to 65 Years of Age Defined as Overweight/Obese [Baseline to month 3]

   Patients were weighed in fasting condition, clothed (underwear, light gown or light clothing), without footwear or heavy jewelry, using a calibrated scale. The same scale should be used throughout the study if possible. The conditions under which patients are weighed should be kept consistent if possible. Overweight/obese patients were defined as follows: patients ≥18 years of age: BMI ≥27 kg/m2 patients 4-17 years of age: BMI ≥90th percentile for the same age and sex

Eligibility Criteria

Criteria

• Ages Eligible for Study: 4 Years to 65 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Confirmed genetic diagnosis of PWS

• Evidence of increased appetite or hyperphagia

• Patient must have a single primary caregiver who should be available for certain durations of the study

• BMI ≤ 65 kg/m2

• Growth hormone treatment permitted if doses have been stable for at least 1 month prior to screening

Exclusion Criteria:

• History of chronic liver disease

• Type 1 diabetes mellitus

• HbA1c > 10%

• Body weight <20 kg

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Alabama at Birmingham	Birmingham	Alabama	United States	35233
2	University of California - Irvine Medical Center	Orange	California	United States	92868
3	Rady Children's Hospital - San Diego	San Diego	California	United States	92123
4	Children's Hospital Colorado	Denver	Colorado	United States	80045
5	University of Kansas Medical Center	Kansas City	Kansas	United States	66160
6	Johns Hopkins University	Baltimore	Maryland	United States	21287
7	Boston Children's Hospital	Boston	Massachusetts	United States	02215
8	University of Michigan	Ann Arbor	Michigan	United States	48109
9	Children's Hospitals and Clinics of Minnesota-Minneapolis	Saint Paul	Minnesota	United States	55102
10	Winthrop University Hospital	Mineola	New York	United States	11501
11	New York Presbyterian Morgan Stanley Children's Hospital	New York	New York	United States	10032
12	Vanderbilt University Medical Center	Nashville	Tennessee	United States	37232
13	University of Texas Southwestern Medical Center	Dallas	Texas	United States	75390
14	Baylor College of Medicine	Houston	Texas	United States	77030
15	Seattle Children's Hospital	Seattle	Washington	United States	98105
16	Royal Prince Alfred Hospital	Camperdown		Australia	2050
17	Austin Health	Melbourne		Australia	3084
18	Perth Children's Hospital	Nedlands		Australia	6009
19	The Childrens Hospital at Westmead	Westmead		Australia	2145
20	Cliniques Universitaires Saint-Luc	Brussels		Belgium	1200
21	Centre Hospitalier Universitaire d'Angers	Angers		France	49100
22	CHU Lyon - Hopital Femmes Mere Enfant	Bron		France	69677
23	Hospital Pitie Salpetriere	Paris		France	75013
24	Hopital Necker-Enfants Malades	Paris		France	75015
25	CHU de Toulouse - Hospital Rangueil	Toulouse		France	31059
26	CHU de Toulouse - Hopital des Enfants	Toulouse		France
27	Azienda Ospedaliera Universitaria Federico II	Napoli		Italy	80131
28	Ospedale Pediatrico Bambino Gesù	Roma		Italy	00165
29	Erasmus University Medical Center	Rotterdam		Netherlands	3015
30	Stichting Kind en Groei	Rotterdam		Netherlands	3016
31	Hospital General Universitario de Alicante	Alicante		Spain	03010
32	Hospital de Cruces	Barakaldo		Spain	48903
33	Hospital Sant Joan de Deu	Barcelona		Spain	08950
34	Corporacio Sanitaria Parc Tauli - Hospital de Sabadell	Sabadell		Spain	08208
35	NHS Tayside	Dundee		United Kingdom	DD1 9SY
36	Chelsea and Westminster Hospital	London		United Kingdom	SW109NH
37	Imperial College London	London		United Kingdom	W12 0NN

Sponsors and Collaborators

• Millendo Therapeutics SAS

Investigators

None specified.

Study Documents (Full-Text)

• Statistical Analysis Plan - Mar 23, 2020
• Study Protocol - Jul 31, 2019

More Information

Publications

None provided.

• Responsible Party: Millendo Therapeutics SAS
• ClinicalTrials.gov Identifier: NCT03790865
• Other Study ID Numbers: AZP01-CLI-003
• First Posted: Jan 2, 2019
• Last Update Posted: Feb 17, 2021
• Last Verified: Feb 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Millendo Therapeutics SAS: Prader-Willi Syndrome; PWS
• Additional relevant MeSH terms: Prader-Willi Syndrome; Syndrome; Hyperphagia

Study Results

Participant Flow

Recruitment Details	Original protocol: for Phase 2b, a total of 50 patients per group will need to be randomized. Amendment v1.2: for Phase 2b, a total of approximately 50 patients per group (8 to 65 years of age) will need to be randomized. In addition to this cohort of 150 patients, a separate cohort of patients 4 to 7 years of age will also be randomized.
Pre-assignment Detail	Screening Period was up to 4 weeks. After signing informed consent, patients with Prader-Willi Syndrome entered the Screening Period to assess preliminary eligibility for the study based on the inclusion and exclusion criteria. In addition, pertinent information was collected such as past medical history, demographic data, and prior and current medications

Arm/Group Title	Low-Dose Livoletide	High-Dose Livoletide	Placebo
Arm/Group Description	Daily subcutaneous injection of ~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of ~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of 0.9% sodium chloride for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period. Placebo: Daily subcutaneous injection
Period Title: Overall Study
STARTED	52	52	54
COMPLETED	52	50	54
NOT COMPLETED	0	2	0

Baseline Characteristics

Arm/Group Title	Low-Dose Livoletide	High-Dose Livoletide	Placebo	Total
Arm/Group Description	Daily subcutaneous injection of ~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of ~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of 0.9% NaCl for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period. Placebo: Daily subcutaneous injection	Total of all reporting groups
Overall Participants	52	52	54	158
Age (Count of Participants)
<=18 years	24 46.2%	26 50%	24 44.4%	74 46.8%
Between 18 and 65 years	28 53.8%	26 50%	30 55.6%	84 53.2%
>=65 years	0 0%	0 0%	0 0%	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	20.7 (9.20)	19.7 (8.27)	19.4 (8.03)	19.9 (8.47)
Sex: Female, Male (Count of Participants)
Female	28 53.8%	28 53.8%	32 59.3%	88 55.7%
Male	24 46.2%	24 46.2%	22 40.7%	70 44.3%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	5 9.6%	5 9.6%	1 1.9%	11 7%
Not Hispanic or Latino	37 71.2%	36 69.2%	37 68.5%	110 69.6%
Unknown or Not Reported	10 19.2%	11 21.2%	16 29.6%	37 23.4%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%	0 0%
Asian	0 0%	3 5.8%	3 5.6%	6 3.8%
Native Hawaiian or Other Pacific Islander	1 1.9%	0 0%	0 0%	1 0.6%
Black or African American	4 7.7%	2 3.8%	1 1.9%	7 4.4%
White	38 73.1%	35 67.3%	36 66.7%	109 69%
More than one race	0 0%	0 0%	0 0%	0 0%
Unknown or Not Reported	9 17.3%	12 23.1%	14 25.9%	35 22.2%
Region of Enrollment (Count of Participants)
Netherlands	3 5.8%	1 1.9%	4 7.4%	8 5.1%
Belgium	1 1.9%	2 3.8%	2 3.7%	5 3.2%
United States	21 40.4%	24 46.2%	21 38.9%	66 41.8%
United Kingdom	2 3.8%	1 1.9%	0 0%	3 1.9%
Italy	1 1.9%	2 3.8%	2 3.7%	5 3.2%
France	9 17.3%	11 21.2%	14 25.9%	34 21.5%
Australia	3 5.8%	3 5.8%	1 1.9%	7 4.4%
Spain	12 23.1%	8 15.4%	10 18.5%	30 19%
Baseline HQ-CT (units on a scale) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [units on a scale]	20.4 (6.37)	19.5 (6.34)	20.5 (5.87)	20.2 (6.15)

Outcome Measures

1. Primary Outcome

Title	Change in Hyperphagia and Food-related Behaviors (Hyperphagia Questionnaire for Clinical Trials; HQ-CT)
Description	Change from baseline to the end of the 3-month Core Period for HQ-CT total score. The HQ-CT score range is 0 to 36 where the higher score represents more severe abnormal food related behaviors.
Time Frame	Baseline to month 3

Outcome Measure Data

Analysis Population Description

The Full Analysis Set (FAS) included all randomized patients. Efficacy analyses for the Phase 2b Core Period were performed on the FAS.

Arm/Group Title	Low-Dose Livoletide	High-Dose Livoletide	Placebo
Arm/Group Description	Daily subcutaneous injection of ~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of ~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of 0.9% NaCl for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period. Placebo: Daily subcutaneous injection
Measure Participants	52	52	54
Mean (Standard Error) [score on a scale]	-5.1 (7.76)	-3.6 (6.08)	-3.3 (5.77)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Low-Dose Livoletide, High-Dose Livoletide, Placebo
	Comments
	Type of Statistical Test	Superiority
	Comments
Statistical Test of Hypothesis	p-Value	<0.025
	Comments
	Method	Mixed Models Analysis
	Comments
Method of Estimation	Estimation Parameter	Mean Difference (Net)
	Estimated Value	4
	Confidence Interval	(2-Sided) %; to
	Parameter Dispersion	Type: Value:
	Estimation Comments

2. Secondary Outcome

Title	Percentage Change From Baseline to Month 3 in Total Body Fat Mass in Patients Eight to 65 Years of Age Defined as Overweight/Obese
Description	Total body fat mass was assessed using dual energy X-ray absorptiometry (DXA) scan. DXAs were conducted at each local facility using standardized procedures and settings. Overweight/obese patients were defined as follows: patients ≥18 years of age: BMI ≥27 kg/m2 patients 4-17 years of age: BMI ≥90th percentile for the same age and sex
Time Frame	Baseline to month 3

Outcome Measure Data

Analysis Population Description

The Full Analysis Set (FAS) included all randomized patients. Efficacy analyses for the Phase 2b Core Period were performed on the FAS. For this endpoint, only patients considered overweight/obese at baseline were included in the analysis. Overweight/obese patients were defined as follows: patients ≥18 years of age: BMI ≥27 kg/m2 patients 4-17 years of age: BMI ≥90th percentile for the same age and sex

Arm/Group Title	Low-Dose Livoletide	High-Dose Livoletide	Placebo
Arm/Group Description	Daily subcutaneous injection of ~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of ~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of 0.9% NaCl for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period. Placebo: Daily subcutaneous injection
Measure Participants	40	39	40
Mean (Standard Deviation) [percent change]	0.33 (3.806)	3.48 (4.429)	-0.36 (4.302)

3. Secondary Outcome

Title	Change From Baseline to Month 3 in Waist Circumference in Patients Eight to 65 Years of Age Defined as Overweight/Obese
Description	The waist circumference was measured in fasting condition at the superior border of iliac crest, according to recommendations from the Anthropometry Procedures Manual of the National Health and Nutrition Examination Survey, Revised 2007. Overweight/obese patients were defined as follows: patients ≥18 years of age: BMI ≥27 kg/m2 patients 4-17 years of age: BMI ≥90th percentile for the same age and sex
Time Frame	Baseline to month 3

Outcome Measure Data

Analysis Population Description

The Full Analysis Set (FAS) included all randomized patients. Efficacy analyses for the Phase 2b Core Period were performed on the FAS. For this endpoint, only patients considered overweight/obese at baseline were included in the analysis. Overweight/obese patients were defined as follows: patients ≥18 years of age: BMI ≥27 kg/m2 patients 4-17 years of age: BMI ≥90th percentile for the same age and sex

Arm/Group Title	Low-Dose Livoletide	High-Dose Livoletide	Placebo
Arm/Group Description	Daily subcutaneous injection of ~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of ~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of 0.9% NaCl for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period. Placebo: Daily subcutaneous injection
Measure Participants	40	39	40
Mean (Standard Deviation) [cm]	0.92 (5.703)	0.11 (4.969)	-0.45 (3.674)

4. Secondary Outcome

Title	Percentage Change From Baseline to Month 3 in Body Weight in Patients Eight to 65 Years of Age Defined as Overweight/Obese
Description	Patients were weighed in fasting condition, clothed (underwear, light gown or light clothing), without footwear or heavy jewelry, using a calibrated scale. The same scale should be used throughout the study if possible. The conditions under which patients are weighed should be kept consistent if possible. Overweight/obese patients were defined as follows: patients ≥18 years of age: BMI ≥27 kg/m2 patients 4-17 years of age: BMI ≥90th percentile for the same age and sex
Time Frame	Baseline to month 3

Outcome Measure Data

Analysis Population Description

The Full Analysis Set (FAS) included all randomized patients. Efficacy analyses for the Phase 2b Core Period were performed on the FAS. For this endpoint, only patients considered overweight/obese at baseline were included in the analysis. Overweight/obese patients were defined as follows: patients ≥18 years of age: BMI ≥27 kg/m2 patients 4-17 years of age: BMI ≥90th percentile for the same age and sex

Arm/Group Title	Low-Dose Livoletide	High-Dose Livoletide	Placebo
Arm/Group Description	Daily subcutaneous injection of ~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of ~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection	Daily subcutaneous injection of 0.9% NaCl for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period. Placebo: Daily subcutaneous injection
Measure Participants	40	39	40
Mean (Standard Deviation) [percent change]	1.39 (3.313)	1.79 (2.079)	1.06 (2.589)

Adverse Events

Time Frame	The analysis of treatment-emergent adverse events (TEAEs) was done starting first dose of study drug and through 30 days after the end of the treatment period
Arm/Group Title	Low-Dose Livoletide		High-Dose Livoletide		Placebo
Arm/Group Description	Daily subcutaneous injection of ~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection		Daily subcutaneous injection of ~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period. Livoletide: Daily subcutaneous injection		Daily subcutaneous injection of 0.9% NaCl for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period. Placebo: Daily subcutaneous injection
All Cause Mortality
	Low-Dose Livoletide		High-Dose Livoletide		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/52 (0%)		0/52 (0%)		0/54 (0%)
Serious Adverse Events
	Low-Dose Livoletide		High-Dose Livoletide		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/52 (3.8%)		1/52 (1.9%)		1/54 (1.9%)
Infections and infestations
Lower respiratory tract infection	1/52 (1.9%)	1	0/52 (0%)	0	0/54 (0%)	0
Nervous system disorders
Syncope	0/52 (0%)	0	1/52 (1.9%)	1	0/54 (0%)	0
Psychiatric disorders
Impulse control disorder	1/52 (1.9%)	1	0/52 (0%)	0	1/54 (1.9%)	1
Other (Not Including Serious) Adverse Events
	Low-Dose Livoletide		High-Dose Livoletide		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	34/52 (65.4%)		34/52 (65.4%)		34/54 (63%)
Gastrointestinal disorders
Diarrhea	8/52 (15.4%)	10	4/52 (7.7%)	4	5/54 (9.3%)	5
Abdominal Pain	1/52 (1.9%)	2	0/52 (0%)	0	2/54 (3.7%)	3
Constipation	2/52 (3.8%)	2	0/52 (0%)	0	1/54 (1.9%)	1
Nausea	1/52 (1.9%)	1	0/52 (0%)	0	2/54 (3.7%)	2
General disorders
Injection site pain	8/52 (15.4%)	11	6/52 (11.5%)	6	2/54 (3.7%)	2
Injection site bruising	3/52 (5.8%)	3	5/52 (9.6%)	5	3/54 (5.6%)	3
Injection site erythma	5/52 (9.6%)	5	2/52 (3.8%)	2	0/54 (0%)	0
Pyrexia	4/52 (7.7%)	4	2/52 (3.8%)	2	1/54 (1.9%)	2
Fatigue	1/52 (1.9%)	1	2/52 (3.8%)	2	1/54 (1.9%)	1
Injection site hematoma	3/52 (5.8%)	6	1/52 (1.9%)	1	0/54 (0%)	0
Injection site mass	2/52 (3.8%)	2	1/52 (1.9%)	1	1/54 (1.9%)	1
Infections and infestations
Nasopharyngitis	2/52 (3.8%)	2	2/52 (3.8%)	2	6/54 (11.1%)	6
Influenza	0/52 (0%)	0	1/52 (1.9%)	1	4/54 (7.4%)	4
Gastroenteritis	1/52 (1.9%)	1	2/52 (3.8%)	2	1/54 (1.9%)	1
Upper respiration infection	0/52 (0%)	0	2/52 (3.8%)	2	1/54 (1.9%)	2
Ear infection	2/52 (3.8%)	2	0/52 (0%)	0	0/54 (0%)	0
Paronychia	2/52 (3.8%)	3	0/52 (0%)	0	0/54 (0%)	0
Sinusitis	0/52 (0%)	0	0/52 (0%)	0	2/54 (3.7%)	2
Injury, poisoning and procedural complications
Ligament sprain	1/52 (1.9%)	1	2/52 (3.8%)	2	0/54 (0%)	0
Contusion	0/52 (0%)	0	0/52 (0%)	0	2/54 (3.7%)	2
Investigations
Blood pressure systolic increased	2/52 (3.8%)	2	0/52 (0%)	0	0/54 (0%)	0
Musculoskeletal and connective tissue disorders
Pain in extremity	1/52 (1.9%)	1	0/52 (0%)	0	2/54 (3.7%)	2
Myalgia	0/52 (0%)	0	0/52 (0%)	0	2/54 (3.7%)	2
Nervous system disorders
Headache	3/52 (5.8%)	6	2/52 (3.8%)	2	3/54 (5.6%)	3
Psychiatric disorders
Dermatillomania	0/52 (0%)	0	2/52 (3.8%)	2	0/54 (0%)	0
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain	4/52 (7.7%)	4	2/52 (3.8%)	2	0/54 (0%)	0

Limitations/Caveats

The double-blind, placebo-controlled phase 2b study did not meet the primary endpoint or any of the secondary endpoints. Therefore, the Sponsor has decided to discontinue further development of livoletide.

More Information

Certain Agreements

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

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.