A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

• Sponsor: PTC Therapeutics (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT03783923
• Collaborator: (none)
• Enrollment: 11
• Locations: 18
• Arm: 1
• Actual Duration (Months): 14.1
• Patients Per Site: 0.6
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment).

Condition or Disease	Intervention/Treatment	Phase
Limb-Girdle Muscular Dystrophy	Drug: Deflazacort	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 11 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Multicenter Open-Label Study on the Safety and Efficacy of Deflazacort (Emflaza) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
• Actual Study Start Date: Oct 31, 2019
• Actual Primary Completion Date: Jan 1, 2021
• Actual Study Completion Date: Jan 1, 2021

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Deflazacort Participants will receive deflazacort 0.6 milligrams per kilograms per day (mg/kg/day) orally. The dose could be reduced in case of tolerability issues. Any participant assigned to placebo prior to the Version 4.0 amendment (prior to or after 01 February 2020) will have the option to be consented under Version 4.0 and will be switched to deflazacort for 26 weeks treatment. Any participant assigned to deflazacort prior to the Version 4.0 amendment (prior to 01 February 2020) will have the option to re-consent under Protocol Version 4.0 and continue for an additional 26 weeks treatment. Any participant assigned to deflazacort prior to the Version 4.0 amendment (after 01 February 2020) will have the option to re-consent under Protocol Version 4.0 at their Week 13 Visit and continue treatment until Week 26. Any new participant enrolled until 31 May 2020 will receive deflazacort for 26 weeks.

Drug: Deflazacort; Deflazacort tablet will be administered as per the dose and schedule specified in the arm.; Other Names: Emflaza®

Outcome Measures

Primary Outcome Measures

1. Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort [Baseline, Week 26]

Secondary Outcome Measures

1. Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort [Baseline, Week 26]

2. Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort [Baseline, Week 26]

3. Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort [Baseline, Week 26]

4. Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort [Baseline, Week 26]

5. Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort [Baseline, Week 26]

6. Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort [Baseline, Week 26]

7. Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort [Baseline, Week 26]

8. Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort [Baseline, Week 26]

9. Number of Participants With Adverse Events (AEs) [Baseline up to Week 52]

   An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an 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); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience.

10. Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort [Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13]

11. Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort [Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13]

12. Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort [Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13]

13. Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort [Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13]

14. Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort [Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Genetic diagnosis of LGMD2I (confirmed mutation in the fukutin-related protein [FKRP] gene).

• Ability to ascend 4 stairs greater than or equal to (≥) 2.5 seconds and be able to complete the ascent and descent both at screening and baseline.

• Ability to understand the nature of the study and the consent form and to comply with study related procedures.

• Must weigh between 35 to 112.5 kilograms (kg).

Exclusion Criteria:

• Received ≥4 weeks of continuous, systemic corticosteroid therapy within 3 months of study screening visit.

• Presence of significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction less than (<) 30 percent [%]) at screening.

• Requires fulltime ventilator support.

• History of chronic systemic fungal or viral infections.

• History of recent bacterial infection (including tuberculosis) per discretion of the Investigator.

• Diagnosis of diabetes mellitus (controlled and/or uncontrolled) defined as glycated hemoglobin (HbA1c) ≥6.5% (based on historical or present diagnosis).

• History of immunosuppression or other contraindications to glucocorticosteroid therapy.

• Requires concomitant use or greater than (>) 1 week of drugs or substances that are moderate to strong cytochrome P3A4 (CYP3A4) inhibitors (for example, clarithromycin, fluconazole, diltiazem, verapamil, grapefruit juice) or moderate or strong CYP3A4 inducers (that is, rifampin, efavirenz, carbamazepine, phenytoin) at baseline.

• Participated in an interventional clinical trial within the last 3 months prior the baseline visit.

• Unable or unwilling to comply with the contraceptive requirements of the protocol.

• Female participants who are pregnant and/or breastfeeding.

• Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, neurologic, psychiatric, or allergic disease.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Rare Disease Research, LLC	Atlanta	Georgia	United States	30324
2	University of Iowa Hospitals and Clinics	Iowa City	Iowa	United States	52242
3	The University of Kansas Medical Center	Kansas City	Kansas	United States	66160
4	Hugo W Moser Research Institute at Kennedy Krieger Institute	Baltimore	Maryland	United States	21205
5	University of Minnesota	Minneapolis	Minnesota	United States	55455
6	Washington University School of Medicine	Saint Louis	Missouri	United States	63110
7	University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104
8	University of Washington	Seattle	Washington	United States	98195
9	University of Alberta	Edmonton	Alberta	Canada	T6G 2G3
10	Ottawa Hospital	Ottawa		Canada	K1Y 4E9
11	Rigshospitalet, University of Copenhagen	Copenhagen		Denmark	2200
12	CHRU de NANCY Service de Neurologie	France		France	54035
13	University Hospital La Timone	Marseille		France	13385
14	Ludwig-Maximilians University Munich, Friedrich-Baur-Institute	Munich		Germany	80801
15	Oslo University Hospital	Oslo		Norway	0424
16	Pirogov Russian National Research Medical University	Moscow		Russian Federation	125412
17	Saint-Petersburg State Pediatric Medical University	Saint Petersburg		Russian Federation	194100
18	Sahlgrenska University Hospital	Gothenburg		Sweden	41345

Sponsors and Collaborators

• PTC Therapeutics

Investigators

• Study Director: Cristobal Passalacqua, MD, PTC Therapeutics

Study Documents (Full-Text)

• Study Protocol - Mar 25, 2020
• Statistical Analysis Plan - Oct 28, 2020

More Information

Publications

None provided.

• Responsible Party: PTC Therapeutics
• ClinicalTrials.gov Identifier: NCT03783923
• Other Study ID Numbers: PTCEMF-GD-004
• First Posted: Dec 21, 2018
• Last Update Posted: Jun 27, 2022
• Last Verified: May 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: Yes
• Additional relevant MeSH terms: Muscular Dystrophies; Muscular Dystrophies, Limb-Girdle; Deflazacort

Study Results

Participant Flow

Pre-assignment Detail	Participants enrolled under protocol v3.0 were 1:1 randomized to get placebo or deflazacort. After protocol v4.0, the study became an open-label study and all participants received deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety and efficacy summaries.

Arm/Group Title	Deflazacort	Placebo
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 milligrams (mg)/kilograms (kg)/day for 26 weeks in placebo-controlled period and for 26 weeks in open-label extension period.	Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.
Period Title: Placebo-Controlled Period (26 Weeks)
STARTED	5	6
Safety Population	5	2
COMPLETED	1	2
NOT COMPLETED	4	4
Period Title: Placebo-Controlled Period (26 Weeks)
STARTED	1	2
COMPLETED	0	0
NOT COMPLETED	1	2

Baseline Characteristics

Arm/Group Title	Deflazacort	Placebo	Total
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.	Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.	Total of all reporting groups
Overall Participants	5	6	11
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%
Between 18 and 65 years	5 100%	5 83.3%	10 90.9%
>=65 years	0 0%	1 16.7%	1 9.1%
Sex: Female, Male (Count of Participants)
Female	4 80%	4 66.7%	8 72.7%
Male	1 20%	2 33.3%	3 27.3%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%
Asian	0 0%	0 0%	0 0%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%
Black or African American	0 0%	0 0%	0 0%
White	5 100%	6 100%	11 100%
More than one race	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%

Outcome Measures

1. Primary Outcome

Title	Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	7
Baseline	5.476 (2.0178)
Change at Week 26	-0.200

2. Secondary Outcome

Title	Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	0

3. Secondary Outcome

Title	Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	7
Baseline	135.4 (26.75)
Change at Week 26	2.0

4. Secondary Outcome

Title	Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	7
Baseline	11.93 (4.743)
Change at Week 26	9.70

5. Secondary Outcome

Title	Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	7
Baseline	3.66 (1.707)
Change at Week 26	0.10

6. Secondary Outcome

Title	Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	7
Baseline	8.53 (1.897)
Change at Week 26	-0.40

7. Secondary Outcome

Title	Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	0

8. Secondary Outcome

Title	Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	0

9. Secondary Outcome

Title	Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort
Time Frame	Baseline, Week 26

Outcome Measure Data

Analysis Population Description

Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	0

10. Secondary Outcome

Title	Number of Participants With Adverse Events (AEs)
Description	An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an 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); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience.
Time Frame	Baseline up to Week 52

Outcome Measure Data

Analysis Population Description

Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo during the study were not included for safety analysis.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	7
Count of Participants [Participants]	5 100%

11. Secondary Outcome

Title	Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time Frame	Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

Outcome Measure Data

Analysis Population Description

The pharmacokinetic (PK) population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	4
21-desacetyl deflazacort: Baseline	401.1 (139.62)
21-desacetyl deflazacort: Week 13	365.6 (NA)
6β-hydroxy-21-desacetyl deflazacort: Week 1	418.2 (58.569)
6β-hydroxy-21-desacetyl deflazacort: Week 13	515.3 (NA)

12. Secondary Outcome

Title	Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time Frame	Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

Outcome Measure Data

Analysis Population Description

The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	4
21-desacetyl deflazacort: Baseline	423.6 (150.16)
6β-hydroxy-21-desacetyl deflazacort: Week 1	520.3 (31.508)

13. Secondary Outcome

Title	Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time Frame	Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

Outcome Measure Data

Analysis Population Description

The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	4
21-desacetyl deflazacort: Baseline	184.8 (49.054)
21-desacetyl deflazacort: Week 13	171.0 (NA)
6β-hydroxy-21-desacetyl deflazacort: Week 1	135.8 (29.205)
6β-hydroxy-21-desacetyl deflazacort: Week 13	162.0 (NA)

14. Secondary Outcome

Title	Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time Frame	Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

Outcome Measure Data

Analysis Population Description

The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	4
21-desacetyl deflazacort: Baseline	0.992
21-desacetyl deflazacort: Week 13	0.525
6β-hydroxy-21-desacetyl deflazacort: Week 1	1.000
6β-hydroxy-21-desacetyl deflazacort: Week 13	1.550

15. Secondary Outcome

Title	Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
Time Frame	Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

Outcome Measure Data

Analysis Population Description

The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.

Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
Measure Participants	4
21-desacetyl deflazacort: Baseline	1.174 (0.0849)
21-desacetyl deflazacort: Week 13	1.235 (NA)
6β-hydroxy-21-desacetyl deflazacort: Week 1	2.358 (0.5825)
6β-hydroxy-21-desacetyl deflazacort: Week 13	1.95

Adverse Events

Time Frame	Baseline up to Week 52
Adverse Event Reporting Description	Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.
Arm/Group Title	Deflazacort
Arm/Group Description	Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.
All Cause Mortality
	Deflazacort
	Affected / at Risk (%)	# Events
Total	0/7 (0%)
Serious Adverse Events
	Deflazacort
	Affected / at Risk (%)	# Events
Total	0/7 (0%)
Other (Not Including Serious) Adverse Events
	Deflazacort
	Affected / at Risk (%)	# Events
Total	5/7 (71.4%)
Endocrine disorders
Cushingoid	1/7 (14.3%)
Gastrointestinal disorders
Abdominal discomfort	1/7 (14.3%)
Dyspepsia	1/7 (14.3%)
General disorders
Fatigue	1/7 (14.3%)
Infections and infestations
Influenza	1/7 (14.3%)
Upper respiratory tract infection	1/7 (14.3%)
Injury, poisoning and procedural complications
Contusion	1/7 (14.3%)
Post-traumatic pain	1/7 (14.3%)
Skin laceration	1/7 (14.3%)
Spinal compression fracture	1/7 (14.3%)
Fall	1/7 (14.3%)
Investigations
Weight increased	2/7 (28.6%)
Metabolism and nutrition disorders
Increased appetite	1/7 (14.3%)
Musculoskeletal and connective tissue disorders
Arthralgia	1/7 (14.3%)
Back pain	1/7 (14.3%)
Muscle tightness	1/7 (14.3%)
Myalgia	1/7 (14.3%)
Nervous system disorders
Headache	1/7 (14.3%)
Hypoaesthesia	1/7 (14.3%)
Tension headache	1/7 (14.3%)
Visual field defect	1/7 (14.3%)
Psychiatric disorders
Apathy	1/7 (14.3%)
Depressed mood	1/7 (14.3%)
Sleep disorder	1/7 (14.3%)
Renal and urinary disorders
Pollakiuria	1/7 (14.3%)
Reproductive system and breast disorders
Amenorrhoea	1/5 (20%)
Menstruation irregular	1/5 (20%)
Skin and subcutaneous tissue disorders
Acne	2/7 (28.6%)
Abnormal hair growth	1/7 (14.3%)
Hirsutism	1/7 (14.3%)

Limitations/Caveats

The study was terminated early due to low enrollment and missing efficacy assessment data due to missed visits related to COVID-19.

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.