Study of Testosterone and rHGH in FSHD (STARFISH)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03123913|
Recruitment Status : Recruiting
First Posted : April 21, 2017
Last Update Posted : April 27, 2020
|Condition or disease||Intervention/treatment||Phase|
|Facioscapulohumeral Muscular Dystrophy||Drug: Testosterone Enanthate Drug: Somatropin||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study|
|Actual Study Start Date :||December 18, 2017|
|Estimated Primary Completion Date :||April 2021|
|Estimated Study Completion Date :||April 2021|
Experimental: Combination therapy
Testosterone Enanthate and Somatropin
Drug: Testosterone Enanthate
Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks.
Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
- Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [ Time Frame: 36 weeks ]Safety and tolerability are monitored by interval laboratory studies, resting echocardiograms, dual energy x-ray absorptiometry (DEXA) studies, and physical examinations. Patients will report any adverse events through in-person and telephone evaluations as well as on patient diaries.
- Serum levels of free and total testosterone, IGF-1, thyroid function, luteinizing hormone and follicle stimulating hormone [ Time Frame: 36 weeks ]Serum levels of free and total testosterone, IGF-1, thyroid function, luteinizing hormone and follicle stimulating hormone will be assessed at each study visit.
- Lean body mass [ Time Frame: 36 weeks ]Lean body mass will be measured at study visits through dual energy x-ray absorptiometry (DEXA) studies.
- Ambulation [ Time Frame: 36 weeks ]Ambulation will be assessed as an exploratory measure with the Six Minute Walk Test.
- Strength [ Time Frame: 36 weeks ]Strength will be assessed as an exploratory measure with manual muscle testing and quantitative muscle testing.
- Pulmonary Function [ Time Frame: 36 weeks ]Pulmonary function will be assessed as an exploratory measures with forced vital capacity testing.
- Patient-Reported Disease Burden [ Time Frame: 36 weeks ]Patient-reported disease burden will be assessed as an exploratory measure with the FSHD-Health Index, PROMIS-57, Beck Depression Inventory, Epworth Sleepiness Scale, Fatigue Severity Scale, and International Prostate Symptoms Score.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03123913
|Contact: Elizabeth A Luebbe, MSfirstname.lastname@example.org|
|United States, New York|
|University of Rochester||Recruiting|
|Rochester, New York, United States, 14642|
|Contact: Chad Heatwole, MD|
|Principal Investigator:||Chad R Heatwole, MD, MS-CI||University of Rochester|