Study of Testosterone and rHGH in FSHD

Study Overview
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Brief Summary
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The purpose of this study is to investigate the safety and tolerability of combination therapy with recombinant human growth hormone (rHGH) and testosterone in adult male patients with facioscapulohumeral muscular dystrophy (FSHD) over 24 weeks.

Detailed Description
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This is a single-center, open-label study of daily human growth hormone (Genotropin®, 5.0 μg/kg via subcutaneous injection) and testosterone (testosterone enanthate, 140mg via intramuscular injection every two weeks) for 24 weeks in men with FSHD with a 12 week washout period. A total of 20 subjects will be enrolled at the University of Rochester Medical Center in Rochester, NY.

Official Title
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Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study

Conditions
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Facioscapulohumeral Muscular Dystrophy

Intervention / Treatment
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* Drug: Testosterone Enanthate
* Drug: Somatropin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: A genetically confirmed diagnosis of FSHD (or clinical symptoms suggestive of FSHD with a first degree relative with genetically confirmed FSHD) Hematocrit of ≤ 50% Prostate-specific antigen ≤ 4.0 ng/ml (or ≤ 3.0 ng/ml if the participant has a first-degree relative with prostate cancer) Fasting blood glucose <126 mg/dl Able to walk continuously for six minutes (cane, walker, orthoses allowed) Able to independently administer intramuscular and subcutaneous injections (or have a family member who is capable and willing to administer these injections) Exclusion Criteria: Diabetes Obesity (BMI>35 kg/m2) Cardiovascular disease (heart failure, coronary artery disease, uncontrolled hypertension, untreated hypercholesterolemia) Untreated thyroid disease Deep vein thrombosis Untreated severe sleep apnea Past pituitary disease Significant musculoskeletal injury and/or pain that affects walking A systolic blood pressure over 160 or a diastolic pressure over 100 Plans to dramatically change exercise habits Liver disease Renal disease Cancer (other than basal cell skin cancer) Plans to conceive Heavy alcohol use (greater than 50g/day) Current testosterone or HGH use Current use of medications that interfere with the growth hormone or gonadal endocrine axis.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Combination therapy<br>Testosterone Enanthate and Somatropin | Drug: Testosterone Enanthate<br>* Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks.<br>* Other names: Testosterone heptanoate;Drug: Somatropin<br>* Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.<br>* Other names: Zorbtive;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Who Experienced an AE | AEs were collected through patient report, interval laboratory studies, resting echocardiograms, dual energy x-ray absorptiometry (DEXA) studies, and physical examinations. | 36 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean Change in Level of Free Testosterone in the Blood |  | Baseline to 24 weeks | 
| Mean Change in Level of Total Testosterone in the Blood |  | Baseline to 24 weeks | 
| Mean Change in Level of IGF-1 in the Blood |  | Baseline to 24 weeks | 
| Mean Change in Level of TSH in the Blood |  | Baseline to 24 weeks | 
| Mean Change in Level of Luteinizing Hormone in the Blood |  | Baseline to 24 weeks | 
| Mean Change in Level of FSH in the Blood |  | Baseline to 24 weeks | 
| Mean Change in Total Lean Body Mass |  | Baseline to 24 weeks |