Study of Testosterone and rHGH in FSHD (STARFISH)

University of Rochester

Status and phase: Completed; Phase 1

Conditions: Facioscapulohumeral Muscular Dystrophy

Treatments: Drug: Testosterone Enanthate; Drug: Somatropin

Study type: Interventional

Funder types: Other

Identifiers

NCT03123913

1R01NS095813-01

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 20 patients

Sex: Male

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Combination therapy

Experimental group

Description:

Testosterone Enanthate and Somatropin

Treatment:

Drug: Somatropin

Drug: Testosterone Enanthate