Study of Testosterone and rHGH in FSHD

• Status: Active, not recruiting
• Trial end date: 2022-04-01
• Target enrollment: 0
• Participant gender: Male

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Rochester
• Treatments: Methyltestosterone; Testosterone; Testosterone 17 beta-cypionate; Testosterone enanthate; Testosterone undecanoate

Criteria

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.