Study of Testosterone and rHGH in FSHD

Phase 1

Completed

• Conditions: Facioscapulohumeral Muscular Dystrophy
• Interventions: Drug: Testosterone Enanthate; Drug: Somatropin

• Registration Number: NCT03123913
• Lead Sponsor: University of Rochester

Brief 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.

Detailed 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.

Study Timeline

• Study First Submitted: 2017-04-12
• Study First Submitted QC: 2017-04-14
• Study First Posted: 2017-04-21
• Study Start: 2017-12-18
• Primary Completion: 2022-03-28
• Study Completion: 2022-03-28
• Results First Submitted: 2023-03-28
• Status Verified: 2023-05
• Results First Submitted QC: 2023-05-12
• Last Update Submitted: 2023-05-12
• Results First Posted: 2023-05-16
• Last Update Posted: 2023-05-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 20

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)

Read More

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.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Combination therapy	Somatropin	Testosterone Enanthate and Somatropin
Combination therapy	Testosterone Enanthate	Testosterone Enanthate and Somatropin

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

University of Rochester; 🇺🇸 Rochester, New York, United States