Study of Testosterone and rHGH in FSHD

Facioscapulohumeral Muscular Dystrophy Clinical Trial

— STARFISH  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03123913
• Other study ID #: 1R01NS095813-01
• Status: Completed
• Phase: Phase 1
• Start date: December 18, 2017
• Est. completion date: March 28, 2022

Study information

• Verified date: May 2023
• Source: University of Rochester
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: March 28, 2022
• Est. primary completion date: March 28, 2022
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Facioscapulohumeral Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Facioscapulohumeral

Intervention

Drug:
• Testosterone Enanthate
Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks.
• Somatropin
Genotropin (5.0 µg/kg/day) delivered via subcutaneous injections.

Locations

Country	Name	City	State
United States	University of Rochester	Rochester	New York

Sponsors (1)

Lead Sponsor	Collaborator
University of Rochester

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Mean Change in Level of Free Testosterone in the Blood		Baseline to 24 weeks
Secondary	Mean Change in Level of Total Testosterone in the Blood		Baseline to 24 weeks
Secondary	Mean Change in Level of IGF-1 in the Blood		Baseline to 24 weeks
Secondary	Mean Change in Level of TSH in the Blood		Baseline to 24 weeks
Secondary	Mean Change in Level of Luteinizing Hormone in the Blood		Baseline to 24 weeks
Secondary	Mean Change in Level of FSH in the Blood		Baseline to 24 weeks
Secondary	Mean Change in Total Lean Body Mass		Baseline to 24 weeks