Study of Testosterone and rHGH in FSHD (STARFISH)

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

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.

Study Overview

• Status: Completed
• Conditions: Facioscapulohumeral Muscular Dystrophy
• Intervention / Treatment: Drug: Testosterone Enanthate; Drug: Somatropin

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 Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • New York
    • Rochester, New York, United States, 14642
      • University of Rochester

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Combination therapy; Testosterone Enanthate and Somatropin

Drug: Testosterone Enanthate; Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks.; Other Names: Delatestryl; Tesostroval; Testro LA; Andro LA; Durathate; Everone; Testrin; Andropository; Testosterone heptanoate; Drug: Somatropin; Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.; Other Names: Genotropin; Norditropin; Humatrope; Serostim; Nutropin; Zorbtive

What is the study measuring?

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	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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ambulation	Ambulation will be assessed as an exploratory measure with the Six Minute Walk Test.	36 weeks
Strength	Strength will be assessed as an exploratory measure with manual muscle testing and quantitative muscle testing.	36 weeks
Pulmonary Function	Pulmonary function will be assessed as an exploratory measures with forced vital capacity testing.	36 weeks
Patient-Reported Disease Burden	Patient-reported disease burden will be assessed as an exploratory measure with the FSHD-Health Index, PROMIS-57, Beck Depression Inventory, Epworth Sleepiness Scale, Fatigue Severity Scale, and International Prostate Symptoms Score.	36 weeks

Collaborators and Investigators

• Sponsor: University of Rochester
• Investigators: Principal Investigator: Chad R Heatwole, MD, MS-CI, University of Rochester

Study record dates

Study Major Dates

• Study Start (Actual): December 18, 2017
• Primary Completion (Actual): March 28, 2022
• Study Completion (Actual): March 28, 2022

Study Registration Dates

• First Submitted: April 12, 2017
• First Submitted That Met QC Criteria: April 14, 2017
• First Posted (Actual): April 21, 2017

Study Record Updates

• Last Update Posted (Actual): May 16, 2023
• Last Update Submitted That Met QC Criteria: May 12, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Facioscapulohumeral; Physiological Effects of Drugs; Antineoplastic Agents; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Androgens; Anabolic Agents; Testosterone; Methyltestosterone; Testosterone undecanoate; Testosterone enanthate; Testosterone 17 beta-cypionate
• Other Study ID Numbers: 1R01NS095813-01 (U.S. NIH Grant/Contract)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No