STARFISH: Study of Testosterone and rHGH in FSHD

University of Rochester (Other)
Overall Status
Completed ID

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 1

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 Design

Study Type:
Actual Enrollment :
20 participants
Intervention Model:
Single Group Assignment
None (Open Label)
Primary Purpose:
Official Title:
Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study
Actual Study Start Date :
Dec 18, 2017
Actual Primary Completion Date :
Feb 28, 2022
Actual Study Completion Date :
Feb 28, 2022

Arms and Interventions

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
  • Humatrope
  • Norditropin
  • Nutropin
  • Serostim
  • Zorbtive
  • Outcome Measures

    Primary Outcome Measures

    1. Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [36 weeks]

      Safety and tolerability are monitored by interval laboratory studies, resting echocardiograms, dual energy x-ray absorptiometry (DEXA) studies, and physical examinations. Patients will report any adverse events through in-person and telephone evaluations as well as on patient diaries.

    Secondary Outcome Measures

    1. Serum levels of free and total testosterone, IGF-1, thyroid function, luteinizing hormone and follicle stimulating hormone [36 weeks]

      Serum levels of free and total testosterone, IGF-1, thyroid function, luteinizing hormone and follicle stimulating hormone will be assessed at each study visit.

    2. Lean body mass [36 weeks]

      Lean body mass will be measured at study visits through dual energy x-ray absorptiometry (DEXA) studies.

    Other Outcome Measures

    1. Ambulation [36 weeks]

      Ambulation will be assessed as an exploratory measure with the Six Minute Walk Test.

    2. Strength [36 weeks]

      Strength will be assessed as an exploratory measure with manual muscle testing and quantitative muscle testing.

    3. Pulmonary Function [36 weeks]

      Pulmonary function will be assessed as an exploratory measures with forced vital capacity testing.

    4. Patient-Reported Disease Burden [36 weeks]

      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.

    Eligibility Criteria


    Ages Eligible for Study:
    18 Years to 65 Years
    Sexes Eligible for Study:
    Accepts 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.

    Contacts and Locations


    Site City State Country Postal Code
    1 University of Rochester Rochester New York United States 14642

    Sponsors and Collaborators

    • University of Rochester


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

    Study Documents (Full-Text)

    None provided.

    More Information


    None provided.
    Responsible Party:
    Chad Heatwole, Associate Professor of Neurology, University of Rochester Identifier:
    Other Study ID Numbers:
    • 1R01NS095813-01
    First Posted:
    Apr 21, 2017
    Last Update Posted:
    Jun 9, 2022
    Last Verified:
    Jun 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    Studies a U.S. FDA-regulated Device Product:
    Product Manufactured in and Exported from the U.S.:
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Jun 9, 2022