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Study of Testosterone and rHGH in FSHD (STARFISH)

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ClinicalTrials.gov Identifier: NCT03123913
Recruitment Status : Recruiting
First Posted : April 21, 2017
Last Update Posted : January 9, 2018
Information provided by (Responsible Party):
Chad Heatwole, 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.

Condition or disease Intervention/treatment Phase
Facioscapulohumeral Muscular Dystrophy Drug: Testosterone Enanthate Drug: Somatropin 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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study
Actual Study Start Date : December 18, 2017
Estimated Primary Completion Date : July 2020
Estimated Study Completion Date : July 2020

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

Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 36 weeks ]
    Lean body mass will be measured at study visits through dual energy x-ray absorptiometry (DEXA) studies.

Other Outcome Measures:
  1. Ambulation [ Time Frame: 36 weeks ]
    Ambulation will be assessed as an exploratory measure with the Six Minute Walk Test.

  2. Strength [ Time Frame: 36 weeks ]
    Strength will be assessed as an exploratory measure with manual muscle testing and quantitative muscle testing.

  3. Pulmonary Function [ Time Frame: 36 weeks ]
    Pulmonary function will be assessed as an exploratory measures with forced vital capacity testing.

  4. Patient-Reported Disease Burden [ Time Frame: 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.

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03123913

Contact: Elizabeth A Luebbe, MS 585-275-7867 elizabeth_luebbe@urmc.rochester.edu

United States, New York
University of Rochester Recruiting
Rochester, New York, United States, 14642
Contact: Chad Heatwole, MD         
Sponsors and Collaborators
University of Rochester
Principal Investigator: Chad R Heatwole, MD, MS-CI University of Rochester

Responsible Party: Chad Heatwole, Associate Professor of Neurology, University of Rochester
ClinicalTrials.gov Identifier: NCT03123913     History of Changes
Other Study ID Numbers: 1R01NS095813-01 ( U.S. NIH Grant/Contract )
First Posted: April 21, 2017    Key Record Dates
Last Update Posted: January 9, 2018
Last Verified: January 2018

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

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Facioscapulohumeral
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Testosterone enanthate
Testosterone undecanoate
Testosterone 17 beta-cypionate
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Anabolic Agents