Study of Testosterone and rHGH in FSHD

NCT ID: NCT03123913

Last Updated: 2023-05-16

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-18

Study Completion Date

2022-03-28

Brief Summary

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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

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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.

Conditions

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Facioscapulohumeral Muscular Dystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Combination therapy

Testosterone Enanthate and Somatropin

Group Type EXPERIMENTAL

Testosterone Enanthate

Intervention Type DRUG

Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks.

Somatropin

Intervention Type DRUG

Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.

Interventions

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Testosterone Enanthate

Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks.

Intervention Type DRUG

Somatropin

Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.

Intervention Type DRUG

Other Intervention Names

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Delatestryl Tesostroval Testro LA Andro LA Durathate Everone Testrin Andropository Testosterone heptanoate Genotropin Humatrope Norditropin Nutropin Serostim Zorbtive

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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University of Rochester

OTHER

Sponsor Role lead

Responsible Party

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Chad Heatwole

Professor of Neurology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Chad R Heatwole, MD, MS-CI

Role: PRINCIPAL_INVESTIGATOR

University of Rochester

Locations

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University of Rochester

Rochester, New York, United States

Site Status

Countries

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United States

References

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Heatwole CR, Luebbe E, Hamel J, Mongiovi PC, Ciafaloni E, Dilek N, Martens WB, Weber DR, Rashid H, Allen McKeown J, Smith CH, Howell S, Rosero SZ, Eichinger K, Baker LS, Dekdebrun JM, Hilbert JE, Varma A, Thornton CA, McDermott MP, Moxley RT 3rd. Study of Testosterone and Recombinant Human Growth Hormone in Facioscapulohumeral Muscular Dystrophy. Neurol Genet. 2025 Aug 27;11(5):e200292. doi: 10.1212/NXG.0000000000200292. eCollection 2025 Oct.

Reference Type DERIVED
PMID: 40900971 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

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1R01NS095813-01

Identifier Type: NIH

Identifier Source: org_study_id

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