STARFISH: Study of Testosterone and rHGH in FSHD

Sponsor

University of Rochester (Other)

Overall Status

Completed

CT.gov ID

NCT03123913

Collaborator

(none)

Enrollment

Location

Arm

50.4

Actual Duration (Months)

0.4

Patients Per Site Per Month

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
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 Design

Study Type:

Interventional

Actual Enrollment :

20 participants

Allocation:

N/A

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 :

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

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

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

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.
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

Ambulation [36 weeks]
Ambulation will be assessed as an exploratory measure with the Six Minute Walk Test.
Strength [36 weeks]
Strength will be assessed as an exploratory measure with manual muscle testing and quantitative muscle testing.
Pulmonary Function [36 weeks]
Pulmonary function will be assessed as an exploratory measures with forced vital capacity testing.
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

Criteria

Ages Eligible for Study:

18 Years to 65 Years

Sexes Eligible for Study:

Male

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.