Clinical trial NCT03123913: Study of Testosterone and rHGH in FSHD
Official title: Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Facioscapulohumeral Muscular Dystrophy
Lead sponsor: University of Rochester
Sponsor class: OTHER
First posted: 2017-04-21
Enrollment: 20
Sex: MALE
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants Who Experienced an AE
timeFrame: 36 weeks
Secondary outcomes:
measure: Mean Change in Level of Free Testosterone in the Blood
timeFrame: Baseline to 24 weeks
measure: Mean Change in Level of Total Testosterone in the Blood
timeFrame: Baseline to 24 weeks
measure: Mean Change in Level of IGF-1 in the Blood
timeFrame: Baseline to 24 weeks
measure: Mean Change in Level of TSH in the Blood
timeFrame: Baseline to 24 weeks
measure: Mean Change in Level of Luteinizing Hormone in the Blood
timeFrame: Baseline to 24 weeks
measure: Mean Change in Level of FSH in the Blood
timeFrame: Baseline to 24 weeks
measure: Mean Change in Total Lean Body Mass
timeFrame: Baseline to 24 weeks
Other outcomes:
Locations:
city: Rochester
country: United States
facility: University of Rochester
state: New York
Study officials:
affiliation: University of Rochester
name: Chad R Heatwole, MD, MS-CI
role: PRINCIPAL_INVESTIGATOR
References: