Study of Testosterone and rHGH in FSHD

Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03123913

Acronym

STARFISH

Enrollment

Registered

2017-04-21

Start date

2017-12-18

Completion date

2022-03-28

Last updated

2023-05-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Facioscapulohumeral Muscular Dystrophy

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.

Interventions

DRUGSomatropin

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

DRUGTestosterone Enanthate

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

MALE

Age

18 Years to 65 Years

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.

Design outcomes

Primary

Measure	Time frame	Description
Number of Participants Who Experienced an AE	36 weeks	AEs were collected through patient report, interval laboratory studies, resting echocardiograms, dual energy x-ray absorptiometry (DEXA) studies, and physical examinations.

Secondary

Measure	Time frame
Mean Change in Level of Total Testosterone in the Blood	Baseline to 24 weeks
Mean Change in Level of IGF-1 in the Blood	Baseline to 24 weeks
Mean Change in Level of TSH in the Blood	Baseline to 24 weeks
Mean Change in Level of Free Testosterone in the Blood	Baseline to 24 weeks
Mean Change in Level of FSH in the Blood	Baseline to 24 weeks
Mean Change in Total Lean Body Mass	Baseline to 24 weeks
Mean Change in Level of Luteinizing Hormone in the Blood	Baseline to 24 weeks

Countries

United States

Participant flow

Participants by arm

Arm	Count
Combination Therapy Testosterone Enanthate and Somatropin Testosterone Enanthate: Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. Somatropin: Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.	20
Total	20

Baseline characteristics

Characteristic	Combination Therapy
Age, Continuous	41 years STANDARD_DEVIATION 10
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	20 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	0 Participants
Race (NIH/OMB) Black or African American	0 Participants
Race (NIH/OMB) More than one race	1 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) White	19 Participants
Region of Enrollment United States	20 participants
Sex: Female, Male Female	0 Participants
Sex: Female, Male Male	20 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 20
other Total, other adverse events	20 / 20
serious Total, serious adverse events	0 / 20

Outcome results

Primary

Number of Participants Who Experienced an AE

AEs were collected through patient report, interval laboratory studies, resting echocardiograms, dual energy x-ray absorptiometry (DEXA) studies, and physical examinations.

Time frame: 36 weeks

Arm	Measure	Value (COUNT_OF_PARTICIPANTS)
Combination Therapy	Number of Participants Who Experienced an AE	14 Participants

Secondary