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Study of Testosterone and rHGH in FSHD (STARFISH)

Primary Purpose

Facioscapulohumeral Muscular Dystrophy

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Testosterone Enanthate
Somatropin
Sponsored by
University of Rochester
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Facioscapulohumeral Muscular Dystrophy

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)MaleDoes not accept 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.

Sites / Locations

  • University of Rochester

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Combination therapy

Arm Description

Testosterone Enanthate and Somatropin

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Mean Change in Level of Free Testosterone in the Blood
Mean Change in Level of Total Testosterone in the Blood
Mean Change in Level of IGF-1 in the Blood
Mean Change in Level of TSH in the Blood
Mean Change in Level of Luteinizing Hormone in the Blood
Mean Change in Level of FSH in the Blood
Mean Change in Total Lean Body Mass

Full Information

First Posted
April 12, 2017
Last Updated
May 12, 2023
Sponsor
University of Rochester
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1. Study Identification

Unique Protocol Identification Number
NCT03123913
Brief Title
Study of Testosterone and rHGH in FSHD
Acronym
STARFISH
Official Title
Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
December 18, 2017 (Actual)
Primary Completion Date
March 28, 2022 (Actual)
Study Completion Date
March 28, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Rochester

4. Oversight

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

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Facioscapulohumeral Muscular Dystrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Combination therapy
Arm Type
Experimental
Arm Description
Testosterone Enanthate and Somatropin
Intervention Type
Drug
Intervention Name(s)
Testosterone Enanthate
Other Intervention Name(s)
Delatestryl, Tesostroval, Testro LA, Andro LA, Durathate, Everone, Testrin, Andropository, Testosterone heptanoate
Intervention Description
Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks.
Intervention Type
Drug
Intervention Name(s)
Somatropin
Other Intervention Name(s)
Genotropin, Humatrope, Norditropin, Nutropin, Serostim, Zorbtive
Intervention Description
Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
Primary Outcome Measure Information:
Title
Number of Participants Who Experienced an AE
Description
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
Secondary Outcome Measure Information:
Title
Mean Change in Level of Free Testosterone in the Blood
Time Frame
Baseline to 24 weeks
Title
Mean Change in Level of Total Testosterone in the Blood
Time Frame
Baseline to 24 weeks
Title
Mean Change in Level of IGF-1 in the Blood
Time Frame
Baseline to 24 weeks
Title
Mean Change in Level of TSH in the Blood
Time Frame
Baseline to 24 weeks
Title
Mean Change in Level of Luteinizing Hormone in the Blood
Time Frame
Baseline to 24 weeks
Title
Mean Change in Level of FSH in the Blood
Time Frame
Baseline to 24 weeks
Title
Mean Change in Total Lean Body Mass
Time Frame
Baseline to 24 weeks

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Chad R Heatwole, MD, MS-CI
Organizational Affiliation
University of Rochester
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Rochester
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States

12. IPD Sharing Statement

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Study of Testosterone and rHGH in FSHD

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