Facioscapulohumeral Muscular DystrophyPhase 1STARFISHNCT03123913
Study of Testosterone and rHGH in FSHD
A Phase 1 study testing Testosterone Enanthate and Somatropin in people with Facioscapulohumeral Muscular Dystrophy, run by University of Rochester at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy
- What is being tested?
- Testosterone Enanthate and Somatropin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Dec 2017to 28 Mar 2022
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
Show all 17Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study
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.
Read the 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.
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Being tested
- Testosterone Enanthate, Somatropin
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Testosterone EnanthateDrug
Also called Delatestryl, Tesostroval, Testro LA, Andro LA
Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks.
SomatropinDrug
Also called Genotropin, Humatrope, Norditropin, Nutropin
Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections.
| Study group | Role | What they receive |
|---|---|---|
| Combination therapy | Experimental | Testosterone Enanthate and Somatropin Receives: Drug: Testosterone Enanthate; Drug: Somatropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 36 weeks
Secondary outcomes (7)
Mean Change in Level of Free Testosterone in the Blood
Measured: Baseline to 24 weeks
Mean Change in Level of Total Testosterone in the Blood
Measured: Baseline to 24 weeks
Mean Change in Level of IGF-1 in the Blood
Measured: Baseline to 24 weeks
Mean Change in Level of TSH in the Blood
Measured: Baseline to 24 weeks
Mean Change in Level of Luteinizing Hormone in the Blood
Measured: Baseline to 24 weeks
Mean Change in Level of FSH in the Blood
Measured: Baseline to 24 weeks
Mean Change in Total Lean Body Mass
Measured: Baseline to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2017
- Study start18 Dec 2017
- Primary completion28 Mar 2022
- Study completion28 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Facioscapulohumeral Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of RochesterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Rochester
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chad Heatwole, University of Rochester
- Investigators
- Chad R Heatwole, MD, MS-CI, Principal investigator, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03123913
- Sponsor's protocol ID
- 1R01NS095813-01
- Acronym
- STARFISH
- Status last verified
- May 2023
- First submitted
- 12 Apr 2017
- Last updated
- 16 May 2023
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 40900971
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study
- ClinicalTrials.gov ID
- NCT03123913
- Acronym / sponsor ID
- STARFISH / 1R01NS095813-01
- Conditions
- Facioscapulohumeral Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University of RochesterUniversity, hospital or other
- Study start
- 18 Dec 2017 (actual)
- Primary completion
- 28 Mar 2022 (actual)
- Study completion
- 28 Mar 2022 (actual)
- First posted
- 21 Apr 2017 (actual)
- Results
- Posted 16 May 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Combination therapy | Experimental | Drug: Testosterone Enanthate, Drug: SomatropinTestosterone Enanthate and Somatropin |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (17 listed)
- Diabetes
- Obesity (BMI>35 kg/m2)
- Cardiovascular disease (heart failure, coronary artery disease, uncontrolled hypertension, untreated hypercholesterolemia)
- Untreated thyroid disease
- Deep vein thrombosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Testosterone EnanthateAlso: Delatestryl, Tesostroval, Testro LA, Andro LATestosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. | Drug | Combination therapy |
| SomatropinAlso: Genotropin, Humatrope, Norditropin, NutropinGenotropin (5.0 μg/kg/day) delivered via subcutaneous injections. | Drug | Combination therapy |
Primary outcome
Number of Participants Who Experienced an AE
Time frame: 36 weeks
AEs were collected through patient report, interval laboratory studies, resting echocardiograms, dual energy x-ray absorptiometry (DEXA) studies, and physical examinations.
7 secondary outcomes
Mean Change in Level of Free Testosterone in the Blood
Time frame: Baseline to 24 weeks
Mean Change in Level of Total Testosterone in the Blood
Time frame: Baseline to 24 weeks
Mean Change in Level of IGF-1 in the Blood
Time frame: Baseline to 24 weeks
Mean Change in Level of TSH in the Blood
Time frame: Baseline to 24 weeks
Mean Change in Level of Luteinizing Hormone in the Blood
Time frame: Baseline to 24 weeks
Mean Change in Level of FSH in the Blood
Time frame: Baseline to 24 weeks
Mean Change in Total Lean Body Mass
Time frame: Baseline to 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40900971Heatwole 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chad R Heatwole, MD, MS-CI principal investigator
University of Rochester
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Testosterone Enanthate and Somatropin in people with Facioscapulohumeral Muscular Dystrophy, run by University of Rochester at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 May 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Experienced an AE (time frame: 36 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03123913, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2023.