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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Combination therapy | Experimental | Testosterone Enanthate and Somatropin |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Testosterone Enanthate | Drug | Testosterone enanthate in oil (140mg) delivered via intramuscular injections every 2 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 36 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change in Level of Free Testosterone in the Blood | Baseline to 24 weeks | |
| Mean Change in Level of Total Testosterone in the Blood | Baseline to 24 weeks | |
| Mean Change in Level of IGF-1 in the Blood |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Chad R Heatwole, MD, MS-CI | University of Rochester | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Rochester | Rochester | New York | 14642 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40900971 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Posted | Count of Participants | Participants | 36 weeks |
|
|
36 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Decreased energy | General disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chad Heatwole, MD, MS-CI | University of Rochester Medical Center | 585-275-7867 | dystrophy_registry@urmc.rochester.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | May 4, 2021 | May 12, 2023 | Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | May 4, 2021 | May 12, 2023 | ICF_001.pdf |
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| ID | Term |
|---|---|
| D020391 | Muscular Dystrophy, Facioscapulohumeral |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| C004648 | testosterone enanthate |
| D043343 | Testosterone Propionate |
| D019382 | Human Growth Hormone |
| D013006 | Growth Hormone |
| ID | Term |
|---|---|
| D013739 | Testosterone |
| D000737 | Androstenols |
| D000736 | Androstenes |
| D000731 | Androstanes |
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| Somatropin | Drug | Genotropin (5.0 μg/kg/day) delivered via subcutaneous injections. |
|
|
| Baseline to 24 weeks |
| Mean Change in Level of TSH in the Blood | Baseline to 24 weeks |
| Mean Change in Level of Luteinizing Hormone 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 |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| Secondary | Mean Change in Level of Free Testosterone in the Blood | Posted | Mean | Standard Deviation | pg/mL | Baseline to 24 weeks |
|
|
|
| Secondary | Mean Change in Level of Total Testosterone in the Blood | Posted | Mean | Standard Deviation | ng/dL | Baseline to 24 weeks |
|
|
|
| Secondary | Mean Change in Level of IGF-1 in the Blood | Posted | Mean | Standard Deviation | ng/mL | Baseline to 24 weeks |
|
|
|
| Secondary | Mean Change in Level of TSH in the Blood | Posted | Mean | Standard Deviation | uIU/mL | Baseline to 24 weeks |
|
|
|
| Secondary | Mean Change in Level of Luteinizing Hormone in the Blood | Posted | Mean | Standard Deviation | mIU/mL | Baseline to 24 weeks |
|
|
|
| Secondary | Mean Change in Level of FSH in the Blood | Posted | Mean | Standard Deviation | mIU/mL | Baseline to 24 weeks |
|
|
|
| Secondary | Mean Change in Total Lean Body Mass | Posted | Mean | Standard Deviation | Kg | Baseline to 24 weeks |
|
|
|
| 0 |
| 20 |
| 0 |
| 20 |
| 20 |
| 20 |
| Exercise fatigue | General disorders | Non-systematic Assessment |
|
| Soreness | General disorders | Non-systematic Assessment |
|
| Increased appetite | General disorders | Non-systematic Assessment |
|
| Itchiness | General disorders | Non-systematic Assessment |
|
| Poison ivy | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Vivid dreams | Psychiatric disorders | Non-systematic Assessment |
|
| Elevated IGF-1 | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Elevated testosterone | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Elevated HbA1c | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Elevated insulin | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Elevated HCT | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Elevated glucose | Blood and lymphatic system disorders | Non-systematic Assessment |
|
| Glycosuria | Renal and urinary disorders | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Parathesia | Nervous system disorders | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Edema limbs | General disorders | Non-systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | Non-systematic Assessment |
|
| Fatigue | General disorders | Non-systematic Assessment |
|
| Flu-like symptoms | General disorders | Non-systematic Assessment |
|
| Headache | Nervous system disorders | Non-systematic Assessment |
|
| Injection site reaction | General disorders | Non-systematic Assessment |
|
| Irritability | Psychiatric disorders | Non-systematic Assessment |
|
| Muscle cramp | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Pain | General disorders | Non-systematic Assessment |
|
| Palpitations | Cardiac disorders | Non-systematic Assessment |
|
| Sore throat | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Urine discoloration | Renal and urinary disorders | Non-systematic Assessment |
|
| Weight gain | Investigations | Non-systematic Assessment |
|
| White patches on tongue | General disorders | Non-systematic Assessment |
|
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| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013256 |
| Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D045165 | Testosterone Congeners |
| D012739 | Gonadal Steroid Hormones |
| D042341 | Gonadal Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |