Phase 1
N=20
Study of Testosterone and rHGH in FSHD
Facioscapulohumeral Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT03123913 ↗Enrolled (actual)
20
Serious AEs
0.0%
Results posted
May 2023
Primary outcome: Primary: Number of Participants Who Experienced an AE — 14 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Testosterone Enanthate (Drug); Somatropin (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- University of Rochester
- Primary completion
- Mar 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Experienced an AE |
14 | — |
| SECONDARY Mean Change in Level of Free Testosterone in the Blood |
17.42 | — |
| SECONDARY Mean Change in Level of Total Testosterone in the Blood |
8.26 | — |
| SECONDARY Mean Change in Level of IGF-1 in the Blood |
89.79 | — |
| SECONDARY Mean Change in Level of TSH in the Blood |
0.37 | — |
| SECONDARY Mean Change in Level of Luteinizing Hormone in the Blood |
-3.48 | — |
| SECONDARY Mean Change in Level of FSH in the Blood |
-3.29 | — |
| SECONDARY Mean Change in Total Lean Body Mass |
2.21 | — |
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.
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 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.
Data sourced from ClinicalTrials.gov (NCT03123913). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.