In the Phase I study called STARFISH, researchers at the University of Rochester evaluated the safety and potential efficacy of a combination treatment of recombinant human growth hormone (rHGH) and testosterone in 20 adult men with facioscapulohumeral muscular dystrophy (FSHD).
- The 19 participants who completed the study did not experience any serious adverse effects.
- After 24 weeks of treatment, the walking distance in the 6-minute walk test increased by more than 37 metres.
- Lean body mass increased by 2.21 kilograms, while fat mass decreased by 1.3 kilograms.
- Overall strength increased by 3%.
- The burden of disease, as measured by the FSHD Health Index, was also reduced.
Larger-scale randomised, double-blind, placebo-controlled trials will be needed to further investigate this promising therapeutic approach.
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ClinicalTrials.gov :Â NCT03123913
