Phase I study suggests safety and efficacy of growth hormone and testosterone combination therapy in FSHD

In the Phase IAu cours d’un essai clinique de phase I un médicament dont l’intérêt thérapeutique a été montré sur des modèles animaux et/ou cellulaires (essais précliniques) est administré pour la première fois à un petit groupe de volontaires sains, plus rarement à des malades, afin d’évaluer leur tolérance à la substance en fonction de la dose (Comment le futur traitement est-il absorbé et éliminé ? Comment se fait sa répartition dans les organes ? Est-il toxique et à quelles doses ? Existe-t-il des effets secondaires ?). 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.

Study of Testosterone and Recombinant Human Growth Hormone in Facioscapulohumeral Muscular Dystrophy. Heatwole CR, Luebbe E, Hamel J et al. Neurol Genet. 2025 August 27. doi: 10.1212/NXG.0000000000200292.

 

ClinicalTrials.gov : NCT03123913