All results of clinical trials generate new knowledge, and helps to advance the development of treatments. This includes the sharing of so-called negative results: study results that do not meet certain endpoints, or where efficacy is not seen. However, publishing negative data is important, as we can learn from this and not make the same mistakes in the future.
For this reason, the World Duchenne Organization is glad to hear that Roche shared two lay summaries on their recently terminated studies. This concerns two clinical studies for an investigational medicine called taldefgrobep alfa (RG6206) in boys with Duchenne Muscular Dystrophy (DMD).
Click on the links below to access the lay summary to the studies:
These summaries were written by Roche after the studies ended. They are intended for members of the public and people who took part in the study.
About the studies
Both studies were not completed in full. A pre-planned analysis of the initial results of the SPITFIRE study – a study of the same investigational drug as in the THUNDERJET one – showed that taldefgrobep alfa (RG6206) was not as effective as had been hoped. It was well tolerated, however. As a result, THUNDERJET was stopped early.
All studies contribute to the better understanding of diseases. Despite the fact that the THUNDERJET study was stopped early, it has provided valuable information about DMD and how potential medicines may work in DMD patients.
About SPITFIRE and THUNDERJET
SPITFIRE was a clinical trial to establish the effectiveness and safety of an investigational medicine called taldefgrobep alfa (RG6206 – an anti-myostatin adnectin) in boys with Duchenne Muscular Dystrophy (DMD).
THUNDERJET was a clinical trial to establish the safety, tolerability and pharmacokinetics (how a medicine works in the body) of different doses of an investigational medicine called taldefgrobep alfa (RG6206 – an anti-myostatin adnectin) in boys with Duchenne Muscular Dystrophy (DMD).