welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
Santhera Completes Enrollment Of Phase 3 SIDEROS Study With Puldysa® (Idebenone) In Duchenne Muscular Dystrophy (DMD)
Clinical Trials and Research

Santhera Completes Enrollment of Phase 3 SIDEROS Study With Puldysa® (Idebenone) in Duchenne Muscular Dystrophy (DMD)

Santhera Pharmaceuticals announces full recruitment of its Phase 3 SIDEROS study with idebenone in Duchenne muscular dystrophy (DMD). The sample-size and variability re-assessment performed according to study protocol demonstrated that with the currently enrolled patients the study has a very high power (>99%). Given the strong powering of SIDEROS, the Company is now assessing the potential of conducting an interim analysis to test for overwhelming efficacy with a view of completing the trial early.

With patient recruitment into the 18-month international SIDEROS trial in its final stages, Santhera performed the planned sample size and variability re-assessment in accordance with the study protocol to confirm adequate study power. This blinded analysis showed that variability is lower than anticipated per protocol and, with the current number of enrolled patients, the SIDEROS study has a very high power of over 99% to detect a treatment difference.

May 20, 2020
Capricor Announces Positive Top-Line Final Results From HOPE-2 Study In Patients With Duchenne Muscular Dystrophy Treated With Lead Candidate CAP-1002
Clinical Trials and Research

Capricor Announces Positive Top-Line Final Results from HOPE-2 Study in Patients With Duchenne Muscular Dystrophy Treated With Lead Candidate CAP-1002

Capricor Therapeutics, a clinical-stage biotechnology company focused on the development of first-in-class biological therapeutics for the treatment and prevention of diseases, announced today positive top-line 12-month results of the HOPE-2 clinical trial using CAP-1002 to treat patients in advanced stages of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Boys and young men typically lose their ability to walk in their teens and generally die of cardiac or respiratory complications by the 3rd decade of life. The data showed improvements in upper limb, cardiac and respiratory function with p-values less than p=0.05 in multiple measures.

The 12-month data from HOPE-2 showed statistically meaningful improvements in the PUL 2.0 in CAP-1002 treated patients (p=0.05) with a mean change of 2.4 points over placebo patients. With the exception of steroids, preservation of function in DMD is uncommon.

 

May 13, 2020