Clinical Trial Alert: Stealth BioTherapeutics Seeks Participants for a Phase 3 Mitochondrial Myopathy Trial

Stealth BioTherapeutics is looking for participants with primary mitochondrial myopathy (PMM) to participate in the MMPOWER-3 Phase 3 trial. This trial is designed to evaluate the efficacy and safety of daily subcutaneous injections of elamipretide, an investigational drug that associates with cardiolipin, a key structural component of the inner mitochondrial membrane, and has shown to . . .

Read More

Clinical Trial Alert: Capricor Seeks Participants for a Phase 2 DMD Study

Researchers at Capricor Therapeutics are looking for ambulatory and non-ambulatory children and adults with Duchenne muscular dystrophy (DMD) to participate in a Phase 2 study. The goal of the study is to evaluate the safety and efficacy of CAP-1002. Researchers hope that this therapy may slow disease progression in heart and skeletal muscle by modulating . . .

Read More

Clinical Trial Alert: Orion Pharma Seeks Participants for a Phase 3 ALS Study

Researchers at Orion Pharma are looking for participants with ALS (amyotrophic lateral sclerosis) to participate in the REFALS Phase 3 study. The study is designed to help researchers evaluate the effects of oral levosimendan (ODM-109) on respiratory function in patients with ALS. Participants will be randomized to receive either levosimendan or placebo to determine whether . . .

Read More

Summit Therapeutics Discontinues ezutromid for DMD after Disappointing Phase 2 Trial Results

This morning, Summit Therapeutics announced that it is discontinuing development of ezutromid for Duchenne muscular dystrophy after primary and secondary endpoints were missed after 48 weeks of treatment in their PhaseOut DMD trial. PhaseOut DMD was a phase 2, multi-center, open-label clinical trial of ezutromid, a utrophin-modulation therapy. Thirty-eight boys enrolled in the trial completed the 48-week regimen. The . . .

Read More

Sarepta Reports Positive Preliminary Findings in DMD Gene Therapy Trial

This week, Sarepta Therapeutics announced encouraging preliminary results from a phase 1/2a gene therapy trial designed to assess the investigational drug AAVrh74.MHCK7.micro-Dystrophin in boys with Duchenne muscular dystrophy (DMD). After 90 days in the trial, the first three participants all showed robust expression of micro-dystrophin — a shortened version of the protein that is absent . . .

Read More

Trial to Test Firdapse in MG Seeks Participants

Researchers are looking for people with myasthenia gravis (MG) to participate in a phase 3 clinical trial to evaluate the safety and effectiveness of the investigational drug amifampridine phosphate (brand name Firdapse). Effects of the drug will be assessed in individuals with muscle-specific kinase (MuSK) antibody positive MG and in a smaller group with acetylcholine . . .

Read More

Trial to Test Drug Targeting Muscle Cramps in CMT Seeks Participants

Researchers are looking for people with Charcot-Marie-Tooth disease (CMT) to participate in a phase 2 clinical trial to evaluate the safety and effectiveness of the investigational drug FLX-787-ODT. FLX-787-ODT, under development by Flex Pharma, is taken in tablet form and is designed to reduce muscle cramps in adults with CMT. Trial length is approximately three . . .

Read More

Trial to Test MG Drug UCB7655 Seeks Participants

Researchers are looking for people with myasthenia gravis (MG) to participate in a multinational phase 2A clinical trial to evaluate the safety, tolerability and effectiveness of the investigational drug UCB7665. UCB7665, under development by UCB Pharma, is administered via subcutaneous (under-the-skin) injection and is designed to reduce the severity of muscle weakness and fatigue in . . .

Read More

Santhera Seeks Participants for DMD Study, Makes Available EAP and Online DMD Resource

With a focus on respiratory care in Duchenne muscular dystrophy (DMD), Santhera Pharmaceuticals has made available new resources for individuals with the disease. In addition, the company is seeking individuals to participate in a phase 3 clinical trial to test a respiratory drug in people with DMD. SIDEROS trial seeks participants Researchers are looking for . . .

Read More