Long-term OLE Study of Casimersen or Golodirsen for Patients with DMD
Research type
Research Study
Full title
Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
IRAS ID
247338
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
PPDi
Eudract number
2017-004625-32
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
This is a long-term, open-label extension study for patients with Duchenne Muscular Dystrophy (DMD) enrolled in clinical trials with casimersen or golodirsen. DMD is a rare, life-threatening, degenerative neuromuscular disease caused by mutations in the gene for dystrophin, a protein that protects muscles from injury.
Existing interventions for patients with DMD are largely supportive in nature and include muscle-stretching exercises, tendon-release surgery, and eventual wheelchair use and assisted breathing. Current pharmacologic treatments, such as glucocorticoids, focus on alleviation of symptoms, but do not address the underlying cause of the disease. The benefits of these treatments are usually temporary, and their use is limited by numerous side effects.
Casimersen and golodirsen are designed to cause cells to ‘skip’ over sections of DNA that code for the dystrophin gene (exons 45 and 53 respectively). Mutations in these regions are found in approximately 8% of patients with DMD, so ‘skipping’ them allows a shorter but functional version of the protein to be produced. Although no clinical study can guarantee an improvement in efficacy outcome measures, parent study findings indicate that continued long-term treatment with casimersen and golodirsen is expected to attenuate the decline in physical functioning that typically occurs with DMD.
An extension study will allow the continued treatment of the patients with either casimersen or golodirsen and the long-term assessment of efficacy and safety endpoints. Patients who have completed the parent studies 4045-101, 404-301, or 4053-101 will be eligible to participate in the extension study. Adverse events, concomitant medications, clinical laboratory tests, physical examinations, 12-lead electrocardiograms, vital signs, and functional assessments (6-minute walk test, North Star Ambulatory Assessment Scale, performance of the upper limb, loss of ambulation, and pulmonary function tests) will be collected to assess safety and efficacy throughout the trial.
Lay summary:
not currently available.REC name
London - Riverside Research Ethics Committee
REC reference
18/LO/1125
Date of REC Opinion
23 Jul 2018
REC opinion
Further Information Favourable Opinion