Managed access to del-zota for Duchenne muscular dystrophy with exon 44 skipping
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This program provides access to an investigational medicine called del-zota (also known as AOC 1044) for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by exon 44 skipping. The medicine aims to help the body produce a shorter but functional version of the dystrophin protein, which may slow muscle damage and improve function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a type of Duchenne muscular dystrophy (DMD) that can be treated with exon 44 skipping. If you're not sure, ask your doctor or contact the study team.
- If you are new to this program (not joining from a previous study), you must live in the US and have a US primary health care provider.
- You must be 2 years old or older when you agree to take part.
- If you previously had gene therapy for DMD, it must have been more than 12 months ago (with the immunosuppressive treatment), and you did not have a satisfactory response.
- You cannot have serious breathing or heart problems, or be near the end of life, and you must not have had treatment with another investigational drug recently.
- If you already completed the EXPLORE44-OLE study, you can join if you had no major tolerability issues with AOC 1044 and you don't have any new health conditions that would affect safety.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study tests whether a new medicine called AOC 1044 (delpacibart zotadirsen) can help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The medicine works by helping the body skip a damaged part of the gene to make a functional protein.
This study tests whether a new medicine called zeleciment rostudirsen can help ambulatory (walking) males with Duchenne muscular dystrophy (DMD). The medicine is given as an intravenous infusion (through a vein) every 4 weeks and works by helping the body produce a protective muscle protein.
This trial tests a new medicine called ENTR-601-44 for people with Duchenne muscular dystrophy whose genetic mutation can be treated by skipping a part of the gene called exon 44. The goal is to see if it is safe and helps with muscle function.
This study tests a gene therapy that aims to help muscles produce a protein they are missing. It is for people with Duchenne muscular dystrophy who are no longer able to walk, and it may help slow the progression of the disease.
This trial tests an experimental drug called ENTR-601-45 for people with Duchenne muscular dystrophy that can be treated with 'exon 45 skipping.' The goal is to see if it's safe and helps improve muscle function. It's for males between 4 and 20 who can still walk and have enough arm strength.
This trial tests a new cell-based treatment for Duchenne muscular dystrophy in adults who can no longer walk. The goal is to see if the therapy can help improve muscle function.
Hear when a new Muscular Disorders, Atrophic trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.