Insmed was announced on the page published on ClinicalTrials.gov with the ID NCT06817382 that the phase 1 trials of INS1201 gene therapy developed for Duchenne Muscular Dystrophy will be initiated.
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INS1201 Gene Therapy Phase 1 Clinical Trial
The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.
What is Insmed INS1201 Gene Therapy?
INS1201 targets the central nervous system directly and is administered intrathecally, in contrast to Elevidys, which needs systemic intravenous injection. The safety and effectiveness issues with systemic gene treatments are intended to be addressed by this focused delivery strategy.
Intrathecal administration dramatically lowers the necessary dose by ten to fifty times, preventing systemic toxicity and off-target effects by concentrating the medication where it is needed. Despite obstacles like spinal cord injury, nerve damage, and the risk for infections, this might provide patients with a safer and possibly more successful option, especially in light of safety issues associated with the large viral vector dosages utilized in intravenous therapy.
INS1201 Clinical Trial – NCT06817382
Study Start (Estimated): 2025-05-31
Primary Completion (Estimated): 2028-01-31
Study Completion (Estimated): 2028-03-31
Number of Participants (Estimated): 12 (Male)
Locations: USA
Ages Eligible for Study: 2 Years to 4 Years (Child)
INS1201 Phase 1 Participation Criteria
People must fulfill the inclusion requirements in order to be eligible. If a person has another ailment or is undergoing treatment that could prevent them from receiving gene therapy, they might not be able to participate. The complete set of inclusion and exclusion criteria can be found by clicking on this study link – NCT06817382 –.
How Can I Contact with Insmed?
Name: Insmed Medical Information
Phone Number: 18444467633
Email: [email protected]
Learn More: Cures of Duchenne (List of All Researches)
