ASCEND Study
Insmed Gene Therapy is conducting a study, titled ASCEND, to test the safety of INS1201 in 2-, 3-, and 4-year-old ambulatory boys with genetically confirmed Duchenne muscular dystrophy (DMD) and the effects of INS1201 on their muscle.
INS1201 is an investigational gene therapy designed to produce a short, but functional, form of the key muscle protein dystrophin that is missing or nonfunctional in children with DMD.
INS1201 is given as a one-time intrathecal (IT) injection of a fixed (not weight-based) dose into the spinal fluid to evaluate the efficiency of a lower amount of adeno-associated virus (AAV) on the ability to get muscle and heart tissue to produce micro-dystrophin. ASCEND Study will investigate the safety of INS1201 using this administration and the resulting effect on the targeted muscles.
ASCEND is a first-in-human experimental research study designed to test the safety of INS1201.
The study will include boys aged 2 to <5 years
INS1201 is given as one-time IT injection into the fluid-filled space around the spinal cord
The effect of INS1201 will be assessed from needle biopsy data and physical function tests
The study will last approximately 2 years with an extended follow-up period totaling 5 years
How to Get Involved
ASCEND is open and now recruiting.
For information about eligibility requirements and locations, go to clinicaltrials.gov.
Study-related support services are available for enrolled families. A support services provider can help with logistics, including lodging and travel.
Learn More
About ASCEND
To learn more about the ASCEND study and the eligibility requirements, please contact:
Insmed Medical Information
[email protected]
Find Out What
to Expect
See what ASCEND Study participants may expect and learn what is required when taking part of this clinical study.
Explore Frequently
Asked Questions
Get answers to frequently asked questions about ASCEND.
As with any clinical study, the participants’ legally authored representatives have the right to withdraw the participant from ASCEND at any time and for any reason without bias to their future medical care by the Investigator or at the institution.