Frequently Asked Questions

Get answers to frequently asked questions about ASCEND.

ASCEND is a study being conducted to test the safety of INS1201 in 2-, 3-, and 4-year-old ambulatory boys with genetically confirmed 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.

ASCEND Phase I will test the safety of INS1201 in 2-, 3-, and 4-year-old ambulatory boys with genetically confirmed DMD and the effects of INS1201 on their muscle.

To participate in this study, your child must meet these and other criteria:

  • Male at birth
  • 2 years to <5 years of age
  • Can walk unassisted for at least 10 meters
  • Definitive diagnosis of DMD prior to screening based on genetic testing, with mutations in DMD gene between or including exons 18 to 58
  • Can cooperate and perform motor assessments

All other study requirements will be explained in detail by the study staff.

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 will investigate the safety of INS1201 using this administration and the resulting effect on the targeted muscles.

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.

Visit clinicaltrials.gov

Get an Overview of ASCEND

Get details about the ASCEND study of investigational treatment INS1201 in 2-, 3-, and 4-year-old ambulatory boys with genetically confirmed Duchenne muscular dystrophy.

Study Overview

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.