A Research Study To Find Out if Giving the IGHMBP2 Gene Back to Individuals in Their Spinal Fluid is Safe
Phase: Recruiting by Invitation
First Posted: March
Condition(s): IGHMBP2 Related Disease (SMARD1, CMT-2S)
NCT Number: NCT05152823 Other Study ID Number(s): STUDY00002143
What Is the Purpose of This Study?
To evaluate the safety and effectiveness of the gene therapy.
Who Can Take Part in This Study?
- Age 2 months or greater and less than 14 years of age
- Confirmation of two pathogenic variants in the IGHMBP2 gene
For a full list of eligibility requirements, visit https://clinicaltrials.gov/study/NCT05152823
What Will Happen During This Study?
- Single dose of gene therapy by intrathecal delivery
- Patients will be evaluated at screening visit, during gene transfer, and for return follow-up visits on Days 7, 14, 30, 60, 90, 180 and Months 12, 18, 24 and 36.
- Duration of the study is 36 months
Will We Be Paid for This Study?
No, participants will not be compensated for their time.
Principal Investigator
Discover More Research
Explore additional studies relating to gene therapyYour Might Also Be Interested In
Spinal Muscular Atrophy
Spinal muscular atrophies (SMA) are a group of genetic (passed down by parents) diseases that affect motor neurons (nerve cells) in the spinal cord, causing the weakening of voluntary muscles (muscles that you control).
Research and Clinical Care: How They Work Together to Treat Children
In this 700 Children's blog post for parents, Nationwide Children's highlights how clinical research and clinical care work together as a key part of our strategic plan to achieve the best outcomes for children. Before delving into these elements work together, the post covers the basics of clinical research.
Stay Updated With Clinical Research. Follow @NCHClinicalResearch on Instagram!
Parents and caregivers can learn more about clinical research on our Instagram channel, @NCHClinicalResearch. You can be part of the clinical research that moves us forward in our journey to best outcomes for all children, everywhere.