Gene Therapy Clinical Research

Investigators at the Jerry R. Mendell Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children's Hospital are currently conducting numerous clinical research studies, described in detail below. The Neurosciences Center at Nationwide Children's offers patients and families a comprehensive approach to the care for children and adolescents with disorders of the brain, spine and nervous system; from initial diagnosis and treatment to rehabilitation and long-term follow-up care.

A child with Duchenne muscular dystrophy sitting on the floor holding dinosaur figurines while being treated at Nationwide Children's.

FDA Approves First Gene Therapy Treatment for SMA

In a landmark moment for the Abigail Wexner Research Institute at Nationwide Children's, the Food and Drug Administration (FDA) approved Zolgensma® for spinal muscular atrophy (SMA) following decades of research in its Jerry R. Mendell Center for Gene Therapy to help patients with neuromuscular diseases. The FDA approved the treatment for pediatric patients less than 2 years of age with spinal muscular atrophy, including those who are pre-symptomatic at diagnosis. Jerry Mendell, MD, principal investigator emeritus in the Mendell Center, led the Phase 1 clinical trial that was the first to study gene therapy for spinal muscular atrophy type 1 (SMA1), the most severe form of SMA.

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Gene Therapy Clinical Studies

Studies Now Enrolling Participants

Open-Label Study Administering One Time Gene Therapy to Patients With Weakened Hearts With Duchenne Muscular Dystrophy (Sardocor Corp.)

This research study is testing whether an experimental drug is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD).

For Boys Aged 1-30 Months With Duchenne Muscular Dystrophy Taking Oral Liquid Prednisolone Once Weekly

This research study's goal to determine if a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.

Observational Study for Patients with LAMA2 Aged 0-5 years old

The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time.

A Study To Determine Why Children With SMA and DMD Are Diagnosed Late and Start Treatment Late

The purpose of this study is to determine which socioeconomic, system and health literacy factors play a role in time to diagnosis and treatment.

A Research Study To Find Out if Giving the IGHMBP2 Gene Back to Individuals in Their Spinal Fluid is Safe

The purpose of this study is to evaluate the safety and effectiveness of the gene therapy.

One Time Gene Therapy Infusion for Patients With LGMD2D/R3 (Sarepta)

The purpose of this study is to assess the safety of SRP-9004.

A Study of Patients With Dystrophinopathy (Duchenne and Becker Muscular Dystrophy) That Seeks to Understand How Different Mutations in the Dystrophin Gene Affect Disease Severity, and How Other Genes Besides the Dystrophin Gene May Influence Progression of Disease

The purpose of this study is to understand how different mutations in the dystrophin gene affect disease severity, and how other genes besides the dystrophin gene may influence progression of dystrophinopathy (Duchenne and Becker muscular dystrophy.

A One-time Infusion of a Adeno-associated Virus (AAV) Vector Carrying a Microdystrophin Gene as a Therapy for Boys with Duchenne Muscular Dystrophy Aged 4 Through 11 Years (Solid Biosciences Inc.)

The purpose of this study is to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of the microdystrophin gene therapy SGT-003 in participants with Duchenne muscular dystrophy.

Evaluation of Muscle, Skin and Other Tissues for the Study of Neuromuscular and Neurologic Diseases

The purpose of this study is to create a collection of muscle, skin and other cells to use as tools in seeking to identify the causes (and potential treatments) of neuromuscular and neurologic diseases.

Active, Not Recruiting Studies

An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects with Duchenne Muscular Dystrophy (ENDEAVOR)

The purpose of this study is to evaluate the safety and protein expression in the muscle from delandistrogene moxeparvovec (ELEVIDYS) infusion in participants with Duchenne muscular dystrophy.

A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects with LGMD2E (β-Sarcoglycan Deficiency)

This study is the first in human, single center, open-label gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E. 

A Multicenter, Open-label, Single-dose, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 on Subjects with Limb Girdle Muscular Dystrophy, Type 2E/R4 (β-Sarcoglycan Deficiency)

The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).

A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects with Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)

This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).

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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.