Neurological Disorders Clinical Trials

Full title:

ASCEND: A Phase 1, Multicenter, Open-label Study to Investigate the Safety and Biodistribution of a Single Intrathecal Injection of INS1201 in Ambulatory Males with Duchenne Muscular Dystrophy

Study goal:

To study INS1201 gene therapy for Duchenne muscular dystrophy (DMD)

Diagnosis:

Duchenne muscular dystrophy

Age:

2–4 years

Study goal:

To learn more about participants' beliefs, attitudes, and questions about gene therapy to help make web-based resources to share information. This could help patients make treatment decisions.

Diagnosis:

Neurological disorders, sickle cell disease, hemophilia, inherited genetic diseases

Age:

18 to 35 years old with rare genetic diseases

Full title:

 Inherited Neuropathies Consortium Rare Disease Clinical Research Network

Study goal:

This long-term study aims to better understand the genetic causes of Charcot-Marie-Tooth disease (CMT) and to learn how different forms of the rare hereditary disease affect people over time.

Diagnosis:

Charcot-Marie-Tooth Disease

Age:

Birth to 22 years

Full title:

Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients with Spinal Muscular Atrophy Treated with SMN-Directed Therapies

Study goal:

To study how different SMA drugs affect muscles and nerve cells and to study the use of MRI to track muscle changes.

Diagnosis:

Spinal muscular atrophy

Age:

5-20 years old

Full title:

NEUROBANK: Investigation of Neurological Disorders with Onset in Childhood

Study goal:

To better understand why neurological diseases start in childhood and how they develop over time

Diagnosis:

A genetic condition with neurological signs and symptoms in childhood; genetic or neurological or neurodevelopmental disorder (inherited); biological relative with a known or suspected genetic neurological or neurodevelopmental disorder

Age:

Any age

Full title:

TBANK: Protocol for Collecting, Banking, and Distributing Human Tissue Samples: St. Jude Children’s Research Hospital Biorepository

Study goal:

To provide a high-quality repository of tumor and normal samples to facilitate translational research performed by St Jude faculty and their collaborators

Diagnosis:

All diseases

Full title:

Friedreich’s Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI)

Study goal:

To collect data about how Friedreich’s ataxia progresses over time and affects the daily life of patients.

Diagnosis:

Friedreich’s ataxia

Age:

Any