Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia

Early Phase 1

Withdrawn

• Conditions: Periventricular Leukomalacia; White Matter Disease; Hypoxic-Ischemic Encephalopathy; Demyelinating Diseases; Neonatal Encephalopathy

• Registration Number: NCT06985303
• Lead Sponsor: MGAM LLC

Brief Summary

The goal of this study is to explore a new treatment that may help repair brain damage in individuals with periventricular leukomalacia (PVL), a condition that affects white matter in the brain. Researchers are testing whether a combination of a novel cell therapy and specific molecular agents can support brain repair.

The main questions the study aims to answer are:

Can the treatment help regrow white matter and improve myelin repair? Does the treatment reduce scarring in the brain? Is the treatment safe and well-tolerated?

The study uses several components, including:

A specific type of neural progenitor cell to form the basis of the therapy. A small molecule compound to support cell function and survival. An agent designed to promote the repair of the myelin sheath. An enzyme intended to break down scar tissue in the brain. Researchers will study how these components work together to protect and repair the brain by influencing key pathways involved in damage and recovery.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-05-14
• Study First Submitted QC: 2025-05-14
• Study First Posted: 2025-05-22
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-17
• Last Update Posted: 2025-07-22
• Study Start: 2027-01-01
• Primary Completion: 2030-01-01
• Study Completion: 2031-12-31

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• Diagnosis of periventricular leukomalacia (PVL) confirmed by MRI
• Clinically stable at time of intervention
• Parental or legal guardian consent if participant is a minor

Exclusion Criteria

• Severe congenital brain malformations unrelated to PVL
• Active CNS infection or systemic inflammatory disease
• History of severe intraventricular hemorrhage (Grade III/IV)
• Known allergy or history of a significant hypersensitivity reaction to the investigational product or any of its components.
• Participation in another interventional study within the past 30 days

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Change in White Matter Integrity	At baseline and 12 weeks post-intervention	Measured using fractional anisotropy (FA) values derived from diffusion tensor imaging (DTI) MRI to assess structural white matter characteristics in brain regions affected by periventricular leukomalacia (PVL).

Secondary Outcome Measures

Name	Time	Method
Change in Functional Motor Score	Baseline, 6 weeks, and 12 weeks post-intervention	Measured using the Gross Motor Function Measure-88 (GMFM-88). The scale ranges from 0 to 100; higher scores indicate better motor function.
Change in Glial Scar Density	12 weeks post-intervention	Quantified using established biomarkers for astrogliosis, such as glial fibrillary acidic protein (GFAP), from cerebrospinal fluid or via advanced imaging. This outcome is intended to measure the biological activity of the therapy's scar-reducing component.