Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia

MGAM LLC

Status and phase: Withdrawn; Early Phase 1

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

Treatments: Combination Product: Investigational Combination Product

Study type: Interventional

Funder types: Industry

Identifiers

NCT06985303

MGAM-001

Details and patient eligibility

About

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.

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Experimental: Investigational Combination Therapy

Experimental group

Description:

Participants will receive an investigational combination therapy. This therapy consists of a proprietary cell-based component administered with a cocktail of molecular and enzymatic agents. These agents are designed to support cell health, promote myelin repair, and reduce inhibitory factors in the damaged brain tissue. The overall goal of the intervention is to promote white matter regeneration in patients with periventricular leukomalacia by targeting key barriers that prevent natural repair.

Treatment:

Combination Product: Investigational Combination Product