Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia

NCT ID: NCT06985303

Last Updated: 2025-07-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

EARLY_PHASE1

Study Classification

INTERVENTIONAL

Study Start Date

2027-01-01

Study Completion Date

2031-12-31

Brief Summary

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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

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Conditions

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

This is a single-group assignment study where all participants receive the investigational therapy. The therapy is a multi-component treatment combining a proprietary cell-based product with several molecular and enzymatic agents. This model allows for an initial assessment of the safety, tolerability, and biological activity of this combination approach for promoting white matter repair in PVL.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Experimental: Investigational Combination Therapy

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.

Group Type EXPERIMENTAL

Investigational Combination Product

Intervention Type COMBINATION_PRODUCT

This investigational therapy combines a proprietary, human-derived cell-based component with a unique blend of small molecules and an enzyme. The components are designed to work synergistically to address the complex pathology of white matter injury. The therapy aims to provide a source for cellular regeneration, support the survival of existing cells, enhance the potential for myelin repair, and modify the inhibitory environment of glial scar tissue. This multi-pronged biological intervention is designed to promote neural regeneration and functional recovery in patients with PVL.

Interventions

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Investigational Combination Product

This investigational therapy combines a proprietary, human-derived cell-based component with a unique blend of small molecules and an enzyme. The components are designed to work synergistically to address the complex pathology of white matter injury. The therapy aims to provide a source for cellular regeneration, support the survival of existing cells, enhance the potential for myelin repair, and modify the inhibitory environment of glial scar tissue. This multi-pronged biological intervention is designed to promote neural regeneration and functional recovery in patients with PVL.

Intervention Type COMBINATION_PRODUCT

Eligibility Criteria

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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
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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MGAM LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Other Identifiers

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MGAM-001

Identifier Type: -

Identifier Source: org_study_id

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