Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia

NCT06985303 | Withdrawn Early Phase 1

• 1 other identifier: MGAM-001
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

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

Keywords

Periventricular Leukomalacia; Myelin Repair; White Matter Injury; Demyelination; Neonatal Brain Injury; Neuroregeneration; CNS Regeneration; Brain Repair; Neonatal Hypoxia

Outcome Measures

Primary Outcomes (1)

• Change in White Matter Integrity

  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).

  At baseline and 12 weeks post-intervention

Secondary Outcomes (2)

• Change in Functional Motor Score

  Baseline, 6 weeks, and 12 weeks post-intervention

• Change in Glial Scar Density

  12 weeks post-intervention

Study Arms (1)

Experimental: Investigational Combination Therapy

EXPERIMENTAL

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.

Combination Product: Investigational Combination Product

Interventions

Investigational Combination Product 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.

Experimental: Investigational Combination Therapy

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• MGAM LLC: lead

MeSH Terms

Conditions

Leukomalacia, Periventricular; Leukoencephalopathies; Hypoxia-Ischemia, Brain; Demyelinating Diseases; Asphyxia Neonatorum

Condition Hierarchy (Ancestors)

Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Encephalomalacia; Vascular Diseases; Cardiovascular Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Brain Ischemia; Hypoxia, Brain; Hypoxia; Signs and Symptoms, Respiratory; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: May 14, 2025
• First Posted: May 22, 2025
• Study Start (Estimated): January 1, 2027
• Primary Completion (Estimated): January 1, 2030
• Study Completion (Estimated): December 31, 2031
• Last Updated: July 22, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share