WithdrawnEarly Phase 1

Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia

Stopped: Based on a review of new preclinical findings, the sponsor has decided to conduct additional foundational research before initiating this clinical study. The study is withdrawn pending further investigation.

0Started 2027-01-01

Plain-language 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.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change in White Matter Integrity

Timeframe: At baseline and 12 weeks post-intervention

Trial details

NCT IDNCT06985303

SponsorMGAM LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2030-01-01

View on ClinicalTrials.gov More Periventricular Leukomalacia trials

Plain-language summary

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.