Trials / Withdrawn
WithdrawnNCT06985303
Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia
Phase 1 Study Evaluating the Safety and Preliminary Efficacy of a Novel Cell-Based and Small Molecule Combination Therapy for White Matter Injury in Patients With Periventricular Leukomalacia
- Status
- Withdrawn
- Phase
- EARLY_Phase 1
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- MGAM LLC · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| COMBINATION_PRODUCT | 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. |
Timeline
- Start date
- 2027-01-01
- Primary completion
- 2030-01-01
- Completion
- 2031-12-31
- First posted
- 2025-05-22
- Last updated
- 2025-07-22
Source: ClinicalTrials.gov record NCT06985303. Inclusion in this directory is not an endorsement.