Demyelinating Diseases, Hypoxic-Ischemic Encephalopathy, Neonatal Encephalopathy, Periventricular Leukomalacia, White Matter Disease
Conditions
Keywords
Periventricular Leukomalacia, Myelin Repair, White Matter Injury, Demyelination, Neonatal Brain Injury, Neuroregeneration, CNS Regeneration, Brain Repair, Neonatal Hypoxia
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.
Interventions
COMBINATION_PRODUCTInvestigational 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.
Sponsors
MGAM LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
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.
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in White Matter Integrity | At baseline and 12 weeks post-intervention | 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Functional Motor Score | Baseline, 6 weeks, and 12 weeks post-intervention | Measured using the Gross Motor Function Measure-88 (GMFM-88). The scale ranges from 0 to 100; higher scores indicate better motor function. |
| Change in Glial Scar Density | 12 weeks post-intervention | Quantified using established biomarkers for astrogliosis, such as glial fibrillary acidic protein (GFAP), from cerebrospinal fluid or via advanced imaging. This outcome is intended to measure the biological activity of the therapy's scar-reducing component. |
Outcome results
None listed