Periventricular LeukomalaciaEarly Phase 1NCT06985303
Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia
A Early Phase 1 study testing Investigational Combination Product in people with Periventricular Leukomalacia and White Matter Disease, run by MGAM LLC.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Periventricular Leukomalacia, White Matter Disease, Hypoxic-Ischemic Encephalopathy
- What is being tested?
- Investigational Combination Product
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2027to 31 Dec 2031
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of periventricular leukomalacia (PVL) confirmed by MRI
- Clinically stable at time of intervention
- Parental or legal guardian consent if participant is a minor
Who cannot join
- 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
About this study
Official title: 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
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:
Read more
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
- Being tested
- Investigational Combination Product
- Keywords
- Periventricular Leukomalacia, Myelin Repair, White Matter Injury, Demyelination, Neonatal Brain Injury, Neuroregeneration, CNS Regeneration, Brain Repair, Neonatal Hypoxia
- Medical subject headings
- Leukomalacia, Periventricular, Leukoencephalopathies, Hypoxia-Ischemia, Brain, Demyelinating Diseases, Asphyxia Neonatorum
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Investigational Combination ProductCombination 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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Combination Product: Investigational Combination Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: At baseline and 12 weeks post-intervention
Secondary outcomes (2)
Change in Functional Motor Score
Measured using the Gross Motor Function Measure-88 (GMFM-88). The scale ranges from 0 to 100; higher scores indicate better motor function.
Measured: Baseline, 6 weeks, and 12 weeks post-intervention
Change in Glial Scar Density
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.
Measured: 12 weeks post-intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2025
- Study start1 Jan 2027 (estimated)
- Primary completion1 Jan 2030 (estimated)
- Study completion31 Dec 2031 (estimated)
Why it 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Periventricular LeukomalaciaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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.
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- MGAM LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06985303
- Sponsor's protocol ID
- MGAM-001
- Status last verified
- Jul 2025
- First submitted
- 14 May 2025
- Last updated
- 22 Jul 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06985303
- Acronym / sponsor ID
- MGAM-001
- 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
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason 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.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- MGAM LLCIndustry (pharma, biotech, devices)
- Study start
- 1 Jan 2027 (estimated)
- Primary completion
- 1 Jan 2030 (estimated)
- Study completion
- 31 Dec 2031 (estimated)
- First posted
- 22 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| Experimental: Investigational Combination Therapy | Experimental | Combination Product: Investigational Combination ProductParticipants 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of periventricular leukomalacia (PVL) confirmed by MRI
- Clinically stable at time of intervention
- Parental or legal guardian consent if participant is a minor
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Investigational Combination ProductThis 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. | Combination product | Experimental: Investigational Combination Therapy |
Primary outcome
Change in White Matter Integrity
Time frame: 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).
2 secondary outcomes
Change in Functional Motor Score
Time frame: Baseline, 6 weeks, and 12 weeks post-intervention
Change in Glial Scar Density
Time frame: 12 weeks post-intervention
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Investigational Combination Product in people with Periventricular Leukomalacia and White Matter Disease, run by MGAM LLC.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 22 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in White Matter Integrity (time frame: At baseline and 12 weeks post-intervention). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2025.