MT2022-01: MSCs for ALD

Study Overview
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Brief Summary
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This is a single-institution study to evaluate the use of intravenously administered allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene therapy, or in patients who are too advanced for gene therapy or HSCT. The intervention will occur in the time between diagnosis of active CALD and transplant which is currently 8-12 weeks.

Official Title
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Intravenous Administration of Mesenchymal Stem Cells (IV-MSC) for the Treatment of Cerebral Adrenoleukodystrophy (cALD)

Conditions
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Cerebral Adrenoleukodystrophy

Intervention / Treatment
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* Biological: Mesenchymal stem cells (IV-MSC)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: age ≥ 3 years diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation evidence of active cerebral disease as determined by the presence of gadolinium enhancement ALD MRI (Loes) score ≥ 1 Patients who have not received prior gene therapy or transplant Life expectancy of > 6 months as determined by the enrolling researcher Have adequate organ function confirmed by laboratory values obtained within 28 days prior to enrollment Exclusion Criteria: Inability to undergo sedation or MRI studies for any reason Other concurrent life-threatening disease (life expectancy <6 months) or eligible for hospice care Known or suspected hypersensitivity to human serum albumin, diphenhydramine, acetaminophen, methylprednisolone, or any of the components of IMS001.

Ages Eligible for Study
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Minimum Age: 3 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: IV-MSC for cALD (Early Disease/Bridge Therapy)<br>Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy. | Biological: Mesenchymal stem cells (IV-MSC)<br>* Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.<br>|
| Experimental: IV-MSC for cALD (Advanced Disease)<br>Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT. | Biological: Mesenchymal stem cells (IV-MSC)<br>* Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC). | Safety and tolerability of cerebral adrenoleukodystrophy (cALD) patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC) assessed by incidence of adverse events. | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC). | Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI. | 6 months |