MT2022-01: MSCs for ALD
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: IV-MSC for cALD (Early Disease/Bridge Therapy) Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy. |
Biological: Mesenchymal stem cells (IV-MSC)
Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.
|
| Experimental: IV-MSC for cALD (Advanced Disease) Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT. |
Biological: Mesenchymal stem cells (IV-MSC)
Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.
|
Outcome Measures
Primary Outcome Measures
- Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC). [6 months]
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.
Secondary Outcome Measures
- Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC). [6 months]
Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI.
Eligibility Criteria
Criteria
Inclusion Criteria:
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age ≥ 3 years
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diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation
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evidence of active cerebral disease as determined by the presence of gadolinium enhancement
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ALD MRI (Loes) score ≥ 1
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Patients who have not received prior gene therapy or transplant
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Life expectancy of > 6 months as determined by the enrolling researcher
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Have adequate organ function confirmed by laboratory values obtained within 28 days prior to enrollment
Exclusion Criteria:
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Inability to undergo sedation or MRI studies for any reason
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Other concurrent life-threatening disease (life expectancy <6 months) or eligible for hospice care
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Known or suspected hypersensitivity to human serum albumin, diphenhydramine, acetaminophen, methylprednisolone, or any of the components of IMS001.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Masonic Cancer Center, University of Minnesota | Minneapolis | Minnesota | United States | 55455 |
Sponsors and Collaborators
- Masonic Cancer Center, University of Minnesota
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 2022LS001