NCT06030648

MT2022-01: MSCs for ALD

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

• Status: Suspended
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Cerebral Adrenoleukodystrophy

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2023-08-28
• Primary completion: 2026-05-01
• Completion: 2028-05-01
• First posted: 2023-09-11
• Last updated: 2025-05-30

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study