MT2022-01: MSCs for ALD

Phase 1

Recruiting

• Conditions: Cerebral Adrenoleukodystrophy
• Interventions: Biological: Mesenchymal stem cells (IV-MSC)

• Registration Number: NCT06030648
• Lead Sponsor: Masonic Cancer Center, University of Minnesota

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-08-28
• Study First Submitted: 2023-08-31
• Status Verified: 2023-09
• Study First Submitted QC: 2023-09-07
• Last Update Submitted: 2023-09-07
• Study First Posted: 2023-09-11
• Last Update Posted: 2023-09-11
• Primary Completion: 2025-05-01
• Study Completion: 2028-05-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 10

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
IV-MSC for cALD (Advanced Disease)	Mesenchymal stem cells (IV-MSC)	Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.
IV-MSC for cALD (Early Disease/Bridge Therapy)	Mesenchymal stem cells (IV-MSC)	Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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.

Trial Locations

Locations (1)

Masonic Cancer Center, University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States