MT2022-01: MSCs for ALD

NCT ID: NCT06030648

Last Updated: 2025-05-30

Basic Information

• Recruitment Status: SUSPENDED
• Clinical Phase: PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-08-28
• Study Completion Date: 2028-05-01

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.

Conditions

Cerebral Adrenoleukodystrophy

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Mesenchymal stem cells (IV-MSC)

Intervention Type: BIOLOGICAL

Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.

IV-MSC for cALD (Advanced Disease)

Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.

Group Type: EXPERIMENTAL

Mesenchymal stem cells (IV-MSC)

Intervention Type: BIOLOGICAL

Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.

Interventions

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.

Intervention Type: BIOLOGICAL

Eligibility Criteria

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.

• Minimum Eligible Age: 3 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Countries

United States

Other Identifiers

2022LS001

Identifier Type: -

Identifier Source: org_study_id