MT2022-01: MSCs for ALD

NCT06030648 | Suspended Phase 1 FDA Drug

• 1 other identifier: 2022LS001
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).

  6 months

Study Arms (2)

IV-MSC for cALD (Early Disease/Bridge Therapy)

EXPERIMENTAL

Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.

Biological: Mesenchymal stem cells (IV-MSC)

IV-MSC for cALD (Advanced Disease)

EXPERIMENTAL

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

Biological: Mesenchymal stem cells (IV-MSC)

Interventions

Mesenchymal stem cells (IV-MSC) 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); IV-MSC for cALD (Early Disease/Bridge Therapy)

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 31, 2023
• First Posted: September 11, 2023
• Study Start: August 28, 2023
• Primary Completion: May 1, 2026
• Study Completion (Estimated): May 1, 2028
• Last Updated: May 30, 2025

Record last verified: 2025-05

Locations

US (1)