MT2022-01: MSCs for ALD

May 28, 2025 updated by: Masonic Cancer Center, University of Minnesota

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

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.

Study Overview

Status

Suspended

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Masonic Cancer Center, University of Minnesota

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
Time Frame: 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.
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
Time Frame: 6 months
Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Masonic Cancer Center, University of Minnesota

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 28, 2023

Primary Completion (Estimated)

May 1, 2026

Study Completion (Estimated)

May 1, 2028

Study Registration Dates

First Submitted

August 31, 2023

First Submitted That Met QC Criteria

September 7, 2023

First Posted (Actual)

September 11, 2023

Study Record Updates

Last Update Posted (Actual)

May 30, 2025

Last Update Submitted That Met QC Criteria

May 28, 2025

Last Verified

May 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2022LS001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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