MT2022-01: MSCs for ALD

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: Recruiting
• Conditions: Cerebral Adrenoleukodystrophy
• Intervention / Treatment: Biological: Mesenchymal stem cells (IV-MSC)

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Troy C Lund, MD; Phone Number: +1 612-626-2778; Email: lundx072@umn.edu

Study Locations

• United States
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • Recruiting
      • Masonic Cancer Center, University of Minnesota
      • Contact: Troy C Lund, MD; Phone Number: 612-626-2778; Email: lundx072@umn.edu

Participation Criteria

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

How is the study designed?

Design Details

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

Number of Arms

2

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).	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).	Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI.	6 months

Collaborators and Investigators

• Sponsor: Masonic Cancer Center, University of Minnesota

Study record dates

Study Major Dates

• Study Start (Actual): August 28, 2023
• Primary Completion (Estimated): May 1, 2025
• 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): September 11, 2023
• Last Update Submitted That Met QC Criteria: September 7, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Demyelinating Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Endocrine System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Leukoencephalopathies; Adrenal Gland Diseases; Hereditary Central Nervous System Demyelinating Diseases; Peroxisomal Disorders; Adrenal Insufficiency; Adrenoleukodystrophy
• 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