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MT2022-01: MSCs for ALD

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ClinicalTrials.gov Identifier: NCT06030648
Recruitment Status : Recruiting
First Posted : September 11, 2023
Last Update Posted : September 11, 2023
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Description
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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.

Condition or disease Intervention/treatment Phase
Cerebral Adrenoleukodystrophy Biological: Mesenchymal stem cells (IV-MSC) Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Intravenous Administration of Mesenchymal Stem Cells (IV-MSC) for the Treatment of Cerebral Adrenoleukodystrophy (cALD)
Actual Study Start Date : August 28, 2023
Estimated Primary Completion Date : May 1, 2025
Estimated Study Completion Date : May 1, 2028

Resource links provided by the National Library of Medicine


Arms and Interventions
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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.


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.


Eligibility Criteria
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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06030648


Contacts
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Contact: Troy C Lund, MD +1 612-626-2778 lundx072@umn.edu

Locations
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United States, Minnesota
Masonic Cancer Center, University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Troy C Lund, MD    612-626-2778    lundx072@umn.edu   
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
More Information
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Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT06030648    
Other Study ID Numbers: 2022LS001
First Posted: September 11, 2023    Key Record Dates
Last Update Posted: September 11, 2023
Last Verified: September 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Adrenoleukodystrophy
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Leukoencephalopathies
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
 Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases