Back to resultsMT2022-01: MSCs for ALD
Primary Purpose
Cerebral Adrenoleukodystrophy
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Mesenchymal stem cells (IV-MSC)
Sponsored by
About this trial
This is an interventional treatment trial for Cerebral Adrenoleukodystrophy
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.
Sites / Locations
- Masonic Cancer Center, University of MinnesotaRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
IV-MSC for cALD (Early Disease/Bridge Therapy)
IV-MSC for cALD (Advanced Disease)
Arm Description
Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.
Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
Full Information
NCT ID
NCT06030648
First Posted
August 31, 2023
Last Updated
September 7, 2023
Sponsor
Masonic Cancer Center, University of Minnesota
1. Study Identification
Unique Protocol Identification Number
NCT06030648
Brief Title
MT2022-01: MSCs for ALD
Official Title
Intravenous Administration of Mesenchymal Stem Cells (IV-MSC) for the Treatment of Cerebral Adrenoleukodystrophy (cALD)
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 28, 2023 (Actual)
Primary Completion Date
May 1, 2025 (Anticipated)
Study Completion Date
May 1, 2028 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Masonic Cancer Center, University of Minnesota
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Adrenoleukodystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
IV-MSC for cALD (Early Disease/Bridge Therapy)
Arm Type
Experimental
Arm Description
Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.
Arm Title
IV-MSC for cALD (Advanced Disease)
Arm Type
Experimental
Arm Description
Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.
Intervention Type
Biological
Intervention Name(s)
Mesenchymal stem cells (IV-MSC)
Intervention Description
Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.
Primary Outcome Measure Information:
Title
Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
Description
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.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
Description
Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI.
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Troy C Lund, MD
Phone
+1 612-626-2778
Email
lundx072@umn.edu
Facility Information:
Facility Name
Masonic Cancer Center, University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Troy C Lund, MD
Phone
612-626-2778
Email
lundx072@umn.edu
12. IPD Sharing Statement
Learn more about this trial
MT2022-01: MSCs for ALD
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