MT2022-01: MSCs for ALD

Cerebral Adrenoleukodystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06030648
• Other study ID #: 2022LS001
• Status: Recruiting
• Phase: Phase 1
• Start date: August 28, 2023
• Est. completion date: May 1, 2028

Study information

• Verified date: September 2023
• Source: Masonic Cancer Center, University of Minnesota
• Contact: Troy C Lund, MD
• Phone: +1 612-626-2778
• Email: lundx072[@]umn.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: May 1, 2028
• Est. primary completion date: May 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Cerebral Adrenoleukodystrophy

Intervention

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.

Locations

Country	Name	City	State
United States	Masonic Cancer Center, University of Minnesota	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Masonic Cancer Center, University of Minnesota

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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