MT2022-01: MSCs for ALD

University of Minnesota (UMN)

Status and phase: Suspended; Phase 1

Conditions: Cerebral Adrenoleukodystrophy

Treatments: Biological: Mesenchymal stem cells (IV-MSC)

Study type: Interventional

Funder types: Other

Identifiers

NCT06030648

2022LS001

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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10 participants in 2 patient groups

IV-MSC for cALD (Early Disease/Bridge Therapy)

Experimental group

Description:

Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.

Treatment:

Biological: Mesenchymal stem cells (IV-MSC)

IV-MSC for cALD (Advanced Disease)

Experimental group

Description:

Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.

Treatment:

Biological: Mesenchymal stem cells (IV-MSC)