MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)

University of Minnesota (UMN)

Status and phase: Withdrawn; Phase 1

Conditions: Cerebral Adrenoleukodystrophy

Treatments: Biological: Mesenchymal Stem Cells

Study type: Interventional

Funder types: Other

Identifiers

NCT02410239

2014LS018

Details and patient eligibility

About

The purpose of this study is to determine the maximum tolerated dose (MTD) of mesenchymal stem cells (IT-MSC) in patients with active, advanced cerebral adrenoleukodystrophy (cALD).

Full description

This is a single-institution dose escalation study to determine the maximum tolerated dose (MTD) of intrathecally administered allogeneic, 3rd party mesenchymal stem cells (IT-MSC) in patients with active, advanced cerebral adrenoleukodystrophy (cALD).

Sex: All

Ages: 4+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age ≥ 4 years at time of study enrollment
• Diagnosis of ALD - the diagnosis of ALD can be made by either biochemical or molecular/genetic evidence (plasma VLCFA or ABCD1 mutation analysis) supportive of ALD as the cause of cerebral demyelination
• Evidence of active cerebral disease - defined as the presence of gadolinium enhancement on a single brain MRI study - MRI used for eligibility determination may be performed at an outside institution; the most recent MRI used to determine eligibility must be within 2 calendar months of the date of enrollment on this study
• ALD MRI (Loes) score ≥ 10
• Off of N-acetylcysteine, systemic immunosuppressive drugs or any other therapeutic intervention (except hydrocortisone and/or fludracortisone for the treatment of adrenal insufficiency) for ≥ 10 days prior to the first IT-MSC dose
• Life expectancy of >6 months as determined by the enrolling researcher and documented in the medical record
• Voluntary written consent provided by parent(s)/guardian(s)

Exclusion criteria

• A candidate for allogeneic hematopoietic stem cell transplantation as determined by the University of Minnesota Inherited Metabolic and Storage Disease group
• Inability to undergo sedation, lumbar puncture or MRI studies for any reason
• Inability to stay in Minnesota for therapy through the day 28 evaluation

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Mesenchymal Stem Cell

Experimental group

Description:

Third party donor Mesenchymal Stem Cells (MSC) will be administered via intrathecal administration at the assigned dose on days 0, 7 and 14. Dose escalation will be guided by a fast track design. One patient is entered per dose level until a DLT is experienced. At that point, two additional patients will be enrolled at the same dose level. Dose levels will be 2.5 x 10e6 MSC/kg per dose, 5 x 10e6 MSC/kg per dose or 7.5 x 10e6 MSC/kg per dose.

Treatment:

Biological: Mesenchymal Stem Cells