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

NCT ID: NCT02410239

Last Updated: 2017-12-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE1

Study Classification

INTERVENTIONAL

Study Start Date

2015-06-30

Study Completion Date

2022-07-31

Brief Summary

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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).

Detailed Description

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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).

Conditions

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Cerebral Adrenoleukodystrophy

Keywords

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Cerebral Adrenoleukodystrophy cALD

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Mesenchymal Stem Cell

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.

Group Type EXPERIMENTAL

Mesenchymal Stem Cells

Intervention Type BIOLOGICAL

Interventions

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Mesenchymal Stem Cells

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

4 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Paul Orchard, MD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Other Identifiers

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2014LS018

Identifier Type: -

Identifier Source: org_study_id