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

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Brief Title

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

Official Title

MT2014-14 Intrathecal Administration of Mesenchymal Stem Cells (IT-MSC) for the Treatment of Advanced Cerebral Adrenoleukodystrophy (cALD)

Brief Summary

      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

      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).

Study Phase

Phase 1

Study Type


Primary Outcome

Maximum Tolerated Dose

Secondary Outcome

 Radiographic Response


Cerebral Adrenoleukodystrophy


Mesenchymal Stem Cells

Study Arms / Comparison Groups

 Mesenchymal Stem Cell
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.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

June 2015

Completion Date

July 2022

Primary Completion Date

July 2017

Eligibility Criteria

        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




4 Years - N/A

Accepts Healthy Volunteers



Paul Orchard, MD, , 

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Masonic Cancer Center, University of Minnesota

Study Sponsor

Paul Orchard, MD, Principal Investigator, Masonic Cancer Center, University of Minnesota

Verification Date

November 2017