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

Primary Purpose

Cerebral Adrenoleukodystrophy

Status
Withdrawn
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Mesenchymal Stem Cells
Sponsored by
Masonic Cancer Center, University of Minnesota
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cerebral Adrenoleukodystrophy focused on measuring Cerebral Adrenoleukodystrophy, cALD

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Mesenchymal Stem Cell

    Arm 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.

    Outcomes

    Primary Outcome Measures

    Maximum Tolerated Dose
    Determine the maximum tolerated dose (MTD) of intrathecally-administered allogeneic, third-party mesenchymal stem cells (IT-MSC) in patients with active, advanced cerebral adrenoleukodystrophy (cALD).

    Secondary Outcome Measures

    Radiographic Response
    Evaluate the incidence of, and time to, radiographic response in patients receiving IT-MSC for active, advanced cerebral adrenoleukodystrophy. Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI, or the absence of an increase in Loes score between the enrollment MRI and 6 month MRI.

    Full Information

    First Posted
    March 27, 2015
    Last Updated
    November 29, 2017
    Sponsor
    Masonic Cancer Center, University of Minnesota
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02410239
    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)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    November 2017
    Overall Recruitment Status
    Withdrawn
    Why Stopped
    Research cancelled
    Study Start Date
    June 2015 (Anticipated)
    Primary Completion Date
    July 2017 (Anticipated)
    Study Completion Date
    July 2022 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Masonic Cancer Center, University of Minnesota

    4. Oversight

    Data Monitoring Committee
    Yes

    5. Study Description

    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).

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cerebral Adrenoleukodystrophy
    Keywords
    Cerebral Adrenoleukodystrophy, cALD

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Mesenchymal Stem Cell
    Arm Type
    Experimental
    Arm 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.
    Intervention Type
    Biological
    Intervention Name(s)
    Mesenchymal Stem Cells
    Primary Outcome Measure Information:
    Title
    Maximum Tolerated Dose
    Description
    Determine the maximum tolerated dose (MTD) of intrathecally-administered allogeneic, third-party mesenchymal stem cells (IT-MSC) in patients with active, advanced cerebral adrenoleukodystrophy (cALD).
    Time Frame
    Day 28 post intrathecal injection of MSC
    Secondary Outcome Measure Information:
    Title
    Radiographic Response
    Description
    Evaluate the incidence of, and time to, radiographic response in patients receiving IT-MSC for active, advanced cerebral adrenoleukodystrophy. Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI, or the absence of an increase in Loes score between the enrollment MRI and 6 month MRI.
    Time Frame
    6 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    4 Years
    Accepts Healthy Volunteers
    No
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Paul Orchard, MD
    Organizational Affiliation
    Masonic Cancer Center, University of Minnesota
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Learn more about this trial

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

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