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
Interventional
Primary Outcome
Maximum Tolerated Dose
Secondary Outcome
Radiographic Response
Condition
Cerebral Adrenoleukodystrophy
Intervention
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.
Publications
* 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
Biological
Estimated Enrollment
0
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
Gender
All
Ages
4 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Paul Orchard, MD, ,
Administrative Informations
NCT ID
NCT02410239
Organization ID
2014LS018
Responsible Party
Sponsor
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