MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)
|ClinicalTrials.gov Identifier: NCT02410239|
Recruitment Status : Withdrawn (Research cancelled)
First Posted : April 7, 2015
Last Update Posted : December 2, 2017
|Condition or disease||Intervention/treatment||Phase|
|Cerebral Adrenoleukodystrophy||Biological: Mesenchymal Stem Cells||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||MT2014-14 Intrathecal Administration of Mesenchymal Stem Cells (IT-MSC) for the Treatment of Advanced Cerebral Adrenoleukodystrophy (cALD)|
|Estimated Study Start Date :||June 2015|
|Estimated Primary Completion Date :||July 2017|
|Estimated Study Completion Date :||July 2022|
Experimental: 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.
Biological: Mesenchymal Stem Cells
- Maximum Tolerated Dose [ Time Frame: Day 28 post intrathecal injection of MSC ]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).
- Radiographic Response [ Time Frame: 6 months ]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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02410239
|Principal Investigator:||Paul Orchard, MD||Masonic Cancer Center, University of Minnesota|