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

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

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 Summary: 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).
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: 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).


Original Primary Outcome: Same as current

Current Secondary Outcome: 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.


Original Secondary Outcome: Same as current

Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: March 27, 2015
Date Started: June 2015
Date Completion: July 2022
Last Updated: February 17, 2017
Last Verified: February 2017