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

NCT02410239 | Withdrawn Phase 1 DMC

• 1 other identifier: 2014LS018
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Cerebral Adrenoleukodystrophy

Keywords

Cerebral Adrenoleukodystrophy; cALD

Outcome Measures

Primary Outcomes (1)

• 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).

  Day 28 post intrathecal injection of MSC

Secondary Outcomes (1)

• Radiographic Response

  6 months

Study Arms (1)

Mesenchymal Stem Cell

EXPERIMENTAL

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

Interventions

Mesenchymal Stem Cells BIOLOGICAL

Mesenchymal Stem Cell

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Officials

• Paul Orchard, MD

  Masonic Cancer Center, University of Minnesota

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 27, 2015
• First Posted: April 7, 2015
• Study Start: June 1, 2015
• Primary Completion: July 1, 2017
• Study Completion: July 1, 2022
• Last Updated: December 2, 2017

Record last verified: 2017-11