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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02410239
• Date of registration: 27/03/2015
• Prospective Registration: Yes
• Primary sponsor: Masonic Cancer Center, University of Minnesota
• Public title: MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)
• Scientific title: MT2014-14 Intrathecal Administration of Mesenchymal Stem Cells (IT-MSC) for the Treatment of Advanced Cerebral Adrenoleukodystrophy (cALD)
• Date of first enrolment: June 2015
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT02410239
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Contacts

• Name: Paul Orchard, MD
• Affiliation: Masonic Cancer Center, University of Minnesota

Key inclusion & exclusion 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



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cerebral Adrenoleukodystrophy

Intervention(s)

Biological: Mesenchymal Stem Cells

Primary Outcome(s)

Maximum Tolerated Dose [Time Frame: Day 28 post intrathecal injection of MSC]

Secondary Outcome(s)

Radiographic Response [Time Frame: 6 months]

Secondary ID(s)

2014LS018

Source(s) of Monetary Support

Please refer to primary and secondary sponsors