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

Cerebral Adrenoleukodystrophy Clinical Trial

Official title:

MT2014-14 Intrathecal Administration of Mesenchymal Stem Cells (IT-MSC) for the Treatment of Advanced Cerebral Adrenoleukodystrophy (cALD)

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02410239
• Other study ID #: 2014LS018
• Status: Withdrawn
• Phase: Phase 1
• First received: March 27, 2015
• Last updated: November 29, 2017
• Start date: June 2015
• Est. completion date: July 2022

Study information

• Verified date: November 2017
• Source: Masonic Cancer Center, University of Minnesota
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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).

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: July 2022
• Est. primary completion date: July 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Cerebral Adrenoleukodystrophy

Intervention

Biological:
• Mesenchymal Stem Cells

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Masonic Cancer Center, University of Minnesota

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Radiographic Response	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.	6 months