- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02410239
MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)
November 29, 2017 updated by: Masonic Cancer Center, University of Minnesota
MT2014-14 Intrathecal Administration of Mesenchymal Stem Cells (IT-MSC) for the Treatment of Advanced Cerebral Adrenoleukodystrophy (cALD)
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).
Study Overview
Status
Withdrawn
Conditions
Intervention / Treatment
Detailed 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).
Study Type
Interventional
Phase
- Phase 1
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
4 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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).
|
Day 28 post intrathecal injection of MSC
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
6 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Anticipated)
June 1, 2015
Primary Completion (Anticipated)
July 1, 2017
Study Completion (Anticipated)
July 1, 2022
Study Registration Dates
First Submitted
March 27, 2015
First Submitted That Met QC Criteria
April 1, 2015
First Posted (Estimate)
April 7, 2015
Study Record Updates
Last Update Posted (Actual)
December 2, 2017
Last Update Submitted That Met QC Criteria
November 29, 2017
Last Verified
November 1, 2017
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Demyelinating Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Endocrine System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Metabolism, Inborn Errors
- Mental Retardation, X-Linked
- Intellectual Disability
- Heredodegenerative Disorders, Nervous System
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Leukoencephalopathies
- Adrenal Gland Diseases
- Hereditary Central Nervous System Demyelinating Diseases
- Peroxisomal Disorders
- Adrenal Insufficiency
- Adrenoleukodystrophy
Other Study ID Numbers
- 2014LS018
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Cerebral Adrenoleukodystrophy
-
bluebird bioTerminatedCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, United Kingdom, Argentina, Canada, Germany, Italy, Netherlands
-
bluebird bioActive, not recruitingCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
-
Masonic Cancer Center, University of MinnesotaRecruitingCerebral Adrenoleukodystrophy | Adrenoleukodystrophy | ALD (Adrenoleukodystrophy)United States
-
Masonic Cancer Center, University of MinnesotaRecruiting
-
Minoryx Therapeutics, S.L.RecruitingCerebral Adrenoleukodystrophy (cALD)United States, Brazil, Argentina
-
bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, France, Germany, Italy, Netherlands, United Kingdom
-
bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, Germany, Argentina, Australia, France, United Kingdom
-
bluebird bioCenter for International Blood and Marrow Transplant ResearchRecruitingCerebral Adrenoleukodystrophy (CALD)United States
-
Minoryx Therapeutics, S.L.Active, not recruitingCerebral AdrenoleukodystrophySpain, Germany, France, Argentina
-
Viking Therapeutics, Inc.RecruitingAdrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)United States, France, Germany, Italy, United Kingdom
Clinical Trials on Mesenchymal Stem Cells
-
Guangzhou General Hospital of Guangzhou Military...UnknownSpinal Cord InjuryChina
-
Guangzhou General Hospital of Guangzhou Military...Guangzhou Municipal Twelfth People's Hospital; Guangdong Prevention and Treatment...Unknown
-
Guangzhou General Hospital of Guangzhou Military...UnknownParkinson's DiseaseChina
-
Hospital ZnojmoUnknown
-
National Research Center for Hematology, RussiaUnknownRelapse | Graft-versus-host DiseaseRussian Federation
-
University College, LondonUnknownTendinopathy | Achilles Tendinitis | Achilles Degeneration | Achilles Tendinitis, Right Leg | Achilles Tendon Thickening | Achilles Tendinitis, Left LegUnited Kingdom
-
Qingdao UniversityUnknownDilated CardiomyopathyChina
-
Qingdao UniversityUnknownUlcerative Colitis | Mesenchymal Stem Cells | Umbilical CordChina
-
Qingdao UniversityUnknownDiabetes Mellitus | Diabetes Mellitus, Type 1 | Mesenchymal Stem Cells | Umbilical CordChina
-
Universidad Catolica Santiago de GuayaquilMaastricht University Medical CenterUnknownOsteo Arthritis KneeEcuador