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

Related Clinical Trial
SMART-ALD – A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD) Quality of Life in Women With X-linked Adrenoleukodystrophy A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) Plasma Exchange With Albumin in AMN Patients Stem Cell Transplant for Inborn Errors of Metabolism Human Placental-Derived Stem Cell Transplantation Precision Exercise in Children With Malignant Hemopathies Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy MD1003-AMN MD1003 in Adrenomyeloneuropathy A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients. Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD) A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children Early Diagnosis Of Childhood Cerebral ALD Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children HSCT for High Risk Inherited Inborn Errors Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD) UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) A Phase III Trial of Lorenzo’s Oil in Adrenomyeloneuropathy Lentiviral Gene Therapy for X-ALD MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD) Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) Newborn Screening for Adrenoleukodystrophy A Study to Prospectively Assess Disease Progression in Male Children With X-ALD Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy Expanded Access for Lorenzo’s Oil (GTO/GTE) in Adrenoleukodystrophy Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Brief Title

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

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 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 Phase

Phase 1

Study Type

Interventional


Primary Outcome

Maximum Tolerated Dose

Secondary Outcome

 Radiographic Response

Condition

Cerebral Adrenoleukodystrophy

Intervention

Mesenchymal Stem Cells

Study Arms / Comparison Groups

 Mesenchymal Stem Cell
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

0

Start Date

June 2015

Completion Date

July 2022

Primary Completion Date

July 2017

Eligibility 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
      

Gender

All

Ages

4 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Paul Orchard, MD, , 



Administrative Informations


NCT ID

NCT02410239

Organization ID

2014LS018


Responsible Party

Sponsor

Study Sponsor

Masonic Cancer Center, University of Minnesota


Study Sponsor

Paul Orchard, MD, Principal Investigator, Masonic Cancer Center, University of Minnesota


Verification Date

November 2017