RECRUITING

UCB Transplant of Inherited Metabolic Diseases with Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Conditions

Adrenoleukodystrophy Batten Disease Mucopolysaccharidosis II Leukodystrophy, Globoid Cell Leukodystrophy, Metachromatic Neimann Pick Disease Pelizaeus-Merzbacher Disease Sandhoff Disease Tay-Sachs Disease Brain Diseases, Metabolic, Inborn Alpha-Mannosidosis Sanfilippo Mucopolysaccharidoses Hunter Syndrome Krabbe Metachromatic Leukodystrophy ALD MLD PMD

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.

Official Title

Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases with Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells

Quick Facts

Study Start:2014-09

Study Completion:2025-10

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT02254863

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.

Ages Eligible for Study:1 Week to 22 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion Criteria
1. Patients must be age ≥1 week to ≤21 years. 2. Patients must have one of the following inherited metabolic diseases detected by enzyme or mutation analysis, and confirmed by repeat testing on a separately obtained sample: 3. Patients must have neurologic evidence of their disease, either clinically or via neuroimaging or neurophysiological testing. Examples of evidence of neurologic involvement include, but are not limited to the following: * Abnormal EEG, Brainstem Auditory Evoked Response (BAER), and/or Visual Evoked Potentials (VEP). * Abnormal brain MRI, ie. increased Loes score (measure of white matter damage, demyelination, and brain atrophy) and/or abnormal corticospinal tracts as assessed by MRI with diffusion tensor imaging (DTI). * Three or more of the early clinical markers: problems sleeping, increased activity, behavior difficulties, seizure-like activity, chewing behavior, inappropriate bladder training, inappropriate bowel training. 4. Patients must have adequate organ function as measured by: * Renal: Serum creatinine ≤ 2.0 mg/dl * Hepatic: Hepatic transaminases (ALT/AST) ≤ 5 x normal, bilirubin ≤ 2.0 mg/dl (except in patients with Gilbert's disease or newborns with physiological or breast milk associated jaundice). * Cardiac: Normal cardiac function by echocardiogram or radionuclide scan (shortening fraction or ejection fraction * 80% of normal value for age). Patients with acquired or congenital cardiomyopathy may receive melphalan as a substitute for cyclophosphamide. * Pulmonary: Pulmonary function tests demonstrating FVC, FEV1, and DLCO ≥ 60% of predicted in patients who can complete the testing. If patient cannot perform PFT's, an O2 sat must be \>90% on room air. 5. Patients must have an available, suitably matched, banked UCB unit for transplant. 6. Patients must have a performance status as follows: Lansky ≥ 40%, or Karnofsky ≥ 40% 7. Patients must have a life expectancy of ≥ 6 months.	1. Prior organ, tissue, or stem cell transplant within 3 years of study entry. 2. Prior participation in any gene or regenerative cell therapy study. 3. Inability to have an MRI scan or lumbar puncture. 4. Intractable seizures. 5. Chronic aspiration. 6. Bleeding disorder. 7. Evidence of HIV infection or HIV positive serology. 8. Uncontrolled bacterial, viral, or fungal infection at the time of pre-UCBT cytoreduction. 9. Inability to obtain patient's, parent's or legal guardian's consent. 10. Requirement of ventilatory support. 11. Pregnant or breastfeeding. 12. Active concurrent malignancy, or receiving concurrent radiotherapy, immunosuppressive medications, or cytotoxic chemotherapy

Inclusion Criteria

Exclusion Criteria

1. Patients must be age ≥1 week to ≤21 years.
2. Patients must have one of the following inherited metabolic diseases detected by enzyme or mutation analysis, and confirmed by repeat testing on a separately obtained sample:
3. Patients must have neurologic evidence of their disease, either clinically or via neuroimaging or neurophysiological testing. Examples of evidence of neurologic involvement include, but are not limited to the following:
* Abnormal EEG, Brainstem Auditory Evoked Response (BAER), and/or Visual Evoked Potentials (VEP).
* Abnormal brain MRI, ie. increased Loes score (measure of white matter damage, demyelination, and brain atrophy) and/or abnormal corticospinal tracts as assessed by MRI with diffusion tensor imaging (DTI).
* Three or more of the early clinical markers: problems sleeping, increased activity, behavior difficulties, seizure-like activity, chewing behavior, inappropriate bladder training, inappropriate bowel training.
4. Patients must have adequate organ function as measured by:
* Renal: Serum creatinine ≤ 2.0 mg/dl
* Hepatic: Hepatic transaminases (ALT/AST) ≤ 5 x normal, bilirubin ≤ 2.0 mg/dl (except in patients with Gilbert's disease or newborns with physiological or breast milk associated jaundice).
* Cardiac: Normal cardiac function by echocardiogram or radionuclide scan (shortening fraction or ejection fraction
* 80% of normal value for age). Patients with acquired or congenital cardiomyopathy may receive melphalan as a substitute for cyclophosphamide.
* Pulmonary: Pulmonary function tests demonstrating FVC, FEV1, and DLCO ≥ 60% of predicted in patients who can complete the testing. If patient cannot perform PFT's, an O2 sat must be \>90% on room air.
5. Patients must have an available, suitably matched, banked UCB unit for transplant.
6. Patients must have a performance status as follows: Lansky ≥ 40%, or Karnofsky ≥ 40%
7. Patients must have a life expectancy of ≥ 6 months.

1. Prior organ, tissue, or stem cell transplant within 3 years of study entry.
2. Prior participation in any gene or regenerative cell therapy study.
3. Inability to have an MRI scan or lumbar puncture.
4. Intractable seizures.
5. Chronic aspiration.
6. Bleeding disorder.
7. Evidence of HIV infection or HIV positive serology.
8. Uncontrolled bacterial, viral, or fungal infection at the time of pre-UCBT cytoreduction.
9. Inability to obtain patient's, parent's or legal guardian's consent.
10. Requirement of ventilatory support.
11. Pregnant or breastfeeding.
12. Active concurrent malignancy, or receiving concurrent radiotherapy, immunosuppressive medications, or cytotoxic chemotherapy

Contacts and Locations

Study Contact

Sydney Crane, RN

CONTACT

cordbloodtherapyinfo@dm.duke.edu

Erin Arbuckle

CONTACT

cordbloodtherapyinfo@dm.duke.edu

Principal Investigator

Joanne Kurtzberg, MD

PRINCIPAL_INVESTIGATOR

Duke University

Study Locations (Sites)

Duke University Medical Center

Durham, North Carolina, 27705

United States

Collaborators and Investigators

Sponsor: Joanne Kurtzberg, MD

Joanne Kurtzberg, MD, PRINCIPAL_INVESTIGATOR, Duke University

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 Registration Dates

Study Start Date2014-09

Study Completion Date2025-10

Study Record Updates

Study Start Date2014-09

Study Completion Date2025-10

Terms related to this study

Keywords Provided by Researchers

Adrenoleukodystrophy
Batten Disease
Hunter Syndrome
Krabbe
Metachromatic Leukodystrophy
ALD
MLD
PMD

Additional Relevant MeSH Terms

Adrenoleukodystrophy
Batten Disease
Mucopolysaccharidosis II
Leukodystrophy, Globoid Cell
Leukodystrophy, Metachromatic
Neimann Pick Disease
Pelizaeus-Merzbacher Disease
Sandhoff Disease
Tay-Sachs Disease
Brain Diseases, Metabolic, Inborn
Alpha-Mannosidosis
Sanfilippo Mucopolysaccharidoses