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UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
NCT02254863 · View on ClinicalTrials.gov ↗
Study Summary
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.
Conditions Studied
Interventions
- BIOLOGICAL DUOC-01
Study Locations (1)
North Carolina
- Duke University Medical Center — Durham
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 40 participants |
| Start Date | 2014-09 |
| Est. Completion | 2026-10 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02254863
The ClinicalTrials.gov registry entry for NCT02254863 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 40 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Joanne Kurtzberg, MD, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 10 conditions, with Batten Disease appearing as the primary indexed condition, and to 1 intervention — of which DUOC-01 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02254863 reports 1 study location spanning 1 distinct geographic area — top geographies include North Carolina. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT02254863 about?
NCT02254863 is a clinical study titled "UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells". 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 standar...
What is the current status of trial NCT02254863?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 40 participants. The study started on 2014-09. Estimated completion is 2026-10.
What conditions does trial NCT02254863 study?
This clinical trial studies the following conditions: Batten Disease, Mucopolysaccharidosis II, Adrenoleukodystrophy, Sandhoff Disease, Tay-Sachs Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT02254863?
The interventions under investigation include: DUOC-01 (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT02254863?
This trial is sponsored by Joanne Kurtzberg, MD, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT02254863 being conducted?
This trial has 1 study location across North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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