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NCT02254863 · ClinicalTrials.gov registry record · Phase 1
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
A Phase 1 study of Batten Disease and Mucopolysaccharidosis II, sponsored by Joanne Kurtzberg, MD.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 40
- Enrollment target
- 1
- Study location
NCT02254863 is a Phase 1 study of Batten Disease and Mucopolysaccharidosis II that is actively recruiting participants, run by Joanne Kurtzberg, MD. The registered enrollment target is 40 participants, below the 2,826-participant average among 11 other Batten Disease trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state.
The verdict
NCT02254863, a Phase 1 study of Batten Disease and Mucopolysaccharidosis II, is actively recruiting participants, sponsored by Joanne Kurtzberg, MD.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 40 participants
- Enrollment target
- 1
- Study location
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 |
Interested in This Trial?
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, categorized as Phase 1. The registered enrollment target is 40 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 2,826-participant average among 11 other Batten Disease trials with a reported enrollment target (99% lower). The listed sponsor is Joanne Kurtzberg, MD, which has 15 total studies on file at ClinicalTrials.gov.
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.
NCT02254863 reports 1 study location spanning 1 distinct geographic area - top geographies include North Carolina.
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, Tay-Sachs Disease, Sandhoff Disease.
What interventions are being tested in trial NCT02254863?
The interventions under investigation include: DUOC-01 (BIOLOGICAL).
Who is sponsoring clinical trial NCT02254863?
This trial is sponsored by Joanne Kurtzberg, MD, which has 15 total clinical trials registered on ClinicalTrials.gov.
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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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.