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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: Recruiting Phase 1 study of Batten Disease and Mucopolysaccharidosis II, sponsored by Joanne Kurtzberg, MD.
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. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Will monitor for fever, vomiting, neck stiffness, seizures, changes in state of consciousness
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 |
What NCT02254863 shows while recruiting
NCT02254863 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 40 participants, a relatively small participant target, below the 2,826-participant average among 11 other Batten Disease trials with a reported enrollment target (99% lower).
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 a single indexed study location in 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.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT02254863's enrollment target sits among peer trials
40 8th of 11 higher than 4 of 11 other Batten Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Batten Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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