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

> **NCT02254863** · PHASE1 · RECRUITING · sponsor: **Joanne Kurtzberg, MD** · enrollment: 40 (estimated)

## Conditions studied

- 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

## Interventions

- **BIOLOGICAL:** DUOC-01

## Key facts

- **NCT ID:** NCT02254863
- **Lead sponsor:** Joanne Kurtzberg, MD
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2014-09
- **Primary completion:** 2026-10
- **Final completion:** 2026-10
- **Target enrollment:** 40 (ESTIMATED)
- **Last updated:** 2025-09-08


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02254863

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02254863, "UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT02254863. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*