A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
Summary
The registered summary isn't in our current data snapshot. Read it on the ClinicalTrials.gov record ↗. Why? →
Investigators
The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.
- Adrian Thrasher, MD, PhD
Great Ormond Street Hospital for Chidren NHS Foundation Trust
Principal Investigator - Christine Duncan, MD
Boston Children's Hospital
Principal Investigator - David Williams, MD
Boston Children's Hospital
Principal Investigator - Florian Eichler, MD
Massachusetts General Hospital
Principal Investigator - Hernan Amartino, MD
Medeos SRL
Principal Investigator - Jakob Sieker, MD.
bluebird bio, Inc.
Study Director - Jorn-Sven Kuhl, MD
University of Leipzig
Principal Investigator - Nicholas Smith, MD
Women and Children's Hospital
Principal Investigator - Patrick Aubourg, MD, PhD
Hôpital Bicêtre
Principal Investigator - Paul Orchard, MD
University of Minnesota
Principal Investigator - Satiro de Oliveira, MD
University of California, Los Angeles
Principal Investigator
A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.
Sites
8 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.
- Medeos Srl
Buenos Aires, Argentina
- Women and Children'S Hospital
North Adelaide, South Australia, Australia
- Hôpital Bicêtre
Le Kremlin-Bicêtre, France
- University of Leipzig
Leipzig, Germany
- Great Ormond Street Hospital for Children Nhs Foundation Trust
London, United Kingdom
- Mattel Children'S Hospital Ucla/ronald Reagan Ucla Medical Center
Los Angeles, California, United States
- Boston Children'S Hospital/massachusetts General Hospital
Boston, Massachusetts, United States
- University of Minnesota
Minneapolis, Minnesota, United States
Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.
Drugs studied
No drug or biological interventions are in our data for this study — it may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →
Registration & key dates
Registry attributes as recorded on the ClinicalTrials.gov study record.
- Start
- 21 Aug 2013
- Primary completion
- 26 Mar 2021
- Completion
- 26 Mar 2021
- Last update posted
- 25 Apr 2022
- Registered enrollment
- 32
- Registry id
- NCT01896102
Cross-registrations
Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 12 Jul 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.