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Completed Phase 2Phase 3 Interventional Results available

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

ClinicalTrials.gov ID: NCT01896102

Public ClinicalTrials.gov record NCT01896102. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Jul 22, 2026, 3:22 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Study identification

NCT ID
NCT01896102
Recruitment status
Completed
Study type
Interventional
Phase
Phase 2, Phase 3
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Enrollment
32 participants

Conditions and interventions

Interventions

  • Lenti-D Drug Product (eli-cel) Genetic

Genetic

Eligibility (public fields only)

Age range
Up to 17 Years
Sex
Male
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Aug 20, 2013
Primary completion
Mar 25, 2021
Completion
Mar 25, 2021
Last update posted
Apr 24, 2022

2013 – 2021

United States locations

U.S. sites
3
U.S. states
3
U.S. cities
3
Facility City State ZIP Site status
Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center Los Angeles California 90095
Boston Children's Hospital/Massachusetts General Hospital Boston Massachusetts 02115
University of Minnesota Minneapolis Minnesota 55455

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT01896102, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Apr 24, 2022 · Synced Jul 22, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01896102 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →