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COMPLETED Phase 2

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

NCT01896102 · View on ClinicalTrials.gov ↗

Study Summary

This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells were collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells were transplanted back into the participant following myeloablative conditioning. Participants in this study will be continuously followed in study LTF-304.

Interventions

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

Study Locations (8)

Other

  • Medeos SRL — Buenos Aires
  • Hôpital Bicêtre — Le Kremlin-Bicêtre
  • University of Leipzig — Leipzig
  • Great Ormond Street Hospital for Children NHS Foundation Trust — London

California

  • Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center — Los Angeles

Massachusetts

  • Boston Children's Hospital/Massachusetts General Hospital — Boston

Minnesota

  • University of Minnesota — Minneapolis

South Australia

  • Women and Children's Hospital — North Adelaide

Trial Details

FieldValue
Enrollment Target 32 participants
Start Date 2013-08-21
Est. Completion 2021-03-26
Phase Phase 2

Sponsor

Genetix Biotherapeutics

33 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT01896102

The ClinicalTrials.gov registry entry for NCT01896102 describes a study currently listed as completed. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genetix Biotherapeutics, which has 33 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

The record links to 1 condition, with Cerebral Adrenoleukodystrophy (CALD) appearing as the primary indexed condition, and to 1 intervention — of which Lenti-D Drug Product (eli-cel) is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT01896102 reports 8 study locations spanning 5 distinct geographic areas — top geographies include Other, California, Massachusetts. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

Frequently Asked Questions

What is clinical trial NCT01896102 about?

NCT01896102 is a clinical study titled "A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)". This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blo...

What is the current status of trial NCT01896102?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 32 participants. The study started on 2013-08-21. Estimated completion is 2021-03-26.

What conditions does trial NCT01896102 study?

This clinical trial studies the following conditions: Cerebral Adrenoleukodystrophy (CALD). These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT01896102?

The interventions under investigation include: Lenti-D Drug Product (eli-cel) (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT01896102?

This trial is sponsored by Genetix Biotherapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT01896102 being conducted?

This trial has 8 study locations across California, Massachusetts, Minnesota, South Australia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial