Cure My Disease

Don't miss your cure!

 Log in / Sign up

One-Time gene therapy for fatal childhood brain disease: does it last?

NCT ID NCT02698579

Cure Sponsor: Genetix Biotherapeutics Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 14, 2026 · Last updated Sep 15, 2026 · Updated 1 time

Summary

Researchers are following 64 people with cerebral adrenoleukodystrophy (CALD) for 15 years after they received a one-time gene therapy called eli-cel in earlier studies. The therapy uses the patient's own stem cells, modified to carry a working copy of the gene that is faulty in CALD. This follow-up study tracks whether the treatment keeps working and monitors for long-term side effects like new cancers, immune problems, or worsening nerve symptoms. No new drug is given; participants simply continue to be evaluated.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
a one-time gene therapy called elivaldogene autotemcel (eli-cel), made from the patient's own modified stem cells
What this could lead to
If the gene therapy holds up, it could offer a lasting, one-time treatment that stops the devastating brain damage of cerebral adrenoleukodystrophy and removes the need for a donor stem cell transplant.
What could go wrong
This is a long-term follow-up study, not a new treatment trial, so it cannot prove the therapy works on its own. Gene therapies carry known risks, including the chance of new blood cancers or immune reactions, and some participants may still need another transplant.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

64 people

The number who actually took part.

Started

Jan 2016

Expected to finish

Aug 2038

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study will be expected to participate in this long-term follow-up study.

Ages

Up to 19 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Provision of written informed consent for this study by the participant or participant's parent(s)/ legal guardian(s) and written informed assent by participant, if applicable * Have received eli-cel in a parent clinical study Exclusion Criteria: * There are no exclusion criteria for this study

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Adrenoleukodystrophy (ALD) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

adrenoleukodystrophy X-linked cerebral adrenoleukodystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Adrenoleukodystrophy (ALD) Cerebral adrenoleukodystrophy (CALD) X-linked adrenoleukodystrophy (X-ald)

Contacts and locations

Locations

  • Boston Children's Hospital/Massachusetts General Hospital

    Boston, Massachusetts, 02115, United States

  • Great Ormond Street Hospital

    London, England, United Kingdom

  • Hospital das Clínicas da Universidade de São Paulo

    São Paulo, 05403-000, Brazil

  • Hôpital Bicêtre

    Le Kremlin-Bicêtre, Cedex, 94275, France

  • Instituto Neurogenia

    Caba, Argentina

  • Lucile Packard Children's Hospital - Stanford

    Palo Alto, California, 94304, United States

  • Mattel Children's Hospital-UCLA

    Los Angeles, California, 90095, United States

  • Ospedale Pediatrico Bambino Gesù

    Rome, 00165, Italy

  • Prinses Maxima Center

    Utrecht, 3584, Netherlands

  • Royal Free London Hospital

    London, England, NW3 2QG, United Kingdom

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • Universitätsklinikum Leipzig AöR

    Leipzig, 04103, Germany

  • Women's and Children's Hospital

    North Adelaide, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.