Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Long-term Safety and Efficacy Study of Elivaldogene Autotemcel for Patients with Cerebral Adrenoleukodystrophy

Verified siteInvestigationalNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on a rare genetic disease called Cerebral Adrenoleukodystrophy (CALD). CALD affects the brain and can lead to severe neurological problems. The study is examining a treatment known as elivaldogene autotemcel, also referred to as Lenti-D. This treatment involves using a patient's own blood stem cells, which are modified to include a healthy version of a gene that is faulty in people with CALD. The modified cells are then infused back into the patient through a process called intravenous infusion.

The purpose of this study is to monitor the long-term safety and effectiveness of the Lenti-D treatment in individuals who have already received it in previous studies. Participants will be followed for up to 15 years after their initial treatment. During this time, researchers will observe how well the treatment works in preventing major disabilities and whether any side effects or new health issues arise. This includes checking for conditions like graft versus host disease (GVHD), which can occur when the infused cells attack the patient's body, and other immune-related or neurological disorders.

Throughout the study, participants will undergo regular health assessments, including MRI scans to monitor changes in the brain. The study aims to provide valuable information on the long-term outcomes of using Lenti-D for treating CALD, helping to understand its potential benefits and risks over an extended period. This information will be crucial for improving treatment strategies for individuals affected by this challenging condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after receiving the Lenti-D Drug Product in a previous clinical study.

    Written informed consent is required from the participant or their legal guardian. If applicable, the participant must also provide written informed assent.

  2. Step 2

    Monitoring phase

    The main objective is to monitor the long-term safety and effectiveness of the Lenti-D Drug Product, also known as elivaldogene autotemcel or eli-cel.

    Monitoring includes checking for major functional disability-free survival over time, up to 15 years after the drug infusion.

    Regular assessments will be conducted to identify any new or worsening health conditions, such as malignancies, immune-related issues, or neurological disorders.

  3. Step 3

    Follow-up assessments

    Interim analyses will occur after 5 and 10 years of follow-up to evaluate survival without major functional disabilities.

    The study will track the number of participants who undergo additional stem cell transplants within 15 years post-infusion.

    Changes in neurological function scores and the presence of gadolinium enhancement on MRI scans will be monitored over time.

  4. Step 4

    Completion of study

    The study is expected to conclude by August 31, 2038.

    Participants will be observed for up to 15 years following the initial drug infusion to ensure comprehensive data collection on long-term outcomes.

Who can join the trial?

4 criteria

  • The patient must have a condition called Cerebral Adrenoleukodystrophy (CALD).
  • The patient or their parent/legal guardian must provide written permission to participate in the study. If the patient is able, they should also give their written agreement.
  • The patient must have already received a treatment called Lenti-D Drug Product in a previous related study.
  • The study is only for male patients.

Who cannot join the trial?

4 criteria

  • Only males can participate in the study. Females are not eligible.
  • Participants must have been involved in the parent clinical studies where the eli-cel drug was administered.
  • Participants must be within the specified age range, which is typically determined by the study.
  • Individuals who are considered part of a vulnerable population are not eligible. This usually means people who might have difficulty giving informed consent or are at higher risk of harm.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Lenti-D Drug Product (eli-cel) is a treatment used for patients with cerebral adrenoleukodystrophy. This therapy involves using a specially designed gene therapy product to help manage the condition. The main goal of this treatment is to monitor its long-term safety and effectiveness in patients who have already received it in previous clinical studies.

What is already known about the treatment

Elivaldogene Autotemcel – This medication is administered through a one-time intravenous infusion. It is currently being studied in clinical trials for its long-term safety and efficacy in treating Cerebral Adrenoleukodystrophy (CALD). The main therapeutic indication is for patients with CALD, a rare genetic disorder. At the molecular level, it works by delivering a functional copy of the ABCD1 gene to the patient's cells, which helps in breaking down very long-chain fatty acids that accumulate in the brain. It is classified as a gene therapy product.

Investigated diseases

Cerebral Adrenoleukodystrophy (CALD) – This is a rare genetic disorder that primarily affects the brain and spinal cord. It is caused by a buildup of very long-chain fatty acids in the brain, which leads to the destruction of the protective sheath around nerve cells. The disease typically begins in childhood and progresses rapidly, leading to a decline in cognitive and motor functions. Symptoms may include behavioral changes, vision and hearing loss, and difficulty with coordination and balance. As the disease progresses, it can lead to severe neurological impairment. CALD is part of a group of disorders known as leukodystrophies, which affect the white matter of the brain.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2024-513904-33-00Protocol codeLTF-304Estimated enrolment64 patientsSponsorGenetix Biotherapeutics Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.