Active not recruitingOBSERVATIONAL

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

This study is looking at the long-term health of male children and young men who have a serious brain condition called cerebral adrenoleukodystrophy, or CALD. They were previously treated with a gene therapy called Lenti-D (also known as eli-cel) in a past study. This current study is simply observing them over many years to see how they are doing and if the treatment continues to be safe and effective. No new medicines or treatments are given during this study. It's a way for doctors and researchers to understand the long-term effects of the gene therapy and help future patients. Participants will be followed for up to 15 years in total after their initial treatment.

At a glance

Status

Active not recruiting

Sponsor

Genetix Biotherapeutics Inc.

Enrolment target

Start

22 Jan 2016

Estimated completion

01 Aug 2038

Cerebral Adrenoleukodystrophy (CALD)Adrenoleukodystrophy (ALD)X-Linked Adrenoleukodystrophy (X-ALD)

What is this study about?

This study is called a 'follow-up study' because it's checking in on participants who were part of an earlier research project. These participants are children and young men who have a very serious genetic condition called cerebral adrenoleukodystrophy, or CALD. This condition affects the brain and can lead to severe problems.

In the earlier studies (called ALD-102 or ALD-104), these participants received a special type of gene therapy treatment called Lenti-D (also known as eli-cel). Gene therapy aims to fix problems caused by faulty genes.

The main goal of this new study is to see how these participants are doing in the long run after receiving the Lenti-D treatment. Researchers want to understand if the treatment continues to be safe and if it keeps helping them over many years. This information is very important for medical science and for developing better treatments for CALD in the future.

Key takeaways

This study is observing male children and young men with CALD.
Participants previously received Lenti-D gene therapy.
No new treatments are given in this follow-up study.
The study tracks health and safety for up to 15 years.
It helps researchers understand long-term effects of the treatment.
Participation requires being part of a previous Lenti-D study.

Who may be eligible?

To take part in this follow-up study, individuals must meet two main requirements. Firstly, the study is for male children and young men, up to 19 years old, who have CALD.

Secondly, and most importantly, participants must have already received the Lenti-D (eli-cel) gene therapy as part of one of the earlier clinical trials (specifically, Study ALD-102 or Study ALD-104). This study is only for people who have already had this treatment.

If you meet these two conditions, and you or your parent/guardian agree to participate and sign the necessary forms, then you can be part of this important follow-up. There aren't any reasons that would prevent someone who meets these criteria from joining this particular follow-up study.

Quick self-check

Are you male?
Are you 19 years old or younger?
Do you have cerebral adrenoleukodystrophy (CALD)?
Did you receive Lenti-D (eli-cel) gene therapy in a past clinical study (ALD-102 or ALD-104)?
Are you, or your parent/guardian, willing to give written consent to participate?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you won't be given any new medication or treatment. Instead, it's about checking your health over a long period. You and your family will continue to have regular check-ups, probably at a clinic or hospital involved in the study. These visits will likely include examinations and tests, which will be similar to the ones you've had before. The study will continue for a total of 15 years, starting from when you first received the Lenti-D treatment. This means that if you've already been in the initial study for two years, you'll be followed for another 13 years.

Potential risks and benefits

The main benefit of taking part in this follow-up study is that it helps doctors and researchers learn more about the long-term effects of the Lenti-D treatment for CALD. This information is crucial for improving care for future patients. Since no new medicines are given, the risks are generally associated with the check-ups and tests, which your care team will explain. You or your parents always have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (13)

Mattel Children's Hospital-UCLA
Los Angeles, United States
Lucile Packard Children's Hospital - Stanford
Palo Alto, United States
Boston Children's Hospital/Massachusetts General Hospital
Boston, United States
University of Minnesota
Minneapolis, United States
Instituto Neurogenia
Caba, Argentina
Women's and Children's Hospital
North Adelaide, Australia
Hospital das Clínicas da Universidade de São Paulo
São Paulo, Brazil
Hôpital Bicêtre
Le Kremlin-Bicêtre, France
Universitätsklinikum Leipzig AöR
Leipzig, Germany
Ospedale Pediatrico Bambino Gesù
Rome, Italy
Prinses Maxima Center
Utrecht, Netherlands
Royal Free London Hospital
London, United Kingdom

+1 more sites — see the official record for the full list.

Common questions

What is cerebral adrenoleukodystrophy (CALD)?

CALD is a serious inherited brain condition that mostly affects boys and young men. It can cause problems with movement, thinking, and even eyesight and hearing.

What is Lenti-D (eli-cel)?

Lenti-D is a special gene therapy treatment. It aims to correct the genetic problem that causes CALD, helping to slow or stop the condition from getting worse.

Will I receive new treatment in this study?

No, you will not receive any new treatment or medication in this study. It's purely a follow-up to see how you are doing after receiving Lenti-D in a previous study.

How long will this study last?

The study will follow participants for a total of 15 years from the time they first received the Lenti-D treatment. So, if you've already been in the earlier study for 2 years, you'd participate for another 13 years.

Who can take part in this study?

This study is for male children and young men, up to 19 years old, who have CALD and have already been treated with Lenti-D (eli-cel) in a previous clinical trial.