Active not recruitingOBSERVATIONAL

Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

This study is a long-term follow-up for individuals, from young children to adults, who have a rare genetic immune condition called Leukocyte Adhesion Deficiency-I (LAD-I). These individuals previously took part in an earlier study where they received a special gene therapy. The main goal is to carefully watch how safe and well this gene therapy works over many years, up to 15 years in total. Researchers are looking at how their bodies respond to the treatment and if there are any long-term effects. This follow-up helps scientists understand the real-world impact of the gene therapy and improve future treatments for LAD-I, ultimately aiming to help people with this condition live healthier lives.

At a glance

Status

Active not recruiting

Sponsor

Rocket Pharmaceuticals Inc.

Enrolment target

Start

09 Mar 2022

Estimated completion

04 Oct 2036

Leukocyte Adhesion Deficiency

What is this study about?

This research study is called a 'long-term follow-up' because it's designed to keep an eye on people who have already had a special treatment called gene therapy for a condition called Leukocyte Adhesion Deficiency-I (LAD-I). LAD-I is a rare illness where certain immune cells, called white blood cells, can't stick to other cells properly. This makes it hard for the body to fight off infections, which can be very serious.

The gene therapy given in the earlier study used a person's own blood stem cells, which are like master cells that can turn into different blood cells. These stem cells were gently changed in a lab to include a missing gene (called ITGB2) that helps the immune cells work correctly. The goal of this follow-up study is to see how safe and effective this changed gene therapy is over a long time, up to 15 years.

By carefully watching participants, doctors and researchers can learn much more about how this new treatment helps people with LAD-I live healthier lives, whether it prevents serious infections, and if there are any side effects that show up later on. This information is really important for developing and improving future treatments for this condition.

Key takeaways

This is a long-term follow-up for people who previously received gene therapy for LAD-I.
The study aims to check the safety and effectiveness of the gene therapy over many years.
Participation involves regular check-ups, some of which may require travel to a study center.
It helps doctors understand how gene therapy works in the long run for LAD-I.
People aged 3 months and older are included if they meet the previous study criteria.

Who may be eligible?

To be able to join this follow-up study, you must have already taken part in the previous research study (called RP-L201-0318) for LAD-I. In that earlier study, you would have received the gene therapy treatment, which involved having your own special blood stem cells changed in a lab and then given back to you.

It's also important that you (or your parents/guardians, if you are a child) are willing and able to come for all the necessary study visits and follow the instructions given by the study team. You'll need to sign a consent form, which explains everything about the study and confirms you agree to take part.

There are no specific reasons that would stop someone from joining this follow-up study, as long as they meet the main requirements mentioned above.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Did you take part in the earlier gene therapy study (RP-L201-0318) for LAD-I?
Did you receive the gene therapy treatment in that study?
Are you able to attend the required study visits?
Are you willing to sign a consent form to join this follow-up study?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be followed for a long time – up to 15 years after you first received the gene therapy. This tracking will continue until the study ends, or if you decide to stop participating, or if you are no longer able to be reached.

Most of your follow-up visits can happen with your local healthcare provider, and blood samples will be sent to the study lab. However, for the first three years, you'll need to visit the main study center once a year. If you need to have a bone marrow sample taken, these visits will always need to happen at the study center throughout the entire study period. Blood and bone marrow samples will be stored and tested only if doctors need more information, for example, if there's a new health concern.

Potential risks and benefits

Taking part in this study offers the benefit of continued close monitoring of your health, specifically related to your LAD-I and the gene therapy you received. This can provide valuable information for your own care and help advance treatments for others. As this is a long-term follow-up study, any direct risks are generally related to the procedures for collecting blood or bone marrow samples, such as minor discomfort or bruising. The gene therapy itself was given in the previous study, and this follow-up aims to monitor potential long-term effects. You are free to withdraw from the study at any time without affecting your medical care.

Locations (3)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of California, Los Angeles (UCLA)
Verified postcode
Los Angeles, United States
Hospital Infantil Universitario Niño Jesús
Verified postcode
Madrid, Spain
University College London Great Ormond Street Institute of Child Health (GOSH)
Verified postcode
London, United Kingdom

Common questions

What is LAD-I?

LAD-I is a rare inherited condition where your body's immune cells can't properly fight off infections.

What is gene therapy?

Gene therapy is a treatment that aims to fix faulty genes that cause diseases, like replacing a missing gene to help your body work better.

How long will I be in this study?

You will be followed for up to 15 years from when you first received the gene therapy in the previous study.

Do I have to travel for all my appointments?

No, many appointments can be done closer to home, but you'll need to visit the study center once a year for the first three years, and for any bone marrow samples.

Can I stop participating in the study?

Yes, you can choose to stop participating at any time, and it will not affect your medical care.