Long-Term Study on the Safety and Effects of RP-L102 Infusion for Patients with Fanconi Anemia Subtype A

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What is this study about?

This clinical trial is focused on studying Fanconi anemia subtype A, a rare genetic disorder that affects the bone marrow, leading to decreased production of blood cells. The treatment being tested is called Fancalen, also known by its code name RP-L102. This is a type of gene therapy that uses the patient’s own blood stem cells, which are modified outside the body to carry a healthy version of the FANCA gene. These modified cells are then infused back into the patient through a process called intravenous infusion.

The purpose of this study is to evaluate the long-term safety and effectiveness of this gene therapy. Participants will receive the infusion of Fancalen and will be monitored over an extended period to observe how well the treatment works and to ensure it is safe. The study will look at how the modified cells persist in the body and whether they help maintain stable blood counts, which is crucial for preventing complications associated with Fanconi anemia.

Throughout the study, researchers will also assess the potential for any long-term side effects, including the risk of developing other blood-related conditions. This trial is part of ongoing research to find better treatments for Fanconi anemia subtype A and aims to provide valuable insights into the benefits and risks of using gene therapy for this condition.

1 joining the study

Upon joining the study, the participant is required to have been previously enrolled in one of the RP-L102 parent studies and have received an infusion of CD34+ enriched cells that were modified with a special vector carrying the FANCA gene.

The participant must agree to follow the study visit schedule and other requirements, and provide written consent to participate.

2 infusion process

The participant receives an infusion of Fancalen, which consists of their own cells that have been modified to carry the FANCA gene. This is done through an intravenous infusion, meaning the cells are delivered directly into the bloodstream.

3 long-term follow-up

The study involves a long-term follow-up to monitor the safety and effectiveness of the treatment. This includes regular check-ups to assess the persistence of the therapeutic vector in the bone marrow and blood.

The follow-up aims to evaluate the stability of blood counts and the absence of bone marrow failure or blood-related cancers. The study also looks at the correction of blood cells and the potential development of any malignancies.

4 monitoring and assessments

Participants will undergo regular assessments to monitor the long-term effects of the treatment. This includes checking for any replication-competent lentivirus in the blood and evaluating the correction of blood cells.

The study will also assess the incidence of any blood-related cancers or other tumors, comparing these findings with known rates in individuals with Fanconi anemia.

Who Can Join the Study?

The person must have been part of one of the earlier studies called RP-L102-0418, RP-L102-0319, or RP-L102-0118.
The person must have received a special treatment in the earlier study. This treatment involved using their own blood cells, which were changed in a lab to include a new gene called FANCA.
The person must be willing and able to attend all the study visits and follow the study rules.
The person must have signed a form agreeing to take part in the study. If they are underage, they must also have given their agreement in a way that is suitable for their age.

Who Cannot Join the Study?

Patients with any other serious medical condition that might interfere with the study.
Patients who have had a previous treatment that could affect the study results.
Patients who are currently participating in another clinical trial.
Patients who have a history of allergic reactions to any component of the study treatment.
Patients who are unable to comply with the study procedures.
Patients who are pregnant or breastfeeding.
Patients with a history of certain types of cancer, such as acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). These are types of blood cancers.
Patients with a history of solid organ tumors, like squamous cell carcinoma of the head and neck. This is a type of skin cancer that can occur in the head and neck area.

Where you can join this trial?

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Trial status

Country	Status	Recruitment Start
Spain	Recruiting	01.11.2022

Trial locations

Investigated drugs:

Autologous Cd34+Enriched Cells From Patients With Fanconi Anemia Subtype A Transduced Ex Vivo With Lentiviral Vector Carrying The Fanconi Anemia Complementation Group A Gene

RP-L102 is a type of gene therapy used in this clinical trial. It involves taking some of the patient’s own blood-forming cells and modifying them outside the body. These cells are changed using a special tool called a lentiviral vector, which helps correct a genetic problem. After the cells are modified, they are put back into the patient’s body. The goal is to help the patient’s body produce healthy blood cells and improve their condition over the long term. This therapy is specifically designed for patients with a condition called Fanconi Anemia, which affects the bone marrow and blood cells.

Fanconi anemia subtype A (FA-A) – Fanconi anemia subtype A is a genetic disorder that affects the bone marrow, leading to decreased production of all types of blood cells. It is characterized by bone marrow failure, which can result in fatigue, frequent infections, and easy bruising or bleeding due to low blood cell counts. Over time, individuals with this condition may develop physical abnormalities, such as short stature, skin discoloration, and skeletal anomalies. The disease progresses with an increased risk of developing certain cancers, particularly blood-related cancers like acute myeloid leukemia and myelodysplastic syndrome. Fanconi anemia subtype A is caused by mutations in the FANCA gene, which is responsible for DNA repair, leading to increased susceptibility to DNA damage. The progression of the disease can vary widely among individuals, with some experiencing more severe symptoms and complications than others.

Trial ID:

2022-501082-52-00

Protocol code:

RP-L102-0221-LTFU

Trial Phase:

Human Pharmacology (Phase I) – Other

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Long-Term Study on the Safety and Effects of RP-L102 Infusion for Patients with Fanconi Anemia Subtype A

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?