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Study on the Safety and Effects of Infusing Modified CD34+ Cells for Patients with Fanconi Anemia Subtype A

InvestigationalNo placebo
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What is this trial about?

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

This clinical trial is focused on studying a rare genetic disorder called Fanconi Anemia Subtype A. This condition affects the body's ability to repair damaged DNA, leading to bone marrow failure and an increased risk of cancer. The study is testing a treatment called Fancalen, which involves using the patient's own blood cells. These cells are collected and modified outside the body using a special tool called a lentiviral vector to carry a healthy version of the FANCA gene. The modified cells are then infused back into the patient through a process called infusion.

The purpose of the study is to evaluate the long-term safety and effectiveness of this treatment. Participants will receive the infusion of their modified cells and will be monitored over time to see how well the treatment works and to check for any side effects. The study will look at how the modified cells behave in the body, including their ability to stay in the bone marrow and blood, and whether they help stabilize blood counts. Researchers will also assess if the treatment reduces the risk of developing blood-related cancers or other tumors.

Participants in this study will have regular follow-up visits to monitor their health and the effects of the treatment. These visits will include blood tests and other assessments to ensure the treatment is working as intended and to identify any potential issues early. The study aims to provide valuable information on the potential benefits and risks of using gene therapy to treat Fanconi Anemia Subtype A.

The research process

The trial runs in 5 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 enrollment in the clinical phase 1/2 study FANCOLEN-I and receiving an infusion of autologous CD34+ enriched gene-corrected hematopoietic cells.

    Written informed consent is required, and adherence to the study visit schedule and protocol requirements is necessary.

  2. Step 2

    Infusion administration

    The treatment involves an infusion of Fancalen, which consists of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene.

    The infusion is administered intravenously.

  3. Step 3

    Safety assessments

    Regular blood-based evaluations are conducted to analyze integration sites in peripheral blood mononuclear cells.

    Assessments include monitoring for replication-competent lentivirus (RCL) in serum and blood cells, and recording any adverse events such as hospitalizations or new therapies for bone marrow failure.

  4. Step 4

    Efficacy assessments

    Blood samples are taken to evaluate peripheral blood counts and assess phenotypic correction through chromosomal fragility tests in T-lymphocytes.

    In cases of hematologic malignancy, additional tests determine if the malignancy originated from gene-corrected or uncorrected cells.

  5. Step 5

    Long-term follow-up

    The study includes long-term follow-up to evaluate the persistence of the therapeutic vector in bone marrow and blood cells.

    The follow-up aims to assess the stability and normalization of blood counts and monitor for any development of hematologic or non-hematologic malignancies.

Who can join the trial?

6 criteria

  • The patient must have been part of the earlier clinical phase 1/2 study called FANCOLEN-I.
  • The patient must have received an infusion of their own blood-forming cells, which were improved with a gene correction, during the FANCOLEN-I study.
  • The patient must be willing and able to follow the schedule for study visits and meet other study requirements.
  • The patient must have given written permission to participate in the current study, following the rules and regulations.
  • Patients who have had a transplant of blood-forming cells from a donor, due to bone marrow failure or certain blood cancers, will also be included in this study.
  • For patients who received a donor transplant, certain tests will not be done if previous tests showed no presence of the gene therapy in any cell samples.

Who cannot join the trial?

9 criteria

  • Patients with any other serious medical condition that could interfere with the study.
  • Patients who have had a recent infection that could affect their participation.
  • Patients who are currently taking medications that might interfere with the study treatment.
  • Patients who have a history of allergic reactions to similar treatments.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial recently.
  • Patients who have a history of substance abuse that could affect their ability to follow the study procedures.
  • Patients who have a mental health condition that might interfere with their ability to participate in the study.
  • Patients who do not agree to follow the study requirements and procedures.
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Investigated drugs

Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene are used in this clinical trial. These are special blood-forming cells taken from the patient's own body. They are modified in the laboratory using a lentiviral vector, which is a tool that helps deliver the FANCA gene into the cells. The FANCA gene is important for repairing DNA damage. This therapy aims to correct the genetic defect in patients with Fanconi Anaemia Subtype A, potentially improving their blood cell production and overall health.

What is already known about the treatment

Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene – This medication is administered through an infusion of genetically modified cells. It is currently being studied in clinical trials for its safety and effectiveness in treating Fanconi anemia subtype A. The main therapeutic indication is to correct the genetic defect in patients with this condition. At the molecular level, the mechanism involves using a lentiviral vector to introduce a functional copy of the FANCA gene into the patient's own hematopoietic stem cells, aiming to restore normal cell function. This treatment falls under the pharmacological classification of gene therapy.

Investigated diseases

Fanconi Anemia (subtype A) – Fanconi anemia is a rare genetic disorder that affects the bone marrow, leading to decreased production of all types of blood cells. It is characterized by physical abnormalities, bone marrow failure, and an increased risk of certain cancers. The disease progresses as the bone marrow becomes less effective at producing blood cells, which can lead to fatigue, frequent infections, and easy bruising or bleeding. Over time, individuals with Fanconi anemia may develop leukemia or other cancers. The condition is inherited in an autosomal recessive pattern, meaning both copies of the gene in each cell have mutations. Subtype A refers to a specific genetic mutation within the broader category of Fanconi anemia.
Trial detailsLast updated 9 Oct 2026
Age0-17PhasePhase ITrial ID2024-511523-33-00Protocol codeRP-L102-0116-LTFUEstimated enrolment9 patientsSponsorRocket Pharmaceuticals Inc.

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