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Long-term Safety and Efficacy Study of Lovotibeglogene Autotemcel Gene Therapy for Patients with Sickle Cell Disease

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What is this trial about?

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

This clinical trial focuses on sickle cell disease, a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study is examining a treatment called lovotibeglogene autotemcel, also known as bb1111 or LentiGlobin BB305 Drug Product for Sickle Cell Disease. This treatment involves a type of gene therapy where a patient's own blood stem cells are modified outside the body using a special tool called a lentiviral vector to help correct the genetic defect causing the disease.

The purpose of the study is to evaluate the long-term safety and effectiveness of this treatment in individuals with sickle cell disease. Participants in the study will receive the treatment and then be monitored over an extended period to observe any changes in their health. The study will track various health outcomes, including the occurrence of immune-related issues, blood disorders, and any new or worsening neurological conditions. Additionally, the study will assess the treatment's impact on the frequency and severity of painful episodes known as vaso-occlusive events, which are common in sickle cell disease.

Participants will be followed for up to 15 years to gather comprehensive data on the treatment's effects. This long-term follow-up will help researchers understand how well the treatment works over time and identify any potential long-term side effects. The study aims to provide valuable insights into the potential benefits and risks of using gene therapy to treat sickle cell disease, ultimately contributing to improved care for individuals living with this condition.

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 providing written informed consent. This consent may be given by the patient or, if applicable, by the patient's parent or legal guardian.

    Eligibility requires prior treatment with the drug product for sickle cell disease in a specific clinical study.

  2. Step 2

    Treatment administration

    The treatment involves the use of lovotibeglogene autotemcel, also known as LentiGlobin BB305 Drug Product for Sickle Cell Disease.

    This medication is administered as a dispersion for infusion through an intravenous route.

  3. Step 3

    Long-term follow-up

    The primary objective is to evaluate the long-term safety and efficacy of the treatment.

    Monitoring includes assessing the number of patients with immune-related adverse events, new or worsening blood disorders, neurological disorders, and malignancies.

  4. Step 4

    Evaluation of outcomes

    Secondary outcomes include the resolution of severe vaso-occlusive events over time, changes in blood markers, and iron levels.

    The follow-up period extends through Year 15, with assessments of various health indicators over this duration.

  5. Step 5

    Completion of study

    The estimated end date for the study is January 31, 2038.

    The study aims to provide comprehensive data on the long-term effects of the treatment for sickle cell disease.

Who can join the trial?

5 criteria

  • The patient must have sickle-cell disease, which is a blood disorder that affects red blood cells.
  • The patient or their parent/legal guardian must provide written informed consent, which means they agree to participate in the study after understanding what it involves.
  • The patient must have been treated with a specific drug product for sickle-cell disease in a previous clinical study sponsored by bluebird bio.
  • The study is open to both male and female patients.
  • The study includes patients who are considered part of a vulnerable population, which means they may need extra protection or care.

Who cannot join the trial?

7 criteria

  • Patients who do not have sickle-cell disease cannot participate. Sickle-cell disease is a condition where red blood cells, which carry oxygen, are shaped like a sickle or crescent, instead of being round.
  • Patients who are not within the specified age range cannot participate. The age range for this study is not specified in the provided data.
  • Patients who are not willing to follow the study procedures or take the study medication cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate. These conditions are not specified in the provided data.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible.
  • Patients who have allergies or reactions to the study medication cannot participate.
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Investigated drugs

Lovocel (lovotibeglogene autotemcel, also known as bb1111 or LentiGlobin BB305) is a gene therapy treatment for individuals with sickle cell disease. This therapy involves using a patient's own blood stem cells, which are collected and then modified outside the body. The modification is done using a lentiviral vector, which helps introduce a new gene into the cells. These modified cells are then returned to the patient's body, with the aim of producing healthy red blood cells that can reduce the symptoms and complications associated with sickle cell disease. The clinical trial is focused on evaluating the long-term safety and effectiveness of this treatment.

What is already known about the treatment

Lovocel (lovotibeglogene autotemcel) – This medication is administered through ex vivo gene therapy, where a patient's own hematopoietic stem cells are modified outside the body using a lentiviral vector and then reintroduced. It is currently being studied in clinical trials for its long-term safety and efficacy in treating sickle cell disease. The main therapeutic indication is for sickle cell disease, a genetic disorder affecting red blood cells. At the molecular level, lovocel works by introducing a modified gene into the patient's stem cells to produce healthier red blood cells. It is classified under gene therapy products, specifically targeting genetic blood disorders.

Investigated diseases

Sickle-cell disease – Sickle-cell disease is a genetic disorder that affects the shape and function of red blood cells. Normally, red blood cells are round and flexible, allowing them to move easily through blood vessels. In sickle-cell disease, these cells become rigid and shaped like a crescent or sickle. This abnormal shape can cause the cells to get stuck in small blood vessels, leading to blockages that slow or stop the flow of blood and oxygen to parts of the body. Over time, this can result in episodes of pain, known as vaso-occlusive crises, and can damage organs and tissues. The disease is chronic and can lead to various complications throughout a person's life.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-513901-30-00Protocol codeLTF-307Estimated enrolment88 patientsSponsorGenetix Biotherapeutics Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).