BEACON
Title of Protocol:
A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (Beam-101) in Patients with Sickle Cell Disease and Severe Vaso-Occlusive Crises
Purpose/Objective of Study
The purpose of Beacon trial is to evaluate the safety and ability of the autologous stem cell transplant with gene-edited stem cells to treat sickle cell disease.
Study Agent(s) and Mechanism of Drug Action/Device Description:
Beam-101 is the investigation product in this trial. Beam-101 is a cellular product consisting of autologous CD34+ hematopoietic stem cells that have undergone ex vivo base editing to introduce a specific A-to-G transition mutation into promoters of the HBG1 to HBG2 genes that encode gamma globin.
Key Eligibility
Potential participants must meet the following key eligibility criteria
- Participant ages between 12 to 35 years of age
- Diagnosis of HbSS, sickle cell beta thalassemia or sickle beta zero thalassemia
- Participants must have had at least 4 severe vaso-occlusive crisis within a 24 month period prior to informed consent
- Participant must not have been diagnosed with moyamoya syndrome
Toxicities/Side Effects
Some possible side effects from this trial included: engraftment failure, development of cancer of the blood, low blood counts, infection, lung or liver injury, sterility, hair loss, mucositis or skin rash.
Time Commitment
30 months
Website link of sponsor
NA
Research Team
PI: Julie Kanter, MD
Clinical Research Administrative Manager: Emily Warner
Site Coordinator: Christina Ordinario, MSN, CRNP, FNP-C
HGB210
Study Status: enrollment complete
Title of Protocol:
A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the BB305 Lentiviral Vector in Subjects with Sickle Cell Disease
Purpose/Objective of Study
The primary purpose of the HGB210 trial is to evaluate the safety and efficacy of treatment with bb1111 drug product and autologous transplant in individuals with severe sickle cell disease.
Study Agent(s) and Mechanism of Drug Action/Device Description:
Bb1111 is the investigation product in this trial. Bb1111 is a cellular product consisting of autologous CD34+ cell enriched population from patients with SCD that contains hematopoietic stem cells transduced with BB305 lentiviral vector encoding βA-T87Q the globin gene, suspended in cryopreservation solution.
Key Eligibility
Potential participants must meet the following key eligibility criteria:
- Participant ages between 12 to 50 years of age
- Diagnosis of HbSS, sickle cell beta thalassemia or sickle beta zero thalassemia
- Participants must have had at least 4 severe vaso-occlusive crisis within a 24 month period prior to informed consent
- Participants mush have either experienced hydroxyurea failure or intolerance
- Participant must not have been diagnosed with stroke or moyamoya syndrome
Toxicities/Side Effects
Some possible side effects from this trial included: engraftment failure, development of cancer of the blood, low blood counts, infection, lung or liver injury, sterility, hair loss, mucositis, or skin rash.
Time Commitment
30 months
Website link of sponsor
NA
Research Team
PI: Julie Kanter, MD
Clinical Research Administrative Manager: Emily Warner
Site Coordinator: Emily Burke, CRNP
LTF-307
Study Status: enrollment open
Title of Protocol:
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector
Purpose/Objective of Study
The primary purpose of this study is to evaluate the long-term safety and efficacy of the gene therapy during product used in bluebird bio-sponsored clinical studies in treated subjects with sickle cell disease.
Study Agent(s) and Mechanism of Drug Action/Device Description:
There is no treatment intervention for this study, long term follow up only from being treated on a bluebird bio clinical trial.
Key Eligibility
Potential participants must meet the following key eligibility criteria
- Participant must have been previously treated with drug product for therapy of sickle cell disease in a bluebird bio sponsored clinical study
Toxicities/Side Effects
There is no treatment intervention for this study, long term follow up only.
Time Commitment
13 years
Website link of sponsor
NA
Research Team
PI: Julie Kanter, MD
Clinical Research Administrative Manager: Emily Warner
Site Coordinator: Emily Burke, CRNP