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Hematopoietic Stem Cell Transplantation
Gene Therapy (bb1111) for Sickle Cell Disease
Phase 1 & 2
Waitlist Available
Research Sponsored by bluebird bio
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up month 3, 6, 12, 18, and 24 post-transplant
Awards & highlights
Study Summary
This trial will assess the safety and effectiveness of a new gene therapy for people with severe sickle cell disease.
Who is the study for?
This trial is for adults and adolescents aged 12 to 50 with severe Sickle Cell Disease (SCD), who have had at least four severe pain episodes in the last two years despite treatment, or cannot tolerate hydroxyurea. Participants must have a performance status indicating they can carry out daily activities. Excluded are those with cancer, immunodeficiency, certain infections like HIV/HBV/HCV, poor bone marrow function, serious brain blood vessel problems, prior transplants or gene therapy, advanced liver disease, pregnancy/breastfeeding without contraception use.Check my eligibility
What is being tested?
The study tests bb1111 (LentiGlobin BB305 Drug Product) used in hematopoietic stem cell transplantation (HSCT) for treating severe SCD. It's an open-label Phase 1/2 trial involving around 50 participants to assess safety and efficacy of this single-dose treatment.See study design
What are the potential side effects?
While specific side effects of bb1111 aren't listed here, HSCT-related side effects generally include risk of infection due to low white blood cells count; nausea; fatigue; mouth sores; hair loss from conditioning chemotherapy; graft-versus-host disease where donor cells attack your body.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ month 3, 6, 12, 18, and 24 post-transplant
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month 3, 6, 12, 18, and 24 post-transplant
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
VOE-CR
Secondary outcome measures
Average and median of HbAT87Q percentage of non-transfused total Hb over time
Average and median of HbS percentage of non-transfused total Hb over time
Average and median of non-HbS percentage of non-transfused total Hb over time
+26 moreTrial Design
3Treatment groups
Experimental Treatment
Group I: Group CExperimental Treatment1 Intervention
Plerixafor mobilization and apheresis will be used for collection of rescue cells, and subjects will receive treatment of bb1111 manufactured with autologous CD34+ hematopoietic stem cells (HSCs) collected by plerixafor mobilization and apheresis transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene.
*No Longer Recruiting
Group II: Group BExperimental Treatment1 Intervention
Group B1:
Subjects will have rescue cells collected by bone marrow harvest, and will receive treatment of bb1111 manufactured with autologous CD34+ hematopoietic stem cells (HSCs) collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene.
*No Longer Recruiting
Group B2:
Plerixafor mobilization and apheresis will be used for collection of rescue cells and exploratory manufacturing development. Subjects will receive treatment of bb1111 manufactured with autologous CD34+ hematopoietic stem cells (HSCs) collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene.
*No Longer Recruiting
Group III: Group AExperimental Treatment1 Intervention
Subjects will have rescue cells collected by bone marrow harvest, and will receive treatment of bb1111 manufactured with autologous CD34+ hematopoietic stem cells (HSCs) collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene.
*No Longer Recruiting
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
bb1111
2015
Completed Phase 2
~50
Find a Location
Who is running the clinical trial?
bluebird bioLead Sponsor
20 Previous Clinical Trials
1,993 Total Patients Enrolled
Richard Colvin, MDStudy Directorbluebird bio, Inc.
1 Previous Clinical Trials
35 Total Patients Enrolled
Anjulika Chawla, MD, FAAPStudy Directorbluebird bio, Inc.
2 Previous Clinical Trials
120 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have had or currently have any type of cancer or immune system disorder, except for non-life threatening skin cancer that has been treated and cured.You have a close family member who has been diagnosed with a type of cancer that runs in the family.You need strong blood-thinning medication during the treatment period, but if you only take a lower dose for prevention, you can still participate.You have received an organ transplant from someone else in the past.You have previously received gene therapy treatment.You cannot receive a red blood cell transfusion.You have a severe form of sickle cell disease (SCD) and have experienced at least 4 very painful episodes in the 2 years before agreeing to participate.
Research Study Groups:
This trial has the following groups:- Group 1: Group A
- Group 2: Group B
- Group 3: Group C
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any vacancies for individuals looking to participate in this research?
"The information posted on clinicaltrials.gov reveals that this particular medical trial is currently not accepting any new patients; it was initially listed in August 2014 with the last update occurring July 15th 2022. Nevertheless, there are 197 alternative trials actively seeking enrolment at this time."
Answered by AI
Who is allowed to join the experiment?
"This research is looking to enrol 50 subjects, aged between 12 and 50 years old, with anemia due to sickle cell. Additionally, these individuals must have experienced four severe vaso-occlusive events in the two months prior to informed consent; or alternatively had difficulty continuing hydroxyurea (HU) treatment per their physician's judgement. Furthermore, they should possess a Karnofsky performance status of at least 60 if over 16 years of age and Lansky score of at least 60 for those below that threshold."
Answered by AI
Does the protocol of this investigation accept individuals aged twenty and above?
"In line with the conditions for inclusion, this trial has a minimum patient age of 12 and a maximum of 50."
Answered by AI
How many venues are partaking in this experiment?
"This research is currently taking place in 11 different sites. These locations include Hyde Park, Chicago and Charleston as well as 8 other facilities. To reduce inconveniences, it's best to choose the trial site closest to you if you decide to participate."
Answered by AI
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