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Gene Therapy

Gene Therapy for Sickle Cell Disease

Phase 3
Recruiting
Research Sponsored by bluebird bio
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Karnofsky performance status of ≥60 (≥16 years of age) or Lansky performance status of ≥60 (<16 years of age)
Treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on sickle cell disease history
Timeline
Screening 3 weeks
Treatment Varies
Follow Up month 6, 12, 18 and 24 post-transplant
Awards & highlights

Study Summary

This trial is studying a gene therapy for sickle cell disease. The FDA has put a partial clinical hold on the trial for patients less than 18 years of age, which means that no new enrollment, cell collection, conditioning, or drug product infusion can be initiated in pediatric patients. The study is still open to patients 18 years of age and older.

Who is the study for?
This trial is for individuals aged 2 to 50 with sickle cell disease (SCD), who have had at least four pain episodes in the last two years and either failed hydroxyurea treatment or can't tolerate it. Participants must weigh over 6 kg, have a certain level of physical ability, and agree to use contraception if applicable. Those with inadequate bone marrow function, prior malignancies, genetic risks for other blood disorders, or severe cerebral vasculopathy cannot join.Check my eligibility
What is being tested?
The study tests a gene therapy called bb1111 in about 35 people with SCD. It's an open-label Phase 3 trial where participants receive one dose of the therapy through hematopoietic stem cell transplantation (HSCT). The goal is to see how well this new treatment works compared to traditional treatments.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include those typical of HSCT such as infection risk due to low white blood cells, bleeding issues from low platelets, reactions from drug infusion like fever or chills, and possible organ damage related to the conditioning regimen used before transplant.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am mostly able to care for myself and carry out daily activities.
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I have been treated for sickle cell disease and followed at a medical center with detailed records for the past 2 years.
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I have had a bad reaction to or did not respond to hydroxyurea.
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I have sickle cell disease with a specific genetic makeup.
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I am between 2 and 50 years old.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month 6, 12, 18 and 24 post-transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and month 6, 12, 18 and 24 post-transplant for reporting.

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
Average and median of HbS percentage of non-transfused total Hb
Average and median of non-HbS percentage of non-transfused total Hb
+31 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: bb1111Experimental Treatment1 Intervention
Subjects will receive treatment with a single dose of Drug Product manufactured with autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and apheresis, transduced with BB305 lentiviral vector (LVV) encoding the human beta-A-T87Q globin gene. Plerixafor mobilization and apheresis will also be used for collection of rescue cells.
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
2,008 Total Patients Enrolled
Richard Colvin, MDStudy Directorbluebird bio, Inc.
1 Previous Clinical Trials
50 Total Patients Enrolled
Anjulika Chawla, MD, FAAPStudy Directorbluebird bio, Inc.
2 Previous Clinical Trials
135 Total Patients Enrolled

Media Library

bb1111 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04293185 — Phase 3
Sickle Cell Disease Research Study Groups: bb1111
Sickle Cell Disease Clinical Trial 2023: bb1111 Highlights & Side Effects. Trial Name: NCT04293185 — Phase 3
bb1111 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04293185 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this research only involve elderly patients?

"This particular study is only enrolling patients that are aged 2-50. In contrast, there are 133 clinical trials for those under 18 and 85 for senior citizens."

Answered by AI

How many individuals are enrolled in this study currently?

"The sponsor, bluebird bio, needs to recruit 35 patients that meet the clinical trial's inclusion criteria in order to operate the study. The trial will be conducted out of various locations including Virginia Commonwealth University (VCU) in Richmond, Virginia and University of Minnesota in Minneapolis, Minnesota."

Answered by AI

Who makes for an ideal candidate to test this new medication?

"This trial is currently seeking 35 young patients, aged 2-50, that suffer from anemia and sickle cell. Eligible applicants must have experienced a minimum of 4 protocol-defined VOEs in the 24 months prior to informed consent, have either experienced HU failure at any point in the past or be intolerant to HU, agree to use 1 method of highly effective contraception from Screening to at least 6 months after drug product infusion, have a diagnosis of SCD with βS/βS, βS/β0, or βS/β+ genotype,, weigh a minimum of 6 kg., have a Karn"

Answered by AI

Is this research being conducted in multiple states across America?

"In addition to Virginia Commonwealth University (VCU) in Richmond, Virginia and the University of Minnesota in Minneapolis, Minnesota; this study is also enrolling patients at Fred Hutchinson Cancer Research Center in Seattle, Washington as well as 8 other locations."

Answered by AI

What are the short-term and long-term effects of bb1111 on human health?

"There is some efficacy data and multiple rounds of safety data, so the Power team gave bb1111 a score of 3."

Answered by AI

Are there any more patients needed for this clinical trial?

"The clinicaltrials.gov website says that this study is still looking for patients. This trial was first announced on February 14th, 2020 and received its most recent update on July 15th, 2022. In total, the study needs 35 enrollees from 8 different hospitals or medical facilities."

Answered by AI

Who else is applying?

What site did they apply to?
University of Alabama
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
2020. "A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04293185.
All > Sickle Cell Disease > Sickle Cell Anemia
~15 spots leftby May 2027
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