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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

Must not have

Female subject is breastfeeding, pregnant, or planning pregnancy

Unable to receive pRBC transfusion

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6-24 months post-transplant

Awards & highlights

Pivotal Trial

No Placebo-Only Group

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently pregnant, breastfeeding, or planning to become pregnant.

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I cannot receive red blood cell transfusions.

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My bone marrow is not working properly.

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I need blood-thinning medication from the start of my treatment until my platelet count recovers.

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I do not have any serious ongoing infections.

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A close family member has or might have a hereditary cancer syndrome.

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I have genetic mutations that affect my α-globin genes.

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My liver disease is in an advanced stage.

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I have severe blood vessel problems in my brain, including a stroke or significant narrowing/blockage.

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I have had an allogeneic transplant or gene therapy before.

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I am not allergic or unable to take plerixafor, busulfan, or similar medications.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6-24 months post-transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6-24 months post-transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6-24 months post-transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

VOE-CR

Secondary study objectives

Proportion of subjects achieving Globin Response

VOE-CR24

sVOE-75

+2 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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.

0 / 16Eligibility criteria met