A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

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Recruiting
participants needed

35
sponsor

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Updated on 19 February 2024

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karnofsky performance status

analgesia

gene therapy

priapism

pediatric

Summary

This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects 2 and 50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) with LentiGlobin BB305 Drug Product for SCD.

Details

Condition	SICKLE CELL ANEMIA
Age	2-50 years
Treatment	LentiGlobin BB305 Drug Product for SCD
Clinical Study Identifier	NCT04293185
Sponsor	bluebird bio
Last Modified on	19 February 2024

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Eligibility		Yes	No	Not Sure
Inclusion Criteria
	Have a diagnosis of SCD, with either S/S, S/0 or S/+ genotype
	Be 2 and 50 years of age at time of consent
	Weigh a minimum of 6 kg
	Have a Karnofsky performance status of 60 (16 years of age) or a Lansky performance status of 60 (<16 years of age)
	Be 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
	Have severe manifestations of SCD. i.e. in the setting of appropriate supportive care measures (e.g., pain management plan), have experienced at least 4 severe VOEs in the 24 months prior to informed consent as defined below. For the purposes of this study, a severe VOE is defined as an event with no medically determined cause other than a vaso-occlusion, requiring a 24 -hour hospital or emergency room (ER) observation unit visit or at least 2 visits to a day unit or ER over 72 hours with both visits requiring intravenous treatment. Exception: priapism does not require hospital admission but does require a medical facility visit; 4 priapism episodes that require a visit to a medical facility (without inpatient admission) are sufficient to meet criterion
	Have either experienced HU failure at any point in the past or must have intolerance to HU (intolerance is defined as the patient being unable to continue to take HU per PI judgment)
Exclusion Criteria
	Applicable to subjects <18 years of age only: Availability of a willing, matched human leukocyte antigen (HLA)-identical sibling HSC donor
	Severe cerebral vasculopathy, defined by any history of: overt ischemic or hemorrhagic stroke, abnormal transcranial Doppler (>200 cm/sec based on central read) requiring chronic transfusion, occlusion or stenosis in the circle of Willis, or presence of Moyamoya disease
	Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B, hepatitis C, human T-lymphotrophic virus-1 (HTLV-1) or -2 (HTLV-2), active syphilis
	Clinically significant, active bacterial, viral, fungal, or parasitic infection
	Advanced liver disease, such as
	clear evidence of liver cirrhosis, active hepatitis or significant fibrosis (based on MRI or liver biopsy)
	liver iron concentration 15 mg/g unless liver biopsy shows no evidence of cirrhosis, active hepatitis or significant fibrosis
	Inadequate bone marrow function, as defined by an absolute neutrophil count of <110^9/L (<0.510^9/L for subjects on hydroxyurea treatment) or a platelet count <10010^9/L
	Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients
	Patients needing therapeutic anticoagulation treatment during the period of conditioning through platelet engraftment
	Unable to receive red blood cell (RBC) transfusion
	Prior receipt of an allogeneic HSC transplant
	Prior receipt of gene therapy
	Any prior or current malignancy or immunodeficiency disorder, except previously treated, non-life threatening, cured tumors such as squamous cell carcinoma of the skin
	Immediate family member with a known or suspected Familial Cancer Syndrome
	Pregnancy, or breastfeeding in a postpartum female, or absence of adequate contraception for fertile subjects
	Any other condition that would render the subject ineligible for HSCT
	Participation in another clinical study with an investigational drug within 30 days of screening

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A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease