Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study

Bellicum Pharmaceuticals

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Thalassemia; Anemia, Aplastic; Hemoglobinopathies; Diamond Blackfan Anemia; Anemia, Sickle Cell; Lymphoma, Non-Hodgkin; Myelodysplastic Syndromes; Primary Immunodeficiency; Leukemia, Acute Myeloid (AML), Child; Fanconi Anemia; Cytopenia; Acute Lymphoblastic Leukemia

Treatments: Drug: Rimiducid; Biological: rivogenlecleucel

Study type: Interventional

Funder types: Industry

Identifiers

NCT03733249

BP-404

Details and patient eligibility

About

This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.

Full description

Subjects enrolled on the BP-004 study who have completed or discontinued from the study, and are beyond Day 180 will be requested to enroll on this long-term follow up protocol. Long term follow up for gene therapy clinical and safety endpoints will continue up to 15 years.

Enrollment: 187 patients

Sex: All

Ages: 1 month to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Signed written informed consent by the patient or the patient's guardian for children who are minors
• Enrolled on BP-004 protocol, received BPX-501 infusion, completed or discontinued from the study, and are beyond Day +180.

Exclusion criteria

• Lack of parents'/guardian's informed consent for children who are minors
• Loss of allograft prior to 6 months

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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187 participants in 1 patient group

Rimiducid and Rivogenlecleucel

Experimental group

Description:

Rimiducid: to treat uncontrolled GVHD in patients who have received rivogenlecleucel Rimiducid will be given at 0.4 mg/kg weight (intravenous infusion) No further rivogenlecleucel infusions are planned. Patients who received rivogenlecleucel in the BP-004 study will be evaluated for long-term safety and efficacy.

Treatment:

Biological: rivogenlecleucel

Drug: Rimiducid