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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders

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

Intervention / Treatment
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* Drug: Rimiducid
* Biological: rivogenlecleucel


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 1 Month
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Rimiducid and Rivogenlecleucel<br>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. | Drug: Rimiducid<br>* Rimiducid is administered to treat chronic graft versus host disease<br>* Other names: AP1903;Biological: rivogenlecleucel<br>* donor T-cells modified with iCasp safety switch<br>* Other names: BPX-501;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Survival | Overall survival (OS) in both malignant and non-malignant subpopulations at 1 and 2 years in the Intent-to-Treat (ITT) Population | 1 and 2 years after rivogenlecleucel infusion | 
| Incidence of Disease-free Survival | KM Parameter Estimates of disease-free survival (DFS) in the non-malignant subpopulation at 1 and 2 years in the Intent-to-Treat (ITT) Population | 1 and 2 years after rivogenlecleucel infusion | 
| Relapse-free Survival | Kaplan-Meier Parameter Estimates of Relapse-free survival rate (number of patients survived without experiencing a recurrence) at the 1-year and 2-year timepoints in the Intent-to-Treat (ITT) Population in the malignant study arm (patients with a malignant reason for their transplant). ITT Population: Includes all patients treated with HSCT who received rivogenlecleucel at the dose of 1×10E6 cells/kg | 1 and 2 years after rivogenlecleucel infusion | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALL, Long term follow up, Gene-modified cells, AML, hematologic neoplasms, hematologic malignancies, anemia, congenital cytopenia, primary immune deficiencies, hemoglobinopathy