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

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.

• Rimiducid (Drug), also known as AP1903: Rimiducid is administered to treat chronic graft versus host disease
• rivogenlecleucel (Biological), also known as BPX-501: donor T-cells modified with iCasp safety switch

Primary outcomes

• Overall Survival [1 and 2 years after rivogenlecleucel infusion]: Overall survival (OS) in both malignant and non-malignant subpopulations at 1 and 2 years in the Intent-to-Treat (ITT) Population
• Incidence of Disease-free Survival [1 and 2 years after rivogenlecleucel infusion]: 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
• Relapse-free Survival [1 and 2 years after rivogenlecleucel infusion]: 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

Sex: All, ages: 1 Month to 18 Years

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

Participant flow

Outcome results

• Overall Survival (Primary), Count of participants, Participants: Malignant 8; Non-malignant 3
• Incidence of Disease-free Survival (Primary), Count of participants, Participants: Non-malignant 4
• Relapse-free Survival (Primary), Count of participants, Participants: Malignant 12

Adverse events

Time frame: BP-404 is the long-term follow-up study of patients in the BP-004 study who were enrolled at the Italian and Saudi Arabia sites. Since a summary of AEs is not available for the long-term studies alone, the below AE data pertain to Study BP-004 (AEs assessed up to180 days post rivogenlecleucel administration for serious AEs (including all-cause mortality) and 30 days post rivogenlecleucel for non-serious AEs [30 and 7 days post rimiducid administration for serious/non-serious AEs respectively]).

Serious adverse events by term

Other adverse events by term

• Study Protocol (PDF, 2020-06-30)
• Statistical Analysis Plan (PDF, 2019-05-08)

• Bellicum Pharmaceuticals, Study director, Bellicum Pharmaceuticals