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

Phase 1

Terminated

• Conditions: Acute Lymphoblastic Leukemia; Leukemia, Acute Myeloid (AML), Child; Lymphoma, Non-Hodgkin; Myelodysplastic Syndromes; Primary Immunodeficiency; Thalassemia; Hemoglobinopathies; Fanconi Anemia; Diamond Blackfan Anemia; Anemia, Sickle Cell
• Interventions: Drug: Rimiducid; Biological: rivogenlecleucel

• Registration Number: NCT03733249
• Lead Sponsor: Bellicum Pharmaceuticals

Brief Summary

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

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.

Study Timeline

• Study Start: 2017-01
• Study First Submitted: 2018-11-02
• Study First Submitted QC: 2018-11-05
• Study First Posted: 2018-11-07
• Primary Completion: 2020-06-30
• Study Completion: 2023-04-14
• Results First Submitted: 2023-06-22
• Status Verified: 2023-09
• Results First Submitted QC: 2023-09-22
• Last Update Submitted: 2023-09-22
• Results First Posted: 2023-09-26
• Last Update Posted: 2023-09-26

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 187

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Rimiducid and Rivogenlecleucel	rivogenlecleucel	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.
Rimiducid and Rivogenlecleucel	Rimiducid	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.

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

IRCCS Ospedale Pediatrico Bambino Gesù; 🇮🇹 Rome, Italy