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

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

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.

Study Overview

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

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 Type: Interventional
• Enrollment (Actual): 187
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Italy
  • Rome, Italy, 00161
    • IRCCS Ospedale Pediatrico Bambino Gesu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse events	Incidence of delayed adverse events suspected to be related to rivogenlecleucel or rimiducid	Up to 15 years after rivogenlecleucel infusion
Rimiducid Pharmacokinetics	Assessment of PK (rimiducid) and PD (BPX-501) profiles after treatment with rimiducid for aGVHD.	Up to 15 years after rivogenlecleucel infusion

Collaborators and Investigators

• Sponsor: Bellicum Pharmaceuticals
• Investigators: Study Director: Bellicum Pharmaceuticals, Bellicum Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2017
• Primary Completion (Actual): June 30, 2020
• Study Completion (Actual): April 14, 2023

Study Registration Dates

• First Submitted: November 2, 2018
• First Submitted That Met QC Criteria: November 5, 2018
• First Posted (Actual): November 7, 2018

Study Record Updates

• Last Update Posted (Actual): September 26, 2023
• Last Update Submitted That Met QC Criteria: September 22, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: AML; anemia; ALL; hematologic malignancies; Long term follow up; Gene-modified cells; hematologic neoplasms; congenital cytopenia; primary immune deficiencies; hemoglobinopathy
• Additional Relevant MeSH Terms: Metabolic Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Genetic Diseases, Inborn; DNA Repair-Deficiency Disorders; Leukemia, Lymphoid; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia, Hypoplastic, Congenital; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Lymphoma; Leukemia, Myeloid; Red-Cell Aplasia, Pure; Myelodysplastic Syndromes; Leukemia; Leukemia, Myeloid, Acute; Lymphoma, Non-Hodgkin; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Primary Immunodeficiency Diseases; Anemia; Anemia, Sickle Cell; Fanconi Anemia; Thalassemia; Anemia, Aplastic; Hemoglobinopathies; Anemia, Diamond-Blackfan
• Other Study ID Numbers: BP-404

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes