Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
Primary Purpose
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin
Status
Terminated
Phase
Phase 1
Locations
Italy
Study Type
Interventional
Intervention
Rimiducid
rivogenlecleucel
Sponsored by
About this trial
This is an interventional treatment trial for Acute Lymphoblastic Leukemia focused on measuring Long term follow up, Gene-modified cells, ALL, AML, hematologic neoplasms, hematologic malignancies, anemia, congenital cytopenia, primary immune deficiencies, hemoglobinopathy
Eligibility Criteria
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
Sites / Locations
- IRCCS Ospedale Pediatrico Bambino Gesù
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Rimiducid and Rivogenlecleucel
Arm 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.
Outcomes
Primary Outcome Measures
Overall Survival
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
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
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
Secondary Outcome Measures
Full Information
NCT ID
NCT03733249
First Posted
November 2, 2018
Last Updated
September 22, 2023
Sponsor
Bellicum Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT03733249
Brief Title
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Terminated
Study Start Date
January 2017 (Actual)
Primary Completion Date
June 30, 2020 (Actual)
Study Completion Date
April 14, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bellicum Pharmaceuticals
4. Oversight
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
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
Keywords
Long term follow up, Gene-modified cells, ALL, AML, hematologic neoplasms, hematologic malignancies, anemia, congenital cytopenia, primary immune deficiencies, hemoglobinopathy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
187 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Rimiducid and Rivogenlecleucel
Arm Type
Experimental
Arm 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.
Intervention Type
Drug
Intervention Name(s)
Rimiducid
Other Intervention Name(s)
AP1903
Intervention Description
Rimiducid is administered to treat chronic graft versus host disease
Intervention Type
Biological
Intervention Name(s)
rivogenlecleucel
Other Intervention Name(s)
BPX-501
Intervention Description
donor T-cells modified with iCasp safety switch
Primary Outcome Measure Information:
Title
Overall Survival
Description
Overall survival (OS) in both malignant and non-malignant subpopulations at 1 and 2 years in the Intent-to-Treat (ITT) Population
Time Frame
1 and 2 years after rivogenlecleucel infusion
Title
Incidence of Disease-free Survival
Description
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
Time Frame
1 and 2 years after rivogenlecleucel infusion
Title
Relapse-free Survival
Description
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
Time Frame
1 and 2 years after rivogenlecleucel infusion
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Month
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bellicum Pharmaceuticals
Organizational Affiliation
Bellicum Pharmaceuticals, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
IRCCS Ospedale Pediatrico Bambino Gesù
City
Rome
ZIP/Postal Code
00161
Country
Italy
12. IPD Sharing Statement
Learn more about this trial
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
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