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

Myelodysplastic Syndromes Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03733249
• Other study ID #: BP-404
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: January 2017
• Est. completion date: April 14, 2023

Study information

• Verified date: September 2023
• Source: Bellicum Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 187
• Est. completion date: April 14, 2023
• Est. primary completion date: June 30, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month to 18 Years

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Drug:
• Rimiducid
Rimiducid is administered to treat chronic graft versus host disease

Biological:
• rivogenlecleucel
donor T-cells modified with iCasp safety switch

Locations

Country	Name	City	State
Italy	IRCCS Ospedale Pediatrico Bambino Gesù	Rome

Sponsors (1)

Lead Sponsor	Collaborator
Bellicum Pharmaceuticals

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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