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

NCT03733249 | Terminated Phase 1 Results FDA Drug

• 1 other identifier: BP-404
• Study Type: interventional
• Target: 187
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (1)

Rimiducid and Rivogenlecleucel

EXPERIMENTAL

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; Biological: rivogenlecleucel

Interventions

Rimiducid DRUG

Rimiducid is administered to treat chronic graft versus host disease

Also known as: AP1903

Rimiducid and Rivogenlecleucel

rivogenlecleucel BIOLOGICAL

donor T-cells modified with iCasp safety switch

Also known as: BPX-501

Rimiducid and Rivogenlecleucel

Eligibility Criteria

• Age: 1 Month - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• Lack of parents'/guardian's informed consent for children who are minors
• Loss of allograft prior to 6 months

Sponsors & Collaborators

• Bellicum Pharmaceuticals: lead

Study Sites (1)

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, 00161, Italy

Location

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia; Leukemia, Myeloid, Acute; Lymphoma, Non-Hodgkin; Myelodysplastic Syndromes; Primary Immunodeficiency Diseases; Anemia, Aplastic; Hemoglobinopathies; Cytopenia; Fanconi Anemia; Anemia, Diamond-Blackfan; Thalassemia; Anemia, Sickle Cell; Hematologic Neoplasms; Anemia

Interventions

AP 1903 reagent

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, Myeloid; Lymphoma; Bone Marrow Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Bone Marrow Failure Disorders; Anemia, Hypoplastic, Congenital; Congenital Bone Marrow Failure Syndromes; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Red-Cell Aplasia, Pure; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Neoplasms by Site

Results Point of Contact

• Title: Rivogenlecleucel Study Team
• Organization: Bellicum Pharmaceuticals

clinicaltrials@bellicum.com

(832) 384-1100

Study Officials

• Bellicum Pharmaceuticals

  Bellicum Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2018
• First Posted: November 7, 2018
• Study Start: January 1, 2017
• Primary Completion: June 30, 2020
• Study Completion: April 14, 2023
• Last Updated: September 26, 2023
• Results First Posted: September 26, 2023

Record last verified: 2023-09

Locations

IT (1)