Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Diseases and Abnormalities at or Before Birth

Immune System Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Nutritional and Metabolic Diseases

Rare Diseases

Syndrome

Leukemia

Preleukemia

Myelodysplastic Syndromes

Cytopenia

Anemia, Sickle Cell

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Anemia

Lymphoma

Lymphoma, Non-Hodgkin

Thalassemia

Hematologic Neoplasms

Immunologic Deficiency Syndromes

Anemia, Aplastic

Hemoglobinopathies

Primary Immunodeficiency Diseases

Hematologic Diseases

Fanconi Syndrome

Fanconi Anemia

Anemia, Diamond-Blackfan

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Neoplasms by Histologic Type

Bone Marrow Diseases

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Hemolysis

Anemia, Hemolytic

Anemia, Hemolytic, Congenital

Genetic Diseases, Inborn

Anemia, Hypoplastic, Congenital

Bone Marrow Failure Disorders

Pancytopenia

Congenital Bone Marrow Failure Syndromes

DNA Repair-Deficiency Disorders

Metabolic Diseases

Red-Cell Aplasia, Pure

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Lymphosarcoma

Acute Graft Versus Host Disease

Congenital Aplastic Anemia

Diamond-Blackfan Anemia

Pure Red Cell Aplasia

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 is administered to treat chronic graft versus host disease

Rimiducid

donor T-cells modified with iCasp safety switch

rivogenlecleucel

Bellicum Pharmaceuticals

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.

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.

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

Overall survival (OS) in both malignant and non-malignant subpopulations at 1 and 2 years in the Intent-to-Treat (ITT) Population

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

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

All patients who received HSCT and went on to receive any dose of rivogenlecleucel.

Rivogenlecleucel (BPX-501) Safety Population

All patients who received at least 1 dose of rimiducid for the treatment of acute or chronic GvHD refractory to SoC treatment after rivogenlecleucel administration.

Rimiducid Safety Population

Rimiducid and Rivogenlecleucel

Subject eligibility criteria: patients less than or equal to 18 years of age and greater than or equal to 1 month of age.

Age, Continuous

< 2 years

2 to < 12 years

12 to ≤ 18 years

18 to ≤ 26 years

Age, Customized

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

HSCT safety population: All enrolled patients who received hematopoietic stem cell transplantation

Rivogenlecleucel Study Team

1 Year

2 Years

Malignant

Non-malignant

ITT Population: Includes all patients treated with HSCT who received rivogenlecleucel at the dose of 1×10E6 cells/kg

Study BP-404 is the long-term follow-up study of patients of the BP-004 study who were enrolled at the Italian and Saudi Arabia sites. There is a second long-term follow-up study (BP-004 UK) for patients enrolled at the UK sites. The below results include data from both the BP-404 study and the BP-004 UK study. Individual results for either study are not available.

Overall Survival

Incidence of Disease-free Survival

Relapse-free Survival

Overall Study

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study	NCT03733249	Acute Lymphobla... Leukemia, Acute... Lymphoma, Non-H... Myelodysplastic... Primary Immunod... Anemia, Aplasti... Hemoglobinopath... Cytopenia Fanconi Anemia Diamond Blackfa... Thalassemia Anemia, Sickle ...	Rimiducid rivogenlecleuce...	1 Month - 18 Years	Bellicum Pharmaceuticals
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study	NCT03733249	Acute Lymphobla... Leukemia, Acute... Lymphoma, Non-H... Myelodysplastic... Primary Immunod... Anemia, Aplasti... Hemoglobinopath... Cytopenia Fanconi Anemia Diamond Blackfa... Thalassemia Anemia, Sickle ...	Rimiducid rivogenlecleuce...	1 Month - 18 Years	Bellicum Pharmaceuticals

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