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

Musculoskeletal Diseases

Skin and Connective Tissue Diseases

Osteopetrosis

Osteosclerosis

Osteochondrodysplasias

Mucopolysaccharidosis IV

Bone Diseases

Bone Diseases, Developmental

Mucopolysaccharidosis Type IV

Hematinics

All Drugs and Chemicals

Liver Extracts

TCR alpha beta depleted graft infusion with addback of BPX-501 T cells (rivogenlecleucel).

Rimiducid/AP1903: Dimerizer drug administered to subjects who develop Grade III-IV acute GVHD, Grade II gut/liver acute GVDH or Grade I/II skin-only acute GvHD which is non-responsive after 7 days of standard of care treatment

BPX-501 T cells

This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (Graft versus host disease).

This is a Phase I/II study evaluating the safety and feasibility of BPX-501 T cells infused after partially mismatched, related, TCR alpha beta T cell depleted hematopoietic stem cell transplant (HSCT) in pediatric patients. The purpose of this clinical trial is to determine whether BPX-501 infusion can enhance immune reconstitution in those patients with hematologic disorders, with the potential for reducing the severity and duration severe acute graft versus host disease (GvHD).

The trial will also evaluate the treatment of GvHD by the infusion of dimerizer drug (AP1903/rimiducid) in those subjects who present with GVHD who progress or do not respond to standard of care treatment.

Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant

Phase I/II Study of CaspaCIDe T Cells (BPX-501; Rivogenlecleucel) From an HLA Partially Matched Family Donor After Negative Selection of TCRαβ+ T Cells in Paediatric Patients Affected by Haematological Disorders

Events included transplant-related mortality (TRM) / non-relapse mortality (NRM), severe GvHD (acute Grades 2-4 organ or extensive chronic GvHD) and life-threatening infections (Grade 4). Time to the first event only is represented in the primary endpoint, if a subsequent event occurred in the same patient this was not captured in this outcome.

There were no protocol-specified primary endpoints for Phase I of the study.

Analysis of All Cause Mortality was performed with the HSCT Safety Population (all patients who received a HSCT).

Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received any dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)

BPX-501 (rivogenlecleucel): TCR alpha beta depleted graft infusion with addback of BPX-501 T cells

BPX-501 T cells: 1x10E6 cells/kg infused on Day 0

BPX-501

Rimiducid/AP1903: Dimerizer drug administered to subjects who develop Grade III-IV acute GVHD, Grade II gut/liver acute GVDH or Grade I/II skin-only acute GvHD which is non-responsive after 7 days of standard of care treatment

Rimiducid: 0.4mg/kg administered IV to treat GVHD

TCR alpha beta depleted graft infusion with addback of BPX-501 T cells (rivogenlecleucel).

Rimiducid/AP1903: Dimerizer drug administered to subjects who develop Grade III-IV acute GVHD, Grade II gut/liver acute GVDH or Grade I/II skin-only acute GvHD which is non-responsive after 7 days of standard of care treatment

BPX-501 T cells: 1x10E6 cells/kg infused on Day 0

Rimiducid: 0.4mg/kg administered IV to treat GVHD

BPX-501 T Cells and Rimiducid

One patient was enrolled under a protocol version for which the inclusion criterion was \<= 21 years and ≥ 3 months. The age range was revised in a subsequent protocol to \< 18 years and ≥ 1 month (\< 1 month upon approval by Sponsor).

All enrolled patients iwho received HSCT (HSCT safety population)

ITT population: All patients treated with HSCT who received 1×10\^6 cells/kg BPX-501

Event-free Survival (EFS) at 180 Days After Transplant

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
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
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant	NCT02065869	Acute Lymphobla... Leukemia, Acute... Lymphoma, Non-H... Myelodysplastic... Primary Immunod... Anemia, Aplasti... Osteopetrosis Hemoglobinopath... Cytopenia Fanconi Anemia Diamond Blackfa... Thalassemia Anemia, Sickle ...	BPX-501 T cells Rimiducid	1 Month - 18 Years	Bellicum Pharmaceuticals
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant	NCT02065869	Acute Lymphobla... Leukemia, Acute... Lymphoma, Non-H... Myelodysplastic... Primary Immunod... Anemia, Aplasti... Osteopetrosis Hemoglobinopath... Cytopenia Fanconi Anemia Diamond Blackfa... Thalassemia Anemia, Sickle ...	BPX-501 T cells Rimiducid	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
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant	NCT02065869	Acute Lymphobla... Leukemia, Acute... Lymphoma, Non-H... Myelodysplastic... Primary Immunod... Anemia, Aplasti... Osteopetrosis Hemoglobinopath... Cytopenia Fanconi Anemia Diamond Blackfa... Thalassemia Anemia, Sickle ...	BPX-501 T cells Rimiducid	1 Month - 18 Years	Bellicum Pharmaceuticals

Spots Global Cancer Trial Database for anemia, sickle cell