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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rimiducid | Drug | Rimiducid is administered to treat chronic graft versus host disease |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Bellicum Pharmaceuticals | Bellicum Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| IRCCS Ospedale Pediatrico Bambino Gesù | Rome | 00161 | Italy |
3 Enrolled patients did not undergo hematopoietic stem cell transplantation (HSCT)
Study BP-404 was the long-term follow-up for patients enrolled in the BP-004 study at the Italian site as well as patients in Saudi Arabia (patients enrolled and treated in the UK who moved back after hematopoietic stem cell transplantation. The below participant flow information pertains to all patients in the BP-004 study (NCT02065869)
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| ID | Title | Description |
|---|---|---|
| FG000 | Rimiducid and Rivogenlecleucel | 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. |
| Title | Milestones | Reasons Not Completed | ||||
|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 30, 2020 | Jun 22, 2023 |
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| rivogenlecleucel | Biological | donor T-cells modified with iCasp safety switch |
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| Patients Receiving Rivogenlecleucel (BPX-501 Safety Population) |
|
| Intent-to-Treat Population | Includes all patients treated with HSCT who received rivogenlecleucel at the dose of 1×10E6 cells/kg. |
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| Patients Receiving at Least 1 Dose of Rimiducid |
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| COMPLETED |
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| NOT COMPLETED |
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HSCT safety population: All enrolled patients who received hematopoietic stem cell transplantation
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| ID | Title | Description |
|---|---|---|
| BG000 | Rimiducid and Rivogenlecleucel | 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. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Subject eligibility criteria: patients less than or equal to 18 years of age and greater than or equal to 1 month of age. | Median | Full Range | years |
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| Age, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 | 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. | Posted | Count of Participants | Participants | 1 and 2 years after rivogenlecleucel infusion |
|
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| |||||||||||||||||||||||||||||||
| 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 | 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. | Posted | Count of Participants | Participants | 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 | 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. | Posted | Count of Participants | Participants | 1 and 2 years after rivogenlecleucel infusion |
|
|
BP-404 is the long-term follow-up study of patients in the BP-004 study who were enrolled at the Italian and Saudi Arabia sites. Since a summary of AEs is not available for the long-term studies alone, the below AE data pertain to Study BP-004 (AEs assessed up to180 days post rivogenlecleucel administration for serious AEs (including all-cause mortality) and 30 days post rivogenlecleucel for non-serious AEs [30 and 7 days post rimiducid administration for serious/non-serious AEs respectively]).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Rivogenlecleucel (BPX-501) Safety Population | All patients who received HSCT and went on to receive any dose of rivogenlecleucel. | 7 | 171 | 52 | 171 | 140 | 171 |
| EG001 | Rimiducid 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. | 0 | 16 | 7 | 16 | 9 | 16 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | MedDRA (18.0) | Systematic Assessment |
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| Cytopenia | Blood and lymphatic system disorders | MedDRA (18.0) | Systematic Assessment |
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| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA (18.0) | Systematic Assessment |
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| Histiocytosis haematophagic | Blood and lymphatic system disorders | MedDRA (18.0) | Systematic Assessment |
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| Neutropenia | Blood and lymphatic system disorders | MedDRA (18.0) | Systematic Assessment |
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| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (18.0) | Systematic Assessment |
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| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA (18.0) | Systematic Assessment |
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| Pericardial effusion | Cardiac disorders | MedDRA (18.0) | Systematic Assessment |
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| Pneumopericardium | Cardiac disorders | MedDRA (18.0) | Systematic Assessment |
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| Diplopia | Eye disorders | MedDRA (18.0) | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Haematemesis | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Pancreatitis | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Parotid gland enlargement | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Device damage | General disorders | MedDRA (18.0) | Systematic Assessment |
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| Malaise | General disorders | MedDRA (18.0) | Systematic Assessment |
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| Medical device complication | General disorders | MedDRA (18.0) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (18.0) | Systematic Assessment |
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| Acute graft versus host disease | Immune system disorders | MedDRA (18.0) | Systematic Assessment |
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| Adenovirus infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Bacterial sepsis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Cytomegalovirus infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Device related infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Encephalitis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Escherichia infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Gastroenteritis rotavirus | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Klebsiella infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Pharyngitis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Pseudomonas infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Respiratory tract infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Sepsis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Septic shock | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Staphylococcal bacteraemia | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Staphylococcal infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Staphylococcal sepsis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Varicella zoster virus infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
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| Juvenile chronic myelomonocytic leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (18.0) | Systematic Assessment |
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| Central nervous system lesion | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
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| Encephalopathy | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
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| Miller Fisher syndrome | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
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| Posterior reversible encephalopathy syndrome | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
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| Cystitis haemorrhagic | Renal and urinary disorders | MedDRA (18.0) | Systematic Assessment |
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| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Cardiopulmonary failure | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Hypotension | Vascular disorders | MedDRA (18.0) | Systematic Assessment |
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| Pelvic venous thrombosis | Vascular disorders | MedDRA (18.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cytomegalovirus infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Adenovirus infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Human herpesvirus 6 infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Acute graft versus host disease | Immune system disorders | MedDRA (18.0) | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Systematic Assessment |
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| Diarrhea | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (18.0) | Systematic Assessment |
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| Chronic graft versus host disease | Immune system disorders | MedDRA (18.0) | Systematic Assessment |
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| Sepsis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Septic shock | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Staphylococcal infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
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| Gastrointestinal hemorrhage | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Parotid gland enlargement | Gastrointestinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Central nervous system lesion | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
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| Encephalopathy | Nervous system disorders | MedDRA (18.0) | Systematic Assessment |
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| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
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| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Right ventricular failure | Cardiac disorders | MedDRA (18.0) | Systematic Assessment |
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| Hypokalemia | Metabolism and nutrition disorders | MedDRA (18.0) | Systematic Assessment |
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| Confusional state | Psychiatric disorders | MedDRA (18.0) | Systematic Assessment |
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| Skin discoloration | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Rivogenlecleucel Study Team | Bellicum Pharmaceuticals | (832) 384-1100 | clinicaltrials@bellicum.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 8, 2019 | Sep 12, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D007938 | Leukemia |
| D015470 | Leukemia, Myeloid, Acute |
| D008228 | Lymphoma, Non-Hodgkin |
| D009190 | Myelodysplastic Syndromes |
| D000081207 | Primary Immunodeficiency Diseases |
| D000741 | Anemia, Aplastic |
| D006453 | Hemoglobinopathies |
| D000095542 | Cytopenia |
| D005199 | Fanconi Anemia |
| D029503 | Anemia, Diamond-Blackfan |
| D013789 | Thalassemia |
| D000755 | Anemia, Sickle Cell |
| D019337 | Hematologic Neoplasms |
| D000740 | Anemia |
| ID | Term |
|---|---|
| D007945 | Leukemia, Lymphoid |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D007951 | Leukemia, Myeloid |
| D008223 | Lymphoma |
| D001855 | Bone Marrow Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007153 | Immunologic Deficiency Syndromes |
| D000080983 | Bone Marrow Failure Disorders |
| D029502 | Anemia, Hypoplastic, Congenital |
| D000080984 | Congenital Bone Marrow Failure Syndromes |
| D049914 | DNA Repair-Deficiency Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D012010 | Red-Cell Aplasia, Pure |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D009371 | Neoplasms by Site |
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| ID | Term |
|---|---|
| C423866 | AP 1903 reagent |
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| 12 to ≤ 18 years |
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| 18 to ≤ 26 years |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
|
| Unknown or Not Reported |
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| At Risk [Defined as: Total participants - (Event Occurred + Censored)] |
|
| 2 Years |
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