Not provided
Not provided
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Primary endpoint would unlikely to be met based on the unplanned interim assessment on the first 20 efficacy evaluable patients.
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This is a prospective, open-label, single-arm study evaluating the safety and efficacy of defibrotide for the prevention of CAR-T-associated neurotoxicity in subjects with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) receiving Yescarta.
Not provided
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Defibrotide | Experimental | Part 1 (lead-in phase) will evaluate a 2.5 mg/kg/dose regimen before escalating to a 6.25 mg/kg/dose regimen. After the Safety Assessment Committee establishes the recommended phase 2 dose based on dose-limiting toxicities during Part 1, Part 2 will enroll subjects at the recommended phase 2 dose. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Defibrotide | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of CAR-T-associated Neurotoxicity of Any Grade, Defined by CTCAE v5.0 by CAR-T Day +30 | The primary efficacy endpoint was the incidence of CAR-T-associated neurotoxicity of any grade defined by CTCAE v5.0 by CAR-T Day +30. The results report the estimated percentage of participants with no CAR-T-associated neurotoxicity of any grade, defined by CTCAE v5.0, which incorporated the 2 stage design. | By CAR-T Day +30 |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of CAR-T-Associated Neurotoxicity Grade 3 or Greater Defined by CTCAE v5.0 by CAR-T Day +30 | The secondary efficacy endpoint was the incidence of CAR-T-associated neurotoxicity Grade 3 or greater defined by CTCAE v5.0 by CAR-T Day +30. The results report the estimated percentage of participants with no CAR-T-associated neurotoxicity (Grade 3 or greater defined by CTCAE v5.0) by CAR-T Day +30. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic | Phoenix | Arizona | 85054 | United States | ||
| University of Maryland |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37399456 | Derived | Jacobson CA, Rosenthal AC, Arnason J, Agarwal S, Zhang P, Wu W, Amber V, Yared JA. A phase 2 trial of defibrotide for the prevention of chimeric antigen receptor T-cell-associated neurotoxicity syndrome. Blood Adv. 2023 Nov 14;7(21):6790-6799. doi: 10.1182/bloodadvances.2023009961. | |
| 33750072 | Derived | Danish H, Santomasso BD. Neurotoxicity Biology and Management. Cancer J. 2021 Mar-Apr 01;27(2):126-133. doi: 10.1097/PPO.0000000000000507. |
Not provided
Not provided
Patient disposition was assessed in the Safety Analysis Set (N=25) which was comprised of the Part 1: Defibrotide 2.5 mg/kg/dose (n=4) and the combined Part 1: Defibrotide 6.25 mg/kg/dose and Phase 2: RP2D 6.25 mg/kg/dose (n=21). The patient disposition is reported in this format as per the Statistical Analysis Plan.
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Part 1: Safety Lead-In, Defibrotide 2.5 mg/kg/Dose | Participants who received the safety (lead in) dose of 2.5 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part 1: Safety Lead In Phase |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 9, 2019 | Sep 22, 2021 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| By CAR-T Day +30 |
| Incidence of CAR-T-Associated Neurotoxicity of Any Grade According to the ASBMT Consensus Grading System by CAR-T Day +30 | The secondary efficacy endpoint was the incidence of CAR-T-associated neurotoxicity of any grade according to ASBMT consensus grading system by CAR-T Day +30. The results report the estimated percentage of participants with no CAR-T-associated neurotoxicity by CAR-T Day +30 summarized descriptively by any grade according to the ASBMT consensus grading system. | By CAR-T Day +30 |
| Incidence of CAR-T-Associated Neurotoxicity of Grade 3 or Greater According to the ASBMT Consensus Grading System by CAR-T Day +30 | The secondary efficacy endpoint was the incidence of CAR-T-associated neurotoxicity grade 3 or greater according to ASBMT consensus grading system by CAR-T Day +30. The results report the estimated percentage of participants with no CAR-T-associated neurotoxicity by CAR-T Day +30 summarized descriptively by grade 3 or greater according to the ASBMT consensus grading system. | By CAR-T Day +30 |
| Incidence of Cytokine Release Syndrome (CRS) of Any Grade According to the ASBMT Consensus Grading System by CAR-T Day +30 | The secondary efficacy endpoint was the incidence of cytokine release syndrome (CRS) of any grade according to ASBMT consensus grading system by CAR-T Day +30. The results report the estimated percentage of participants with no CRS any grade according to ASBMT criteria and was summarized descriptively using the ASBMT consensus grading system by CAR-T Day +30. | By CAR-T Day +30 |
| Use of High Dose Steroid By CAR-T Day +30 | The percentage of participants using high dose steroids was summarized descriptively. The use of high dose steroids was defined as a dose of dexamethasone of at least 7.5 mg/day or equivalent. Only the Overall Defibrotide: 6.25 mg/kg/dose group was analyzed for this outcome. | By CAR-T Day +30 |
| Baltimore |
| Maryland |
| 21201 |
| United States |
| Beth Israel Deaconess Medical Center | Boston | Massachusetts | 02215 | United States |
| Dana Farber Cancer Institute | Boston | Massachusetts | 02215 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Phase 2: RP2D, Defibrotide 6.25 mg/kg/Dose |
Participants who received the recommended phase 2 dose of 6.25 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). |
| COMPLETED |
|
| NOT COMPLETED |
|
| Phase 2: Treatment at RP2D |
|
Baseline demographics were assessed in the Safety Analysis Set (N=25) which was comprised of the Part 1: Defibrotide 2.5 mg/kg (n=4) and the combined Part 1: Defibrotide 6.25 mg/kg and Phase 2: RP2D 6.25 mg/kg (n=21). The baseline demographics are reported in this format as per the Statistical Analysis Plan.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Part 1: Safety Lead-In, Defibrotide 2.5 mg/kg/Dose | Participants who received the safety (lead in) dose of 2.5 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). |
| BG001 | Phase 2: RP2D, Defibrotide 6.25 mg/kg/Dose | Participants who received the recommended phase 2 dose of 6.25 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| 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 | Incidence of CAR-T-associated Neurotoxicity of Any Grade, Defined by CTCAE v5.0 by CAR-T Day +30 | The primary efficacy endpoint was the incidence of CAR-T-associated neurotoxicity of any grade defined by CTCAE v5.0 by CAR-T Day +30. The results report the estimated percentage of participants with no CAR-T-associated neurotoxicity of any grade, defined by CTCAE v5.0, which incorporated the 2 stage design. | The Efficacy Evaluable Analysis Set consisted of all participants in the Enrolled (RP2D) Analysis Set who received at least 18 doses (of all 35) of defibrotide and either developed CAR-T-associated neurotoxicity on or before CAR-T Day +30; OR completed the CAR-T Day +30 neurological assessment and discontinued defibrotide due to CAR-T-associated neurotoxicity before receiving 18 doses of defibrotide. Yescarta infusion must NOT have been delayed by more than 2 days from the original schedule. | Posted | Number | 90% Confidence Interval | percentage of participants | By CAR-T Day +30 |
|
|
| |||||||||||||||||||||||||||||||
| Secondary | Incidence of CAR-T-Associated Neurotoxicity Grade 3 or Greater Defined by CTCAE v5.0 by CAR-T Day +30 | The secondary efficacy endpoint was the incidence of CAR-T-associated neurotoxicity Grade 3 or greater defined by CTCAE v5.0 by CAR-T Day +30. The results report the estimated percentage of participants with no CAR-T-associated neurotoxicity (Grade 3 or greater defined by CTCAE v5.0) by CAR-T Day +30. | The Efficacy Evaluable Analysis Set consisted of all participants in the Enrolled (RP2D) Analysis Set who received at least 18 doses (of all 35) of defibrotide and either developed CAR-T-associated neurotoxicity on or before CAR-T Day +30; OR completed the CAR-T Day +30 neurological assessment and discontinued defibrotide due to CAR-T-associated neurotoxicity before receiving 18 doses of defibrotide. Yescarta infusion must NOT have been delayed by more than 2 days from the original schedule. | Posted | Number | percentage of participants | By CAR-T Day +30 |
| ||||||||||||||||||||||||||||||||||
| Secondary | Incidence of CAR-T-Associated Neurotoxicity of Any Grade According to the ASBMT Consensus Grading System by CAR-T Day +30 | The secondary efficacy endpoint was the incidence of CAR-T-associated neurotoxicity of any grade according to ASBMT consensus grading system by CAR-T Day +30. The results report the estimated percentage of participants with no CAR-T-associated neurotoxicity by CAR-T Day +30 summarized descriptively by any grade according to the ASBMT consensus grading system. | The Efficacy Evaluable Analysis Set consisted of all participants in the Enrolled (RP2D) Analysis Set who received at least 18 doses (of all 35) of defibrotide and either developed CAR-T-associated neurotoxicity on or before CAR-T Day +30; OR completed the CAR-T Day +30 neurological assessment and discontinued defibrotide due to CAR-T-associated neurotoxicity before receiving 18 doses of defibrotide. Yescarta infusion must NOT have been delayed by more than 2 days from the original schedule. | Posted | Number | percentage of participants | By CAR-T Day +30 |
| ||||||||||||||||||||||||||||||||||
| Secondary | Incidence of CAR-T-Associated Neurotoxicity of Grade 3 or Greater According to the ASBMT Consensus Grading System by CAR-T Day +30 | The secondary efficacy endpoint was the incidence of CAR-T-associated neurotoxicity grade 3 or greater according to ASBMT consensus grading system by CAR-T Day +30. The results report the estimated percentage of participants with no CAR-T-associated neurotoxicity by CAR-T Day +30 summarized descriptively by grade 3 or greater according to the ASBMT consensus grading system. | The Efficacy Evaluable Analysis Set consisted of all participants in the Enrolled (RP2D) Analysis Set who received at least 18 doses (of all 35) of defibrotide and either developed CAR-T-associated neurotoxicity on or before CAR-T Day +30; OR completed the CAR-T Day +30 neurological assessment and discontinued defibrotide due to CAR-T-associated neurotoxicity before receiving 18 doses of defibrotide. Yescarta infusion must NOT have been delayed by more than 2 days from the original schedule. | Posted | Number | percentage of participants | By CAR-T Day +30 |
| ||||||||||||||||||||||||||||||||||
| Secondary | Incidence of Cytokine Release Syndrome (CRS) of Any Grade According to the ASBMT Consensus Grading System by CAR-T Day +30 | The secondary efficacy endpoint was the incidence of cytokine release syndrome (CRS) of any grade according to ASBMT consensus grading system by CAR-T Day +30. The results report the estimated percentage of participants with no CRS any grade according to ASBMT criteria and was summarized descriptively using the ASBMT consensus grading system by CAR-T Day +30. | The Efficacy Evaluable Analysis Set consisted of all participants in the Enrolled (RP2D) Analysis Set who received at least 18 doses (of all 35) of defibrotide and either developed CAR-T-associated neurotoxicity on or before CAR-T Day +30; OR completed the CAR-T Day +30 neurological assessment and discontinued defibrotide due to CAR-T-associated neurotoxicity before receiving 18 doses of defibrotide. Yescarta infusion must NOT have been delayed by more than 2 days from the original schedule. | Posted | Number | percentage of participants | By CAR-T Day +30 |
| ||||||||||||||||||||||||||||||||||
| Secondary | Use of High Dose Steroid By CAR-T Day +30 | The percentage of participants using high dose steroids was summarized descriptively. The use of high dose steroids was defined as a dose of dexamethasone of at least 7.5 mg/day or equivalent. Only the Overall Defibrotide: 6.25 mg/kg/dose group was analyzed for this outcome. | The Efficacy Evaluable Analysis Set consisted of all participants in the Enrolled (RP2D) Analysis Set who received at least 18 doses (of all 35) of defibrotide and either developed CAR-T-associated neurotoxicity on or before CAR-T Day +30; OR completed the CAR-T Day +30 neurological assessment and discontinued defibrotide due to CAR-T-associated neurotoxicity before receiving 18 doses of defibrotide. Yescarta infusion must NOT have been delayed by more than 2 days from the original schedule. | Posted | Number | percentage of participants | By CAR-T Day +30 |
|
|
Adverse Events (AEs) and Serious Adverse Events (SAEs) were recorded from the time the subject signed informed consent until the final study visit or Early Termination (ET).
Adverse events (AEs), SAEs, and all-cause mortality were assessed in the Safety Analysis Set (N=25) which comprised of the Part 1: Defibrotide 2.5 mg/kg/dose (n=4) and the combined Part 1: Defibrotide 6.25 mg/kg/dose and Phase 2: RP2D 6.25 mg/kg/dose (n=21). The safety data are reported in this format as per the Statistical Analysis Plan.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part 1: Safety Lead-In, Defibrotide 2.5 mg/kg/Dose | Participants who received the safety (lead in) dose of 2.5 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). | 0 | 4 | 4 | 4 | 4 | 4 |
| EG001 | Phase 2: RP2D, 6.25 mg/kg/Dose | Participants who received the recommended phase 2 dose of 6.25 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). | 0 | 21 | 9 | 21 | 21 | 21 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Cytokine release syndrome | Immune system disorders | MedDRA | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Tumour lysis syndrome | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Neurotoxicity | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Disseminated intravascular coagulation | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Sinus bradycardia | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Sinus Tachycardia | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Ventricular tachycardia | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Anal incontinence | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Anorectal discomfort | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Vommiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA | Systematic Assessment |
| |
| Chills | General disorders | MedDRA | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA | Systematic Assessment |
| |
| Generalized oedema | General disorders | MedDRA | Systematic Assessment |
| |
| Localized oedema | General disorders | MedDRA | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA | Systematic Assessment |
| |
| Pain | General disorders | MedDRA | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA | Systematic Assessment |
| |
| Cytokine release syndrome | Immune system disorders | MedDRA | Systematic Assessment |
| |
| Candida infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Lung infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Incision site pain | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood fibrinogen decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood glucose increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood sodium decrreased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood thyroid stimulating hormone decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood urea decreased | Investigations | MedDRA | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA | Systematic Assessment |
| |
| Clostridium test | Investigations | MedDRA | Systematic Assessment |
| |
| Clostridium test positive | Investigations | MedDRA | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA | Systematic Assessment |
| |
| Acidosis | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Fluid overload | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypokalemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypophagia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Arthalgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Muscle Spasma | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Aphasia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Atazia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Cognative disorder | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Depressed level of consciousness | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Neurotoxicity | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Dysphemia | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Incontinence | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Micturition urgency | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Stress unrinary incontinence | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Penile haemorrhage | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Dyspnoes | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Hiccups | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Laryngeal haemorrage | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Tachypnoes | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA | Systematic Assessment |
|
The sponsor can review trial results communications prior to public release and can embargo such communications for a period of at least 60 days from the time submitted to sponsor for review. If requested by sponsor, the PI will withhold publication for up to an additional 30 days. Furthermore, the first publication of study results must be a joint publication of all study sites unless a joint manuscript has not been submitted for publication within 12 months of completion of the study.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Director, Clinical Trial Disclosure & Transparency | Jazz Pharmaceuticals | 2158709177 | ClinicalTrialDisclosure@JazzPharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 21, 2021 | Sep 22, 2021 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D020258 | Neurotoxicity Syndromes |
| ID | Term |
|---|---|
| D009422 | Nervous System Diseases |
| D011041 | Poisoning |
| D064419 | Chemically-Induced Disorders |
Not provided
Not provided
| ID | Term |
|---|---|
| C036901 | defibrotide |
Not provided
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
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Participants who received the RP2D of 6.25 mg/kg/dose defibrotide in Part 2 (n=10) once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). Stage 2 participants include the additional efficacy evaluable participants enrolled after the completion of Stage 1.
| OG002 | Overall: Defibrotide, 6.25 mg/kg/Dose | All participants who received 6.25 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7) in Stage 1 and Stage 2. |
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Participants who received the RP2D of 6.25 mg/kg/dose defibrotide in Part 2 (n=10) once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). Stage 2 participants include the additional efficacy evaluable participants enrolled after the completion of Stage 1. |
| OG002 | Overall: Defibrotide, 6.25 mg/kg/Dose | All participants who received 6.25 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7) in Stage 1 and Stage 2. |
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Participants who received the RP2D of 6.25 mg/kg/dose defibrotide in Part 2 (n=10) once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). Stage 2 participants include the additional efficacy evaluable participants enrolled after the completion of Stage 1. |
| OG002 | Overall: Defibrotide, 6.25 mg/kg/Dose | All participants who received 6.25 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7) in Stage 1 and Stage 2. |
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Participants who received the RP2D of 6.25 mg/kg/dose defibrotide in Part 2 (n=10) once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7). Stage 2 participants include the additional efficacy evaluable participants enrolled after the completion of Stage 1. |
| OG002 | Overall: Defibrotide, 6.25 mg/kg/Dose | All participants who received 6.25 mg/kg/dose defibrotide once daily as a single dose on CAR-T Day -5, -4, and -3 prior to lymphodepletion and then every 6 hours daily for 8 days (CAR-T Day 0 to Day 7) in Stage 1 and Stage 2. |
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