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| Name | Class |
|---|---|
| Johnson & Johnson Pharmaceutical Research & Development, L.L.C. | INDUSTRY |
This is a phase 1/2 open-label, dose-escalation study investigating single-agent therapy with VELCADE in patients with previously treated systemic AL-amyloidosis who require further treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental | VELCADE |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VELCADE | Drug | Once weekly at: 0.7, 1.0, 1.3 or 1.6 mg/m2 Or Twice-weekly at: 0.7, 1.0, or 1.3 mg/m2 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Tolerated Dose | Maximum Tolerated Dose (MTD) was defined as the highest dose level that has 0/1 out of 6 patients experiences Dose Limited Toxicity (DLT). MTD is defined separately for QW and BIQ dose cohorts. DLT was defined as adverse events occurring during Cycle 1 and: (1) related to VELCADE, (2) Grade 4 thrombocytopenia or neutropenia, (3) Grade 3 or higher nonhematologic toxicity. | 5 weeks in once weekly (QW) dose cohorts and 3 weeks in twice weekly (BIW) dose cohorts |
| Subjects With Treatment Emergent Adverse Events | Treatment emergent adverse events observed during outcome measure time frame | from first study-related procedure to 30 days after last dose of study medication |
| Subjects With Serious Treatment Emergent Adverse Events | Serious treatment emergent adverse events observed during outcome measure time frame | from first study-related procedure to 30 days after last dose of study medication |
| Subjects Grade 3/4/5 Treatment Emergent Adverse Events | Grade 3/4/5 treatment emergent adverse events observed during outcome measure time frame. Grade is determined according to Common Terminology Criteria for Adverse Event (CTCAE) Version 3.0. | from first study-related procedure to 30 days after last dose of study medication |
| Subjects With Treatment Emergent Adverse Events Leading to Treatment Termination | Treatment emergent adverse events observed during outcome measure time frame leading to treatment termination | from first study-related procedure to 30 days after last dose of study medication |
| Measure | Description | Time Frame |
|---|---|---|
| Best Confirmed Hematologic Responders | Hematologic response was determined by the investigator per the response criteria for immunoglobulin light chain amyloidosis by Gertz (2005). It include Complete and Partial Responders (CR+PR). CR requires serum and urine negative for a monoclonal protein by immunofixation and free light chain ratio normal. PR requires: 1. reduction in quantitative serum M-protein by 50% if baseline value is at least 0.5 g/dL, 2. if light chain is detected in the urine (with a consistent peak and >100 mg/ 24 hours), then 50% reduction is required, 3. if free light chain >10 mg/dL, reduction by 50% is required. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Millennium Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cedars-Sinai Medical Center, Samuel Oschin Comprehensive Cancer Institute | Los Angeles | California | 90049 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25202139 | Derived | Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Blade J, Fermand JP, Hassoun H, Heffner L, Kukreti V, Vescio RA, Pei L, Enny C, Esseltine DL, van de Velde H, Cakana A, Comenzo RL. Long-term follow-up from a phase 1/2 study of single-agent bortezomib in relapsed systemic AL amyloidosis. Blood. 2014 Oct 16;124(16):2498-506. doi: 10.1182/blood-2014-04-568329. Epub 2014 Sep 8. | |
| 21562045 |
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| ID | Title | Description |
|---|---|---|
| FG000 | Single Agent VELCADE | Bortezomib 0.7, 1.0, 1.3 and 1.6 mg/m^2 once weekly (QW) 4 doses in a 5 week cycle, and 0.7, 1.0, 1.3 mg/m^2 twice weekly (BIW) 4 doses in a 3 week cycle |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Single Agent VELCADE | Bortezomib 0.7, 1.0, 1.3 and 1.6 mg/m^2 once weekly (QW) 4 doses in a 5 week cycle, and 0.7, 1.0, 1.3 mg/m^2 twice weekly (BIW) 4 doses in a 3 week cycle |
| 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 |
| 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 | Maximum Tolerated Dose | Maximum Tolerated Dose (MTD) was defined as the highest dose level that has 0/1 out of 6 patients experiences Dose Limited Toxicity (DLT). MTD is defined separately for QW and BIQ dose cohorts. DLT was defined as adverse events occurring during Cycle 1 and: (1) related to VELCADE, (2) Grade 4 thrombocytopenia or neutropenia, (3) Grade 3 or higher nonhematologic toxicity. | Phase 1 Safety Population includes all subjects who received at least one dose of VELCADE in phase 1 dose escalation cohorts | Posted | Number | participants with DLT | 5 weeks in once weekly (QW) dose cohorts and 3 weeks in twice weekly (BIW) dose cohorts |
|
from first study-related procedure to 30 days after last dose of study medication
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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 | Single Agent VELCADE | Bortezomib 0.7, 1.0, 1.3 and 1.6 mg/m^2 once weekly (QW) 4 doses in a 5 week cycle, and 0.7, 1.0, 1.3 mg/m^2 twice weekly (BIW) 4 doses in a 3 week cycle |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (11.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (11.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Helgi van de Velde | Johnson & Johnson Pharmaceutical Research & Development | HVDVELDE@ITS.JNJ.COM |
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| ID | Term |
|---|---|
| D000686 | Amyloidosis |
| ID | Term |
|---|---|
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| D000069286 | Bortezomib |
| ID | Term |
|---|---|
| D001897 | Boronic Acids |
| D000148 | Acids, Noncarboxylic |
| D000143 | Acids |
| D007287 | Inorganic Chemicals |
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| from first dose of study medication to end of study visit |
| Winship Cancer Center - Emory Clinic School of Medicine |
| Atlanta |
| Georgia |
| 30322 |
| United States |
| Boston Medical Center | Boston | Massachusetts | 02118 | United States |
| MSKCC | New York | New York | 10017 | United States |
| Derived |
| Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Blade J, Fermand JP, Hassoun H, Heffner L, Vescio RA, Liu K, Enny C, Esseltine DL, van de Velde H, Cakana A, Comenzo RL. Efficacy and safety of once-weekly and twice-weekly bortezomib in patients with relapsed systemic AL amyloidosis: results of a phase 1/2 study. Blood. 2011 Jul 28;118(4):865-73. doi: 10.1182/blood-2011-02-334227. Epub 2011 May 11. |
| 19498019 | Derived | Reece DE, Sanchorawala V, Hegenbart U, Merlini G, Palladini G, Fermand JP, Vescio RA, Liu X, Elsayed YA, Cakana A, Comenzo RL; VELCADE CAN2007 Study Group. Weekly and twice-weekly bortezomib in patients with systemic AL amyloidosis: results of a phase 1 dose-escalation study. Blood. 2009 Aug 20;114(8):1489-97. doi: 10.1182/blood-2009-02-203398. Epub 2009 Jun 4. |
| Treatment ongoing |
|
| Progression of amyloid markers |
|
| Deterioration in KPS and organ function |
|
| Subjects overall condition deterioration |
|
| Other |
|
| Participants |
|
| Age Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| Primary | Subjects With Treatment Emergent Adverse Events | Treatment emergent adverse events observed during outcome measure time frame | Safety population | Posted | Number | participants | from first study-related procedure to 30 days after last dose of study medication |
|
|
|
| Secondary | Best Confirmed Hematologic Responders | Hematologic response was determined by the investigator per the response criteria for immunoglobulin light chain amyloidosis by Gertz (2005). It include Complete and Partial Responders (CR+PR). CR requires serum and urine negative for a monoclonal protein by immunofixation and free light chain ratio normal. PR requires: 1. reduction in quantitative serum M-protein by 50% if baseline value is at least 0.5 g/dL, 2. if light chain is detected in the urine (with a consistent peak and >100 mg/ 24 hours), then 50% reduction is required, 3. if free light chain >10 mg/dL, reduction by 50% is required. | Efficacy population included all treated subjects with an evaluable post baseline resposne assessment in the MTD cohorts (1.6 mg/m^2 QW and 1.3 mg/m^2 BIW). | Posted | Number | participants responded | from first dose of study medication to end of study visit |
|
|
|
| Primary | Subjects With Serious Treatment Emergent Adverse Events | Serious treatment emergent adverse events observed during outcome measure time frame | Safety population | Posted | Number | participants | from first study-related procedure to 30 days after last dose of study medication |
|
|
|
| Primary | Subjects Grade 3/4/5 Treatment Emergent Adverse Events | Grade 3/4/5 treatment emergent adverse events observed during outcome measure time frame. Grade is determined according to Common Terminology Criteria for Adverse Event (CTCAE) Version 3.0. | Safety population | Posted | Number | participants | from first study-related procedure to 30 days after last dose of study medication |
|
|
|
| Primary | Subjects With Treatment Emergent Adverse Events Leading to Treatment Termination | Treatment emergent adverse events observed during outcome measure time frame leading to treatment termination | Safety population | Posted | Number | participants | from first study-related procedure to 30 days after last dose of study medication |
|
|
|
| 25 |
| 70 |
| 70 |
| 70 |
| Atrial fibrillation | Cardiac disorders | MedDRA (11.0) | Systematic Assessment |
|
| Cardiac failure acute | Cardiac disorders | MedDRA (11.0) | Systematic Assessment |
|
| Cardiac failure cogestive | Cardiac disorders | MedDRA (11.0) | Systematic Assessment |
|
| Restrictive cardiomyopathy | Cardiac disorders | MedDRA (11.0) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Ileus | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Ileus paralytic | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Volvulus | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Bacteraemia | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Escherichia bacteraemia | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Herpes zoster | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Lobar pneumonia | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Staphylococcal bacteraemia | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA (11.0) | Systematic Assessment |
|
| Hip fracture | Injury, poisoning and procedural complications | MedDRA (11.0) | Systematic Assessment |
|
| Fluid retention | Metabolism and nutrition disorders | MedDRA (11.0) | Systematic Assessment |
|
| Autonomic neuropathy | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Cerebral ischaemia | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Loss of consciousness | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Neuralgia | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA (11.0) | Systematic Assessment |
|
| Renal failure | Renal and urinary disorders | MedDRA (11.0) | Systematic Assessment |
|
| Renal failure acute | Renal and urinary disorders | MedDRA (11.0) | Systematic Assessment |
|
| Penal impairment | Renal and urinary disorders | MedDRA (11.0) | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Rash generalized | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA (11.0) | Systematic Assessment |
|
| Orthostatic hypotension | Vascular disorders | MedDRA (11.0) | Systematic Assessment |
|
| Leukopenia | Blood and lymphatic system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Palpitations | Cardiac disorders | MedDRA (11.0) | Systematic Assessment |
|
| Conjunctival haemorrhage | Eye disorders | MedDRA (11.0) | Systematic Assessment |
|
| Conjunctivitis | Eye disorders | MedDRA (11.0) | Systematic Assessment |
|
| Dry eye | Eye disorders | MedDRA (11.0) | Systematic Assessment |
|
| Vision blurred | Eye disorders | MedDRA (11.0) | Systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Dry mouth | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Chills | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Early satiety | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Oedema | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Pain | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (11.0) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Cystitis | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA (11.0) | Systematic Assessment |
|
| Periorbital haematoma | Injury, poisoning and procedural complications | MedDRA (11.0) | Systematic Assessment |
|
| Anorexia | Metabolism and nutrition disorders | MedDRA (11.0) | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (11.0) | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA (11.0) | Systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (11.0) | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Hypoaesthesia | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Neuropathy peripheral | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Peripheral sensory neuropathy | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Somnolence | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA (11.0) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Systematic Assessment |
|
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA (11.0) | Systematic Assessment |
|
| Phlebitis | Vascular disorders | MedDRA (11.0) | Systematic Assessment |
|
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| D001896 |
| Boron Compounds |
| D009930 | Organic Chemicals |
| D011719 | Pyrazines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |