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This study was stopped after Part 1 completed and was terminated because the predefined criteria of efficacy was not reached ( >50% better then placebo)
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To evaluate the safety and efficacy of QGE031 versus placebo in patients with bullous pemphigoid. Efficacy will be assessed as a reduction of disease activity. How QGE031 is broken down by the body and the impact it has on different blood and tissue markers will also be explored.
This study was planned to be divided into 2 distinct parts. Part 1 was a multicenter, randomized, placebo-controlled study evaluating the efficacy, safety, PK and PD of multiple, subcutaneous doses of QGE031 in the treatment of patients with BP with disease refractory to oral steroid treatment. Patients were treated with QGE031 or placebo in a 2:1 ratio.
Part 2 of this study was planned to be a multi-center, open label, dose range finding study evaluating the efficacy, safety, PK and PD of multiple, subcutaneous doses of QGE031 in the treatment of patients with BP with disease refractory to oral steroid treatment.
This study was stopped after Part 1 completed and was terminated because the predefined criteria of efficacy was not reached ( >50% better then placebo)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| QGE031 | Experimental | QGE031 240 mg Q2W s.c. |
|
| Placebo | Placebo Comparator | Placebo to Match Q2W s.c. |
|
| Open Label QGE031 | Experimental | Open Label QGE031 Q2W s.c. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| QGE031 | Drug | QGE031 will be evaluated at various dose levels and regimens, based on the impact on disease of the next highest dose level and regimen. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12 | Clinical Global Assessment of Change (CGA-C) responder rate was the responder rate at 12 weeks based on the CGA-C in bullous pemphigoid (BP). A patient with a CGA-C score of 3 or 4 indicating 'at least marked improvement from baseline' at 12 weeks was considered a responder. The CGA-C is an investigator assessment of change from baseline and is scored as follows: -4 = Very marked worsening (100% worsening); -3 = Marked worsening (67-99% worsening); -2 = Moderate worsening (34-66% worsening); -1 = Slight worsening (1-33% worsening); 1= Slight improvement (1-33% improvement); 2 = Moderate improvement (34-66% improvement); 3 = Marked improvement (67-99% improvement); 4 = Complete clearance (100% improvement) | 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks | Clinical Global Assessment of Change (CGA-C) responder rate was the responder rate at 6 weeks based on the CGA-C score in bullous pemphigoid (BP). A patient with a CGA-C score of 3 or 4 indicating marked improvement from baseline at 6 weeks was considered a responder. The CGA-C is an investigator assessment of change from baseline and is scored as follows: -4 = Very marked worsening (100% worsening); -3 = Marked worsening (67-99% worsening); -2 = Moderate worsening (34-66% worsening); -1 = Slight worsening (1-33% worsening); 1= Slight improvement (1-33% improvement); 2 = Moderate improvement (34-66% improvement); 3 = Marked improvement (67-99% improvement); 4 = Complete clearance (100% improvement) |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Iowa City | Iowa | 52242 | United States | ||
| Novartis Investigative Site |
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| ID | Title | Description |
|---|---|---|
| FG000 | QGE031 | QGE031 240 mg Q2W s.c. |
| FG001 | Placebo | Placebo to Match Q2W s.c. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Placebo | Drug | Placebo will be used to control for normal variability in disease severity. |
|
| 6 weeks |
| Number of Patients Investigator Global Assessment Score Over 12 Weeks | Investigator's Global Assessment (IGA) - (scale of 0 to 4, where 0=clear, 1=almost clear, 2=mild, 3=moderate and 4=severe) | Baseline (week 0), week 6 and week 12 |
| Durham |
| North Carolina |
| 27710 |
| United States |
| Novartis Investigative Site | Vienna | A 1040 | Austria |
| Novartis Investigative Site | Rouen | Cedex | 76031 | France |
| Novartis Investigative Site | Dresden | 01307 | Germany |
| Novartis Investigative Site | Freiburg im Breisgau | 79104 | Germany |
| Novartis Investigative Site | Marburg | 35039 | Germany |
| Novartis Investigative Site | Shinjuku-ku | Tokyo | 160-8582 | Japan |
| Novartis Investigative Site | Taipei | Taiwan | 10002 | Taiwan |
| Safety Follow-up |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | QGE031 | QGE031 240 mg Q2W s.c. |
| BG001 | Placebo | Placebo to Match Q2W s.c. |
| 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, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | 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 | Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12 | Clinical Global Assessment of Change (CGA-C) responder rate was the responder rate at 12 weeks based on the CGA-C in bullous pemphigoid (BP). A patient with a CGA-C score of 3 or 4 indicating 'at least marked improvement from baseline' at 12 weeks was considered a responder. The CGA-C is an investigator assessment of change from baseline and is scored as follows: -4 = Very marked worsening (100% worsening); -3 = Marked worsening (67-99% worsening); -2 = Moderate worsening (34-66% worsening); -1 = Slight worsening (1-33% worsening); 1= Slight improvement (1-33% improvement); 2 = Moderate improvement (34-66% improvement); 3 = Marked improvement (67-99% improvement); 4 = Complete clearance (100% improvement) | Pharmacodynamics (PD) analysis set included all randomized patients. | Posted | Number | number of participants | 12 weeks |
|
|
| |||||||||||||||||||||||||||||
| Secondary | Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks | Clinical Global Assessment of Change (CGA-C) responder rate was the responder rate at 6 weeks based on the CGA-C score in bullous pemphigoid (BP). A patient with a CGA-C score of 3 or 4 indicating marked improvement from baseline at 6 weeks was considered a responder. The CGA-C is an investigator assessment of change from baseline and is scored as follows: -4 = Very marked worsening (100% worsening); -3 = Marked worsening (67-99% worsening); -2 = Moderate worsening (34-66% worsening); -1 = Slight worsening (1-33% worsening); 1= Slight improvement (1-33% improvement); 2 = Moderate improvement (34-66% improvement); 3 = Marked improvement (67-99% improvement); 4 = Complete clearance (100% improvement) | Pharmacodynamics (PD) analysis set included all randomized patients. | Posted | Number | number of participants | 6 weeks |
|
| ||||||||||||||||||||||||||||||
| Secondary | Number of Patients Investigator Global Assessment Score Over 12 Weeks | Investigator's Global Assessment (IGA) - (scale of 0 to 4, where 0=clear, 1=almost clear, 2=mild, 3=moderate and 4=severe) | Pharmacodynamics (PD) analysis set included all randomized patients. | Posted | Number | Number of participants | Baseline (week 0), week 6 and week 12 |
|
|
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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 | QGE031 | QGE031 240 mg Q2W s.c | 6 | 13 | 11 | 13 | ||
| EG001 | Placebo | Placebo to Match Q2W s.c. | 2 | 7 | 6 | 7 | ||
| EG002 | Open Label QGE031 | Open Label QGE031 Q2W s.c. | 1 | 4 | 2 | 4 | ||
| EG003 | Follow-up Period: QGE031 | Follow-up Period after completion of Part 1 QGE031 240 mg Q2W s.c. | 2 | 11 | 10 | 11 | ||
| EG004 | Follow-up Period: Placebo | Follow-up Period after completion of Part 1 Placebo to Match Q2W s.c. | 0 | 5 | 3 | 5 | ||
| EG005 | Follow-up Period: Open Label QGE031 | Follow-up Period after completion of Part 1 Open Label QGE031 Q2W s.c. | 0 | 4 | 2 | 4 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute myocardial infarction | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Cardiogenic shock | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Coronary artery disease | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Hepatic failure | Hepatobiliary disorders | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Cauda equina syndrome | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Pemphigoid | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Cushingoid | Endocrine disorders | MedDRA | Systematic Assessment |
| |
| Conjunctivitis allergic | Eye disorders | MedDRA | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Aphthous stomatitis | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA | Systematic Assessment |
| |
| Injection site haematoma | General disorders | MedDRA | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA | Systematic Assessment |
| |
| Xerosis | General disorders | MedDRA | Systematic Assessment |
| |
| Hepatitis | Hepatobiliary disorders | MedDRA | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA | Systematic Assessment |
| |
| Acarodermatitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Furuncle | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Ophthalmic herpes simplex | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Skin candida | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Accidental overdose | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Wound | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood triglycerides increased | Investigations | MedDRA | Systematic Assessment |
| |
| Eosinophil count increased | Investigations | MedDRA | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Haemoglobin urine present | Investigations | MedDRA | Systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Neutrophil count increased | Investigations | MedDRA | Systematic Assessment |
| |
| Neutrophil morphology abnormal | Investigations | MedDRA | Systematic Assessment |
| |
| Staphylococcus test positive | Investigations | MedDRA | Systematic Assessment |
| |
| White blood cells urine positive | Investigations | MedDRA | Systematic Assessment |
| |
| Cachexia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Glucose tolerance impaired | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Burning sensation | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Sciatic nerve palsy | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Vascular dementia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA | Systematic Assessment |
| |
| Glycosuria | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| Benign prostatic hyperplasia | Reproductive system and breast disorders | MedDRA | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Miliaria | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Pemphigoid | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Purpura senile | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 |
| ID | Term |
|---|---|
| D010391 | Pemphigoid, Bullous |
| ID | Term |
|---|---|
| D012872 | Skin Diseases, Vesiculobullous |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
Not provided
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| ID | Term |
|---|---|
| C000598891 | ligelizumab |
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| Male |
|
|
|