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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-515622-80-00 | EU Trial (CTIS) Number |
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This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von Willebrand disease (VWD). Participants on prior standard of care (SOC) on-demand therapy will be assessed via a randomized comparison (Arm A - emicizumab prophylaxis and Arm B - continuation of SOC on-demand therapy), while participants on prior SOC prophylactic therapy (Arm C - emicizumab prophylaxis) will be assessed via intra-participant analysis with data obtained from the preceding non-interventional study (NIS), WP45335 (NCT06883240).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm A (Prior On-Demand SOC): Emicizumab Prophylaxis for 24 Weeks | Experimental | Participants who are randomized to Arm A, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will receive emicizumab SC prophylaxis. |
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| Arm B (Prior On-Demand SOC): On-Demand SOC for 24 Weeks | Active Comparator | Participants who are randomized to Arm B, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will continue to receive their current SOC on-demand treatment until Week 24. |
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| Arm C (Prior Prophylaxis SOC): Emicizumab Prophylaxis for 24 Weeks | Experimental | Participants who enroll in Arm C, taking SOC prophylactic treatment at the time of study entry and for at least 24 weeks of observation during the preceding NIS WP45335, will receive emicizumab SC prophylaxis. |
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| Treatment Extension Period for All Arms: Emicizumab Prophylaxis | Experimental | Participants in Arms A and C who have completed 24 weeks of emicizumab prophylaxis and who derive benefit from emicizumab will have the opportunity to continue to receive emicizumab prophylaxis in the extension period. Participants in Arm B who have completed 24 weeks of SOC on-demand treatment will have the opportunity to receive emicizumab prophylaxis in the extension period. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Emicizumab | Drug | Participants will receive emicizumab 3 milligrams per kilogram (mg/kg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg/kg SC once every 2 weeks (Q2W). During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators. |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Bleed Rate (ABR) for Treated Bleeds in the Randomized Arms | From Baseline to at least 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| ABR for All Bleeds in the Randomized Arms | From Baseline to at least 24 weeks | |
| ABR for Treated Spontaneous Bleeds in the Randomized Arms | From Baseline to at least 24 weeks | |
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Inclusion Criteria:
Additional Inclusion Criteria for Arms A and B:
Additional Inclusion Criteria for Arm C:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Reference Study ID Number: WP45338 https://forpatients.roche.com/ | Contact | 888-662-6728 (U.S. Only) | global-roche-genentech-trials@gene.com | |
| Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry | Contact |
| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC Davis | Recruiting | Sacramento | California | 95817 | United States | |
| University of Florida |
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| Label | URL |
|---|---|
| Please use this form to submit your questions for a faster response: https://www.gene.com/contact-us/submit-medical-inquiry. Do not include or attach any medical records when emailing or completing the form. A nurse will respond within 24 business hours. | View source |
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For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
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Participants on prior SOC on-demand therapy will be randomized into Arms A and B. Participants on prior SOC prophylactic therapy, who participated in the NIS WP45335 (NCT06883240) and meet the eligibility criteria for this study, will be enrolled into Arm C.
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| von Willebrand Factor (VWF) Concentrates | Drug | Used according to local labeling or local treatment guidelines. |
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| Factor VIII (FVIII) Concentrates | Drug | Used according to local labeling or local treatment guidelines. |
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| von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates | Drug | Used according to local labeling or local treatment guidelines. |
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| Bypassing Agents | Drug | Used according to local labeling or local treatment guidelines. |
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| ABR for Treated Joint Bleeds in the Randomized Arms |
| From Baseline to at least 24 weeks |
| Intra-Participant Comparison of the ABR for Treated Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding Non-Interventional Study (NIS) WP45335 | From Baseline to at least 24 weeks |
| Intra-Participant Comparison of the ABR for All Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding NIS WP45335 | From Baseline to at least 24 weeks |
| Intra-Participant Comparison of the ABR for Treated Spontaneous Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding NIS WP45335 | From Baseline to at least 24 weeks |
| Intra-Participant Comparison of the ABR for Treated Joint Bleeds with Prophylactic Emicizumab Versus Prophylactic SOC from the Preceeding NIS WP45335 | From Baseline to at least 24 weeks |
| Incidence and Severity of Adverse Events, with Severity Determined According to the World Health Organization (WHO) Toxicity Grading Scale | From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months) |
| Incidence and Severity of Thromboembolic Events | From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months) |
| Incidence and Severity of Thrombotic Microangiopathy Events | From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months) |
| Incidence and Severity of Injection-Site Reactions | From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months) |
| Incidence of Adverse Events Leading to Drug Discontinuation | From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months) |
| Incidence of Severe Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions | From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months) |
| Incidence of Clinical Laboratory Abnormalities | From first dose of study treatment until 24 weeks after final dose of study treatment (up to 3 years, 11 months) |
| Trough Plasma Concentration of Emicizumab at Prespecified Timepoints During the Treatment Period | Predose and at prespecified timepoints from first dose of emicizumab until study completion (up to 3 years, 11 months) |
| Percentage of Participants with Anti-Drug Antibodies (ADAs) to Emicizumab at Baseline and with ADAs to Emicizumab During the Treatment Period | Baseline and at prespecified timepoints from first dose of emicizumab until study completion (up to 3 years, 11 months) |
| Change from Baseline in Respiratory Rate Over Time | Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months) |
| Change from Baseline in Pulse Rate Over Time | Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months) |
| Change from Baseline in Systolic Blood Pressure Over Time | Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months) |
| Change from Baseline in Diastolic Blood Pressure Over Time | Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months) |
| Change from Baseline in Body Temperature Over Time | Baseline, Weeks 1, 2, 25, and every 12 weeks thereafter (weeks after switch to emicizumab for Arm B only) until study completion (up to 3 years, 11 months) |
| Change from Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcF, RR, PR, and QRS Intervals | Baseline and study completion (up to 3 years, 11 months) |
| Change from Baseline in Heart Rate Over Time, as Measured by Electrocardiogram (ECG) | Baseline and study completion (up to 3 years, 11 months) |
| Change from Baseline in the PROMIS-29 Questionnaire Pain Interference Domain Score Over Time | PROMIS-29 stands for Patient-Reported Outcomes Measurement Information System-29 | Baseline and at prespecified timepoints until study completion (up to 3 years, 11 months) |
| Change from Baseline in the PROMIS-29 Questionnaire Fatigue Domain Score Over Time | PROMIS-29 stands for Patient-Reported Outcomes Measurement Information System-29 | Baseline and at prespecified timepoints until study completion (up to 3 years, 11 months) |
| Recruiting |
| Gainesville |
| Florida |
| 32610 |
| United States |
| University of Minnesota Medical Center | Recruiting | Minneapolis | Minnesota | 55455 | United States |
| Washington University School of Medicine | Recruiting | St Louis | Missouri | 63110 | United States |
| Virginia Commonwealth University | Recruiting | Richmond | Virginia | 23298-0461 | United States |
| UZ Leuven Gasthuisberg | Recruiting | Leuven | 3000 | Belgium |
| The Hospital for Sick Children | Recruiting | Toronto | Ontario | M5G 1X8 | Canada |
| McGill University Health Center | Recruiting | Montreal | Quebec | H4A 3J1 | Canada |
| IPS SURA Industriales Medellín | Recruiting | Medellín | Colombia |
| Hopital Claude Huriez - CHU Lille | Recruiting | Lille | 59037 | France |
| Groupe Hospitalier Necker Enfants Malades | Recruiting | Paris | 75015 | France |
| Universitätsklinikum Bonn | Recruiting | Bonn | 53127 | Germany |
| Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz | Recruiting | Duisburg | 47051 | Germany |
| Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin | Recruiting | Frankfurt/M. | 60590 | Germany |
| Universita' Degli Studi La Sapienza-Ist.Di Ematologia | Recruiting | Rome | Lazio | 00161 | Italy |
| IRCCS Ca' Granda Ospedale Maggiore Policlinico | Recruiting | Milan | Lombardy | 20122 | Italy |
| AOU Careggi | Recruiting | Florence | Tuscany | 50134 | Italy |
| Kurume University Hospital | Recruiting | Fukuoka | 830-0011 | Japan |
| Nagoya University Hospital | Recruiting | Nagoya | 466-8560 | Japan |
| Erasmus MC | Recruiting | Rotterdam | 3015 GD | Netherlands |
| Instytut Hematologii i Transfuzjologii | Recruiting | Warsaw | 02-776 | Poland |
| Charlotte Maxeke Johannesburg Academic Hospital | Recruiting | Johannesburg | 2193 | South Africa |
| Hospital Universitario la Paz | Recruiting | Madrid | 28046 | Spain |
| Hospital Universitario Virgen del Rocio | Recruiting | Seville | 41013 | Spain |
| Sahlgrenska Universitetssjukhuset | Recruiting | Gothenburg | S-413 45 | Sweden |
| St Thomas' Hospital | Recruiting | London | SE1 7EH | United Kingdom |
| Great Ormond Street Hospital | Recruiting | London | WC1N 3JH | United Kingdom |
| Manchester Royal Infirmary | Recruiting | Manchester | M13 9WL | United Kingdom |
| ID | Term |
|---|---|
| D056729 | von Willebrand Disease, Type 3 |
| ID | Term |
|---|---|
| D014842 | von Willebrand Diseases |
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000608208 | emicizumab |
| D014841 | von Willebrand Factor |
| D005169 | Factor VIII |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D001685 | Biological Factors |
| D011498 | Protein Precursors |
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