Protocol No: | ECCT/21/02/02 | Date of Protocol: | 15-04-2020 |
Study Title: | PROSPECTIVE NON-INTERVENTIONAL MULTI-CENTER STUDY EVALUATING BLEEDING INCIDENCE AND HEALTH-RELATED QUALITY OF LIFE IN PATIENTS WITH HEMOPHILIA A WITH INHIBITORS AND NON-INHIBITORS TREATED WITH EMICIZUMAB AND OTHER HEMOPHILIA TREATMENTS IN A REAL-LIFE SETTING |
PROSPECTIVE NON-INTERVENTIONAL MULTI-CENTER STUDY EVALUATING BLEEDING INCIDENCE AND HEALTH-RELATED QUALITY OF LIFE IN PATIENTS WITH HEMOPHILIA A WITH INHIBITORS AND NON-INHIBITORS TREATED WITH EMICIZUMAB AND OTHER HEMOPHILIA TREATMENTS IN A REAL-LIFE SETTING
RESHAPE: REAL-WORLD TREATMENT PRACTICE OF SEVERE HEMOPHILIA A PATIENTS | |
Study Objectives: | Effectiveness Objectives The primary effectiveness objective for this study is as follows:
The secondary effectiveness objectives for this study are as follows:
Safety Objectives The safety objectives for this study are as follows:
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Laymans Summary: | This a Non-interventional study in Real world setting. The study will evaluate bleeding incidence and quality of life for Hemophilia A (with or without inhibitors) patient receiving hemophilia treatment. The main objective is to analyze the treated bleeds according to the treatment the hemophilia A patient received within two patients groups (Cohort A: patients without FVIII inhibitors and Cohort B: patients with FVIII inhibitors) under routine clinical practice. Observation over all patients will last for 1 year (52 weeks) from their first data collection. If the patient has changed his treatment regimen within the initial 52 weeks observation period, additional 1 year (52 weeks) of observation will be added. Patients will collect detailed information about any bleeding event (date and start time of bleeding, location of bleeding, type of bleeding) via a Bleeds & Medication Questionnaire (BMQ), which will be installed on smart devices provided by the Sponsor. Paper based EQ-5D-5L questionnaire will be collected approximately each 3 months (13 weeks). Other data will be collected from the patients’ clinical notes. The study will be conducted at approximately 80 sites in approximately 25 countries. Approximately 705 patients will be included (609 patients with no inhibitors against FVIII; 96 patients with inhibitors).
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2 | This a Non-interventional study in Real world setting. The study will evaluate bleeding incidence and quality of life for Hemophilia A (with or without inhibitors) patient receiving hemophilia treatment. The main objective is to analyze the treated bleeds according to the treatment the hemophilia A patient received within two patients groups (Cohort A: patients without FVIII inhibitors and Cohort B: patients with FVIII inhibitors) under routine clinical practice. Observation over all patients will last for 1 year (52 weeks) from their first data collection. If the patient has changed his treatment regimen within the initial 52 weeks observation period, additional 1 year (52 weeks) of observation will be added. Patients will collect detailed information about any bleeding event (date and start time of bleeding, location of bleeding, type of bleeding) via a Bleeds & Medication Questionnaire (BMQ), which will be installed on smart devices provided by the Sponsor. Paper based EQ-5D-5L questionnaire will be collected approximately each 3 months (13 weeks). Other data will be collected from the patients’ clinical notes. The study will be conducted at approximately 80 sites in approximately 25 countries. Approximately 705 patients will be included (609 patients with no inhibitors against FVIII; 96 patients with inhibitors). |
Abstract of Study: | SSStudy Title: Prospective No-Interventional Multi-Center Study Evaluating Bleeding Incidence and Health-Related Quality of Life in Patients with Hemophilia A with Inhibitors and Non-Inhibitors Treated with Emicizumab and other Hemophilia A Treatments in a real-Life Setting.
Name of Principal Investigator: Festus Njuguna, MD
Co-investigators: Carole Kilach, MD
Supervisors: Cyrus Njuguna, Gabriel Omwalo, Huwaida Bulhan
Name of Organization: AMPATH/MTRH
Funding: ROCHE Pharmaceuticals
Broad Objectives: To analyze all treated bleeds as measured by annualized bleeding rate (ABR) in hemophilia A patients according to treatment type within two patients’ groups (Cohort A: patients without FVIII inhibitors and Cohort B: patients with FVIII inhibitors) under routine clinical practice.
Specific Objectives:
Study setting: Hemophilia Clinic in Moi Teaching and Referral Hospital
Study population: Patients with congenital Severe Hemophilia A with or without inhibitors under treatment with Emicizumab and other hemophilia A treatment options.
Number of subjects to be contacted: In Kenya, 20 participants will be recruited from MTRH as the only participating site nationally. Globally, approximately 705 participants will be recruited.
Study procedures (main) done on participants:
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2 | Study Title: Prospective No-Interventional Multi-Center Study Evaluating Bleeding Incidence and Health-Related Quality of Life in Patients with Hemophilia A with Inhibitors and Non-Inhibitors Treated with Emicizumab and other Hemophilia A Treatments in a real-Life Setting.
Name of Principal Investigator: Festus Njuguna, MD
Co-investigators: Carole Kilach, MD
Supervisors: Gabriel Omwalo, Huwaida Bulhan
Name of Organization: AMPATH/MTRH
Funding: ROCHE Pharmaceuticals
Broad Objectives: To analyze all treated bleeds as measured by annualized bleeding rate (ABR) in hemophilia A patients according to treatment type within two patients’ groups (Cohort A: patients without FVIII inhibitors and Cohort B: patients with FVIII inhibitors) under routine clinical practice.
Specific Objectives:
Study setting: Hemophilia Clinic in Moi Teaching and Referral Hospital
Study population: Patients with congenital Severe Hemophilia A with or without inhibitors under treatment with Emicizumab and other hemophilia A treatment options.
Number of subjects to be contacted: In Kenya, 20 participants will be recruited from MTRH as the only participating site nationally. Globally, approximately 705 participants will be recruited.
Study procedures (main) done on participants:
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