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

Study Objectives:

Effectiveness Objectives

The primary effectiveness objective for this study is as follows:

  • 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.

The secondary effectiveness objectives for this study are as follows:

  • To analyze the difference in number of all treated bleeds in hemophilia A patients before and after receiving emicizumab (intra-patient analysis). This analysis will only apply for the patients who are switched to emicizumab at least 6 months after study entry.
  • To analyze the number of all bleeds (treated and not treated), including joint bleeds, major bleeds, traumatic and spontaneous bleeds, in HA patients within two patients groups (Cohort A: patients without FVIII inhibitors and Cohort B: patients with FVIII inhibitors) under routine clinical practice.
  • To analyze the percentage of patients with zero bleeding rate.

Safety Objectives

The safety objectives for this study are as follows:

  • To collect information on safety in HA patients within two patients groups (Cohort A: patients without FVIII inhibitors and Cohort B: patients with FVIII inhibitors) under routine clinical practice.

 

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).

 

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:

  • To analyze the difference in number of all treated bleeds in Hemophilia A patients before and after receiving emicizumab (intra-patient analysis). This analysis will only apply for the patients who are switched to emicizumab at least 6 months after study entry. 
  • To analyze the number of all bleeds (treated and not treated), including joint bleeds, major bleeds, traumatic and spontaneous bleeds, in Hemophilia A patients within two patients’ groups (Cohort A: patients without FVIII inhibitors and Cohort B: patients with FVIII inhibitors) under routine clinical practice. 
  • To analyze the percentage of patients with zero bleeding rate. 
  • To collect information on safety in Hemophilia A patients within two patients’ groups (Cohort A: patients without FVIII inhibitors and Cohort B: patients with FVIII inhibitors) under routine clinical practice. 

 

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: 


 

  • Blood specimens drawn: N/A


 

  • Surgical procedure: N/A


 

  • Questionnaires: Yes


 

  • FGDs:  N/A


 

  • Informed consent: Written