Acquired Hemophilia A (AHA) MedDRA version: 22.1 Level: LLT Classification code 10053761 Term: Acquired hemophilia with anti FVIII, XI, or XIII System Organ Class: 100000004851 MedDRA version: 20.0 Level: LLT Classification code 10053760 Term: Acquired hemophilia System Organ Class: 100000004851
Conditions
Interventions
Trade Name: Hemlibra®
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Emicizumab
Other descriptive name: RO5534262 EMICIZUMAB
Concentration unit: mg/ml milligram(s)/millilitre
Concent
Sponsors
GWT-TUD GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Patients diagnosed with AHA based on a reduced FVIII activity (0.6 BU/ml) at the time of diagnosis (local laboratory) 2) Signed informed consent form by the participant or a person who is legally authorized to sign on behalf of the participant before any study specific tests or procedures 3) Male or female patients aged 18 years or older at the time of informed consent 4) Ability to understand and follow study-related instructions 5) Current bleeds due to AHA at the time of screening Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 24 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 23
Exclusion criteria
Exclusion criteria: 1) Congenital hemophilia A 2) Partial or complete remission of AHA (defined as FVIII:C =50% and no bleeding and no hemostatic therapy) at the time of screening 3) Treatment with aPCC within the last 48 h before first study treatment or planned treatment with aPCC during the course of the study 4) Treatment of AHA within the days before study enrollment with more than 100 mg prednisolone (or equivalent) per day or prednisolone for more than 2 days or with other immunosuppressive drugs (e.g. rituximab, cyclophosphamide). IST for other concomitant disorders (e.g. autoimmune disorders) is not an exclusion criterion and can be continued at the investigator’s discretion. 5) Therapy (current or planned during the emicizumab treatment period) with immunosuppressive or immune modulating drugs that were not already given on a regular basis before first diagnosis of AHA 6) Positive lupus anticoagulant at the time of screening 7) Severe uncontrolled infection at the time of screening 8) Signs of active disseminated intravascular coagulation at the time of screening 9) Current treatment for thromboembolic disease or signs of current thromboembolic disease at time of screening 10) Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator’s judgment 11) Known severe congenital or acquired thrombophilia 12) Life expectancy 40 U/mL) b. Postoperatively (six weeks after bilateral ovariectomy with or without hysterectomy) c. Regular and correct use of a contraceptive method with error rate <1% per year such as implants, depot injections, oral contraceptives or intrauterine devices d. Sexual abstinence e. Vasectomy of the partner 21) Subject is in custody by order of an authority or a court of law 22) Receipt of an investigational drug concurrently or within 5 half-lives before administration of the study drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the present study is to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).;Secondary Objective: - To study the safety of emicizumab in patients with AHA - To compare bleeding and adverse events with the historic GTH-AH 01/2010 cohort - To compare bleeding and adverse events with a parallel US study - To evaluate the pharmacokinetics (PK) of emicizumab in patients with AHA ;Primary end point(s): The primary efficacy objective is to evaluate the efficacy of prophylactic emicizumab which will be evaluated by determination of the rate of clinically significant bleeds per patient-week until death or week 12 after starting emicizumab treatment, whatever occurs first.;Timepoint(s) of evaluation of this end point: 12 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary study objective to study the safety of emicizumab in patients with AHA will be evaluated on the basis of the incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy in the 12 weeks after starting emicizumab treatment and mortality in the 24 weeks after starting emicizumab. Further secondary endpoints include the bleeding-free survival in the 12 weeks after starting emicizumab treatment, mortality and cause of death, days of treatment with and dose of bypassing agents, days in hospital and number of patients archieving partial remission in the 24 weeks after starting emicizumab treatment. The additional exploratory objective to compare bleeding and adverse events with the historic GTH-AH 01/2010 cohort will be evaluated on basis of 1) the number of clinically significant bleeds per patient-week until death or week 12 after starting treatment, whatever occurs first; 2) the incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy until week 12 after starting treatment and mortality after 24 weeks, and 3) bleeding-free survival until week 12 after starting treatment. The additional exploratory objective to compare bleeding and adverse events with a parallel US study will be evaluated on basis of 1) the number of clinically significant bleeds per patient-week until death or week 12 after starting emicizumab treatment, whatever occurs first; 2) the incidence and severity of adverse events, thromboembolic events, thrombotic microangiopathy until week 12 after starting emicizumab treatment and mortality after 24 weeks, and 3) bleeding-free survival until week 12 after starting emicizumab treatment. PK parameters derived by emicizumab plasma levels will be analyzed and descriptively summarized. ;Timepoint(s) of evaluation of this end point: 12 weeks 24 weeks | — |
Countries
Austria, Germany
Contacts
Public ContactMedical Consulting
GWT-TUD GmbH
Outcome results
None listed