NCT04158648CompletedPhase 3
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors
Source: ClinicalTrials.gov · Last updated 27 Aug 2026
Brief summary (registry, in English)
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against factor VIII (FVIII).
- Status (registry)
- Completed
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 73 (actual)
- First submitted
- 7 Nov 2019
- Start date
- Feb 2020
- Primary completion date
- Oct 2021
- Lead sponsor
- Hoffmann-La Roche
- Diseases
- Haemophilia
- NCT number
- NCT04158648
- EU CT number
- 2023-506610-52-00
- EudraCT number
- 2019-002179-32
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 73
- From Feb 2020 to the primary completion date, Oct 2021
- 20 sites in 10 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator's judgment
- Previous (within the last 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease
- Planned surgery during the emicizumab loading dose phase (surgeries in participants on emicizumab from Week 5 onwards are allowed)
- Receipt of any of the following: An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration with the exception of prior emicizumab prophylaxis; A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter; or Any other investigational drug currently being administered or planned to be administered
Other illness
- Inherited or acquired bleeding disorder other than mild or moderate congenital hemophilia A
- Other conditions that may currently increase the risk of bleeding or thrombosis
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
Lab values
- Known HIV infection with CD4 counts \<200 cells per microlitre (/μL)
- Concomitant disease, condition, significant abnormality on screening evaluation or laboratory tests, or treatment that could interfere with the conduct of the study, or that would in the opinion of the investigator, pose an additional unacceptable risk in administering study drug to the participant
- Pregnant or breastfeeding, or intending to become pregnant during the study (women of childbearing potential must have a negative serum pregnancy test result within 7 days prior to initiation of study drug)
Other
- Inability to comply with the study protocol in the opinion of the investigator
Who the study is looking for
- Diagnosis of mild (FVIII level between \>5% and \<40%) or moderate (FVIII level between ≥1% and ≤5%) congenital Hemophilia A without FVIII inhibitors
- Weight ≥3 kilograms (kg)
- Need for prophylaxis based on investigator assessment
- A negative test for inhibitor (i.e., \<0.6 Bethesda Units per milliliter \[BU/mL\]) within 8 weeks prior to enrollment
- No documented inhibitor (i.e., \<0.6 BU/mL), FVIII half-life \<6 hours, or FVIII recovery \<66% in the last 5 years
- Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks prior to enrollment
- Adequate hematologic, hepatic, and renal function
- For women of childbearing potential: agreement to remain abstinent or use contraception (as defined in the protocol) during the treatment period and for at least 24 weeks after the final dose of study drug
Sex: All · Does not accept healthy volunteers
Sites (20)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| UZ Leuven Coordinator of this site? Confirm its status | Leuven | Belgium | Completed |
| Cliniques Universitaires Saint-Luc Coordinator of this site? Confirm its status | Sint-Lambrechts-Woluwe | Belgium | Completed |
| Kaye Edmonton Clinic Coordinator of this site? Confirm its status | Edmonton | Canada | Unknown status |
| Eastern Health - General Hospital Coordinator of this site? Confirm its status | St. John's | Canada | Unknown status |
| Universitaetsklinikum Bonn AöR Coordinator of this site? Confirm its status | Bonn | Germany | Completed |
| Hospital Universitario La Paz Coordinator of this site? Confirm its status | Madrid | Spain | Unknown status |
| Hospital Universitario Virgen del Rocio Coordinator of this site? Confirm its status | Seville | Spain | Unknown status |
| Assistance Publique Hopitaux De Paris Coordinator of this site? Confirm its status | Le Kremlin-Bicetre | France | Completed |
| Assistance Publique Hopitaux de Paris Coordinator of this site? Confirm its status | Paris | France | Completed |
| Cardiff and Vale NHS Trust Coordinator of this site? Confirm its status | Cardiff | United Kingdom | Unknown status |
| Royal Free Hospital Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Great Ormond Street Hospital For Children NHS Foundation Trust Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Amsterdam UMC, location AMC Coordinator of this site? Confirm its status | Amsterdam | Netherlands | Unknown status |
| Instytut Hematologii I Transfuzjologii Coordinator of this site? Confirm its status | Warsaw | Poland | Unknown status |
| University of Michigan, C.S. Mott Children's Hospital Coordinator of this site? Confirm its status | Ann Arbor | United States | Unknown status |
| Hemophilia of Georgia Center for Bleeding & Clotting Disorders Coordinator of this site? Confirm its status | Atlanta | United States | Unknown status |
| Indiana Hemophilia & Thrombosis Center Coordinator of this site? Confirm its status | Indianapolis | United States | Unknown status |
| Childrens Hospital LA Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Washington Institute for Coagulation Coordinator of this site? Confirm its status | Seattle | United States | Unknown status |
| Charlotte Maxeke Johannesburg Hospital Coordinator of this site? Confirm its status | Johannesburg | South Africa | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.