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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

Full criteria on ClinicalTrials.gov

Sites (20)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
UZ Leuven
Coordinator of this site? Confirm its status
LeuvenBelgiumCompleted
Cliniques Universitaires Saint-Luc
Coordinator of this site? Confirm its status
Sint-Lambrechts-WoluweBelgiumCompleted
Kaye Edmonton Clinic
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EdmontonCanadaUnknown status
Eastern Health - General Hospital
Coordinator of this site? Confirm its status
St. John'sCanadaUnknown status
Universitaetsklinikum Bonn AöR
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BonnGermanyCompleted
Hospital Universitario La Paz
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MadridSpainUnknown status
Hospital Universitario Virgen del Rocio
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SevilleSpainUnknown status
Assistance Publique Hopitaux De Paris
Coordinator of this site? Confirm its status
Le Kremlin-BicetreFranceCompleted
Assistance Publique Hopitaux de Paris
Coordinator of this site? Confirm its status
ParisFranceCompleted
Cardiff and Vale NHS Trust
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CardiffUnited KingdomUnknown status
Royal Free Hospital
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LondonUnited KingdomUnknown status
Great Ormond Street Hospital For Children NHS Foundation Trust
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LondonUnited KingdomUnknown status
Amsterdam UMC, location AMC
Coordinator of this site? Confirm its status
AmsterdamNetherlandsUnknown status
Instytut Hematologii I Transfuzjologii
Coordinator of this site? Confirm its status
WarsawPolandUnknown status
University of Michigan, C.S. Mott Children's Hospital
Coordinator of this site? Confirm its status
Ann ArborUnited StatesUnknown status
Hemophilia of Georgia Center for Bleeding & Clotting Disorders
Coordinator of this site? Confirm its status
AtlantaUnited StatesUnknown status
Indiana Hemophilia & Thrombosis Center
Coordinator of this site? Confirm its status
IndianapolisUnited StatesUnknown status
Childrens Hospital LA
Coordinator of this site? Confirm its status
Los AngelesUnited StatesUnknown status
Washington Institute for Coagulation
Coordinator of this site? Confirm its status
SeattleUnited StatesUnknown status
Charlotte Maxeke Johannesburg Hospital
Coordinator of this site? Confirm its status
JohannesburgSouth AfricaUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.