New shot cuts bleeding in hemophilia a patients
NCT ID NCT03315455
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tested emicizumab (Hemlibra) in 85 people with hemophilia A, a condition where blood doesn't clot properly, causing frequent bleeds. Participants received either emicizumab injections or no preventive treatment. The study found that those on emicizumab had significantly fewer bleeding episodes, suggesting it could be a valuable option for managing the disease.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- emicizumab (Hemlibra)
- What this could lead to
- If successful, this could provide a more convenient and effective way to prevent bleeding episodes in people with hemophilia A, reducing the need for frequent intravenous treatments.
- What could go wrong
- The trial is completed and results are positive, but long-term safety and effectiveness in broader populations still need monitoring. Some patients may still experience breakthrough bleeds or side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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85 people
The number who actually took part.
- Started
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Apr 2018
- Finished
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Aug 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: Inclusion Criteria for Arms A, B, and C: * Diagnosis of severe congenital hemophilia A or hemophilia A with FVIII inhibitors * Aged 12 years or older at the time of informed consent * Body weight ≥40 kilograms (kg) at the time of screening * Participants without FVIII inhibitors (\<0.6 Bethesda unit per milliliter \[BU/mL\]) who completed successful immune tolerance induction (ITI) must have done so at least 5 years before screening and have no evidence of inhibitor recurrence (permanent or temporary) * Documentation of the details of episodic therapy (FVIII or bypassing agents) and of number of bleeding episodes for at least the last 24 weeks and ≥5 bleeds in the last 24 weeks prior to study entry * Adequate hematologic, hepatic, and renal function * For women of child bearing potential: agreement to remain abstinent or use a protocol defined contraceptive measure during the treatment period and for at least 5 elimination half-lives (24 weeks) after the last dose of study drug Inclusion Criteria for Arm D: * Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (i.e., ≥5 BU/mL) * Children \<12 years old at time of informed consent * Body weight \>3 kg at time of informed consent * Requires treatment with bypassing agents * Adequate hematologic, hepatic, and renal function * For female participants who are of childbearing potential, follow the same contraception criteria as listed above for Arms A, B, and C Exclusion Criteria: Exclusion Criteria for Arms A, B, and C: * Inherited or acquired bleeding disorder other than hemophilia A * At high risk for thrombotic microangiopathy, in the investigator's judgment * History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator's judgment * Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease * Other conditions that may increase risk of bleeding or thrombosis * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Known human immuno-deficiency virus (HIV) infection with cluster of differentiation 4 (CD4) count \<200 cells/microliter (cells/mcL) within 24 weeks prior to screening. Participants with HIV infection who have CD4 \>200 cells/mcL and meet all other criteria are eligible * Use of systemic immunomodulators at enrollment or planned use during the study, with the exception of anti-retroviral therapy * Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study * Planned surgery (excluding minor procedures such as tooth extraction or incision and drainage) during the study * Receipt of: Emicizumab in a prior investigational study; An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration; A non-hemophilia-related investigational drug concurrently, within last 30 days or 5 half-lives, whichever is shorter * Pregnant or lactating, or intending to become pregnant during the study Exclusion Criteria for Arm D: * Inherited or acquired bleeding disorder other than hemophilia A * Ongoing (or plan to receive during the study) ITI therapy or prophylaxis treatment with FVIII * Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease * Other diseases that may increase risk of bleeding or thrombosis * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Known infection with HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV) * At high risk for thrombotic microangiopathy, in the investigator's judgment * Use of systemic immunomodulators at enrollment or planned use during the study * Planned surgery (excluding minor procedures such as tooth extraction or incision and drainage) during the study * Inability (or unwillingness by caregiver) to receive (allow receipt of) blood or blood products (or any standard-of-care treatment for a life-threatening condition) * Receipt of: Emicizumab in a prior investigational study; An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration; A non-hemophilia-related investigational drug concurrently, within last 30 days or 5 half-lives, whichever is shorter * Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study * Pregnant or lactating, or intending to become pregnant during the study
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Beijing Children's Hospital, Capital Medical University
Beijing, 100045, China
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Peking Union Medical College Hospital
Beijing, 100730, China
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Penang General Hospital
George Town, Pulau Pinang, 10990, Malaysia
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Prince of Wales Hospital
Shatin, Hong Kong
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Queen Elizabeth Hospital
Sabah, Sabah, 88586, Malaysia
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Queen Mary Hospital
Hong Kong, Hong Kong
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Ramathibodi Hospital
Bangkok, 10400, Thailand
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Ruijin Hospital Shanghai Jiaotong University School of Medicine
Shanghai, 200025, China
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Southern Medical University Nanfang Hospital
Guangdong Province Guangzhou City, 510515, China
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Tianjin Institute of Hematology & Blood Diseases Hospital
Tianjin, 300020, China
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Tongji Hosp, Tongji Med. Col, Huazhong Univ. of Sci. & Tech
Wuhan, 430030, China
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Xiangya Hospital of Centre-South University
Changsha, 410008, China
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Xiehe Hospital, Tongji Medical College Huazhong University of Science & Technology
Wuhan, 430022, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a newer clotting factor keep its effectiveness in hemophilia a?
- Can a new injection tame hemophilia a bleeding?
- A Once-a-Week shot could transform hemophilia Care—Even for those with inhibitors
- Can a new clotting factor offer better bleed protection for severe hemophilia?
- Do newer hemophilia drugs protect joints better? study aims to find out
- Newborn screening study aims to catch rare diseases at birth