A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

Study Identifier:
EFC17905
ClinicalTrials.gov Identifier:
NCT07285460
EudraCT Identifier:
N/A
Recruiting

Study Details

Medical Condition
  • Hemophilia
Study Drug
  • Drug: Fitusiran
  • Biological: Clotting factor concentrates (CFC) or bypassing agents (BPA)
  • Biological: Antithrombin concentrate (ATIIIC)
Date
Dec 2025 - Aug 2029
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 1 Year - 11 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Participants not previously exposed to fitusiran are eligible to be included in the study only if all of the following criteria apply:
  • Participant must be 1 to \<12 years of age at the time of enrollment.
  • Participants must have severe hemophilia A or B (FVIII \<1% or FIX ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
  • Participants must meet inhibitor or non-inhibitor status as defined below:
  • Inhibitor:
  • Requiring use of BPA for prophylaxis or BPA as on-demand therapy for any bleeding episodes for at least the last 3 months prior to screening, and meet one of the following Nijmegen-modified Bethesda assay results criteria:
  • Inhibitor titer of ≥0.6 BU/mL at screening, OR
  • Inhibitor titer of \<0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, OR
  • Inhibitor titer of \<0.6 BU/mL at screening with medical record evidence of 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response, or severe allergic reaction (eg, anaphylaxis) or nephrotic syndrome
  • Non-inhibitor:
  • Requiring use of clotting factor concentrates (CFCs) for prophylaxis or CFCs as on-demand therapy for any bleeding episodes for at least the last 3 months prior to screening, and meet each of the following criterion:
  • Nijmegen-modified Bethesda assay inhibitor titer of \<0.6 BU/mL at screening, AND
  • No use of BPA to treat bleeding episodes for at least the last 3 months prior to screening
  • Participants must have adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol.
  • Male: There are no contraceptive requirements for this study except where required by local regulations.
  • Capable of giving signed informed consent/assent. A signed written informed consent must be obtained from parent(s)/legal guardian (hereafter referred to as the "parent"), as well as a written or oral assent obtained from participant, per local and national requirements.
Exclusion Criteria
  • Participants not previously exposed to fitusiran are excluded from the study if any of the following criteria apply:
  • Known co-existing bleeding disorders other than hemophilia A or B.
  • Presence of clinically significant liver disease.
  • History of antiphospholipid antibody syndrome.
  • History of arterial or venous thromboembolism, unrelated to an indwelling venous access
  • Any condition (eg, medical concern), which in the opinion of the Investigator, would make the participant unsuitable for dosing or which could interfere with the study compliance, the participant's safety and/or the participant's participation in the completion of the treatment period of the study.
  • History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
  • Subjects with a central or peripheral indwelling catheter, with a history of venous access complications (such as infections, thrombosis) leading to hospitalization and/or systemic anticoagulation therapy in the last 12 months.
  • At screening, anticipated need of surgery during the study or planned surgery scheduled to occur during the study.
  • Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional BPA infusion for postoperative hemostasis.
  • History of intolerance to SC injection(s).
  • Current participation in ITI therapy.
  • The use of emicizumab (Hemlibra®) or any non-factor bleed management treatment within 6 months prior to screening
  • Prior gene therapy
  • Current or future participation in another clinical study, scheduled to occur during this study, involving an investigational product other than fitusiran or an investigational device.
  • AT activity \<60% at screening, as determined by central laboratory analysis.
  • Co-existing thrombophilic disorder.
  • Presence of an active Hepatitis C virus infection
  • Presence of acute hepatitis A or Hepatitis E virus infection.
  • Presence of acute or chronic hepatitis B virus infection.
  • Platelet count ≤100 000/μL.
  • Presence of acute infection at screening.
  • Human immunodeficiency virus (HIV) positive with a CD4 count of \<400 cells/μL.
  • Estimated glomerular filtration rate ≤45 mL/min/1.73 m2 (using the Schwartz formula).
  • The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Protocol Summary

This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

Trial Locations

Location
Status
Location
The Luskin Orthopaedic Institute for Children- Site Number : 8400013
Los Angeles, California, United States, 90007
Status
Recruiting
Location
The Center for Inherited Blood Disorders- Site Number : 8400009
Orange, California, United States, 92868
Status
Recruiting
Location
Cure 4 The Kids Foundation- Site Number : 8400001
Las Vegas, Nevada, United States, 89147
Status
Recruiting
Location
Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400008
Hackensack, New Jersey, United States, 07601
Status
Recruiting
Location
Investigational Site Number : 0560002
Brussels, Belgium, 1020
Status
Recruiting
Location
Investigational Site Number : 0560001
Brussels, Belgium, 1200
Status
Recruiting
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