A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

Last updated: December 19, 2024
Sponsor: Pfizer
Overall Status: Active - Recruiting

Phase

3

Condition

Hemophilia

Treatment

marstacimab

Clinical Study ID

NCT05611801
B7841008
2022-500495-65-00
ANTI-TFPI PEDIATRIC STUDY
  • Ages 1-17
  • Male

Study Summary

The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.

This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.

This study will enroll participants who:

  • have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors)

  • have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study

  • if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry

  • if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry

All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff.

To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia.

Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Male participants of appropriate age and required minimum weight

  • Participants aged 12 to 17 years must be at least 25 kgs at time of consent.

  • Participants aged 6 to 11 years must be at least 19 kgs at time of consent.

  • Minimum weight requirement for participants aged 1 to 5 years is to be determined.

  • Participants with a diagnosis of severe hemophilia A or moderately severe to severehemophilia B

  • Participants must have at least 1 year of diary and/or medical records available inwhich exogenous FVIII or FIX replacement or bypass agent infusions and hemophilicbleeding episodes were consistently documented over the 12 months prior to the timeof consent.

Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:

  • No current detectable inhibitor and no documented history of inhibitors in the 5years prior to consent

  • Must have at least 50 exposure days to FVIII/FIX replacement products

  • Must be at least 80% compliant with a stable and effective routine prophylaxisregimen with FVIII/FIX replacement products, for at least 12 months prior to consent

Participants who are enrolled into the Inhibitor Cohort must also meet the following criteria:

  • Documentation of current high titer inhibitor (≥5 BU/mL); or current low titerinhibitor (<5 BU/mL) refractory to FVIII or FIX replacement and with FVIII or FIXrecovery <60% of expected within previous 12 months prior to the time of consent

  • Participants who have documented inhibitors while on factor-replacement therapy butwho do not meet the high quantitative inhibitor criteria described in the priorbullet at the time of screening (eg, participant with a previously documentedhigh-titer inhibitor ≥5 BU/mL) and whose condition precludes re-challenge with FVIIIor FIX replacement may be considered for eligibility on a case-by-case basis withdiscussion and agreement from the Pfizer medical monitor.

  • Hemophilia A participants with on-demand treatment regimen with ≥12 bleedingepisodes or hemophilia B participants with on-demand treatment regimen with ≥8bleeding episodes (spontaneous or traumatic) necessitating treatment with bypassfactor in the 12 months prior to informed consent

  • Participants must be on an on-demand bypass treatment regimen during the 12 monthsprior to informed consent

Exclusion

Exclusion Criteria:

  • Known coronary artery, thrombotic, or ischemic disease, or current evidence ofcongenital or acquired thrombophilic disease such as Anti-thrombin III deficiency,Factor V Leiden mutation, prothrombin 20210 mutation, protein C deficiency, proteinS deficiency and antiphospholipid syndrome.

  • Known planned surgical procedure during the planned study period

  • Known hemostatic defect other than hemophilia A or B

  • Abnormal hematology, renal or hepatic function laboratory results at screening

  • Other acute or chronic medical or psychiatric condition that may increase the riskassociated with study participation or investigational product administration or mayinterfere with the interpretation of study results and, in the judgment of theinvestigator

  • Individuals with known allergic reaction or hypersensitivity to hamster protein orother components of the study intervention

  • Current routine prophylaxis with bypassing agent, non-coagulation non-factorreplacement therapy (eg, emicizumab), or any previous treatment with a gene therapyproduct for treatment of hemophilia

  • Participants with inhibitors who are being treated using a prophylaxis treatmentregimen with a bypass agent, and, participants who have previously receivednon-factor-based hemophilia therapy (eg, fitusiran, concizumab, emicizumab) will beconsidered on a case-by-case basis, only after discussion and agreement between theinvestigator and the Pfizer medical monitor

  • Regular use of immunomodulatory medications (eg, IVIG, routine systemiccorticosteroids, rituximab)

  • Use of systemic antifibrinolytics, medications that may increase the risk ofbleeding, and certain non-steroidal anti-inflammatory drugs within 120 hours offirst dose of study intervention and while on study

  • Ongoing or planned use of ITI, or prophylaxis with FVIII or FIX replacement at anytime after initiation of treatment with study intervention

  • Participation in other studies involving investigational drug(s) or investigationalvaccine(s) within 30 days (or as determined by local requirements) or 5 half-livesprior to study entry or during study participation

  • Previous exposure to marstacimab during participation in other marstacimab clinicalstudies

  • CD4 cell count ≤200/uL if HIV-positive

  • Abnormal ECG of clinical relevance that may affect participant safety orinterpretation of study results

  • Investigator site staff members directly involved in the conduct of the study andtheir family members, site staff members otherwise supervised by the investigator,and sponsor and sponsor delegate employees directly involved in the conduct of thestudy and their family members

Study Design

Total Participants: 100
Treatment Group(s): 1
Primary Treatment: marstacimab
Phase: 3
Study Start date:
December 09, 2022
Estimated Completion Date:
September 10, 2028

Connect with a study center

  • Arbesu Hematología

    Mendoza, M5501
    Argentina

    Active - Recruiting

  • Sydney Children's Hospital

    Randwick, New South Wales 2031
    Australia

    Site Not Available

  • Royal Children's Hospital

    Melbourne, Victoria 3052
    Australia

    Active - Recruiting

  • Medizinische Universität Wien

    Vienna, Wien 1090
    Austria

    Active - Recruiting

  • HEMOES

    Vitoria, Espírito Santo 29047-105
    Brazil

    Active - Recruiting

  • Stollery Children's Hospital

    Edmonton, Alberta T6G 2B7
    Canada

    Active - Recruiting

  • CancerCare Manitoba

    Winnipeg, Manitoba R3E 0V9
    Canada

    Active - Recruiting

  • Hamilton Health Sciences - McMaster University Medical Centre

    Hamilton, Ontario/canada L8N 3Z5
    Canada

    Active - Recruiting

  • Beijing Children's hospital, Capital Medical University

    Beijing, Beijing 100045
    China

    Active - Recruiting

  • Southern Medical University Nanfang Hospital

    Guangzhou, Guangdong 510515
    China

    Active - Recruiting

  • The Affiliated Hospital of Guizhou Medical University

    Guiyang, Guizhou 550004
    China

    Active - Recruiting

  • Tongji Hospital of Tongji Medical College of HUST/Pediatric Hematology Department Pharmacy

    Wuhan, Hubei 430030
    China

    Active - Recruiting

  • Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology

    Wuhan, Hubei 430030
    China

    Active - Recruiting

  • Jiangxi Provincial People's Hospital

    Nanchang, Jiangxi 330006
    China

    Active - Recruiting

  • Institute of hematology&blood disease hospital

    Tianjin, Tianjin
    China

    Active - Recruiting

  • Detska nemocnice FN Brno

    Brno, Brno-město 613 00
    Czechia

    Active - Recruiting

  • Rigshospitalet

    Copenhagen, Hovedstaden 2100
    Denmark

    Active - Recruiting

  • Aarhus Universitetshospital, Skejby

    Aarhus, Midtjylland 8200
    Denmark

    Site Not Available

  • Hôpital Universitaire Necker Enfants Malades

    Paris, 75015
    France

    Active - Recruiting

  • Charité Campus Virchow-Klinikum

    Berlin, 13353
    Germany

    Active - Recruiting

  • Nirmal Hospital Pvt Ltd.

    Surat, Gujarat 395002
    India

    Active - Recruiting

  • K.J. Somaiya Hospital

    Mumbai, Maharashtra 400022
    India

    Active - Recruiting

  • Nil Ratan Sircar Medical College and Hospital

    Kolkata, WEST Bengal 700014
    India

    Active - Recruiting

  • Sheba Medical Center

    Ramat Gan, Hamerkaz 5262100
    Israel

    Active - Recruiting

  • IRCCS Istituto Giannina Gaslini

    Genova, Liguria 16147
    Italy

    Active - Recruiting

  • Istituto Clinico Humanitas

    Rozzano, Milano 20089
    Italy

    Active - Recruiting

  • Ospedale Pediatrico Bambino Gesù IRCCS

    Rome, Roma 00165
    Italy

    Site Not Available

  • Azienda Ospedaliero Universitaria di Parma

    Parma, 43126
    Italy

    Site Not Available

  • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino

    Torino, 10126
    Italy

    Site Not Available

  • Hyogo prefectural Kobe Children's Hospital

    Kobe, Hyogo 650-0047
    Japan

    Active - Recruiting

  • Nagano Children's Hospital

    Azumino, Nagano 399-8288
    Japan

    Active - Recruiting

  • Nara Medical University Hospital

    Kashihara, Nara 634-8522
    Japan

    Active - Recruiting

  • Saitama Prefectural Children's Medical Center

    Saitama-shi, Saitama 330-8777
    Japan

    Active - Recruiting

  • Saga University Hospital

    Saga, 849-8501
    Japan

    Active - Recruiting

  • Kyung Hee University Hospital at Gangdong

    Seoul, Seoul-teukbyeolsi [seoul] 05278
    Korea, Republic of

    Active - Recruiting

  • Severance Hospital, Yonsei University Health System

    Seoul, Seoul-teukbyeolsi [seoul] 03722
    Korea, Republic of

    Active - Recruiting

  • Kyungpook National University Hospital

    Daegu, Taegu-kwangyǒkshi 41944
    Korea, Republic of

    Active - Recruiting

  • Arké SMO S.A de C.V

    Veracruz, 91910
    Mexico

    Site Not Available

  • King Fahad Specialist Hospital - Dammam

    Dammam, ASH Sharqīyah 31444
    Saudi Arabia

    Site Not Available

  • King Faisal Specialist Hospital & Research Center

    An Narjis,
    Saudi Arabia

    Site Not Available

  • King Faisal Specialist Hospital & Research Center

    An Narjis, Riyadh,
    Saudi Arabia

    Site Not Available

  • King Faisal Specialist Hospital & Research Center

    Riyadh,
    Saudi Arabia

    Site Not Available

  • Univerzitna nemocnica Bratislava, Nemocnica sv. Cyrila a Metoda

    Bratislava, 851 07
    Slovakia

    Active - Recruiting

  • Detska fakultna nemocnica Kosice

    Kosice, 040 11
    Slovakia

    Active - Recruiting

  • Univerzitna nemocnica Martin

    Martin, 036 59
    Slovakia

    Active - Recruiting

  • Worthwhile Clinical Trials

    Benoni, Gauteng 1500
    South Africa

    Site Not Available

  • Charlotte Maxeke Johannesburg Academic Hospital

    Johannesburg, Gauteng 2193
    South Africa

    Site Not Available

  • Haemophilia Comprehensive Care Centre

    Johannesburg, Gauteng 2193
    South Africa

    Site Not Available

  • Hospital Universitario La Paz

    Madrid, 28046
    Spain

    Active - Recruiting

  • Hospital Universitario Miguel Servet

    Zaragoza, 50009
    Spain

    Active - Recruiting

  • Changhua Christian Hospital

    Changhua County, Changhua 50006
    Taiwan

    Active - Recruiting

  • Taichung Veterans General Hospital

    Taichung, 407
    Taiwan

    Active - Recruiting

  • National Taiwan University Hospital

    Taipei, 10002
    Taiwan

    Active - Recruiting

  • Erciyes University

    Talas, Kayseri 38039
    Turkey

    Active - Recruiting

  • Istanbul Universitesi Istanbul Tıp Fakultesi Hastanesi

    İstanbul, İ̇stanbul 34093
    Turkey

    Active - Recruiting

  • Ege Universitesi Hastanesi

    Izmir, İ̇zmir 35100
    Turkey

    Active - Recruiting

  • Acibadem Adana Hospital

    Adana, 01130
    Turkey

    Active - Recruiting

  • Gazi University Health Research and Application Center Gazi Hospital

    Ankara, 06560
    Turkey

    Active - Recruiting

  • Erciyes Universitesi Tıp Fakultesi Hastaneleri

    Kayseri, 38039
    Turkey

    Active - Recruiting

  • Ondokuz Mayıs Universitesi

    Samsun, 55200
    Turkey

    Active - Recruiting

  • Royal Victoria Infirmary

    Newcastle upon Tyne, England NE1 4LP
    United Kingdom

    Active - Recruiting

  • Birmingham Children's Hospital

    Birmingham, B4 6NH
    United Kingdom

    Site Not Available

  • Leeds General Infirmary

    Leeds, LS1 3EX
    United Kingdom

    Site Not Available

  • Evelina London Children's Hospital

    London, SE1 7EH
    United Kingdom

    Active - Recruiting

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL
    United Kingdom

    Active - Recruiting

  • Intermountain - Primary Children's Hospital

    Salt Lake City, Utah 84113
    United States

    Active - Recruiting

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