Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
- Conditions
- Hemophilia AHemophilia B
- Interventions
- Drug: PF-06741086
- Registration Number
- NCT05145127
- Lead Sponsor
- Pfizer
- Brief Summary
Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study B7841005 and from the Phase 3 Study B7841008.
Study B7841005: approximately 145 adolescent and adult participants 12 to \<75 years of age with severe hemophilia A or moderately severe to severe hemophilia B (defined as FVIII activity \<1% or FIX activity ≤2%, respectively) with or without inhibitors are expected to be enrolled in Study B7841005 during which they will receive prophylaxis (defined as treatment by SC injection of marstacimab).
Study B7841008: this is an ongoing Phase 3, open-label study in pediatric participants \<18 years of age with severe hemophilia A (FVIII Coagulation Factor Activity \<1%) or moderately severe to severe hemophilia B (FIX Coagulation Factor Activity ≤2%). A sequential approach will be used in enrolling at least 100 pediatric participants, at least 20 of which will be aged ≥12 to \<18 years and at least 80 participants will be aged ≥1 to \<12 years. At the start of study B7841008, the dosing and data available in adolescent and adult participants in Study B7841005 supported the initiation of B7841008 study in participants aged ≥12 to \<18 years. Subsequently, additional safety and efficacy data from adolescent participants in Study B7841005 became available for benefit/risk assessment in support of dosing participants aged ≥6 to \<12 years. Based on the positive benefit/risk assessment conducted by both internal Pfizer review and eDMC review, dosing of the ≥6 to \<12 years age group was initiated in June 2023 in B7841008 Study. Data from participants ≥6 years from B7841008 Study and Study B7841005 will support the dosing of participants aged ≥1 to \<6 years.
All participants will be provided the prefilled pen (PFP) for administration of marstacimab in the study. Use of the prefilled syringe (PFS) will be permitted at the investigator's discretion for those participants who have difficulty with administration of the PFP. Additionally, participants will be provided the PFS for use in this study in countries where the PFS is anticipated to be the only presentation available commercially. An optional, open-label, single arm, substudy using the PFP was completed in the first 23 participants rolled over from Study B7841005 who agreed to participate in the substudy.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 245
- All participants will have a minimum body weight as defined by parent studies
- Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.
- Participants have successfully completed participation in parent studies, defined as did not require "Early Termination"
- Previous or current treatment for or history of coronary artery disease, venous or arterial thrombosis (CTCAE Grade >3), or ischemic disease (except catheter-associated thrombosis)
- Abnormal renal function as defined by eGFR <30 mL.min/1.73 m(2)
- Known planned surgical procedure during the planned study period
- Unstable hepatic function as determined by the Investigator clinical assessment and review of the participant's most recent laboratory results, which would make the participant inappropriate for the study
- For participants known to be HIV+, worsening disease status as determined by the Investigator clinical assessment and review of participant's most recent laboratory results, to include recent locally available CD4 count (if available), which would make the participant inappropriate for the study
- Regular, concomitant therapy with immunomodulatory drugs (eg, IVIG, and routine systemic corticosteroids, rituximab)
- Ongoing or planned use of immune tolerance induction or prophylaxis with FVIII or FIX replacement during the study
- Participation in other study involving investigational drug(s) or investigational vaccine(s) within 30 days or 5 half-lives prior to or during study participation, with the exception of participation in parent studies
- Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the Investigator, and their respective family members
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description PF-06741086 PF-06741086 For participants aged ≥12 years 300 milligrams(mg) subcutaneous (sc) loading dose followed by 150 mg sq once weekly (qw). 300 mg sc qw is prescribed for participants who meet dose escalation criteria. For participants aged ≥6 to \<12 years is marstacimab 150 mg SC for initial loading dose followed by 75 mg SC QW. 150 mg sc qw is prescribed for participants who meet dose escalation criteria.
- Primary Outcome Measures
Name Time Method Number of subject reporting Adverse Events Baseline up to 7 years Number of subjects reporting Serious Adverse Events Baseline up to 7 years Incidence and severity of thrombotic events Baseline up to 7 years Incidence and severity of thrombotic microangiopathy Baseline up to 7 years Number of subjects reporting Disseminated intravascular coagulalopathy/consumption coagulopathy Baseline up to 7 years Incidence of clinically significant persistent NAb against marstacimab Baseline up to 7 years Incidence and severity of injection site reaction Baseline up to 7 years Clinically significant changes in vital signs from baseline Baseline up to 7 years Incidence of clinically significant laboratory value abnormalities Baseline up to 7 years Incidence of severe hypersensitivity and anaphylactic reactions Baseline up to 7 years
- Secondary Outcome Measures
Name Time Method Annualized rate of bleeding episodes Baseline up to 7 years Derived for each subject for each treatment period by using the following formula: ABR = number of bleeds requiring treatments/ (days on treatment period / 365.25)
Total coagulation factor product consumption Baseline up to 7 years Incidence of joint bleeds Baseline up to 7 years Incidence of spontaneous bleeds Baseline up to 7 years Incidence of target joint bleeds Baseline up to 7 years Incidence of total bleeds (treated and untreated) Baseline up to 7 year Change in joints measured by the HJHS Baseline up to 7 years Change in joints as measured by the HJHS for participants ≥4 years of age
Change in number of target joints per subject from baseline Baseline up to 7 years Changes in Health Utilities Measure questionnaire data Baseline up to 7 years Changes in Haem-A-QoL questionnaire data for participants ≥17 years of age Baseline up to 7 years Changes in Haemo-QoL questionnaire data Baseline up to 7 years Haemo-QoL CII (Ages 8 to \<12 years), Haemo-QoL (Ages 12 to \<17),
Total bypass product consumption Baseline up to 7 years Changes in EQ-5D questionnaire data Baseline up to 7 years EQ-5D-Y Proxy (Ages ≥ 4 to ≤ 6 years), EQ-5D-Y Self (Ages ≥ 7 to ≤ 11 years), EQ-5D-5L (Ages ≥12)
Trial Locations
- Locations (52)
University of Iowa
🇺🇸Iowa City, Iowa, United States
Northwell Health HTC
🇺🇸New Hyde Park, New York, United States
Washington Institute for Coagulation d/b/a WACBD
🇺🇸Seattle, Washington, United States
Royal Children's Hospital
🇦🇺Melbourne, Victoria, Australia
Stollery Children's Hospital
🇨🇦Edmonton, Alberta, Canada
Hamilton Health Sciences - McMaster University Medical Centre
🇨🇦Hamilton, Ontario, Canada
Nanfang Hospital, Southern Medical University
🇨🇳Guangzhou, Guangdong, China
The Affiliated Hospital of Guizhou Medical University
🇨🇳Guiyang, Guizhou, China
Tongji Hospital Tongji Medical,Science & Technology
🇨🇳Wuhan, Hubei, China
Jiangxi Provincial People's Hospital
🇨🇳Nanchang, Jiangxi, China
Institute of hematology&blood disease hospital
🇨🇳Tianjin, Tianjin, China
Beijing Children's Hospital, Capital Medical University
🇨🇳Beijing, China
Klinicki bolnicki centar Zagreb
🇭🇷Zagreb, Croatia
Hôpital Universitaire Necker Enfants Malades
🇫🇷Paris, France
Prince of Wales Hospital
🇭🇰Hong Kong, Hong Kong
Hospital Universitario A Coruna
🇪🇸A Coruna, Spain
Hospital Universitario Vall d´Hebron
🇪🇸Barcelona, Spain
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Hospital Universitario Miguel Servet
🇪🇸Zaragoza, Spain
ChangHua Christian Hospital
🇨🇳Changhua City, Changhua County, Taiwan
Taichung Veterans General Hospital
🇨🇳Taichung, Taiwan
Acibadem Adana Hospital
🇹🇷Adana, Turkey
Hacettepe University Faculty of Medicine
🇹🇷Ankara, Turkey
Gazi University Health Research and Application Center Gazi Hospital
🇹🇷Ankara, Turkey
Gaziantep University Şahinbey Research and Practice Hospital
🇹🇷Gaziantep, Turkey
Istanbul University Oncology Institute
🇹🇷Istanbul, Turkey
Queen Mary Hospital
🇭🇰Hong Kong, Hong Kong
Nirmal Hospital Pvt Ltd.
🇮🇳Surat, Gjuarat, India
K.J. Somaiya Hospital
🇮🇳Mumbai, Maharashtra, India
Sahyadri Super Speciality Hospital
🇮🇳Pune, Maharashtra, India
Nil Ratan Sircar Medical College and Hospital
🇮🇳Kolkata, WEST Bengal, India
Sheba Medical Center
🇮🇱Ramat Gan, Hamerkaz, Israel
AOU Policlinico Umberto I
🇮🇹Roma, RM, Italy
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Nagoya University Hospital - Transfusion Medicine
🇯🇵Nagoya, Aichi, Japan
Nagano Children's Hospital
🇯🇵Azumino, Nagano, Japan
Nara Medical University Hospital
🇯🇵Kashihara, Nara, Japan
Saitama Children's Medical Center
🇯🇵Saitama-shi, Saitama, Japan
Hiroshima University Hospital
🇯🇵Hiroshima, Japan
Severance Hospital, Yonsei University Health System
🇰🇷Seoul, Seoul-teukbyeolsi[seoul], Korea, Republic of
Kyung Hee University Hospital at Gangdong
🇰🇷Seoul, Seoul-teukbyeolsi [seoul], Korea, Republic of
Kyungpook National University Hospital
🇰🇷Daegu, Taegu-kwangyǒkshi, Korea, Republic of
Centro Multidisciplinario para el Desarrollo Especializado de la Investigacion Clinica en Yucatan
🇲🇽Merida, Yucatán, Mexico
Centro Multidisciplinario para el Desarrollo Especializado de la Investigacion Clinica en Yucatán S.
🇲🇽Merida, Yucatán, Mexico
Sultan Qaboos University Hospital
🇴🇲Muscat, Oman
Institute for Mother and Child healthcare "Dr Vukan Cupic"
🇷🇸Belgrade, Serbia
Clinical Center Nis
🇷🇸Nis, Serbia
Worthwhile Clinical Trials
🇿🇦Benoni, South Africa
Charlotte Maxeke Johannesburg Academic Hospital
🇿🇦Johannesburg, South Africa
Ege University Faculty of Medicine
🇹🇷Izmir, Turkey
Erciyes University Faculty of Medicine
🇹🇷Kayseri, Turkey
Ondokuz Mayıs University Healthcare Practice and Research Center
🇹🇷Samsun, Turkey
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