Clinical trial NCT02492984: PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A
Official title: An Open-label, Single-arm, Post- Authorization Pragmatic Clinical Trial On The Safety And Efficacy Of Xyntha (Moroctocog-alfa (Af-cc), Recombinant Fviii) In Subjects With Hemophilia A In Usual Care Settings In China
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemophilia A
Keywords: FVIII inhibitor
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2015-07-09
Enrollment: 85
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: An open-label, single-arm, post- authorization pragmatic clinical trial on the safety and efficacy of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in subjects with hemophilia A in usual care settings in China for approximately 6 months or or approximately 50 exposure days whichever occurs first

Detailed description: The purpose of this post-approval study is to provide supplementary information relating to the use of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in Chinese subjects with hemophilia A, especially on the safety and efficacy in different populations of Chinese hemophilia A patients, in particular in pediatric patients \<6 years of age, pediatric patients ≥6 to ≤12 years of age, Previously Untreated Patients (PUPs) , subjects receiving prophylaxis treatment after enrollment in the study, and severe patients (FVIII:C \<1%).

Eligibility criteria: Inclusion Criteria:

* Male and/or Female subjects with Hemophilia A.
* Subjects/parents/legal representatives must be able to comply with registry procedures (informed consent/assent process, clinical visits, reporting of infusion and bleed data, reporting of adverse events, etc).
* Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative, parent(s)/legal guardian) has been informed of all pertinent aspects of the study.

Exclusion Criteria:

* Presence of any other bleeding disorder in addition to hemophilia A.
* Treatment with immunomodulatory therapy (e.g., intravenous immunoglobulin, routine systemic corticosteroids, cyclosporins, anti-TNF agents) within 30 days prior to study entry or planned use for the duration of their study participation.
* Subjects with a past history of, or current factor VIII inhibitor. For laboratory-based assessments, any Bethesda inhibitor titer greater than the laboratory's normal range or ≥0.6 BU/mL.
* Subjects with known hypersensitivity to the active substance or to any of the excipients of Xyntha.
* Subjects with a known hypersensitivity to Chinese Hamster Ovary cell proteins.
* Unwilling or unable to follow the terms of the protocol.
* Any condition which may compromise the subject's ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; expectation of poor compliance in provision of observations for study-related documentation), in the opinion of the Investigator.
* Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins and/or during study participation (exception for studies on Xyntha).
* Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are Pfizer employees directly involved in the conduct of the study.

Primary outcomes:
measure: Percentage of Participants With Factor VIII (FVIII) Inhibitors
timeFrame: From Day 1 up to 28 calendar days after End of Treatment (participants had received treatment for 6 months or when participants had achieved 50 exposure days [EDs] whichever occurred first).
Secondary outcomes:
measure: Number of Participants With All Causality Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)
timeFrame: From Day 1 up to 28 calendar days after End of Treatment (participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first).
measure: Response Assessment of On-Demand Treatment of Bleeds
timeFrame: From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.
measure: Number of Infusions Needed to Treat Each New Bleed for On-Demand Treatment
timeFrame: From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.
measure: Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group
timeFrame: From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.
measure: Hemostatic Efficacy for Surgical Prophylaxis Treatment
timeFrame: From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery)
measure: Actual Estimated Blood Loss for Surgical Prophylaxis Treatment
timeFrame: From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery)
measure: Number of Participants With Transfusion Requirement for Surgical Prophylaxis Treatment
timeFrame: From day of surgery to postoperative period (at least 1-3 days post operation or until adequate wound healing for minor surgery or 4-6 days post operation or until threat resolved or adequate wound healing for major surgery)
measure: Average Infusion Dose and Total Factor VIII Consumption for On-Demand Treatment and Surgical Prophylaxis Treatment
timeFrame: On-Demand Group: Day 1 up to 6 months or 50 EDs whichever occurred first. Surgical Prophylaxis Group: Day of surgery to postoperative period. The duration of postoperative period is specified in previous endpoints.
measure: Percentage of Less Than Expected Therapeutic Effect (LETE) in the On-Demand Setting
timeFrame: From Day 1 up to participants had received treatment for 6 months or participants had achieved 50 EDs whichever occurred first.
measure: Number of Confirmed LETE in the Low Recovery Setting
timeFrame: From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.
Other outcomes:
Locations:
city: Guiyang
country: China
facility: The Affiliated Hospital of Guizhou Medical University
state: Guizhou
city: Zhengzhou
country: China
facility: Henan Provincial People's Hospital
state: Henan
city: Wuhan
country: China
facility: Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology
state: Hubei
city: Suzhou
country: China
facility: Department of Hematology,The First Affiliated Hospital of Soochow University
state: Jiangsu
city: Nanchang
country: China
facility: Department of Hematology,Jiangxi Provincial People's Hospital
state: Jiangxi
city: Jinan
country: China
facility: Blood Center of Shandong Province
state: Shandong
city: Chengdu
country: China
facility: Chengdu Women's and Children's Central Hospital
state: Sichuan
city: Tianjin
country: China
facility: Blood Diseases Hospital, Chinese Academy of Medical Science (Institute of Hematology)
state: Tianjin
city: Kunming
country: China
facility: Department of Hematology,The First Affiliated Hospital of Kunming Medical University
state: Yunnan
city: Beijing
country: China
facility: Beijing Children's hospital
city: Chongqing
country: China
facility: The second affiliated hospital of chongqing medical university
city: Chongqing
country: China
facility: Children's Hospital of Chongqing Medical University
city: Guang Zhou
country: China
facility: Hematology Department, Nanfang Hospital, Southern Medical University
city: Shanghai
country: China
facility: Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine/Hematology Department
city: Shanghai
country: China
facility: Department of Hematology/Children's Hospital of Shanghai
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Yang R, Zhao Y, Wang X, Sun J, Wu R, Jin C, Jin J, Wu D, Rendo P, Sun F, Rupon J, Huard F, Korth-Bradley JM, Xu L, Luo B, Liu YC. Safety and Efficacy of Moroctocog Alfa (AF-CC) in Chinese Patients with Hemophilia A: Results of Two Open-Label Studies. J Blood Med. 2020 Nov 25;11:439-448. doi: 10.2147/JBM.S241605. eCollection 2020.