PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A

Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02492984
• Other study ID #: B1831083
• Secondary ID: 2015-005040-33
• Status: Completed
• Phase: Phase 4
• First received: March 19, 2015
• Last updated: September 26, 2016
• Start date: April 2015
• Est. completion date: August 2016

Study information

• Verified date: September 2016
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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

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%).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 85
• Est. completion date: August 2016
• Est. primary completion date: July 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Intravenous infusions of Xyntha
Enrolled subjects will be treated with intravenous infusions of Xyntha for: • On-Demand treatment, • Surgical Prophylaxis at a dose and frequency prescribed by the subject's treating physician in accordance with the Xyntha label and will be adjusted solely according to medical and therapeutic necessity.

Locations

Country	Name	City	State
China	Beijing Children's hospital	Beijing
China	Chengdu Women's and Children's Central Hospital	Chengdu	Sichuan
China	Children's Hospital of Chongqing Medical University	Chongqing
China	The second affiliated hospital of chongqing medical university	Chongqing
China	Hematology Department, Nanfang Hospital, Southern Medical University	Guang Zhou
China	Affiliate Hospital of Guiyang Medical College	Guiyang	Guizhou
China	Blood Center of Shandong Province	Jinan	Shandong
China	Department of Hematology,The First Affiliated Hospital of Kunming Medical University	Kunming	Yunnan
China	Department of Hematology,Jiangxi Provincial People's Hospital	Nanchang	Jiangxi
China	Department of Hematology/Children's Hospital of Shanghai	Shanghai
China	Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine/Hematology Department	Shanghai
China	Department of Hematology,The First Affiliated Hospital of Soochow University	Suzhou	Jiangsu
China	Blood Diseases Hospital, Chinese Academy of Medical Science (Institute of Hematology)	Tianjin	Tianjin
China	Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology	Wuhan	Hubei
China	Henan Provincial People's Hospital	Zhengzhou	Henan

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Safety as measured by Pediatric Quality of Life Inventory (PedsQL) questionnaire for on-demand treatment pediatric patients		Up to approximately 7 months	Yes
Primary	Safety as measured by FVIII inhibitor development	To evaluate the product medically important events (FVIII inhibitor development during the study) in Chinese hemophilia A subjects during treatment with Xyntha.	Up to approximately 7 months	Yes
Secondary	The overall safety of Xyntha, including the occurrence of AE s and SAEs.	To evaluate the overall safety of Xyntha, including the occurrence of AE s and SAEs	Up to approximately 7 months	Yes
Secondary	Efficacy as measured by the responses to all on-demand treatment with Xyntha for all bleeds		Up to approximately 6 months or approximately 50 exposure days whichever occurs first.	No
Secondary	Efficacy as measured by number of Xyntha infusions to treat each new bleed		Up to approximately 6 months (±7 days) or approximately 50 exposure days whichever occurs first	No
Secondary	Efficay as measured by the responses to all Surgical prophylaxis treatment with Xyntha		Treatment duration is to be determined according to the investigator's medical judgment based on the nature of the surgery carried out and subject conditions, not to exceed 3 weeks.	No
Secondary	Efficacy as measured by Blood loss in Surgical prophylaxis subjects		Treatment duration is to be determined according to the investigator's medical judgment based on the nature of the surgery carried out and subject conditions, not to exceed 3 weeks.	No
Secondary	Efficacy as measured by transfusion requirements in Surgical prophylaxis subjects		Treatment duration is to be determined according to the investigator's medical judgment based on the nature of the surgery carried out and subject conditions, not to exceed 3 weeks.	No
Secondary	Efficacy as measured by The average infusion dose and total factor VIII consumption		Up to 6months or 50 exposure days which occurs first and for surgical prophylaxis is to be determined according to the investigator's medical judgment based on the nature of the surgery carried out and subject conditions, not to exceed 3 weeks.	No
Secondary	Efficacy as measured by the incidence of LETE in the on-demand and low recovery (when available) settings		Up to approximately 6 months or approximately 50 exposure days whichever occurs first.	No

External Links

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