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

Pfizer

Status and phase: Completed; Phase 4

Conditions: Hemophilia A

Treatments: Drug: Intravenous infusions of Xyntha

Study type: Interventional

Funder types: Industry

Identifiers

NCT02492984

2015-005040-33 (EudraCT Number)

B1831083

Details and patient eligibility

About

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

Full 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%).

Enrollment: 85 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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85 participants in 1 patient group

Intravenous infusions of Xyntha

Experimental group

Description:

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.

Treatment:

Drug: Intravenous infusions of Xyntha