NCT02492984

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
85

participants targeted

Target at P50-P75 for phase_4

Timeline
Completed

Started Apr 2015

Geographic Reach
1 country

15 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 19, 2015

Completed
13 days until next milestone

Study Start

First participant enrolled

April 1, 2015

Completed
3 months until next milestone

First Posted

Study publicly available on registry

July 9, 2015

Completed
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2016

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2016

Completed
8 months until next milestone

Results Posted

Study results publicly available

April 4, 2017

Completed
Last Updated

April 4, 2017

Status Verified

February 1, 2017

Enrollment Period

1.3 years

First QC Date

March 19, 2015

Results QC Date

December 19, 2016

Last Update Submit

February 16, 2017

Conditions

Keywords

FVIII inhibitor

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants With Factor VIII (FVIII) Inhibitors

    Percentage of participants with the product medically important event (MIE) (FVIII inhibitor development during the study).

    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 (10)

  • Number of Participants With All Causality Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)

    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).

  • Response Assessment of On-Demand Treatment of Bleeds

    From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.

  • Number of Infusions Needed to Treat Each New Bleed for On-Demand Treatment

    From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.

  • Frequency of Xyntha Infusions to Treat Each New Bleed for On-Demand Group

    From Day 1 up to participants had received treatment for 6 months or when participants had achieved 50 EDs whichever occurred first.

  • Hemostatic Efficacy for Surgical Prophylaxis Treatment

    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)

  • +5 more secondary outcomes

Study Arms (1)

Intravenous infusions of Xyntha

EXPERIMENTAL

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.

Drug: Intravenous infusions of Xyntha

Interventions

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.

Also known as: Xyntha (Moroctocog-alfa (AF-CC)
Intravenous infusions of Xyntha

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

The Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, 550004, China

Location

Henan Provincial People's Hospital

Zhengzhou, Henan, 450003, China

Location

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

Wuhan, Hubei, 430030, China

Location

Department of Hematology,The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

Location

Department of Hematology,Jiangxi Provincial People's Hospital

Nanchang, Jiangxi, 330006, China

Location

Blood Center of Shandong Province

Jinan, Shandong, 250014, China

Location

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, 610073, China

Location

Blood Diseases Hospital, Chinese Academy of Medical Science (Institute of Hematology)

Tianjin, Tianjin Municipality, 300020, China

Location

Department of Hematology,The First Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, 650032, China

Location

Beijing Children's hospital

Beijing, 100045, China

Location

The second affiliated hospital of chongqing medical university

Chongqing, 400010, China

Location

Children's Hospital of Chongqing Medical University

Chongqing, 400014, China

Location

Hematology Department, Nanfang Hospital, Southern Medical University

Guangzhou, 510515, China

Location

Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine/Hematology Department

Shanghai, 200025, China

Location

Department of Hematology/Children's Hospital of Shanghai

Shanghai, 200040, China

Location

Related Publications (1)

  • 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.

Related Links

MeSH Terms

Conditions

Hemophilia A

Interventions

F8 protein, humanFactor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Results Point of Contact

Title
Pfizer ClinicalTrials.gov Call Center
Organization
Pfizer, Inc.

Study Officials

  • Pfizer CT.gov Call Center

    Pfizer

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 19, 2015

First Posted

July 9, 2015

Study Start

April 1, 2015

Primary Completion

July 1, 2016

Study Completion

August 1, 2016

Last Updated

April 4, 2017

Results First Posted

April 4, 2017

Record last verified: 2017-02

Locations