Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

Hemophilia A Clinical Trial

Official title:

An Evaluation Of The Safety And Efficacy Of On-Demand Treatment With Xyntha (B Domain Deleted Recombinant Factor VIII, Albumin Free) In Chinese Subjects With Hemophilia A

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00868530
• Other study ID #: 3082B2-3316
• Secondary ID: B1831015
• Status: Completed
• Phase: Phase 3
• First received: March 19, 2009
• Last updated: April 1, 2011
• Start date: September 2008
• Est. completion date: December 2009

Study information

• Verified date: April 2011
• Source: Wyeth is now a wholly owned subsidiary of Pfizer
• Contact: n/a
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the safety and efficacy of on-demand treatment with Xyntha in Chinese hemophilia A subjects.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 53
• Est. completion date: December 2009
• Est. primary completion date: December 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

• Subjects equal or more than 6 years of age with mild, moderate or severe hemophilia A (FVIII activity: more than 5%, 1-5%, or less than 1%, respectively)

• Subjects with previous exposure to FVIII replacement therapy

• If human immunodeficiency virus (HIV) positive, documented cluster of differentiation (CD4) count more than 200/µL within 6 months of study entry

Exclusion Criteria:

• Diagnosed with any bleeding disorder in addition to hemophilia A

• Current FVIII inhibitor or history of FVIII inhibitor (defined as positive result of the reporting laboratory)

• Subject has no history of exposure to FVIII products (previously untreated patient [PUP])

• Subject is currently utilizing primary FVIII prophylaxis

• Subjects anticipating elective surgery that may be planned to occur in the 6 months following study entry

• Treated with immunomodulatory therapy within 30 days prior to study entry or planned use for the duration of their study participation

• Participated in another investigational drug or device study within 30 days prior to study entry or planned participation for the duration of their study participation

• Subjects with a known hypersensitivity to hamster protein

• Significant hepatic or renal impairment (alanine aminotransferase [ALT] and aspartate aminotransferase [AST] >5 x upper limit of normal [ULN], bilirubin >2 mg/dL or serum creatinine >1.25 x ULN)

• Prothrombin Time >1.5 x ULN

• Platelet count <80,000 / µL

• Pregnant or breastfeeding women

• 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, in the opinion of the Investigator or Sponsor

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• Xyntha
Xyntha for on-demand treatment of bleeding episodes were according to investigator prescription during the 6 months observation period. The recovery assessed by determining the Factor VIII (FVIII) concentration (FVIII:C) levels in individual subjects at the initial and final visits. The dose of Xyntha for recovery assessments is: single 50 IU/kg (±5 IU/kg) IV bolus infusion. All Xyntha administrations occurred in the clinic (hospital).

Locations

Country	Name	City	State
China	Pfizer Investigational Site	Beijing
China	Pfizer Investigational Site	Guangzhou	Guangzhou
China	Pfizer Investigational Site	Hangzhou	Zhejiang
China	Pfizer Investigational Site	Heping District	Tianjin
China	Pfizer Investigational Site	Shanghai
China	Pfizer Investigational Site	Suzhou	Jiangsu

Sponsors (1)

Lead Sponsor	Collaborator
Wyeth is now a wholly owned subsidiary of Pfizer

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion	The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).	8 hours post infusion	No
Primary	Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion	The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).	24 hours post infusion	No
Primary	Number of Participants With Factor VIII (FVIII) Inhibitor Development	Incidence of FVIII inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory. Incidence was stratified by participant exposure history: Minimally Treated Patients (MTPs): those who had received at least 1 prior FVIII infusion, and <= 100 documented Exposure Days (EDs), while Previously Treated Patients (PTPs): those who had received >100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.	Day 1 and Month 6 or Early Termination Visit	Yes
Secondary	FVIII Recovery : Change From Baseline in FVIII Concentration	FVIII recovery was assessed by evaluating the change in FVIII concentration at 6 months compared to baseline.	Day 1 and Month 6 or Early Termination Visit	No
Secondary	Number of Participants With Less Than Expected Therapeutic Effect (LETE)	The incidence of LETE, defined for on-demand treatment as no response after each of 2 successive infusions within 24 hours for the same bleeding event in the absence of confounding factors.	24 hours after each of 2 successive infusion, up to 6 months	No
Secondary	Number of Participants With Thrombosis Allergic-Type Reactions		Baseline up to 6 months	Yes
Secondary	Number of Participants With Thrombosis		Baseline up to 6 months	Yes

External Links

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