Brief Title
Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A
Official Title
An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of rFVIII in the Prevention and Treatment of Bleeding Episodes in Chinese With Hemophilia A
Brief Summary
Efficacy, Safety and Pharmacokinetics Study of a rFVIII in Chinese subjects with Hemophilia
A.To assess efficacy and safety of rFVIII administered as treatment and as on-demand therapy
in adult and adolescent (12-65 years) patients with severe or moderately severe Hemophilia A.
To determine the pharmacokinetic (PK) parameters of rFVIII.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Recovery rate = (change actual value of rFVIII activity before and after infusion )/(change expected value of rFVIII activity before and after infusion)*100%
Secondary Outcome
The proportion of subjects who achieved the expected effect after the first infusion of the rFVIII
Condition
Hemophilia A
Intervention
Recombinant Factor VIII (50 IU/kg)
Study Arms / Comparison Groups
Pharmacokinetic parameters
Description: Pharmacokinetic parameters of rFVIII measured in subset of 10 participants, consisting of:18 Years to 65 Years. In Part 1 of the study, subjects received a single intravenous infusion of 50 IU/kg rFVIII preceded by a 72 hours washout period.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
60
Start Date
August 2016
Completion Date
December 2017
Primary Completion Date
October 2017
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of hemophilia A
- Age of 12 Years to 65 Years，Diagnosis of severe （defined as <1% FVIII:C documented in
medical records） or moderately severe（defined as 1%-5% FVIII:C documented in medical
records） hemophilia A .Subjects who（Age of 18 Years to 65 Years） have received or are
currently receiving FVIII products (plasma-derived and/or recombinant FVIII) and have
had >150 exposure days (EDs) with a FVIII product；The Callan (Age of 12 Years to 17)
have received FVIII products and have had>50 EDs a FVIII product.
- Subjects without a past history of, or current no factor VIII inhibitor. For
laboratory-based assessments, any Bethesda inhibitor titer Lower than the laboratory's
normal range or <0.6 BU/mL (BU:Bethesda Units ).
- Liver and kidney function in accordance with the standard
- Subjects of childbearing potential should agree to use and utilize an adequate method
of contraception throughout treatment and for at least 28 days after study is stopped
- Evidence of a personally or legally acceptable representative (legally acceptable
representative is only applicable to Callan subjects) signed and dated informed
consent document indicating that the subject has been informed of all pertinent
aspects of the study
- The part one of subjects subjects who are willing and able to comply with scheduled
visits, treatment plan, laboratory tests, and other study procedures; Subjects must be
in a non bleeding state before the administration of rFVIII on Day 1; Subjects should
not have received an infusion of any FVIII products for at least 3 days (at least 72
hours) before the administration of rFVIII on Day 1
Exclusion Criteria:
- Current FVIII inhibitor or history of FVIII inhibitor (>0.6 BU/mL )
- Diagnosed with any bleeding disorder in addition to hemophilia
- Documented Human Immunodeficiency Virus (HIV)
- Subjects anticipating elective surgery or other invasive procedure within 1 month
following study entry
- Treatment with an immunomodulatory within 30 days or 5 half lives preceding Day 1,
whichever is longer
- Subjects with known hypersensitivity to the active substance or to any of the
excipients of rFVIII. Subjects with a known hypersensitivity to Chinese Human
embryonic kidney cell proteins
- Subjects with severe anemia requiring blood transfusion
- Subjects with significant hepatic or renal impairment (alanine aminotransferase (ALT)
or aspartate aminotransferase (AST) >5 x ULN, or total bilirubin >2 x ULN or serum
creatinine >2 x ULN), prothrombin time >1.5 x ULN, platelet count <80,000 μL. History
of sensitivity to heparin or heparin induced thrombocytopenia or others
thrombocytopenia
- Patients with heart surgery history requires anticoagulation therapy; Subjects with
severe heart disease, including myocardial infarction or heart failure Grade 3 or
higher(NYHA Classification)
- Blood pressure unable to be controlled ideally(systolic pressure>150 mmHg，diastolic
pressure>90 mmHg)
- Other severe acute or chronic medical or psychiatric condition or laboratory
abnormality that may increase the risk associated with study participation or
investigational product administration or may interfere with the interpretation of
study results and, in the judgment of the investigator, would make the subject
inappropriate for entry into this study
Gender
All
Ages
12 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
, , [email protected]
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT02930317
Organization ID
CTTQ-NXBYZ
Responsible Party
Sponsor
Study Sponsor
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Study Sponsor
, ,
Verification Date
October 2016