China ADVATE PTP Study

NCT02170402 | Completed Phase 4

• 1 other identifier: 061301
• Study Type: interventional
• Target: 82
• Locations: 1 country
• Sites: 11

Brief Summary

The purpose of this study is to assess efficacy, safety and pharmacokinetics of ADVATE in the treatment and prevention of bleeding episodes (BEs)

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Percentage of reduction in annualized bleed rate (ABR) during prophylactic treatment compared to ABR during on demand treatment

  Computed as: {\[median ABR on-demand - median ABR prophylaxis\]÷\[median ABR on-demand\]}\*100% The ABR, will be assumed to have a negative binomial distribution. The 2 treatment regimens (on-demand and prophylaxis) will be compared in terms of mean ABR within a generalized linear model framework (with a logarithmic link function which is the default for the negative binomial distribution), accounting for the fixed effect of study arm and the follow-up time (in years) as an offset. Ratios between treatment means (95% CI) will be estimated within this model.

  12 months

Secondary Outcomes (12)

• Number of units per kg body weight of ADVATE required to resolve a bleeding episode (BE)

  12 months

• Number of infusions of ADVATE required to resolve a bleeding episode (BE)

  12 months

• Overall evaluation of efficacy on a four-point scale (Excellent-Good-Fair-Poor)

  12 months

• Annualized bleeding episode rates (ABR) according to bleed type and bleed etiology summarized by treatment regimen

  12 months

• Inhibitor incidence

  13 months

Study Arms (1)

Previously Treated Patients (PTPs)

EXPERIMENTAL

PTPs will participate sequentially with: Part 1: Pharmacokinetic parameters of ADVATE measured in subset of 24 participants, consisting of: * 12 adults (\>12 years of age) * 12 children (≤12 years of age) Part 2: On-demand treatment with ADVATE for 6 months Part 3: Prophylaxis regimen with ADVATE for 6 months

Biological: Octocog alfa (recombinant human coagulation factor VIII)

Interventions

Octocog alfa (recombinant human coagulation factor VIII) BIOLOGICAL

* Part 1: Pharmacokinetic (PK) analysis - Subset of 24 participants * Part 2: On-demand treatment regimen * Part 3: Prophylaxis treatment regimen

Also known as: ADVATE

Previously Treated Patients (PTPs)

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Ethnic Chinese
• is of any age
• has a documented diagnosis of severe or moderately severe hemophilia A (congenital FVIII deficiency: baseline Factor VIII (FVIII) ≤ 2%)
• has documented and verified \>50 exposure days (EDs) to FVIII (recombinant or plasma derived)
• is receiving on-demand treatment with FVIII at the time of enrolment in this study
• has negative history of inhibitor development
• is HIV negative or HIV positive with stable disease and CD4+ count ≥ 200 cells per mm\^3
• is negative for Hepatitis C virus (HCV); Or participant is HCV positive with chronic stable hepatitis as assessed by investigator

You may not qualify if:

• has prior history of hypersensitivity or anaphylaxis associated with receipt of FVIII
• is diagnosed with other bleeding disorder(s) other than hemophilia A, including but not limited to thrombocytopenia (platelet count \< 100000 /mL)
• has been exposed to an investigational product (IP) within 30 days prior to the screening visit or is scheduled to participate in another clinical study involving an IP or investigational device during participation in the study
• is planned, or likely to have surgery during the study period
• has end-stage renal failure or evidence of a severe or uncontrolled systemic disease as judged by the investigator
• has active hepatic disease (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels \> 5 times the upper limit of normal)
• has clinical or laboratory evidence of severe liver impairment including (but not limited to) a recent \& persistent international normalized ratio (INR) \>1.4, and/or the presence of splenomegaly and/or significant spider angioma on physical exam, and/or a history of esophageal hemorrhage or documented esophageal varices
• is a family member of the investigator or site staff

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (11)

Peking Union Medical College Hospital

Dongcheng, Beijing Municipality, 100730, China

Location

Fujian Medical University Union Hospital

Fuzhou, Fujian, 350001, China

Location

Cangzhou Central Hospital

Cangzhou, Hebei, 061001, China

Location

Tongji Hosp, Tongji Med. Col, Huazhong Univ. of Sci. & Tech/ Wuhan Union Hospital

Wuhan, Hubei, 430022, China

Location

Tongji Hospital of Tongji Medical College of Hongzhong Science and Techology University

Wuhan, Hubei, 430030, China

Location

Xiangya Hospital Central South University

Changsha, Hunan, 410008, China

Location

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

Location

The First Affiliated Hospital of College of Medicine, Zhengjiang University

Hangzhou, Zhejiang, 310003, China

Location

Beijing Children's Hospital Affiliated to Capital University of Medical Sciences

Beijing, 100045, China

Location

Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, 200025, China

Location

Hospital of Blood Disease, Chinese Academy of Medical Sciences

Tianjin, 300020, China

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

F8 protein, human; Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: June 20, 2014
• First Posted: June 23, 2014
• Study Start: June 26, 2014
• Primary Completion: May 31, 2016
• Study Completion: May 31, 2016
• Last Updated: May 3, 2021

Record last verified: 2021-04

Locations

CN (11)