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Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02954575
Recruitment Status : Completed
First Posted : November 3, 2016
Last Update Posted : April 18, 2018
Sponsor:
Information provided by (Responsible Party):
Octapharma

Tracking Information
First Submitted Date  ICMJE October 27, 2016
First Posted Date  ICMJE November 3, 2016
Last Update Posted Date April 18, 2018
Actual Study Start Date  ICMJE December 2016
Actual Primary Completion Date March 29, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 1, 2016)
Reduction of total annualized bleeding rate [ Time Frame: 6 months ]
50% reduction of the total annualized bleeding rate (TABR) observed in the GENA-01 study, with a total of 58.1 BEs per patient per year.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02954575 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 28, 2017)
  • Spontaneous annualized bleeding rate (SABR) [ Time Frame: 6 months ]
  • Efficacy of Wilate in the treatment of breakthrough BEs based on the proportion of BEs successfully treated with Wilate [ Time Frame: 6 months ]
  • Wilate consumption data (FVIII IU/kg per week per patient) for prophylaxis [ Time Frame: 6 months ]
  • PK assessment (Area under of curve) of FVIII:C using the one-stage (OS) assays and actual IMP potencies [ Time Frame: 6 months ]
  • PK assessment (In vivo half life) of FVIII:C using the one-stage (OS) assays and actual IMP potencies [ Time Frame: 6 months ]
  • PK assessment (Maximum plasma concentration (Cmax))) of FVIII:C using the one-stage (OS) assays and actual IMP potencies [ Time Frame: 6 months ]
  • Incremental IVR of Wilate over time (at baseline, and at 3 and 6 months of treatment) [ Time Frame: 6 months ]
  • Association between ABO blood type and the FVIII:C half-life of Wilate [ Time Frame: 6 months ]
  • Association between VWF:Ag concentration and the FVIII:C half-life of Wilate [ Time Frame: 6 months ]
  • Safety and tolerability of Wilate by monitoring adverse events (AEs) throughout the study [ Time Frame: 6 months ]
  • Immunogenicity of Wilate by testing for FVIII inhibitors [ Time Frame: 6 months ]
  • Virus safety to be measured by the number of incidence of parvovirus B19 seroconversions between baseline and end of study [ Time Frame: 6 months ]
Original Secondary Outcome Measures  ICMJE
 (submitted: November 1, 2016)
  • Spontaneous annualized bleeding rate (SABR) [ Time Frame: 6 months ]
  • Efficacy of Wilate in the treatment of breakthrough BEs based on the proportion of BEs successfully treated with Wilate [ Time Frame: 6 months ]
  • Efficacy of Wilate in surgical prophylaxis assessed by surgeon and hematologist using predefined assessment criteria [ Time Frame: 6 months ]
  • Wilate consumption data (FVIII IU/kg per week per patient) for prophylaxis [ Time Frame: 6 months ]
  • PK assessment (Area under of curve) of FVIII:C using the one-stage (OS) assays and actual IMP potencies [ Time Frame: 6 months ]
  • PK assessment (In vivo half life) of FVIII:C using the one-stage (OS) assays and actual IMP potencies [ Time Frame: 6 months ]
  • PK assessment (Maximum plasma concentration (Cmax))) of FVIII:C using the one-stage (OS) assays and actual IMP potencies [ Time Frame: 6 months ]
  • Incremental IVR of Wilate over time (at baseline, and at 3 and 6 months of treatment) [ Time Frame: 6 months ]
  • Association between ABO blood type and the FVIII:C half-life of Wilate [ Time Frame: 6 months ]
  • Association between VWF:Ag concentration and the FVIII:C half-life of Wilate [ Time Frame: 6 months ]
  • Safety and tolerability of Wilate by monitoring adverse events (AEs) throughout the study [ Time Frame: 6 months ]
  • Immunogenicity of Wilate by testing for FVIII inhibitors [ Time Frame: 6 months ]
  • Virus safety to be measured by the number of incidence of parvovirus B19 seroconversions between baseline and end of study [ Time Frame: 6 months ]
Current Other Pre-specified Outcome Measures
 (submitted: June 28, 2017)
Efficacy of Wilate in surgical prophylaxis assessed by surgeon and hematologist using predefined assessment criteria [ Time Frame: 6 months ]
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
Official Title  ICMJE Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Patients With Severe Hemophilia A
Brief Summary The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Severe Hemophilia A
Intervention  ICMJE Drug: Wilate
Other Name: von Willebrand factor / Factor VIII (plasma derived)
Study Arms  ICMJE Experimental: All patients
All patients will receive Wilate for prophylactic treatment
Intervention: Drug: Wilate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 1, 2016)
55
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE March 29, 2018
Actual Primary Completion Date March 29, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Severe hemophilia A (<1% FVIII:C) according to medical history
  2. Male patients aged ≥12 years
  3. Previous treatment with a FVIII concentrate for at least 150 exposure days (EDs)
  4. Immunocompetence (CD4+ count >200/µL)
  5. Good documentation of the historical bleeding rate (at least for the 6 months preceding study start)
  6. Voluntarily given, fully informed written and signed consent obtained by the patient (or parent/legal guardian in case of adolescents) before any study-related procedures are conducted

Whenever possible, the interval between the Screening Visit and the PK or Non-PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/µL for patients to be enrolled (i.e., exclusion criterion no. 4).

Exclusion Criteria:

  1. Any coagulation disorders other than hemophilia A
  2. History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory anti-bodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory
  3. Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine>120 µmol/L)
  4. Patients receiving or scheduled to receive immunomodulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs
  5. Treatment with any investigational medicinal product in another interventional clinical study currently or within 4 weeks before enrollment
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Bulgaria,   Hungary,   Poland,   Romania,   Russian Federation
Removed Location Countries Serbia,   Ukraine
 
Administrative Information
NCT Number  ICMJE NCT02954575
Other Study ID Numbers  ICMJE WIL-27
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Octapharma
Study Sponsor  ICMJE Octapharma
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Cristina Solomon, MD Octapharma
PRS Account Octapharma
Verification Date April 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP