Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients

• ClinicalTrials.gov Identifier: NCT01712438
• Recruitment Status: Completed
• First Posted: October 23, 2012
• Results First Posted: October 21, 2019
• Last Update Posted: January 19, 2021
• Sponsor: Octapharma
• Information provided by (Responsible Party): Octapharma

Tracking Information

• First Submitted Date: October 18, 2012
• First Posted Date: October 23, 2012
• Results First Submitted Date: September 25, 2019
• Results First Posted Date: October 21, 2019
• Last Update Posted Date: January 19, 2021
• Study Start Date: February 2013
• Actual Primary Completion Date: December 14, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 18, 2019)

Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors [ Time Frame: maximum 5 years (100 exposure days) ]

The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development using the modified Bethesda assay (Nijmegen modification). The definitions for thresholds were ≥0.6 to <5 BU/mL for a "low titre" inhibitor and ≥5 BU/mL for a "high-titre" inhibitor.

Original Primary Outcome Measures (submitted: October 19, 2012)

Immunogenicity [ Time Frame: maximum 5 years (100 exposure days) ]

Number of patients that develop an inhibitor

Current Secondary Outcome Measures (submitted: October 18, 2019)

• Frequency of Spontaneous Break-through Bleeds [ Time Frame: Maximum 5 years (100 exposure days) ]
  The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII
• Efficacy of Human-cl rhFVIII for the Treatment of Bleeds [ Time Frame: Maximum 5 years (100 exposure days) ]
  A personal efficacy assessment to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes. Efficacy was assessed using a four-point scale (excellent, good, moderate, none).
• Efficacy of Human-cl rhFVIII for Surgical Prophylaxis [ Time Frame: Maximum 5 years (100 exposure days) ]
  An overall efficacy assessment to assess the efficacy of human-cl rhFVIII in surgical prophylaxis of minor and major surgeries. The efficacy assessment was analyzed using a four-point scale (excellent, good, moderate, none).

Original Secondary Outcome Measures (submitted: October 19, 2012)

Efficacy [ Time Frame: Maximum 5 years (100 exposure days) ]

Efficacy of human cl rhFVIII during prophylactic treatment (frequency of break thorugh blleds) Efficacy of human cl rhFVIII during treatment of bleeds (Number of injections needed to stop a bleed) Efficacy of human cl rhFVIII during surgical prophylaxis (compare expected estimated blood loss versus actual estimated blood loss)

Current Other Pre-specified Outcome Measures (submitted: October 18, 2019)

The Occurrence of Any Adverse Event (AE) [ Time Frame: 5 years ]

The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.

Original Other Pre-specified Outcome Measures (submitted: October 19, 2012)

Safety and tolerability [ Time Frame: 5 years ]

Measure vital signs, Adverse events, and standard hematology and chemistry laboratory parameters. This assessment is descriptive)

Descriptive Information

• Brief Title: Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
• Official Title: Immunogenicity, Efficacy and Safety of Treatment With Human-cl-rhFVIII in Previously Untreated Patients With Severe Hemophilia A
• Brief Summary: Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Severe Hemophilia A
• Intervention: Biological: Human cl rhFVIII

Study Arms

Experimental: Human cl rhFVIII

Intervention: Biological: Human cl rhFVIII

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: October 5, 2016): 110
• Original Estimated Enrollment (submitted: October 19, 2012): 100
• Actual Study Completion Date: December 20, 2019
• Actual Primary Completion Date: December 14, 2018 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Male patients
• Severe Hemophilia A (FVIII:C <1%)
• No previous treatment with FVIII concentrates or other blood products containing FVIII

Exclusion Criteria:

• Diagnosis with a coagulation disorder other than Hemophilia A
• Severe liver or kidney disease
• Concomitant treatment with any systemic immunosuppressive drug

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Belarus, Canada, France, Georgia, Germany, India, Italy, Moldova, Republic of, Morocco, Poland, Portugal, Russian Federation, Slovenia, Spain, Ukraine, United Kingdom, United States

Administrative Information

• NCT Number: NCT01712438
• Other Study ID Numbers: GENA-05; 2012-002554-23 ( EudraCT Number )
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Octapharma
• Original Responsible Party: Same as current
• Current Study Sponsor: Octapharma
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Sigurd Knaub; Octapharma

• PRS Account: Octapharma
• Verification Date: December 2020