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

Phase 3

Completed

• Conditions: Severe Hemophilia A

• Registration Number: NCT01712438
• Lead Sponsor: Octapharma

Brief Summary

Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-10-18
• Study First Submitted QC: 2012-10-19
• Study First Posted: 2012-10-23
• Study Start: 2013-02
• Primary Completion: 2018-12-14
• Results First Submitted: 2019-09-25
• Results First Submitted QC: 2019-10-18
• Results First Posted: 2019-10-21
• Study Completion: 2019-12-20
• Status Verified: 2020-12
• Last Update Submitted: 2020-12-21
• Last Update Posted: 2021-01-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 110

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors	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.

Secondary Outcome Measures

Name	Time	Method
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds	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	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).
Frequency of Spontaneous Break-through Bleeds	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

Trial Locations

Locations (37)

UC Davis; 🇺🇸 Sacramento, California, United States

All Children's Hospital; 🇺🇸 Saint Petersburg, Florida, United States

Harvard Children's Hospital Boston; 🇺🇸 Boston, Massachusetts, United States

Republican Scientific Practical Center for Pediatric Oncology and Hematology; 🇧🇾 Minsk, Belarus

University of Alberta; 🇨🇦 Edmonton, Alberta, Canada

BC Children's Hospital; 🇨🇦 Vancouver, British Columbia, Canada

Mc Master Children's Hospital; 🇨🇦 Hamilton, Ontario, Canada

Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada

Hopital Ste-Justine; 🇨🇦 Montreal, Quebec, Canada

L'hôpital Côte de Nacre - CHU de Caen; 🇫🇷 Caen, France

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