Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

Phase 3

Completed

• Conditions: Severe Hemophilia A
• Interventions: Biological: Human-cl rhFVIII

• Registration Number: NCT01992549
• Lead Sponsor: Octapharma

Brief Summary

The purpose of the study is to collect long-term data on 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: 2013-11-19
• Study First Submitted QC: 2013-11-19
• Study First Posted: 2013-11-25
• Study Start: 2014-04
• Primary Completion: 2018-12-27
• Study Completion: 2018-12-27
• Results First Submitted: 2019-11-27
• Results First Submitted QC: 2019-11-27
• Results First Posted: 2019-12-17
• Status Verified: 2020-12
• Last Update Submitted: 2020-12-21
• Last Update Posted: 2021-01-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 48

Inclusion Criteria

1. Patients who completed GENA-05 in accordance with the study protocol

Exclusion Criteria

1. Severe liver or kidney disease
2. Concomitant treatment with any systemic immunosuppressive drug;
3. Other FVIII concentrate than Human-cl rhFVIII was received between completion visit of GENA-05 and start of GENA-15 (except emergency cases).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Human-cl rhFVIII	Human-cl rhFVIII	-

Primary Outcome Measures

Name	Time	Method
Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors	Maximum two years	The number of patients developing FVIII inhibitors was observed during the observation period by assessing inhibitor development by the modified Bethesda assay (Nijmegen modification) using congenital FVIII-deficient human plasma spiked with Human-cl rhFVIII. The definition threshold for a "positive" inhibitor was if the modified Bethesda assay resulted in a titre ≥0.6 BU/mL at any time point during the observation period.

Secondary Outcome Measures

Name	Time	Method
The Occurrence of Any Adverse Event (AE)	Maximum 2 years	The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.
Efficacy of Human-cl rhFVIII for Surgical Prophylaxis	Maximum 2 years	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). If surgeries could not be assessed due to limited data available or having taken place outside the study site, the results were classified as "not done".
Frequency of Spontaneous Break-through Bleeds	Maximum 2 years	The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs).
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds	Maximum 2 years	A personal efficacy assessment (final outcome) to assess the efficacy of Human-cl rhFVIII for the on-demand treatment of bleeding episodes (BEs) at the end of a BE. Efficacy was assessed using a four-point scale (excellent, good, moderate, none) by the patient's parent(s)/legal guardian(s) together with the investigator in case of on site treatment.

Trial Locations

Locations (15)

University of Alberta; 🇨🇦 Edmonton, Alberta, Canada

UC Davis Medical Center; 🇺🇸 Sacramento, California, United States

McMaster Children's Hospital; 🇨🇦 Hamilton, Ontario, Canada

Hopital de la Timone; 🇫🇷 Marseille, France

Hôpital Kremlin Bicètre; 🇫🇷 Paris, France

Hospital for Sick Children; 🇨🇦 Toronto, Canada

Institute of Hematology and Transfusiology; 🇬🇪 Tbilisi, Georgia

Sahyadri Speciality Hospital; 🇮🇳 Pune, India

IMSP Mother and Child Institute; 🇲🇩 Chişinău, Moldova, Republic of

University Medical School; 🇵🇱 Warsaw, Poland

The National Children Specialized Hospital "OHMATDET"; 🇺🇦 Kiev, Ukraine

Great Ormond Street Hospital for Children; 🇬🇧 London, United Kingdom

Christian Medical College; 🇮🇳 Vellore, India

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

Danylo Halytsky Lviv National Medical University; 🇺🇦 Lviv, Ukraine