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

Extension Study for Patients Who Completed GENA-05 (NuProtect)- to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

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.

Study Overview

• Status: Completed
• Conditions: Severe Hemophilia A
• Intervention / Treatment: Biological: Human-cl rhFVIII

• Study Type: Interventional
• Enrollment (Actual): 48
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Toronto, Canada
    • Hospital for Sick Children
  • Alberta
    • Edmonton, Alberta, Canada
      • University of Alberta
  • British Columbia
    • Vancouver, British Columbia, Canada, V6H 3V4
      • BC Children's Hospital
  • Ontario
    • Hamilton, Ontario, Canada, L8S4K1
      • McMaster Children's Hospital
• France
  • Marseille, France
    • Hôpital de la Timone
  • Paris, France
    • Hôpital Kremlin Bicêtre
• Georgia
  • Tbilisi, Georgia
    • Institute of Hematology and Transfusiology
• India
  • Pune, India, 411004
    • Sahyadri Speciality Hospital
  • Vellore, India, 632004
    • Christian Medical College
• Moldova, Republic of
  • Chişinău, Moldova, Republic of
    • IMSP Mother and Child Institute
• Poland
  • Warsaw, Poland
    • University Medical School
• Ukraine
  • Kiev, Ukraine
    • The National Children Specialized Hospital "OHMATDET"
  • Lviv, Ukraine
    • Danylo Halytsky Lviv National Medical University
• United Kingdom
  • London, United Kingdom, WC1N 3JH
    • Great Ormond Street Hospital for Children
• United States
  • California
    • Sacramento, California, United States, 95817
      • UC Davis Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Human-cl rhFVIII	Biological: Human-cl rhFVIII

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors	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.	Maximum two years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency of Spontaneous Break-through Bleeds	The annualized bleeding rate (ABR) was calculated during the time of prophylactic treatment with Human-cl rhFVIII for spontaneous bleeding events (BEs).	Maximum 2 years
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds	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.	Maximum 2 years
Efficacy of Human-cl rhFVIII for Surgical Prophylaxis	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".	Maximum 2 years
The Occurrence of Any Adverse Event (AE)	The frequency of AEs, as monitored throughout the whole study by the number of patients with at least one adverse event occurrence.	Maximum 2 years

Collaborators and Investigators

• Sponsor: Octapharma
• Investigators: Study Director: Sigurd Knaub, PhD, Octapharma

Study record dates

Study Major Dates

• Study Start: April 1, 2014
• Primary Completion (Actual): December 27, 2018
• Study Completion (Actual): December 27, 2018

Study Registration Dates

• First Submitted: November 19, 2013
• First Submitted That Met QC Criteria: November 19, 2013
• First Posted (Estimate): November 25, 2013

Study Record Updates

• Last Update Posted (Actual): January 19, 2021
• Last Update Submitted That Met QC Criteria: December 21, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: GENA-15