NCT01992549

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
48

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Apr 2014

Longer than P75 for phase_3

Geographic Reach
9 countries

15 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 19, 2013

Completed
6 days until next milestone

First Posted

Study publicly available on registry

November 25, 2013

Completed
4 months until next milestone

Study Start

First participant enrolled

April 1, 2014

Completed
4.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 27, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 27, 2018

Completed
12 months until next milestone

Results Posted

Study results publicly available

December 17, 2019

Completed
Last Updated

January 19, 2021

Status Verified

December 1, 2020

Enrollment Period

4.7 years

First QC Date

November 19, 2013

Results QC Date

November 27, 2019

Last Update Submit

December 21, 2020

Conditions

Severe Hemophilia A

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (4)

  • Frequency of Spontaneous Break-through Bleeds

    Maximum 2 years

  • Efficacy of Human-cl rhFVIII for the Treatment of Bleeds

    Maximum 2 years

  • Efficacy of Human-cl rhFVIII for Surgical Prophylaxis

    Maximum 2 years

  • The Occurrence of Any Adverse Event (AE)

    Maximum 2 years

Study Arms (1)

Human-cl rhFVIII

EXPERIMENTAL
Biological: Human-cl rhFVIII

Interventions

Human-cl rhFVIIIBIOLOGICAL
Human-cl rhFVIII

Eligibility Criteria

Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

University of Alberta

Edmonton, Alberta, Canada

Location

BC Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

McMaster Children's Hospital

Hamilton, Ontario, L8S4K1, Canada

Location

Hospital for Sick Children

Toronto, Canada

Location

Hopital de la Timone

Marseille, France

Location

Hôpital Kremlin Bicètre

Paris, France

Location

Institute of Hematology and Transfusiology

Tbilisi, Georgia

Location

Sahyadri Speciality Hospital

Pune, 411004, India

Location

Christian Medical College

Vellore, 632004, India

Location

IMSP Mother and Child Institute

Chisinau, Moldova

Location

University Medical School

Warsaw, Poland

Location

The National Children Specialized Hospital "OHMATDET"

Kiev, Ukraine

Location

Danylo Halytsky Lviv National Medical University

Lviv, Ukraine

Location

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title
Sylvia Werner
Organization
Octapharma
sylvia.werner@octapharma.com
415 260-9577

Study Officials

  • Sigurd Knaub, PhD

    Octapharma

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 19, 2013

First Posted

November 25, 2013

Study Start

April 1, 2014

Primary Completion

December 27, 2018

Study Completion

December 27, 2018

Last Updated

January 19, 2021

Results First Posted

December 17, 2019

Record last verified: 2020-12

Locations

US(1)MO(1)CA(4)GB(1)IN(2)UA(2)FR(2)PL(1)GE(1)