Severe Hemophilia APhase 3NCT01712438
Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
A Phase 3 study testing Human cl rhFVIII in people with Severe Hemophilia A, run by Octapharma at 37 sites in 17 countries.
Trial summary
Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Severe Hemophilia A
- What is being tested?
- Human cl rhFVIII
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 110 participants
- Where
- 37 sitesin 17 countries
- Study dates
- Feb 2013to 20 Dec 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male patients
- Severe Hemophilia A (FVIII:C <1%)
- No previous treatment with FVIII concentrates or other blood products containing FVIII
Who cannot join
- Diagnosis with a coagulation disorder other than Hemophilia A
- Severe liver or kidney disease
- Concomitant treatment with any systemic immunosuppressive drug
Trial locations and contacts
37 sites in 17 countries.
37 sites in 17 countries
France7 sites
L'hôpital Côte de Nacre - CHU de Caen
Caen
Centre de traitement de l'hemophilie, Hôpital Bicêtre
Le Kremlin-Bicêtre
Hopital de la Timone
Marseille
Hôtel-Dieu de Nantes, Centre Regional de Traitement de l'hemophilie
Nantes
Hôpital Necker
Paris
CHU de Rennes - Hôpital Pontchaillou
Rennes
Hopital Trousseau - CHU Tours
Tours
Canada5 sites
University of Alberta
Edmonton, Alberta, T6G2V2
Mc Master Children's Hospital
Hamilton, Ontario, L8S4K1
Hopital Ste-Justine
Montreal, Quebec, H3T 1C5
Hospital for Sick Children
Toronto, Ontario, M5G 1X8
BC Children's Hospital
Vancouver, British Columbia, V6H 3V4
United States3 sites
Harvard Children's Hospital Boston
Boston, Massachusetts, 02115
UC Davis
Sacramento, California, 95817
All Children's Hospital
St. Petersburg, Florida, 33701
Germany3 sites
Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT)
Bonn
University Hospital Frankfurt/M
Frankfurt, 60590
Universitätsmedizin der Johannes-Gutenberg-Universität Mainz
Mainz
India3 sites
Kasturba Medical College, Dr. TMA Pai Hospital
Manipal, Karnataka
Sahyadri Speciality Hospital, Haematology & BMT Unit
Pune
Christian Medical College & Hospital, Dept of Haematology
Vellore
Italy2 sites
Univ. Di Perugia
Perugia
Centro di Referimento per le Malattie Emorragiche e Trombotiche
Torino
Spain2 sites
Unitat d'hemofilia, Hospital Universitari Vall d'Hebron
Barcelona
Hospital Universitario La Paz
Madrid
Ukraine2 sites
National Children's Specialized Hospital "OHMATDET"
Kiev
Institute of Blood Pathology and Transfusion Medicine
Lviv
United Kingdom2 sites
Cambridge University Hospital
Cambridge, CB2 0QQ
Great Ormond Street Hospital for Children
London, WC1N3JH
Belarus1 site
Republican Scientific Practical Center for Pediatric Oncology and Hematology
Minsk
Moldova1 site
Scientific Research Institute of Mother and Child Health Care
Chisinau
About this study
Official title: Immunogenicity, Efficacy and Safety of Treatment With Human-cl-rhFVIII in Previously Untreated Patients With Severe Hemophilia A
Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.
- Conditions
- Severe Hemophilia A
- Being tested
- Human cl rhFVIII
- Keywords
- Previously untreated patients
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human cl rhFVIIIBiological
| Study group | Role | What they receive |
|---|---|---|
| Human cl rhFVIII | Experimental | Not described Receives: Biological: Human cl rhFVIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 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.
Measured: maximum 5 years (100 exposure days)
Secondary outcomes (3)
Frequency of Spontaneous Break-through Bleeds
The annualized bleeding rate (ABR) was calculated during inhibitor-free periods for spontaneous bleeding events (BEs) during prophylactic treatment with Human cl rhFVIII
Measured: Maximum 5 years (100 exposure days)
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
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).
Measured: Maximum 5 years (100 exposure days)
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).
Measured: Maximum 5 years (100 exposure days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2012 (estimated)
- Study startFeb 2013
- Primary completion14 Dec 2018
- Study completion20 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Severe Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 110 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sigurd Knaub, Study director, Octapharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01712438
- Sponsor's protocol ID
- GENA-05
- Other IDs
- 2012-002554-23
- Status last verified
- Dec 2020
- First submitted
- 18 Oct 2012
- Last updated
- 19 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Immunogenicity, Efficacy and Safety of Treatment With Human-cl-rhFVIII in Previously Untreated Patients With Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT01712438
- Acronym / sponsor ID
- GENA-05
- Conditions
- Severe Hemophilia A
- Keywords
- Previously untreated patients
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 110 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- Feb 2013
- Primary completion
- 14 Dec 2018 (actual)
- Study completion
- 20 Dec 2019 (actual)
- First posted
- 23 Oct 2012 (estimated)
- Results
- Posted 21 Oct 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Human cl rhFVIII | Experimental | Biological: Human cl rhFVIII |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Male patients
- Severe Hemophilia A (FVIII:C <1%)
- No previous treatment with FVIII concentrates or other blood products containing FVIII
Key exclusion criteria (3 listed)
- Diagnosis with a coagulation disorder other than Hemophilia A
- Severe liver or kidney disease
- Concomitant treatment with any systemic immunosuppressive drug
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Human cl rhFVIII | Biological | Human cl rhFVIII |
Primary outcome
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.
3 secondary outcomes
Frequency of Spontaneous Break-through Bleeds
Time frame: Maximum 5 years (100 exposure days)
Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
Time frame: Maximum 5 years (100 exposure days)
Efficacy of Human-cl rhFVIII for Surgical Prophylaxis
Time frame: Maximum 5 years (100 exposure days)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sigurd Knaub study director
Octapharma
37 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Human cl rhFVIII in people with Severe Hemophilia A, run by Octapharma at 37 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in France, Canada, United States, Germany, India and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Oct 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors (time frame: maximum 5 years (100 exposure days)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jan 2021.