Severe Hemophilia APhase 3NCT01125813
Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
A Phase 3 study testing recombinant Factor VIII in people with Severe Hemophilia A, run by Octapharma at 11 sites in 4 countries.
Trial summary
This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis. Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Severe Hemophilia A
- What is being tested?
- recombinant Factor VIII
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 11 sitesin 4 countries
- Study dates
- Jun 2010to Jan 2012
- 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 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Severe hemophilia A ((FVIII:C <= 1%)
- Male subjects >= 12 years of age
- Previously treated with FVIII concentrate, at least 50 EDs
- Immunocompetent (CD4+ count > 200/ul)
- Negative for anti- HIV; if positive, viral load < 200 particles/u; or <400,000 copies/mL
Who cannot join
- Other coagulation disorder than hemophilia A
- Present of past FVIII inhibitor activity (.= 0.6 BU)
- Severe liver and kidney disease
- Receiving of scheduled to receive immuno-modulating drugs
Trial locations and contacts
11 sites in 4 countries.
11 sites in 4 countries
United Kingdom5 sites
Basingstoke & North Hampshire NHS Foundation Trust
Basingstoke, RG24 9NA
University Hospital of Wales
Cardiff
Royal Free Hospital
London
Manchester Royal Infirmary
Manchester
Royal Hallamshire Hospital
Sheffield
Germany4 sites
Universitaetsklinikum
Bonn
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246
Werlhof Institut fuer Haemostaseologie GmbH
Hanover, Lower Saxony, 30159
SRH Kurpfalzkrankenhaus Heidelberg
Heidelberg, 69123
About this study
Official title: Clinical Study to Investigate the Efficacy, Safety, and Immunogenicity of Human-cl rhFVIII in Previously Treated Patients With Severe Hemophilia A
This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.
- Conditions
- Severe Hemophilia A
- Being tested
- recombinant Factor VIII
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
recombinant Factor VIIIBiological
Also called human-cl rhFVIII
intravenous infusion of factor FVIII every other day.
| Study group | Role | What they receive |
|---|---|---|
| human cl-rhFVIII | Experimental | Not described Receives: Biological: recombinant Factor VIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months
Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.
Measured: At least 50 Exposure Days and at least 6 months
Efficacy of Treating Bleeding Episodes
At the end of a bleeding episode, efficacy was assessed as:
- Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion
- Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an infusion requiring up to 2 infusions for complete resolution
- Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution
- None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution Efficacy was rated
Measured: After each bleeding episode, up to 6 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2010 (estimated)
- Study startJun 2010
- Primary completionJan 2012
- Study completionJan 2012
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.
- More trials run by OctapharmaThe sponsor's other registered studies.
- 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Johannes Oldenburg, Prof., Principal investigator, Universitaetsklinikum Bonn
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01125813
- Sponsor's protocol ID
- GENA-08
- Status last verified
- Aug 2017
- First submitted
- 17 May 2010
- Last updated
- 11 Sep 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study to Investigate the Efficacy, Safety, and Immunogenicity of Human-cl rhFVIII in Previously Treated Patients With Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT01125813
- Acronym / sponsor ID
- GENA-08
- Conditions
- Severe Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- Jun 2010
- Primary completion
- Jan 2012 (actual)
- Study completion
- Jan 2012 (actual)
- First posted
- 18 May 2010 (estimated)
- Results
- Posted 22 Apr 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| human cl-rhFVIII | Experimental | Biological: recombinant Factor VIII |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Severe hemophilia A ((FVIII:C <= 1%)
- Male subjects >= 12 years of age
- Previously treated with FVIII concentrate, at least 50 EDs
- Immunocompetent (CD4+ count > 200/ul)
- Negative for anti- HIV; if positive, viral load < 200 particles/u; or <400,000 copies/mL
Key exclusion criteria (4 listed)
- Other coagulation disorder than hemophilia A
- Present of past FVIII inhibitor activity (.= 0.6 BU)
- Severe liver and kidney disease
- Receiving of scheduled to receive immuno-modulating drugs
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 |
|---|---|---|
| recombinant Factor VIIIAlso: human-cl rhFVIIIintravenous infusion of factor FVIII every other day. | Biological | human cl-rhFVIII |
Primary outcome
Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months
Time frame: At least 50 Exposure Days and at least 6 months
Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.
Efficacy of Treating Bleeding Episodes
Time frame: After each bleeding episode, up to 6 month
At the end of a bleeding episode, efficacy was assessed as: * Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion * Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an infusion requiring up to 2 infusions for complete resolution * Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution * None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution Efficacy was rated
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2017.
- 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.
Johannes Oldenburg, Prof. principal investigator
Universitaetsklinikum Bonn
11 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing recombinant Factor VIII in people with Severe Hemophilia A, run by Octapharma at 11 sites in 4 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 5 inclusion and 4 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 11 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United Kingdom, Germany, Austria, Bulgaria. 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 22 Apr 2013). 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?
Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months (time frame: At least 50 Exposure Days and at least 6 months). The study lists 2 primary outcomes in all. 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 11 Sep 2017.