Haemophilia BPhase 3NCT02231944
An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients
A Phase 3 study testing Replenine®-VF (High Purity Factor IX) in people with Haemophilia B, run by Bio Products Laboratory at 7 sites in 2 countries.
Trial summary
To compare the first and second recovery assessments on Replenine®-VF and to evaluate recovery of different batches if patients changed batches during the study. To evaluate Replenine®-VF in terms of long-term clinical efficacy, tolerance and safety Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Haemophilia B
- What is being tested?
- Replenine®-VF (High Purity Factor IX)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- Not given
- Where
- 7 sitesin 2 countries
- Study dates
- Jul 1997to Oct 2003
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Previously treated patients
- At least 12 years of age
- Severe Haemophilia B and without inhibitor to factor IX
Who cannot join
- -
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
United Kingdom5 sites
Addenbrooke's Hospital, Hills Road
Cambridge
University Hospital of Wales, Heath Park
Cardiff
The Royal London Hospital
London
Manchester Royal Infirmary, Oxford Road
Manchester
Norwich and Norfolk Hospital
Norwich
Poland2 sites
Klinika Hematologii Instytutu Medycyny Wewnetrznej
Lodz
Klinika Hematologii AM Lublin, ul.Jaczewskiego 8
Lublin
About this study
Official title: An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients.
To compare the first and second recovery assessments on Replenine®-VF and to evaluate recovery of different batches if patients changed batches during the study.
To evaluate Replenine®-VF in terms of long-term clinical efficacy, tolerance and safety
- Conditions
- Haemophilia B
- Being tested
- Replenine®-VF (High Purity Factor IX)
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Replenine®-VF (High Purity Factor IX)Biological
| Study group | Role | What they receive |
|---|---|---|
| Replenine®-VF | Experimental | Not described Receives: Biological: Replenine®-VF (High Purity Factor IX) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recovery calculated from the first infusion of Replenine®-VF compared with that calculated 12 weeks later.
Safety assessments included:
- assessment of tolerance at injection site
- assessment of Factor IX inhibitor development
- assessment of frequency and type of adverse event
- routine biochemistry and haematology at the start and end of Stage 1 and at the end of the study (Stage 2, if applicable)
- screening for markers of viral infection at the start and end of each stage of the study (and if patients changed batches).
Measured: Baseline and 3 months post-baseline.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Sep 2014 (estimated)
- Study startJul 1997
- Primary completionOct 2003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia BStudies 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
Sponsor and investigators
- Lead sponsor
- Bio Products Laboratory
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02231944
- Sponsor's protocol ID
- R9VFSE
- Status last verified
- Feb 2018
- First submitted
- 2 Sep 2014
- Last updated
- 22 Feb 2018
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients.
- ClinicalTrials.gov ID
- NCT02231944
- Acronym / sponsor ID
- R9VFSE
- Conditions
- Haemophilia B
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- Bio Products LaboratoryUniversity, hospital or other
- Study start
- Jul 1997
- Primary completion
- Oct 2003 (actual)
- First posted
- 4 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To compare the first and second recovery assessments on Replenine®-VF and to evaluate recovery of different batches if patients changed batches during the study.
To evaluate Replenine®-VF in terms of long-term clinical efficacy, tolerance and safety
Study design
- Primary purpose
- Treatment
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Replenine®-VF | Experimental | Biological: Replenine®-VF (High Purity Factor IX) |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Previously treated patients
- At least 12 years of age
- Severe Haemophilia B and without inhibitor to factor IX
Key exclusion criteria (1 listed)
- -
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 |
|---|---|---|
| Replenine®-VF (High Purity Factor IX) | Biological | Replenine®-VF |
Primary outcome
Recovery calculated from the first infusion of Replenine®-VF compared with that calculated 12 weeks later.
Time frame: Baseline and 3 months post-baseline.
Safety assessments included: * assessment of tolerance at injection site * assessment of Factor IX inhibitor development * assessment of frequency and type of adverse event * routine biochemistry and haematology at the start and end of Stage 1 and at the end of the study (Stage 2, if applicable) * screening for markers of viral infection at the start and end of each stage of the study (and if patients changed batches).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2018.
- Related InfoLink provided by the sponsor.
Related trials for Haemophilia B
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia
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.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Replenine®-VF (High Purity Factor IX) in people with Haemophilia B, run by Bio Products Laboratory at 7 sites in 2 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 1 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 22 Feb 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United Kingdom, Poland. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Recovery calculated from the first infusion of Replenine®-VF compared with that calculated 12 weeks later. (time frame: Baseline and 3 months post-baseline.). 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 22 Feb 2018.