Haemophilia APhase 2NCT00245297
Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.
A Phase 2 study testing Kogenate FS, reconstituted in a suspension of liposomes in people with Haemophilia A, run by Recoly N.V. at 3 sites in Russia.
Trial summary
Primary efficacy endpoint: To study whether there is a difference in the length of the bleeding free periods between infusion of FVIII that is reconstituted with 22.1, 12.6, or 4.2 mg of pegylated liposomes per kg bwt compared with standard formulation. Read more about this study
- Who is it for?
- Ages 18 to 60 years, men only
- Conditions the study is for
- Haemophilia A
- What is being tested?
- Kogenate FS, reconstituted in a suspension of liposomes
- How many people
- 16 participants
- Where
- 3 sitesRussia
- Study dates
- Oct 2005to Apr 2006
- Phase
- Phase 2
- 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 18 to 60 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- Between 18 and 60 years of age
- Severe haemophilia A (≤ 1% of baseline Factor VIII activity)
- At least 250 treatment cumulative exposure-days (CEDs) to previous products
- At least 25 cumulative exposure-days (CEDs) to previous products during one year prior to study start
- If HIV positive, CD4 lymphocytes ≥ 400/µl
- Subjects on demand treatment, and with a minimum bleeding/treatment pattern of a four episodes per month, evenly distributed within each month, during the three month preceding study start
- Subjects who have given their written informed consent.
Who cannot join
- Inhibitors or history of inhibitors
- History of adverse reactions related to Factor VIII
- Platelet count <90,000 /µl
- Subjects on prophylaxis treatment
- Subjects with concomitant debilitating disease (e.g. cancer, non-controlled diabetes, heart insufficiency, renal failure)
- Subjects with known sensitivity to blood products
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- Subjects who have participated in another Ethical Committee approved Clinical Trial (including medical device studies) within the past 30 days
- Subjects with a weight over 86 kg or below 50 kg
- Subjects who do not understand or are not willing to comply with the requirement of the study protocol
- Subjects who cannot differentiate a bleeding episode from other causes of joint pain
Trial locations and contacts
3 sites in 1 country.
Russia3 sites
Center for Hematological Research, Department of Reconstructive Orthopedic Surgery
Moscow, 125167
The Russian Academy of Medical Sciences, Haemophilia Center
Moscow, 125167
Republic Haemophilia Center.
Saint Petersburg, 191186
About this study
Official title: A Patient-Blinded, Randomized, Comparative, Multicenter, Crossover, (Liposome) Dose Finding Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.
Primary efficacy endpoint: To study whether there is a difference in the length of the bleeding free periods between infusion of FVIII that is reconstituted with 22.1, 12.6, or 4.2 mg of pegylated liposomes per kg bwt compared with standard formulation.
Safety endpoint: To study the safety and tolerability of Kogenate FS reconstituted with Pegylated liposomes
Read the detailed description
Each subject will receive treatment for three bleeding episodes ("wash-in", on demand injections) followed by a minimum 4 day wash-out. Following this each subject will be randomized to a specific treatment order. Each order consists of four identical blocks. In each block the following three injections will be given: 1 One prophylactic treatment (randomized solution for dissolution) 2 and 3. Two standard on-demand treatments for a spontaneous bleeding episode (standard Kogenate FS). Each block will be followed by a four day wash-out. In total each subject should receive 3+4x3=15 injections. It is estimated that each subject will be in the study for about 4 months. A treatment for a spontaneous bleeding episode may require more than one infusion.
- Conditions
- Haemophilia A
- Being tested
- Kogenate FS, reconstituted in a suspension of liposomes
- Keywords
- Bleeding disorder, severe Haemophilia A, Prophylaxis
- Medical subject headings
- Hemophilia A, Hemostatic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Kogenate FS, reconstituted in a suspension of liposomesDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary efficacy parameter is the number of bleeding free days (without a bleeding/treatment) between a randomized prophylactic infusion and following on demand injection.
Measured: Bleeding free time following four single doses will be studies
Secondary outcomes (1)
In vivo recovery (IVR).
Measured: IVR will be performed at the first and last dose.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2005 (estimated)
- Study startOct 2005
- Study completionApr 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Haemophilia 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 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Recoly N.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Jack Spira, MD. PhD., Study director, Recoly C/o InSpira Medical AB, Nasbyv 38, 13553 Tyreso Sweden
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00245297
- Sponsor's protocol ID
- OMRI Lip-FVIII-05-1
- Status last verified
- Oct 2007
- First submitted
- 26 Oct 2005
- Last updated
- 16 Oct 2007
- Results posted
- No
- Data monitoring committee
- No
Publications
Baru M, Carmel-Goren L, Barenholz Y, Dayan I, Ostropolets S, Slepoy I, Gvirtzer N, Fukson V, Spira J. Factor VIII efficient and specific non-covalent binding to PEGylated liposomes enables prolongation of its circulation time and haemostatic efficacy. Thromb Haemost. 2005 Jun;93(6):1061-8. doi: 10.1160/TH04-08-0485.
PubMed 15968389
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Patient-Blinded, Randomized, Comparative, Multicenter, Crossover, (Liposome) Dose Finding Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.
- ClinicalTrials.gov ID
- NCT00245297
- Acronym / sponsor ID
- OMRI Lip-FVIII-05-1
- Conditions
- Haemophilia A
- Keywords
- Bleeding disorder, severe Haemophilia A, Prophylaxis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Recoly N.V.Industry (pharma, biotech, devices)
- Study start
- Oct 2005
- Study completion
- Apr 2006 (actual)
- First posted
- 27 Oct 2005 (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.
Primary efficacy endpoint: To study whether there is a difference in the length of the bleeding free periods between infusion of FVIII that is reconstituted with 22.1, 12.6, or 4.2 mg of pegylated liposomes per kg bwt compared with standard formulation.
Safety endpoint: To study the safety and tolerability of Kogenate FS reconstituted with Pegylated liposomes
Detailed description
Each subject will receive treatment for three bleeding episodes ("wash-in", on demand injections) followed by a minimum 4 day wash-out. Following this each subject will be randomized to a specific treatment order. Each order consists of four identical blocks. In each block the following three injections will be given: 1 One prophylactic treatment (randomized solution for dissolution) 2 and 3. Two standard on-demand treatments for a spontaneous bleeding episode (standard Kogenate FS). Each block will be followed by a four day wash-out. In total each subject should receive 3+4x3=15 injections. It is estimated that each subject will be in the study for about 4 months. A treatment for a spontaneous bleeding episode may require more than one infusion.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
Who may qualify
- Ages
- 18 to 60 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Between 18 and 60 years of age
- Severe haemophilia A (≤ 1% of baseline Factor VIII activity)
- At least 250 treatment cumulative exposure-days (CEDs) to previous products
- At least 25 cumulative exposure-days (CEDs) to previous products during one year prior to study start
- If HIV positive, CD4 lymphocytes ≥ 400/µl
Key exclusion criteria (10 listed)
- Inhibitors or history of inhibitors
- History of adverse reactions related to Factor VIII
- Platelet count <90,000 /µl
- Subjects on prophylaxis treatment
- Subjects with concomitant debilitating disease (e.g. cancer, non-controlled diabetes, heart insufficiency, renal failure)
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 |
|---|---|---|
| Kogenate FS, reconstituted in a suspension of liposomes | Drug | – |
Primary outcome
The primary efficacy parameter is the number of bleeding free days (without a bleeding/treatment) between a randomized prophylactic infusion and following on demand injection.
Time frame: Bleeding free time following four single doses will be studies
1 secondary outcome
In vivo recovery (IVR).
Time frame: IVR will be performed at the first and last dose.
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 16 Oct 2007.
- Background: PubMed 15968389Baru M, Carmel-Goren L, Barenholz Y, Dayan I, Ostropolets S, Slepoy I, Gvirtzer N, Fukson V, Spira J. Factor VIII efficient and specific non-covalent binding to PEGylated liposomes enables prolongation of its circulation time and haemostatic efficacy. Thromb Haemost. 2005 Jun;93(6):1061-8. doi: 10.1160/TH04-08-0485.
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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.
Jack Spira, MD. PhD. study director
Recoly C/o InSpira Medical AB, Nasbyv 38, 13553 Tyreso Sweden
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Kogenate FS, reconstituted in a suspension of liposomes in people with Haemophilia A, run by Recoly N.V. at 3 sites in Russia.
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 7 inclusion and 10 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 16 Oct 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Russia. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The primary efficacy parameter is the number of bleeding free days (without a bleeding/treatment) between a randomized prophylactic infusion and following on demand injection. (time frame: Bleeding free time following four single doses will be studies). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00245297, which also lists 1 linked publication.
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 16 Oct 2007.