Von Willebrand DiseasePhase 4NCT00555555
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
A Phase 4 study testing Alphanate SD/HT in people with Von Willebrand Disease, run by Grifols Biologicals, LLC.
Trial summary
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures. Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Von Willebrand Disease
- What is being tested?
- Alphanate SD/HT
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- No sites listed yet
- Study dates
- 8 Jul 2008to 6 Feb 2012
- Phase
- Phase 4
- 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 7 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female 7 years of age or older
- The subject has been diagnosed of inherited VWD of Type 3 as determined by subject's medical records.
- The subject needs a surgical procedure (at least 10 surgical procedures have to be considered as "Major" according to the criteria of the protocol).
- The subject is expected to respond to exogenously administered FVIII/VWF according to Investigator's judgment.
- The subject freely gives written informed consent. Subjects who are not legally permitted to provide written consent must sign a form of assent for study participation, and written consent must be provided by a parent or legal guardian.
Who cannot join
- The subject has been diagnosed of Type 1, Type 2, or acquired VWD.
- The subject is known to have history of intolerance to any Alphanate® containing substance.
- The subject is known to have history of anaphylactic reaction(s) to blood or blood components.
- Liver function tests (AST, ALT, bilirubin) > 2.5 x upper limit of normal (ULN).
- Renal function test (creatinine) > 1.5 x ULN.
- The subject is known or suspected to have present or past inhibitor activity (antibodies) directed against FVIII or VWF.
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- The subject is known to abuse alcohol or illicit drug use within the past 12 months.
- The subject is participating in another clinical study involving an investigational treatment, or participated within the past 4 weeks. Studies consisting of data and blood sampling collections on a regular or long-term basis are exempt from this exclusion.
- The subject is unlikely to adhere to the protocol requirements of the study.
About this study
Official title: A Post-marketing Observational Study to Assess the Efficacy and Safety of the FVIII/VWF Complex (Human), Alphanate®, in Preventing Excessive Bleeding During Surgery in Subjects With Congenital Type 3 Von Willebrand Disease
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
Read the detailed description
For the treatment of surgical procedures the intended dose of Alphanate® will be given as a single dose or as multiple doses over several days, depending on the clinical situation, and according to the Full Prescribing Information guideline and the investigator's judgment. For each treated event, the subject's treatment period will be finished when, in the opinion of the local Investigator, the participating subject would not benefit from further infusions of the study concentrate.
- Conditions
- Von Willebrand Disease
- Being tested
- Alphanate SD/HT
- Keywords
- Factor VIII deficiency, Von Willebrand disease, FVIII/VWF concentrate, Bleeding disorders, Blood disorders
- Medical subject headings
- von Willebrand Diseases, Hemophilia A, Hemostatic Disorders, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alphanate SD/HTBiological
Also called Alphanate® Factor VIII/VWF concentrate (Human)
A general guideline based on the product Full Prescribing Information is recommended with a maximum dose of 80 VWF:RCof IU/kg. The number of administrations before, during, and after the surgery procedure depends on the subject's clinical condition and the type of surgery itself. Single administrations or multiple doses may be appropriate. The dose of Alphanate® administered to each subject will be recorded as IU of VWF:RCof and also as IU of FVIII:C. The lot number for each vial of concentrate administered will also be recorded.
| Study group | Role | What they receive |
|---|---|---|
| Coagulation FVIII/VWF | Experimental | Anti-Hemophilic/von Willebrand Factor VIII (Human) Alphanate SD/HT Receives: Biological: Alphanate SD/HT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Investigator Overall Rating as a Measure of the Overall Treatment Outcome for Each Treated Surgical Procedure
The local Investigator to rate the replacement treatment outcome (Investigator Overall Rating - Verbal Rating Scale) as a measure of overall clinical efficacy for each treated event.
It is rated using a 2-point Verbal Rating Scale as: Excellent/Good and Poor/None.
Measured: Up to 30 days
Secondary outcomes (1)
Number of Surgical Procedures at Both Visits Rated as Excellent/Good by the Investigator
The number of surgical procedures rated by the local Investigator for the treatment outcomes at both visits.
Measured: Day 0 (surgery day) and Day 1 (post-surgery day)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2007 (estimated)
- Study start8 Jul 2008
- Primary completion6 Feb 2012
- Study completion6 Feb 2012
Why it stopped: Due to recruitment challenges during the trial, this study was terminated early by the Sponsor.
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 Von Willebrand DiseaseStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grifols Biologicals, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sandra Camprubi, Study director, Instituto Grifols, S.A.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00555555
- Sponsor's protocol ID
- GBI0703
- Status last verified
- Sep 2026
- First submitted
- 7 Nov 2007
- Last updated
- 30 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Post-marketing Observational Study to Assess the Efficacy and Safety of the FVIII/VWF Complex (Human), Alphanate®, in Preventing Excessive Bleeding During Surgery in Subjects With Congenital Type 3 Von Willebrand Disease
- ClinicalTrials.gov ID
- NCT00555555
- Acronym / sponsor ID
- GBI0703
- Conditions
- Von Willebrand Disease
- Keywords
- Factor VIII deficiency, Von Willebrand disease, FVIII/VWF concentrate, Bleeding disorders, Blood disorders
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to recruitment challenges during the trial, this study was terminated early by the Sponsor.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Grifols Biologicals, LLCIndustry (pharma, biotech, devices)
- Study start
- 8 Jul 2008 (actual)
- Primary completion
- 6 Feb 2012 (actual)
- Study completion
- 6 Feb 2012 (actual)
- First posted
- 8 Nov 2007 (estimated)
- Results
- Posted 30 Sep 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
Detailed description
For the treatment of surgical procedures the intended dose of Alphanate® will be given as a single dose or as multiple doses over several days, depending on the clinical situation, and according to the Full Prescribing Information guideline and the investigator's judgment. For each treated event, the subject's treatment period will be finished when, in the opinion of the local Investigator, the participating subject would not benefit from further infusions of the study concentrate.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Coagulation FVIII/VWF | Experimental | Biological: Alphanate SD/HTAnti-Hemophilic/von Willebrand Factor VIII (Human) Alphanate SD/HT |
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female 7 years of age or older
- The subject has been diagnosed of inherited VWD of Type 3 as determined by subject's medical records.
- The subject needs a surgical procedure (at least 10 surgical procedures have to be considered as "Major" according to the criteria of the protocol).
- The subject is expected to respond to exogenously administered FVIII/VWF according to Investigator's judgment.
- The subject freely gives written informed consent. Subjects who are not legally permitted to provide written consent must sign a form of assent for study participation, and written consent must be provided by a parent or legal guardian.
Key exclusion criteria (9 listed)
- The subject has been diagnosed of Type 1, Type 2, or acquired VWD.
- The subject is known to have history of intolerance to any Alphanate® containing substance.
- The subject is known to have history of anaphylactic reaction(s) to blood or blood components.
- Liver function tests (AST, ALT, bilirubin) > 2.5 x upper limit of normal (ULN).
- Renal function test (creatinine) > 1.5 x ULN.
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 |
|---|---|---|
| Alphanate SD/HTAlso: Alphanate® Factor VIII/VWF concentrate (Human)A general guideline based on the product Full Prescribing Information is recommended with a maximum dose of 80 VWF:RCof IU/kg. The number of administrations before, during, and after the surgery procedure depends on the subject's clinical condition and the type of surgery itself. Single administrations or multiple doses may be appropriate. The dose of Alphanate® administered to each subject will be recorded as IU of VWF:RCof and also as IU of FVIII:C. The lot number for each vial of concentrate administered will also be recorded. | Biological | Coagulation FVIII/VWF |
Primary outcome
Investigator Overall Rating as a Measure of the Overall Treatment Outcome for Each Treated Surgical Procedure
Time frame: Up to 30 days
The local Investigator to rate the replacement treatment outcome (Investigator Overall Rating - Verbal Rating Scale) as a measure of overall clinical efficacy for each treated event. It is rated using a 2-point Verbal Rating Scale as: Excellent/Good and Poor/None.
1 secondary outcome
Number of Surgical Procedures at Both Visits Rated as Excellent/Good by the Investigator
Time frame: Day 0 (surgery day) and Day 1 (post-surgery day)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Sandra Camprubi study director
Instituto Grifols, S.A.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Alphanate SD/HT in people with Von Willebrand Disease, run by Grifols Biologicals, LLC.
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 9 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: stopped early, as of the sponsor's last update on 30 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 30 Sep 2026). 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?
Investigator Overall Rating as a Measure of the Overall Treatment Outcome for Each Treated Surgical Procedure (time frame: Up to 30 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 30 Sep 2026; AzTrials last refreshed its copy on 8 Nov 2026.