Hemophilia ANCT02093741
ADVATE 2 mL Post-Authorization Safety Surveillance (PASS)
An observational study of Hemophilia A and Congenital Factor VIII (FVIII) Deficiency, run by Baxalta now part of Shire at 17 sites in 4 countries.
Trial summary
This is a Post-Authorization Safety Surveillance (PASS) study designed to collect data on the safety and effectiveness of ADVATE reconstituted in 2 mL Sterile water for injection (SWFI) during routine clinical practice in children until 12 years of age. Read more about this study
- Who is it for?
- Ages Up to 12 years
- Conditions the study is for
- Hemophilia A, Congenital Factor VIII (FVIII) Deficiency
- What is being tested?
- Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]
- How many people
- 65 participants
- Where
- 17 sitesin 4 countries
- Study dates
- 20 Sep 2013to 20 Jan 2016
- Phase
- Observational
- 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 Up to 12 years
- All sexes
- No healthy volunteers
- Child
Study population: The study population will include 60 evaluable participants aged = 12 years with severe or moderately severe hemophilia A (Factor VIII (FVIII) = 2%) with documented prior exposure to FVIII concentrates.
Who may be able to join
- Severe or moderately severe hemophilia A (baseline Factor VIII (FVIII) ≤ 2%)
- ≤12 years of age
- Participant's legally authorized representative(s) has provided written informed consent
- Participant is prescribed ADVATE and will only receive ADVATE reconstituted in 2 mL sterile water for injection (SWFI)
- Documented history of prior exposure to ADVATE
- Documented evidence of negative inhibitor test result during ≤10 EDs prior to study entry
Who cannot join
- Known hypersensitivity to the active substance or to any of the excipients
- Known allergic reaction to mouse or hamster proteins
- Participant has a requirement for a major surgical procedure at the time of enrollment
- Participant has no prior exposure to a FVIII concentrate
- Participant currently being treated with an immune tolerance induction (ITI) regimen
- Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand disease)
- Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device or PASS registry during the course of this study
Trial locations and contacts
17 sites in 4 countries.
17 sites in 4 countries
France8 sites
CHRU Pellegrin, Hématologie - CRTH
Bordeaux, 33076
Centre Hospitalier Générale, CTH
Chambéry, 73011
CRTH Laboratoire d'Hématologie CHRU - Hôpital du Bocage
Dijon, 21034
American Memorial Hospital, Service de Pédiatrie
Reims, 51092
CHU de Rennes Hôpital Pontchaillou
Rennes, 35033
Hôpital Nord, Pédiatrie
Saint-Priest-en-Jarez, 42270
CHRU Purpan, CRTH - Pavillon Sénac
Toulouse, 31059
CHU de Nancy - Hôpital de Brabois- CRTH - Laboratoire Hémato-Hémostase
Vandœuvre-lès-Nancy, 54511
Hungary4 sites
Heim Pál Children's Hospital, Department of Oncology
Budapest, 1131
Borsod-Abaúj-Zemplén County Hospital, Pediatric Dep
Miskolc, 3629
Mohacsi Korhaz, Department of Pediatrics
Mohács, 7700
Josa Andras Egyetemi Oktatokorhaz, Department of Pediatrics
Nyíregyháza, 4400
United Kingdom3 sites
Birmingham Children's Hospital NHS Trust
Birmingham, B4 6NH
Leeds General Infirmary
Leeds, West Yorkshire, LS1 3EX
Great Ormond Street Hospital for Children NHS Trust
London, WC1N 3JH
Germany2 sites
Universitätskliniken des Saarlandes, Klinik für pädiatrische Onkologie und Hämatologie
Homburg, Saarland, 66421
Klinikum Stuttgart, Olgahospital, Pädiatrie 5
Stuttgart, 70176
About this study
Official title: ADVATE 2 mL (Reconstituted in 2 mL SWFI) POST-AUTHORIZATION SAFETY SURVEILLANCE STUDY
This is a Post-Authorization Safety Surveillance (PASS) study designed to collect data on the safety and effectiveness of ADVATE reconstituted in 2 mL Sterile water for injection (SWFI) during routine clinical practice in children until 12 years of age. This surveillance study is a post-licensure commitment for ADVATE reconstituted in 2 mL SWFI.
- Being tested
- Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]Biological
Also called Antihemophilic Factor (Recombinant)- Plasma/albumin free method (rAHF-PFM), ADVATE
The investigators shall determine all treatment regimens according to product labeling information and standard practice.
| Study group | Role | What they receive |
|---|---|---|
| ADVATE - 2mL | Not described Receives: Biological: Octocog alfa (recombinant human coagulation factor VIII) [ADVATE] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of all local and general, hypersensitivity and infusion-related reactions, irrespective of product-related causality for the adverse events (AEs).
Measured: 6 months
Secondary outcomes (11)
Number and type of adverse events (or adverse experiences) (AEs) considered by the investigator to be causally related to ADVATE reconstituted in 2 mL sterile water for injection (SWFI)
Causally related = possibly or probably related
Measured: 6 months
Number of Factor VIII (FVIII) inhibitors in all participants
Measured: 6 months
Number of Factor VIII (FVIII) inhibitors in Previously Treated Patients (PTPs) (> 50 Exposure Days (EDs)) with baseline Factor VIII (FVIII) < 1% and no history of FVIII inhibitors prior to study entry
Measured: 6 months
Number of Factor VIII (FVIII) inhibitors in Previously Treated Patients (PTPs) (> 50 Exposure Days (EDs)) with baseline Factor VIII (FVIII) ≤ 2% and no history of FVIII inhibitors prior to study entry
Measured: 6 months
Subjective hemostatic effectiveness rating of excellent, good, fair, or none for each bleeding episode treated
Measured: 6 months
Number of bleeding episodes treated with 1, 2, 3, ≥ 4 infusions of ADVATE reconstituted in 2 mL sterile water for injection (SWFI)
Measured: 6 months
Total units of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) administered to treat each bleeding episode
Measured: 6 months
Overall effectiveness of prophylaxis in participants who are on a prophylactic regimen
Measured: 6 months
Global assessment rating of hemostatic effectiveness of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) in surgical or dental procedures
Global assessment rating = excellent, good, fair, or none
Measured: 6 months
Change in Factor VIII (FVIII) treatment satisfaction and preference ratings from caregiver between ADVATE reconstituted in 5 mL and 2 mL sterile water for injection (SWFI)
Measured: 6 months
Change in Factor VIII (FVIII) infusion volume and time to mix and infuse FVIII treatment between ADVATE reconstituted in 5 mL and 2 mL sterile water for injection (SWFI)
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2014 (estimated)
- Study start20 Sep 2013
- Primary completion20 Jan 2016
- Study completion20 Jan 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02093741
- Sponsor's protocol ID
- 061101
- Status last verified
- Mar 2021
- First submitted
- 19 Mar 2014
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ADVATE 2 mL (Reconstituted in 2 mL SWFI) POST-AUTHORIZATION SAFETY SURVEILLANCE STUDY
- ClinicalTrials.gov ID
- NCT02093741
- Acronym / sponsor ID
- 061101
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 20 Sep 2013 (actual)
- Primary completion
- 20 Jan 2016 (actual)
- Study completion
- 20 Jan 2016 (actual)
- First posted
- 21 Mar 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.
This is a Post-Authorization Safety Surveillance (PASS) study designed to collect data on the safety and effectiveness of ADVATE reconstituted in 2 mL Sterile water for injection (SWFI) during routine clinical practice in children until 12 years of age. This surveillance study is a post-licensure commitment for ADVATE reconstituted in 2 mL SWFI.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ADVATE - 2mL | – | Biological: Octocog alfa (recombinant human coagulation factor VIII) [ADVATE] |
Who may qualify
- Ages
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- The study population will include 60 evaluable participants aged = 12 years with severe or moderately severe hemophilia A (Factor VIII (FVIII) = 2%) with documented prior exposure to FVIII concentrates.
Key inclusion criteria (6 listed)
- Severe or moderately severe hemophilia A (baseline Factor VIII (FVIII) ≤ 2%)
- ≤12 years of age
- Participant's legally authorized representative(s) has provided written informed consent
- Participant is prescribed ADVATE and will only receive ADVATE reconstituted in 2 mL sterile water for injection (SWFI)
- Documented history of prior exposure to ADVATE
Key exclusion criteria (7 listed)
- Known hypersensitivity to the active substance or to any of the excipients
- Known allergic reaction to mouse or hamster proteins
- Participant has a requirement for a major surgical procedure at the time of enrollment
- Participant has no prior exposure to a FVIII concentrate
- Participant currently being treated with an immune tolerance induction (ITI) regimen
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 |
|---|---|---|
| Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]Also: Antihemophilic Factor (Recombinant)- Plasma/albumin free method (rAHF-PFM), ADVATEThe investigators shall determine all treatment regimens according to product labeling information and standard practice. | Biological | ADVATE - 2mL |
Primary outcome
Incidence of all local and general, hypersensitivity and infusion-related reactions, irrespective of product-related causality for the adverse events (AEs).
Time frame: 6 months
11 secondary outcomes
Number and type of adverse events (or adverse experiences) (AEs) considered by the investigator to be causally related to ADVATE reconstituted in 2 mL sterile water for injection (SWFI)
Time frame: 6 months
Number of Factor VIII (FVIII) inhibitors in all participants
Time frame: 6 months
Number of Factor VIII (FVIII) inhibitors in Previously Treated Patients (PTPs) (> 50 Exposure Days (EDs)) with baseline Factor VIII (FVIII) < 1% and no history of FVIII inhibitors prior to study entry
Time frame: 6 months
Number of Factor VIII (FVIII) inhibitors in Previously Treated Patients (PTPs) (> 50 Exposure Days (EDs)) with baseline Factor VIII (FVIII) ≤ 2% and no history of FVIII inhibitors prior to study entry
Time frame: 6 months
Subjective hemostatic effectiveness rating of excellent, good, fair, or none for each bleeding episode treated
Time frame: 6 months
Number of bleeding episodes treated with 1, 2, 3, ≥ 4 infusions of ADVATE reconstituted in 2 mL sterile water for injection (SWFI)
Time frame: 6 months
Total units of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) administered to treat each bleeding episode
Time frame: 6 months
Overall effectiveness of prophylaxis in participants who are on a prophylactic regimen
Time frame: 6 months
Global assessment rating of hemostatic effectiveness of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) in surgical or dental procedures
Time frame: 6 months
Change in Factor VIII (FVIII) treatment satisfaction and preference ratings from caregiver between ADVATE reconstituted in 5 mL and 2 mL sterile water for injection (SWFI)
Time frame: 6 months
Change in Factor VIII (FVIII) infusion volume and time to mix and infuse FVIII treatment between ADVATE reconstituted in 5 mL and 2 mL sterile water for injection (SWFI)
Time frame: 6 months
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 17 Mar 2021.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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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.
Study Director study director
Takeda
17 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A and Congenital Factor VIII (FVIII) Deficiency, run by Baxalta now part of Shire at 17 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 6 inclusion and 7 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 17 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in France, Hungary, United Kingdom, Germany. 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.
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?
Incidence of all local and general, hypersensitivity and infusion-related reactions, irrespective of product-related causality for the adverse events (AEs). (time frame: 6 months). 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 17 Mar 2021.