Congenital Bleeding DisorderNCT01476423
Observational Registry of the Treatment of Glanzmann's Thrombasthenia
An observational study of Congenital Bleeding Disorder and Glanzmann's Disease, run by Novo Nordisk A/S at 15 sites in 15 countries.
Trial summary
This observational registry is conducted in Europe, Asia, Africa and the United States of America (USA). The purpose of the registry is to evaluate the efficacy and safety of activated recombinant human factor VII (rFVIIa) during bleeding episodes and for the prevention of bleeding during invasive procedures/surgery… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Congenital Bleeding Disorder, Glanzmann's Disease
- What is being tested?
- activated recombinant human factor VII
- How many people
- 218 participants
- Where
- 15 sitesin 15 countries
- Study dates
- Jan 2004to Oct 2011
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with GT. There is no limit to the number of bleeding episodes or preventions of bleeding during invasive procedures/surgery that can be entered for each patient within the lifetime of the registry. Patients will receive standard care according to local practice, thus any systemic haemostatic treatment (with or without antifibrinolytic drugs or other agents) considered useful by the centres for treatment of GT, are included in the registry.
Who may be able to join
- Patients with congenital GT defined as patients with lifelong bleeding tendency characterised by impaired or absent platelet aggregation, impaired clot retraction and prolonged bleeding time or prolonged platelet function analyser closure time. The patient has normal platelet counts and platelet morphology. Optional diagnosis criteria are quantitative or qualitative evaluation of GP (Glycoprotein) IIb/IIIa receptor including flow cytometry and identification of gene defects
- Signed informed consent by the patient or next of kin or legally acceptable representative to collect data on treatment of a given bleeding episode or surgical event as specified in the protocol. If informed consent is provided by the next of kin or legally acceptable representative, consent must also be obtained from the patient as soon as he/she is able to do so. Informed consent must be obtained before entry of data into the registry
Who cannot join
- Patients with acquired thrombasthenic states caused by autoimmune disorders (acute or chronic) or drugs
Trial locations and contacts
15 sites in 15 countries.
15 sites in 15 countries
United States1 site
Novo Nordisk Clinical Trial Call Center
Princeton, New Jersey, 08540
About this study
Official title: Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry
This observational registry is conducted in Europe, Asia, Africa and the United States of America (USA).
The purpose of the registry is to evaluate the efficacy and safety of activated recombinant human factor VII (rFVIIa) during bleeding episodes and for the prevention of bleeding during invasive procedures/surgery in patients with Glanzmann's thrombasthenia (GT) with past or present refractoriness to platelet transfusions. Attention will be directed towards complications related to thrombo-embolic events and concomitant medications especially antifibrinolytics.
- Conditions
- Congenital Bleeding Disorder, Glanzmann's Disease
- Being tested
- activated recombinant human factor VII
- Keywords
- Glanzmann's Thrombasthenia
- Medical subject headings
- Thrombasthenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
activated recombinant human factor VIIDrug
A prospective, observational multi-national registry collecting data and evaluating the efficacy and safety of rFVIIa in patients with GT with past or present refractoriness to platelet transfusions. The registry will also collect data from a broader range of GT patients treated with systemic haemostatic treatment (with or without antifibrinolytic drugs or other agents) used in the clinics. Data collection will continue for a maximum of six years. Baseline data as well as data obtained during either bleeding episodes or invasive procedures/surgeries will be recorded in the registry.
| Study group | Role | What they receive |
|---|---|---|
| A | Not described Receives: Drug: activated recombinant human factor VII |
What is being measured
The primary outcome is the main question the study is designed to answer.
For bleeding episodes: Overall efficacy evaluated by the caregiver/patient
Measured: within 30 days of end of treatment
For surgery including invasive and dental procedures: Haemoglobin level
Measured: prior to surgery and 24 hours after surgery
For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon
Measured: 24 hours after surgery
Secondary outcomes (3)
Changes in laboratory parameters (prothrombin time, platelet count, fibrinogen), if available
Measured: at the time of administration and two hours after the administration of rFVIIa
Adverse Events (AEs)
Measured: during treatment episodes
Serious Adverse Events (SAEs)
Measured: during treatment episodes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Nov 2011 (estimated)
- Study startJan 2004
- Primary completionOct 2011
- Study completionOct 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Congenital Bleeding DisorderStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 218 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01476423
- Sponsor's protocol ID
- F7HAEM-3521
- Other IDs
- U1111-1122-5019
- Status last verified
- Dec 2014
- First submitted
- 7 Oct 2011
- Last updated
- 23 Dec 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Poon MC, D'Oiron R, Baby S, Zotz RB, Di Minno G. The Glanzmann Thrombasthenia Registry: safety of platelet therapy in patients with Glanzmann thrombasthenia and changes in alloimmunization status. Haematologica. 2023 Oct 1;108(10):2855-2858. doi: 10.3324/haematol.2022.281973. No abstract available.
PubMed 36924249Zotz RB, Poon MC, Di Minno G, D'Oiron R; Glanzmann Thrombasthenia Registry Investigators. The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes. TH Open. 2019 Sep 12;3(3):e286-e294. doi: 10.1055/s-0039-1696657. eCollection 2019 Jul.
PubMed 31523745Di Minno G, Zotz RB, d'Oiron R, Bindslev N, Di Minno MN, Poon MC; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes of non-surgical bleeding episodes in patients with Glanzmann thrombasthenia. Haematologica. 2015 Aug;100(8):1031-7. doi: 10.3324/haematol.2014.121475. Epub 2015 May 22.
PubMed 26001793Poon MC, d'Oiron R, Zotz RB, Bindslev N, Di Minno MN, Di Minno G; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment and outcomes in surgical intervention. Haematologica. 2015 Aug;100(8):1038-44. doi: 10.3324/haematol.2014.121384. Epub 2015 May 22.
PubMed 26001792
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry
- ClinicalTrials.gov ID
- NCT01476423
- Acronym / sponsor ID
- F7HAEM-3521
- Conditions
- Congenital Bleeding Disorder, Glanzmann's Disease
- Keywords
- Glanzmann's Thrombasthenia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 218 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Jan 2004
- Primary completion
- Oct 2011 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 22 Nov 2011 (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 observational registry is conducted in Europe, Asia, Africa and the United States of America (USA).
The purpose of the registry is to evaluate the efficacy and safety of activated recombinant human factor VII (rFVIIa) during bleeding episodes and for the prevention of bleeding during invasive procedures/surgery in patients with Glanzmann's thrombasthenia (GT) with past or present refractoriness to platelet transfusions. Attention will be directed towards complications related to thrombo-embolic events and concomitant medications especially antifibrinolytics.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | – | Drug: activated recombinant human factor VII |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with GT. There is no limit to the number of bleeding episodes or preventions of bleeding during invasive procedures/surgery that can be entered for each patient within the lifetime of the registry. Patients will receive standard care according to local practice, thus any systemic haemostatic treatment (with or without antifibrinolytic drugs or other agents) considered useful by the centres for treatment of GT, are included in the registry.
Key inclusion criteria (2 listed)
- Patients with congenital GT defined as patients with lifelong bleeding tendency characterised by impaired or absent platelet aggregation, impaired clot retraction and prolonged bleeding time or prolonged platelet function analyser closure time. The patient has normal platelet counts and platelet morphology. Optional diagnosis criteria are quantitative or qualitative evaluation of GP (Glycoprotein) IIb/IIIa receptor including flow cytometry and identification of gene defects
- Signed informed consent by the patient or next of kin or legally acceptable representative to collect data on treatment of a given bleeding episode or surgical event as specified in the protocol. If informed consent is provided by the next of kin or legally acceptable representative, consent must also be obtained from the patient as soon as he/she is able to do so. Informed consent must be obtained before entry of data into the registry
Key exclusion criteria (1 listed)
- Patients with acquired thrombasthenic states caused by autoimmune disorders (acute or chronic) or 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 |
|---|---|---|
| activated recombinant human factor VIIA prospective, observational multi-national registry collecting data and evaluating the efficacy and safety of rFVIIa in patients with GT with past or present refractoriness to platelet transfusions. The registry will also collect data from a broader range of GT patients treated with systemic haemostatic treatment (with or without antifibrinolytic drugs or other agents) used in the clinics. Data collection will continue for a maximum of six years. Baseline data as well as data obtained during either bleeding episodes or invasive procedures/surgeries will be recorded in the registry. | Drug | A |
Primary outcome
For bleeding episodes: Overall efficacy evaluated by the caregiver/patient
Time frame: within 30 days of end of treatment
For surgery including invasive and dental procedures: Haemoglobin level
Time frame: prior to surgery and 24 hours after surgery
For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon
Time frame: 24 hours after surgery
3 secondary outcomes
Changes in laboratory parameters (prothrombin time, platelet count, fibrinogen), if available
Time frame: at the time of administration and two hours after the administration of rFVIIa
Adverse Events (AEs)
Time frame: during treatment episodes
Serious Adverse Events (SAEs)
Time frame: during treatment episodes
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 23 Dec 2014.
- Derived from this study: PubMed 36924249Poon MC, D'Oiron R, Baby S, Zotz RB, Di Minno G. The Glanzmann Thrombasthenia Registry: safety of platelet therapy in patients with Glanzmann thrombasthenia and changes in alloimmunization status. Haematologica. 2023 Oct 1;108(10):2855-2858. doi: 10.3324/haematol.2022.281973. No abstract available.
- Derived from this study: PubMed 31523745Zotz RB, Poon MC, Di Minno G, D'Oiron R; Glanzmann Thrombasthenia Registry Investigators. The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes. TH Open. 2019 Sep 12;3(3):e286-e294. doi: 10.1055/s-0039-1696657. eCollection 2019 Jul.
- Derived from this study: PubMed 26001793Di Minno G, Zotz RB, d'Oiron R, Bindslev N, Di Minno MN, Poon MC; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes of non-surgical bleeding episodes in patients with Glanzmann thrombasthenia. Haematologica. 2015 Aug;100(8):1031-7. doi: 10.3324/haematol.2014.121475. Epub 2015 May 22.
- Derived from this study: PubMed 26001792Poon MC, d'Oiron R, Zotz RB, Bindslev N, Di Minno MN, Di Minno G; Glanzmann Thrombasthenia Registry Investigators. The international, prospective Glanzmann Thrombasthenia Registry: treatment and outcomes in surgical intervention. Haematologica. 2015 Aug;100(8):1038-44. doi: 10.3324/haematol.2014.121384. Epub 2015 May 22.
- Clinical Trials at Novo NordiskLink provided by the sponsor.
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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.
Global Clinical Registry (GCR, 1452) study director
Novo Nordisk A/S
15 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Bleeding Disorder and Glanzmann's Disease, run by Novo Nordisk A/S at 15 sites in 15 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 2 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 23 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Algeria, Austria, Belgium, Bulgaria and 10 more countries. 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?
For bleeding episodes: Overall efficacy evaluated by the caregiver/patient (time frame: within 30 days of end of treatment). The study lists 3 primary outcomes in all. 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 NCT01476423, which also lists 4 linked publications.
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 23 Dec 2014.