Afibrinogenemia, CongenitalQualyAFIBNCT03484065
Quality of Life in Patients With Congenital Afibrinogenemia
An observational study of Afibrinogenemia, Congenital, run by University Hospital, Geneva at 28 sites in 21 countries.
Trial summary
The aim of this observational study is to evaluate the quality of life in patients with congenital afibrinogenemia using the Haemo-QoL SF for kids and the Haem-A-QoL for adult patients. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Afibrinogenemia, Congenital
- What is being tested?
- Questionnaire quality of life
- How many people
- 250 participants
- Where
- 28 sitesin 21 countries
- Study dates
- Jun 2016to Jun 2020
- 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 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients suffering from congenital afibrinogenemia
Who may be able to join
- Congenital afibrinogenemia confirmed by biology (absence of circulating fibrinogen) and genotype
Who cannot join
- Lack of participant's consent
- Patient unable to understand the questionnaire
Trial locations and contacts
28 sites in 21 countries.
28 sites in 21 countries
Turkey (Türkiye)3 sites
Uludag University
Bursa
Cerrahpasa Faculty of Medicine
Istanbul
Erciyes University
Kayseri
United States2 sites
Cohen Children's Medical Center
New Hyde Park, New York, 11040
Children's Hospital of Orange Count
Orange, California, 92868
Germany2 sites
Universitats Kilinikum Frankfurt
Frankfurt
Dr von Haumer Children's Hospital
Munich
India2 sites
St John Medical College Hospital
Bangalore
All India Institute of Medical Sciences
New Delhi
Pakistan1 site
National Institute Of Blood Disease and Bone Marrow Transplantation
Karachi
About this study
The aim of this observational study is to evaluate the quality of life in patients with congenital afibrinogenemia using the Haemo-QoL SF for kids and the Haem-A-QoL for adult patients.
Read the detailed description
In this observational study, children and adults suffering from congenital afibrinogenemia confirmed by biology will be enrolled. All patients will receive a questionnaire on quality of life in their own language during a routine visit and filled out by the patient at home. A general questionnaire will be filled out by the patient's physician.
- Conditions
- Afibrinogenemia, Congenital
- Being tested
- Questionnaire quality of life
- Keywords
- Congenital fibrinogen disorders
- Medical subject headings
- Afibrinogenemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Questionnaire quality of lifeOther
| Study group | Role | What they receive |
|---|---|---|
| Afibrinogenemia | Not described Receives: Other: Questionnaire quality of life |
What is being measured
The primary outcome is the main question the study is designed to answer.
The influence of the afibrinogenemia on the patients' quality of life assessed by the Haemo-QoL SF questionnaire (for children) and the Haem-A-QoL questionnaire (for adult)
The quality of life questionnaire includes item assessing:
- Physical health
- Feeling
- View
- Family
- Friends
- Others
- Sport and school
- Treatment
- Perceived support
- Dealing
- Future
- Relationship
Measured: At inclusion
Secondary outcomes (1)
Impact of the afibrinogenemic patient's clinical phenotype on the patients' quality of life
The clinical phenotype will be assessed by a general questionnaire including data on:
- Bleeding events
- Bleeding events treatment
- Thrombotic events
- Thrombotic events treatment
- Fibrinogen replacement (type of product and modality of replacement)
Measured: At Inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2018
- Study startJun 2016
- Primary completionJul 2019
- Study completionJun 2020
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 Afibrinogenemia, CongenitalStudies of the same condition, open and under way.
- More trials run by University Hospital, GenevaThe 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
- Cross-sectional
- Enrolment
- 250 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Geneva
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Casini Alessandro, University Hospital, Geneva
- Investigators
- Alessandro Casini, MD, Principal investigator, University Hospitals of Geneva
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03484065
- Sponsor's protocol ID
- 2016-00447
- Other IDs
- 2016
- Acronym
- QualyAFIB
- Status last verified
- Nov 2020
- First submitted
- 14 Mar 2018
- Last updated
- 12 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Casini A, von Mackensen S, Santoro C, Djambas Khayat C, Belhani M, Ross C, Dorgalaleh A, Naz A, Unal E, Abdelwahab M, Lozeron ED, Trillot N, Susen S, Peyvandi F, de Moerloose P; QualyAfib Study Group. Clinical phenotype, fibrinogen supplementation, and health-related quality of life in patients with afibrinogenemia. Blood. 2021 Jun 3;137(22):3127-3136. doi: 10.1182/blood.2020009472.
PubMed 33512441
Study at a glance
From the registry record, last updated by the sponsor on 12 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Quality of Life in Patients With Congenital Afibrinogenemia
- ClinicalTrials.gov ID
- NCT03484065
- Acronym / sponsor ID
- QualyAFIB / 2016-00447
- Conditions
- Afibrinogenemia, Congenital
- Keywords
- Congenital fibrinogen disorders
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 250 participants (actual)
- Lead sponsor
- University Hospital, GenevaUniversity, hospital or other
- Study start
- Jun 2016 (actual)
- Primary completion
- Jul 2019 (actual)
- Study completion
- Jun 2020 (actual)
- First posted
- 30 Mar 2018 (actual)
- 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.
The aim of this observational study is to evaluate the quality of life in patients with congenital afibrinogenemia using the Haemo-QoL SF for kids and the Haem-A-QoL for adult patients.
Detailed description
In this observational study, children and adults suffering from congenital afibrinogenemia confirmed by biology will be enrolled. All patients will receive a questionnaire on quality of life in their own language during a routine visit and filled out by the patient at home. A general questionnaire will be filled out by the patient's physician.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Afibrinogenemia | – | Other: Questionnaire quality of life |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients suffering from congenital afibrinogenemia
Key inclusion criteria (1 listed)
- Congenital afibrinogenemia confirmed by biology (absence of circulating fibrinogen) and genotype
Key exclusion criteria (2 listed)
- Lack of participant's consent
- Patient unable to understand the questionnaire
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 |
|---|---|---|
| Questionnaire quality of life | Other | Afibrinogenemia |
Primary outcome
The influence of the afibrinogenemia on the patients' quality of life assessed by the Haemo-QoL SF questionnaire (for children) and the Haem-A-QoL questionnaire (for adult)
Time frame: At inclusion
The quality of life questionnaire includes item assessing: * Physical health * Feeling * View * Family * Friends * Others * Sport and school * Treatment * Perceived support * Dealing * Future * Relationship
1 secondary outcome
Impact of the afibrinogenemic patient's clinical phenotype on the patients' quality of life
Time frame: At Inclusion
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 12 Nov 2020.
- Derived from this study: PubMed 33512441Casini A, von Mackensen S, Santoro C, Djambas Khayat C, Belhani M, Ross C, Dorgalaleh A, Naz A, Unal E, Abdelwahab M, Lozeron ED, Trillot N, Susen S, Peyvandi F, de Moerloose P; QualyAfib Study Group. Clinical phenotype, fibrinogen supplementation, and health-related quality of life in patients with afibrinogenemia. Blood. 2021 Jun 3;137(22):3127-3136. doi: 10.1182/blood.2020009472.
- 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.
Alessandro Casini, MD principal investigator
University Hospitals of Geneva
28 sites in 21 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Afibrinogenemia, Congenital, run by University Hospital, Geneva at 28 sites in 21 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 1 inclusion and 2 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 12 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in Turkey (Türkiye), United States, Germany, India, Lebanon and 16 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?
The influence of the afibrinogenemia on the patients' quality of life assessed by the Haemo-QoL SF questionnaire (for children) and the Haem-A-QoL questionnaire (for adult) (time frame: At inclusion). 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 NCT03484065, 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 12 Nov 2020.