Congenital Bleeding DisorderPUCHSNCT01959555

Project to Update the Study of Congenital Haemophilia in Spain

An observational study of Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 1 site in Spain.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

This study is conducted in Europe. The aim of the study is to update the knowledge about the prevalence and severity of congenital haemophilia (A and B) in Spain. Read more about this study

Who is it for?: Any age, men only
Conditions the study is for: Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
What is being tested?: No treatment given
How many people: 1,157 participants
Where: 1 siteSpain
Study dates: Mar 2014to Dec 2014
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
Men only
No healthy volunteers
Child, Adult, Older adult

Study population: Patients in active follow-up in HTCs (Haemophilia Treatment Centres) will be included in this study. A patient is considered as active if a visit to the HTC is performed in the period of January 2013 to December 2013.

Who may be able to join

Informed consent obtained before any study-related activities. (Study-related activities are any procedure related to recording of data according to the protocol)
Male patients of any age diagnosed with congenital haemophilia A or B who visited their HTC at least once during the period of January 2013 to December 2013

Who cannot join

Previous participation in this study
Female patients
Acquired haemophilia

Trial locations and contacts

1 site in 1 country.

Spain1 site

Study site
Madrid, 28033

Other trials in Spain

About this study

This study is conducted in Europe. The aim of the study is to update the knowledge about the prevalence and severity of congenital haemophilia (A and B) in Spain.

Conditions: Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
Being tested: No treatment given
Medical subject headings: Hemophilia A, Hemophilia B

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

No treatment givenOther
Patients will not be treated with any specific product. Data collection only.

Study groups in NCT01959555
Study group	Role	What they receive
Retrospective collection of data		Not described Receives: Other: No treatment given

What is being measured

The primary outcome is the main question the study is designed to answer.

Prevalence of congenital haemophilia A and B in Spain in 2013, described as proportion (%) of patients in the total population (extracted from the last available census of the Spanish population for the regions considered for the study)
Measured: Assessed at study visit (day 1)
Severity (mild, moderate or severe) of congenital haemophilia A and B in Spain during 2013, described as proportion (%) per category of the total number of patients with haemophilia A or B
Measured: Assessed at study visit (day 1)

Secondary outcomes (18)

Mean current age at the time of visit to the HTC in 2013
Measured: Assessed at study visit (day 1)
Mean age at diagnosis
Measured: Assessed at study visit (day 1)
Proportion (%) of patients with/without genetic screening performed
Measured: Assessed at study visit (day 1)
Proportion (%) of patients who needed support of a reference Haemophilia Healthcare Centre in 2013
Measured: Assessed at study visit (day 1)
Proportion (%) of patients who received plasmatic or recombinant treatment
Measured: Assessed at study visit (day 1)
Proportion (%) of patients who received on demand or prophylaxis treatment
Measured: Assessed at study visit (day 1)
Mean number of bleeding episodes
Measured: Assessed at study visit (day 1)
Mean number of joint bleeds
Measured: Assessed at study visit (day 1)
Proportion (%) of patients with established arthropathy
Measured: Assessed at study visit (day 1)
Proportion (%) of patients with infectious complications
Measured: Assessed at study visit (day 1)
Proportion (%) of patients with HIV (Human immunodeficiency virus)
Measured: Assessed at study visit (day 1)
Proportion (%) of patients with presence of inhibitors
Measured: Assessed at study visit (day 1)
Proportion (%) of patients with immune tolerance induction treatment performed
Measured: Assessed at study visit (day 1)
Total number of surgery interventions performed
Measured: ThAssessed at study visit (day 1)
Proportion (%) of patients with quality of life questionnaire (QoLQ) performed in 2013
Measured: Assessed at study visit (day 1)
Mean number of days of absenteeism to school/university in children and adolescents in 2013
Measured: Assessed at study visit (day 1)
Mean number of days of absenteeism from work in 2013
Measured: Assessed at study visit (day 1)
Proportion (%) of patients with treatment administered at home
Measured: Assessed at study visit (day 1)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Mar 2014Dec 2014

First posted10 Oct 2013 (estimated)
Study startMar 2014
Primary completionDec 2014
Study completionDec 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
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
Time perspective: Retrospective
Enrolment: 1,157 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01959555
Sponsor's protocol ID: HAEM-4076
Other IDs: U1111-1136-7052, NOV-HEM-2013-01
Acronym: PUCHS
Status last verified: Nov 2015
First submitted: 8 Oct 2013
Last updated: 30 Nov 2015
Results posted: No
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
Not yet recruitingPhase 1
Topical TOR-582 Treatment of Epistaxis in HHT
Hereditary Hemorrhagic Telangiectasia (HHT), Epistaxis
RecruitingPhase 1/2
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Recruiting
Optimization of Saturation Targets And Resuscitation Trial (OptiSTART)
Premature Infants, Bronchopulmonary Dysplasia

All Congenital Bleeding Disorder trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Nov 2015. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Project to Update the Study of Congenital Haemophilia in Spain
ClinicalTrials.gov ID: NCT01959555
Acronym / sponsor ID: PUCHS / HAEM-4076
Conditions: Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
Study type: Observational
Recruitment status: Completed
Enrolment: 1,157 participants (actual)
Lead sponsor: Novo Nordisk A/SIndustry (pharma, biotech, devices)
Study start: Mar 2014
Primary completion: Dec 2014 (actual)
Study completion: Dec 2014 (actual)
First posted: 10 Oct 2013 (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 study is conducted in Europe. The aim of the study is to update the knowledge about the prevalence and severity of congenital haemophilia (A and B) in Spain.

Study design

Time perspective: Retrospective
Number of arms: 1

Arm	Type	Receives
Retrospective collection of data	–	Other: No treatment given

Who may qualify

Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: Patients in active follow-up in HTCs (Haemophilia Treatment Centres) will be included in this study. A patient is considered as active if a visit to the HTC is performed in the period of January 2013 to December 2013.

Key inclusion criteria (2 listed)

Informed consent obtained before any study-related activities. (Study-related activities are any procedure related to recording of data according to the protocol)
Male patients of any age diagnosed with congenital haemophilia A or B who visited their HTC at least once during the period of January 2013 to December 2013

Key exclusion criteria (3 listed)

Previous participation in this study
Female patients
Acquired haemophilia

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
No treatment givenPatients will not be treated with any specific product. Data collection only.	Other	Retrospective collection of data

Primary outcome

Prevalence of congenital haemophilia A and B in Spain in 2013, described as proportion (%) of patients in the total population (extracted from the last available census of the Spanish population for the regions considered for the study)
Time frame: Assessed at study visit (day 1)
Severity (mild, moderate or severe) of congenital haemophilia A and B in Spain during 2013, described as proportion (%) per category of the total number of patients with haemophilia A or B
Time frame: Assessed at study visit (day 1)

18 secondary outcomes

Mean current age at the time of visit to the HTC in 2013
Time frame: Assessed at study visit (day 1)
Mean age at diagnosis
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients with/without genetic screening performed
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients who needed support of a reference Haemophilia Healthcare Centre in 2013
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients who received plasmatic or recombinant treatment
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients who received on demand or prophylaxis treatment
Time frame: Assessed at study visit (day 1)
Mean number of bleeding episodes
Time frame: Assessed at study visit (day 1)
Mean number of joint bleeds
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients with established arthropathy
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients with infectious complications
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients with HIV (Human immunodeficiency virus)
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients with presence of inhibitors
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients with immune tolerance induction treatment performed
Time frame: Assessed at study visit (day 1)
Total number of surgery interventions performed
Time frame: ThAssessed at study visit (day 1)
Proportion (%) of patients with quality of life questionnaire (QoLQ) performed in 2013
Time frame: Assessed at study visit (day 1)
Mean number of days of absenteeism to school/university in children and adolescents in 2013
Time frame: Assessed at study visit (day 1)
Mean number of days of absenteeism from work in 2013
Time frame: Assessed at study visit (day 1)
Proportion (%) of patients with treatment administered at home
Time frame: Assessed at study visit (day 1)

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 30 Nov 2015.
Clinical Trials at Novo NordiskLink provided by the sponsor.

Related trials for Congenital Bleeding Disorder

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
Not yet recruitingPhase 1
Topical TOR-582 Treatment of Epistaxis in HHT
Hereditary Hemorrhagic Telangiectasia (HHT), Epistaxis
RecruitingPhase 1/2
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Recruiting
Optimization of Saturation Targets And Resuscitation Trial (OptiSTART)
Premature Infants, Bronchopulmonary Dysplasia

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

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 1 site in Spain.

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 3 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 30 Nov 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Spain. 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?

Prevalence of congenital haemophilia A and B in Spain in 2013, described as proportion (%) of patients in the total population (extracted from the last available census of the Spanish population for the regions considered for the study) (time frame: Assessed at study visit (day 1)). The study lists 2 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 NCT01959555.

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 Nov 2015.