Project to Update the Study of Congenital Haemophilia in Spain (PUCHS)

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01959555
First received: October 8, 2013
Last updated: March 19, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention
Congenital Bleeding Disorder
Haemophilia A
Haemophilia B
Other: No treatment given

Study Type: Observational
Study Design: Time Perspective: Retrospective
Official Title: Project to Update the Study of Congenital Haemophilia in Spain

Resource links provided by NLM:


Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:
  • 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) ] [ Designated as safety issue: No ]
  • 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) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Mean current age at the time of visit to the HTC in 2013 [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Mean age at diagnosis [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients with/without genetic screening performed [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients who needed support of a reference Haemophilia Healthcare Centre in 2013 [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients who received plasmatic or recombinant treatment [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients who received on demand or prophylaxis treatment [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Mean number of bleeding episodes [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Mean number of joint bleeds [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients with established arthropathy [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients with infectious complications [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients with HIV (Human immunodeficiency virus) [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients with presence of inhibitors [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients with immune tolerance induction treatment performed [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Total number of surgery interventions performed [ Time Frame: ThAssessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients with quality of life questionnaire (QoLQ) performed in 2013 [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Mean number of days of absenteeism to school/university in children and adolescents in 2013 [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Mean number of days of absenteeism from work in 2013 [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]
  • Proportion (%) of patients with treatment administered at home [ Time Frame: Assessed at study visit (day 1) ] [ Designated as safety issue: No ]

Estimated Enrollment: 2500
Study Start Date: March 2014
Estimated Study Completion Date: September 2014
Estimated Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Retrospective collection of data Other: No treatment given
Patients will not be treated with any specific product. Data collection only.

  Eligibility

Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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.

Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Previous participation in this study
  • Female patients
  • Acquired haemophilia
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01959555

Locations
Spain
Madrid, Spain, 28033
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
No publications provided

Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01959555     History of Changes
Other Study ID Numbers: HAEM-4076, U1111-1147-5668, NOV-HEM-2013-01
Study First Received: October 8, 2013
Last Updated: March 19, 2014
Health Authority: Spain: Spanish Agency of Medicines

Additional relevant MeSH terms:
Blood Coagulation Disorders
Hemostatic Disorders
Hemorrhagic Disorders
Hemophilia B
Hemophilia A
Hemorrhage
Hematologic Diseases
Vascular Diseases
Cardiovascular Diseases
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Pathologic Processes

ClinicalTrials.gov processed this record on July 23, 2014