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NCT Number: NCT06691581

Italian Study for Congenital Platelet Disorders

Inherited platelet disorders (IPD) are a heterogeneous group of rare bleeding diseases associated with a reduction of platelet number and/or function and with a bleeding tendency ranging from mild to severe. The frequency of inherited thrombocytopenias has been estimated to be 2.7/100,000 while the prevalence of inherited platelet function disorders is unknown, partly because they are frequently overlooked due to their difficult diagnosis.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Platelet Disorder Blood Platelet Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Fondazione Policlinico Universitario A.Gemelli IRCCS, Roma, Italy

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About this study

This a no-profit national multicenter ambispectic (retrospective and prospective) observational study.

After collection of informed consent form each patient, each center will enroll the patient and will collect general, laboratory and clinical data on an electronic CRF on a REDCAP platform. Each patient will receive a unique identification number. All clinical events will be reported in the data base.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • All patients with established diagnosis of inherited number or function platelet disorder (IPN and IPD, respectively), on the basis of internationally established clinical, laboratory and genetic criteria will be enrolled.
  • For patients <18 ys, consensus will be signed by parents or legal responsible

Exclusion criteria

  • No written consent
  • Undocumented or uncertain diagnosis

Treatment and study plan

Primary outcomes

  1. congenital platelet diseases

    Time frame: 48 months

    The main objectives of the study are to collect information on the diagnosis and management of these rare disorders and to create clinical and scientific collaborations between participating centres, and the aim of this project is to create for the first time an ambiseptic multicentre database on clinical and laboratory data on patients with congenital platelet disorders and to observe the prevalence of different congenital platelet disorders

Secondary outcomes

  1. QoL

    Time frame: 48 months

    description of different symptoms and therapeutical options and evaluation of health-related quality of life through validated questionnaires that record the patient's self-assessment of health on a vertical visual analogue scale in which the endpoints are labelled 'best imaginable health' and 'worst imaginable health'

Sponsors and collaborators

Lead sponsor

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Other

Registry information

Official study title

Multicenter Observational Ambispective Study for Congenital Platelet Disorders

Acronym: SIPaP

Important dates

Study start
2024
Primary completion
2028
Study completion
2028
First posted
Nov 15, 2024
Registry last updated
Nov 15, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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