Italian Study for Congenital Platelet Disorders

NCT06691581 | Active Not Recruiting

• 1 other identifier: 7003
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Platelet Disorder

Outcome Measures

Primary Outcomes (1)

• congenital platelet diseases

  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

  48 months

Secondary Outcomes (1)

• QoL

  48 months

Study Arms (1)

All patients with established diagnosis will be enrolled.

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.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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

You may not qualify if:

• No written consent
• Undocumented or uncertain diagnosis

Sponsors & Collaborators

• Fondazione Policlinico Universitario Agostino Gemelli IRCCS: lead

Study Sites (2)

Fondazione Policlinico Universitario A.Gemelli IRCCS

Roma, Roma, 00168, Italy

Location

Fondazione Policlinico Universitario A.Gemelli IRCCS, Roma, Roma 00168

Rome, rome, 00168, Italy

Location

MeSH Terms

Conditions

Blood Platelet Disorders

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Erica De Candia, MD

  Fondazione Policlinico A. Gemelli IRCCS

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Internal Medicine

Study Record Dates

• First Submitted: November 11, 2024
• First Posted: November 15, 2024
• Study Start: October 15, 2024
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): October 30, 2028
• Last Updated: November 15, 2024

Record last verified: 2024-10

Locations

IT (2)