Platelet DisorderSIPaPNCT06691581
Italian Study for Congenital Platelet Disorders
An observational study of Platelet Disorder, run by Fondazione Policlinico Universitario Agostino Gemelli IRCCS at 2 sites in Italy.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Platelet Disorder
- How many people
- 200 participants
- Where
- 2 sitesItaly
- Study dates
- 15 Oct 2024to 30 Oct 2028
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- No written consent
- Undocumented or uncertain diagnosis
Trial locations and contacts
2 sites in 1 country.
Italy2 sites
Fondazione Policlinico Universitario A.Gemelli IRCCS
Roma, Roma, 00168
Fondazione Policlinico Universitario A.Gemelli IRCCS, Roma, Roma 00168
Rome, rome, 00168
About this study
Official title: Multicenter Observational Ambispective 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.
Read the detailed description
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.
- Conditions
- Platelet Disorder
- Medical subject headings
- Blood Platelet Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 48 months
Secondary outcomes (1)
QoL
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'
Measured: 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2024
- Study start15 Oct 2024
- Primary completion1 Oct 2028 (estimated)
- Study completion30 Oct 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Platelet DisorderStudies of the same condition, open and under way.
- More trials run by Fondazione Policlinico Universitario Agostino Gemelli IRCCSThe 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
- Prospective
- Enrolment
- 200 participants (estimated)
- Target duration
- 2 Years
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, De Candia Erica, Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Investigators
- Erica De Candia, MD, Principal investigator, Fondazione Policlinico A. Gemelli IRCCS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06691581
- Sponsor's protocol ID
- 7003
- Acronym
- SIPaP
- Status last verified
- Oct 2024
- First submitted
- 11 Nov 2024
- Last updated
- 15 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Observational Ambispective Study for Congenital Platelet Disorders
- ClinicalTrials.gov ID
- NCT06691581
- Acronym / sponsor ID
- SIPaP / 7003
- Conditions
- Platelet Disorder
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCSUniversity, hospital or other
- Study start
- 15 Oct 2024 (actual)
- Primary completion
- 1 Oct 2028 (estimated)
- Study completion
- 30 Oct 2028 (estimated)
- First posted
- 15 Nov 2024 (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.
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.
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 2 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (2 listed)
- No written consent
- Undocumented or uncertain diagnosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
1 secondary outcome
QoL
Time frame: 48 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Nov 2024.
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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.
Erica De Candia, MD principal investigator
Fondazione Policlinico A. Gemelli IRCCS
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Platelet Disorder, run by Fondazione Policlinico Universitario Agostino Gemelli IRCCS at 2 sites in Italy.
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 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: under way, not looking for participants, as of the sponsor's last update on 15 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. 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?
This study is still under way. Results are not available in the source record yet.
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?
congenital platelet diseases (time frame: 48 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Nov 2024.