Purpura, ThrombocytopenicRE-PTTNCT05914441
Spanish-Portuguese Thrombotic Thrombocytopenic Purpura Registry
An observational study of Purpura, Thrombocytopenic, run by Fundación Española de Hematología y Hemoterapía at 7 sites in Spain.
Trial summary
REPTT is an observational, prospective, multi-country, multicentre and non-interventional registry in which at least 300 patients with Thrombotic thrombocytopenic purpura (TTP) in Spain and Portugal will be evaluated. Read more about this study
- Who is it for?
- Ages Up to 99 years
- Conditions the study is for
- Purpura, Thrombocytopenic
- What is being tested?
- Standard clinical practice
- How many people
- 300 participants
- Where
- 7 sitesSpain
- Study dates
- 22 Mar 2024to Dec 2026
- How long will I take part?
- 3 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 Up to 99 years
- All sexes
- Child, Adult, Older adult
Study population: Patients diagnosed with Thrombotic thrombocytopenic purpura (TTP)
Who may be able to join
- Patients with diagnosis of TTP according to International Consensus criteria from centres in Spain and Portugal.
- Patients that voluntarily sign the informed consent. For subjects unable to provide informed consent, a fully recognized medical authority may be used according to local laws.
- Patients between 0 to 99 years old at the time of diagnosis.
- Note: Decision was taken to treat the patient with an specific treatment prior and independently of patient inclusion in this non interventional study.
Who cannot join
- Inability to comply with study procedures and follow-up exams.
- Patients with any type of alteration that compromises their ability to grant written informed consent.
- Patients that do not consent to participate in the study and do not sign informed consent.
- Patients that do not meet the criteria previously mentioned for TTP.
Trial locations and contacts
7 sites in 1 country. Contact the study team or a site near you to ask about taking part.
7 sites in 1 country
Spain7 sites, 7 recruiting
- Recruiting
Hospital Clínic Barcelona
Barcelona, 08036
A responsible person Designated by the sponsor, 934344412, investigacion@mfar.net
A Principal Investigator Designated by the sponsor, M.D.
- Recruiting
Hospital Universitario de Guadalajara
Guadalajara, 19002
A responsible person Designated by the sponsor, 934344412, investigacion@mfar.net
A Principal Investigator Designated by the sponsor, M.D.
- Recruiting
Hospital Universitario Bellvitge
L'Hospitalet de Llobregat, Barcelona, 08907
A responsible person Designated by the Sponsor, 934344412, investigacion@mfar.net
A Principal Investigator Designated by the Sponsor, M.D.
- Recruiting
Hospital Universitario La Paz
Madrid, 28046
A responsible person Designated by the sponsor, 934344412, investigacion@mfar.net
A Principal Investigator Designated by the Sponsor, M.D.
- Recruiting
Hospital Virgen de la Arrixaca
Murcia, 30120
A responsible person Designated by the Sponsor, 934344412, investigacion@mfar.net
A Principal Investigator Designated by the Sponsor, M.D.
- Recruiting
Hospital Universitario Virgen del Rocío
Seville, Andalusia, 41013
A responsible person Designated by the sponsor, 934344412, investigacion@mfar.net
A Principal Investigator Designated by the sponsor, M.D.
- Recruiting
Hospital Clínico Universitario de Valencia
Valencia, 46010
A responsible person Designated by the sponsor, 934344412, investigacion@mfar.net
A Principal Investigator Designated by the sponsor, M.D.
About this study
Official title: Development Of Spanish-Portuguese Thrombotic Thrombocytopenic Purpura Registry (REPTT): A Study Proposal Of The Spanish Society Of Hematology And Hemotherapy (SEHH) Whit The Portuguese Society Of Hematology (SPH)
REPTT is an observational, prospective, multi-country, multicentre and non-interventional registry in which at least 300 patients with Thrombotic thrombocytopenic purpura (TTP) in Spain and Portugal will be evaluated.
The study will be carried out in the context of the usual clinical practice conditions, not imposing restrictions on the participating physician or influencing their normal clinical practice.
Read the detailed description
TTP is a rare life-threatening haematological disease characterised by thrombotic microangiopathy (TMA) with an average annual prevalence of approximately 10 cases/million people worldwide and an annual incidence between 1.5 and 6.0 cases per million according to different studies conducted in France, the United States and in the United Kingdom. In Spain the incidence is 2,67 cases / million population per year.
Acute TTP episodes cause sequelae like vascular disease or kidney damage along with other symptoms more subtle like small neurocognitive deficits and myocardial infarction.
Thus, prompt resolution of acute episodes along with a better understanding of the cardiac abnormalities may allow to prevent further complications, to develop targeted rehabilitation techniques for TTP patients and to improve their quality of life.
This project will collect a big database capable of providing better answers to questions related with treatment efficacy, associated morbidity and mortality, and the possible neurocognitive and cardiac sequelae derived from relapses and acute episodes. Additionally, this project will be linked to obtaining biological samples for a serum and DNA library from patients with TTP.
Patients will be recruited by medical researchers specialized in haematology or by other investigators specialized in thrombotic microangiopathies disease management. This recruitment will be performed in a competitive manner. The collection period will be at least 3 years with the possibility of extending it.
REPTT aims to evaluate new scores and prognostic factors of morbidity and mortality in TTP patients.
The final aim is to establish guidelines and recommendation to improve the global management, diagnosis and treatment of patients with TTP in real-life.
- Conditions
- Purpura, Thrombocytopenic
- Being tested
- Standard clinical practice
- Keywords
- registry, Thrombotic thrombocytopenic purpura, real-world, observational
- Medical subject headings
- Purpura, Thrombocytopenic, Purpura, Thrombotic Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Standard clinical practiceOther
The study will be carried out in the context of the usual clinical practice conditions, not imposing restrictions on the participating physician or influencing their normal clinical practice.
| Study group | Role | What they receive |
|---|---|---|
| Thrombotic thrombocytopenic purpura patients | Including all patients enrolled in the study Receives: Other: Standard clinical practice |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of TTP in Spain/Portugal
Number of patients diagnosed with TTP and enrolled in the registry per year
Measured: Throughout the study period, calculated for the 3 years of expected duration.
Morbidity of TTP in Spain/Portugal
Percentage of patients diagnosed with TTP and enrolled in the registry among spanish / portuguese population per year
Measured: Throughout the study period, calculated for the 3 years of expected duration.
Mortality of TTP in Spain/Portugal
Percentage of patients diagnosed with TTP and enrolled in the registry per year who died due to the disease
Measured: Throughout the study period, calculated for the 3 years of expected duration.
Secondary outcomes (12)
Clinical response to treatment rate
Defined as sustained normalization of platelet counts above the lower limit of the established reference range and of lactate dehydrogenase (LDH) after cessation of plasma exchange. The number of patients that achieved clinical response and their frequency will be reported.
Measured: Throughout the study period, approximately 3 years per patient
TTP Clinical remission rate
Defined as a clinical response after cessation of plasma exchange, maintained for > 30 days. The number of patients that achieved clinical remission and their frequency will be reported
Measured: Throughout the study period, approximately 3 years per patient
TTP Exacerbation rate
Defined as a reduction in platelet count to below the lower limit of the established reference range, an increased LDH level, and the need to restart plasma exchange within 30 days of the last plasma exchange after a clinical response to plasma exchange. The number of patients with exacerbations and their frequency will be reported
Measured: Throughout the study period, approximately 3 years per patient
TTP Relapse rate
Defined as a fall in platelet count to below the lower limit of the established reference range, with or without clinical symptoms, > 30 days after stopping of plasma exchange for an acute TTP episode, requiring reinitiation of therapy. This is usually associated with a new increase in the LDH level. The number of patients with relapse and their frequency will be reported.
Measured: Throughout the study period, approximately 3 years per patient
Refractory TTP rate
defined as persistent thrombocytopenia, lack of a sustained platelet count increment or low platelet counts and a persistently raised LDH level despite five plasma exchanges 44 and steroid treatment. The number of patients with refractory TTP and their frequency will be reported.
Measured: Throughout the study period, approximately 3 years per patient
Time-to-response (TTR)
Defined as the time from the date of first administration of treatment until the date of clinical response. Patients who die, are lost to follow-up, or reach the time point of analysis without a known record of response will have the TTR censored at the date of death, last assessment or last contact of a follow-up, whichever occurs last. Patients who received a new treatment for TTP whatever the type of treatment before disease response will be censored at the start date of this new treatment. The cumulative incidence of TTR will be estimated by the method of Kaplan-Meier.
Measured: Throughout the study period, approximately 3 years per patient
Duration of response (DoR)
DoR will be calculated among those patients that achieve a clinical response from the time that measurement criteria are first met until the date of exacerbation, relapse, appearance of refractory TTP or death by any cause. Patients who are lost to follow-up, or reach the time point of analysis without a known record of TTP recurrence or death will have the DoR censored at the date of last assessment or last contact of a follow-up, whichever occurs last. Patients who received a new treatment for TTP, whatever the type of treatment, will be censored at the start date of this new treatment.
Measured: Throughout the study period, approximately 3 years per patient
Relapse-free survival (RFS)
RFS will be calculated from the date of first administration of treatment until the date of TTP relapse or the date of death due to any cause. Patients who are lost to follow-up, or reach the time point of analysis without a known record of relapse or death will have the RFS censored at the date of last assessment or last contact, whichever occurs last. Patients who received a new treatment for TTP, whatever the type of treatment, before disease relapse or death will be censored at the start date of this new treatment. The cumulative incidence of relapse will be estimated by the method of Kaplan-Meier
Measured: Throughout the study period, approximately 3 years per patient
Overall survival (OS)
OS will be calculated from the date of the first episode recorded until the date of death due to any cause. Patients who are lost to follow-up or reach the time point of analysis without a known record of death will have the OS censored at the date of last contact. The cumulative incidence of OS will be estimated by the method of Kaplan-Meier.
Measured: Throughout the study period, approximately 3 years per patient
Frequency of serious adverse events (SAEs)
Percentage of patients who experience SAEs during the study
Measured: Throughout the study period, approximately 3 years per patient
Rate of complications associated with plasma exchange treatment
Percentage of patients who experience complications associated with plasma exchange
Measured: Throughout the study period, approximately 3 years per patient
Frequency of complications associated with plasma exchange treatment
Number of complications associated with plasma exchange per TTP event
Measured: Throughout the study period, approximately 3 years per patient
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2023
- Study start22 Mar 2024
- Primary completionSep 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Purpura, ThrombocytopenicStudies of the same condition, open and under way.
- 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
- 300 participants (estimated)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- Fundación Española de Hematología y Hemoterapía
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dr. María Eva Mingot Castellano, M.D. Ph.D., Study chair, Hospital Universitario Virgen del Rocío. Sevilla, España.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05914441
- Sponsor's protocol ID
- RE-PTT
- Acronym
- RE-PTT
- Status last verified
- May 2024
- First submitted
- 13 Jun 2023
- Last updated
- 29 May 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development Of Spanish-Portuguese Thrombotic Thrombocytopenic Purpura Registry (REPTT): A Study Proposal Of The Spanish Society Of Hematology And Hemotherapy (SEHH) Whit The Portuguese Society Of Hematology (SPH)
- ClinicalTrials.gov ID
- NCT05914441
- Acronym / sponsor ID
- RE-PTT / RE-PTT
- Conditions
- Purpura, Thrombocytopenic
- Keywords
- registry, Thrombotic thrombocytopenic purpura, real-world, observational
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Fundación Española de Hematología y HemoterapíaUniversity, hospital or other
- Study start
- 22 Mar 2024 (actual)
- Primary completion
- Sep 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 22 Jun 2023 (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.
REPTT is an observational, prospective, multi-country, multicentre and non-interventional registry in which at least 300 patients with Thrombotic thrombocytopenic purpura (TTP) in Spain and Portugal will be evaluated.
The study will be carried out in the context of the usual clinical practice conditions, not imposing restrictions on the participating physician or influencing their normal clinical practice.
Detailed description
TTP is a rare life-threatening haematological disease characterised by thrombotic microangiopathy (TMA) with an average annual prevalence of approximately 10 cases/million people worldwide and an annual incidence between 1.5 and 6.0 cases per million according to different studies conducted in France, the United States and in the United Kingdom. In Spain the incidence is 2,67 cases / million population per year.
Acute TTP episodes cause sequelae like vascular disease or kidney damage along with other symptoms more subtle like small neurocognitive deficits and myocardial infarction.
Thus, prompt resolution of acute episodes along with a better understanding of the cardiac abnormalities may allow to prevent further complications, to develop targeted rehabilitation techniques for TTP patients and to improve their quality of life.
This project will collect a big database capable of providing better answers to questions related with treatment efficacy, associated morbidity and mortality, and the possible neurocognitive and cardiac sequelae derived from relapses and acute episodes. Additionally, this project will be linked to obtaining biological samples for a serum and DNA library from patients with TTP.
Patients will be recruited by medical researchers specialized in haematology or by other investigators specialized in thrombotic microangiopathies disease management. This recruitment will be performed in a competitive manner. The collection period will be at least 3 years with the possibility of extending it.
REPTT aims to evaluate new scores and prognostic factors of morbidity and mortality in TTP patients.
The final aim is to establish guidelines and recommendation to improve the global management, diagnosis and treatment of patients with TTP in real-life.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thrombotic thrombocytopenic purpura patients | – | Other: Standard clinical practiceIncluding all patients enrolled in the study |
Who may qualify
- Ages
- Up to 99 years
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Patients diagnosed with Thrombotic thrombocytopenic purpura (TTP)
Key inclusion criteria (4 listed)
- Patients with diagnosis of TTP according to International Consensus criteria from centres in Spain and Portugal.
- Patients that voluntarily sign the informed consent. For subjects unable to provide informed consent, a fully recognized medical authority may be used according to local laws.
- Patients between 0 to 99 years old at the time of diagnosis.
- Note: Decision was taken to treat the patient with an specific treatment prior and independently of patient inclusion in this non interventional study.
Key exclusion criteria (4 listed)
- Inability to comply with study procedures and follow-up exams.
- Patients with any type of alteration that compromises their ability to grant written informed consent.
- Patients that do not consent to participate in the study and do not sign informed consent.
- Patients that do not meet the criteria previously mentioned for TTP.
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 |
|---|---|---|
| Standard clinical practiceThe study will be carried out in the context of the usual clinical practice conditions, not imposing restrictions on the participating physician or influencing their normal clinical practice. | Other | Thrombotic thrombocytopenic purpura patients |
Primary outcome
Incidence of TTP in Spain/Portugal
Time frame: Throughout the study period, calculated for the 3 years of expected duration.
Number of patients diagnosed with TTP and enrolled in the registry per year
Morbidity of TTP in Spain/Portugal
Time frame: Throughout the study period, calculated for the 3 years of expected duration.
Percentage of patients diagnosed with TTP and enrolled in the registry among spanish / portuguese population per year
Mortality of TTP in Spain/Portugal
Time frame: Throughout the study period, calculated for the 3 years of expected duration.
Percentage of patients diagnosed with TTP and enrolled in the registry per year who died due to the disease
12 secondary outcomes
Clinical response to treatment rate
Time frame: Throughout the study period, approximately 3 years per patient
TTP Clinical remission rate
Time frame: Throughout the study period, approximately 3 years per patient
TTP Exacerbation rate
Time frame: Throughout the study period, approximately 3 years per patient
TTP Relapse rate
Time frame: Throughout the study period, approximately 3 years per patient
Refractory TTP rate
Time frame: Throughout the study period, approximately 3 years per patient
Time-to-response (TTR)
Time frame: Throughout the study period, approximately 3 years per patient
Duration of response (DoR)
Time frame: Throughout the study period, approximately 3 years per patient
Relapse-free survival (RFS)
Time frame: Throughout the study period, approximately 3 years per patient
Overall survival (OS)
Time frame: Throughout the study period, approximately 3 years per patient
Frequency of serious adverse events (SAEs)
Time frame: Throughout the study period, approximately 3 years per patient
Rate of complications associated with plasma exchange treatment
Time frame: Throughout the study period, approximately 3 years per patient
Frequency of complications associated with plasma exchange treatment
Time frame: Throughout the study period, approximately 3 years per patient
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 29 May 2024.
- Individual participant dataSharing planned: Undecided
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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.
Dr. María Eva Mingot Castellano, M.D. Ph.D. study chair
Hospital Universitario Virgen del Rocío. Sevilla, España.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Purpura, Thrombocytopenic, run by Fundación Española de Hematología y Hemoterapía at 7 sites 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 4 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 29 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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?
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?
Contact Contact person Designated by the Sponsor (+34934344412, investigacion@mfar.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of TTP in Spain/Portugal (time frame: Throughout the study period, calculated for the 3 years of expected duration.). The study lists 3 primary outcomes in all. 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 29 May 2024.