Response Rate and Duration After Discontinuation Thrombopoietin Receptor Agonists Primary Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

Official title:

Response Rate and Response Duration After Discontinuation of Treatment With Thrombopoietin Receptor Agonists in Patients Affected by Primary Immune Thrombocytopenia (pITP): Retrospective Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02298075
• Other study ID #: ITP0714
• Status: Recruiting
• Start date: April 2016
• Est. completion date: December 2018

Study information

• Verified date: October 2018
• Source: Gruppo Italiano Malattie EMatologiche dell'Adulto
• Contact: Enrico Crea
• Email: e.crea[@]gimema.it
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The primary objective of the study is the evaluation of sustained response rate after discontinuation of treatment with TPO-RAs, Eltrombopag and Romiplostim, in persistent or chronic pITP patients who failed one or more therapy lines, splenectomy included.

Description:

Increasing evidence exists in the literature on the persistent response after TPO-RAs discontinuation. However, the available data consist of description of case reports from different patients' series. At this time, systematic data collections on this topic do not exist and the real incidence of such persistent response is unknown. Therefore, because of the increasing interest in this new and challenging therapeutic field, also for the therapeutic implications, we would like to investigate the impact of TPO- RAs on the achievement of sustained and persistent responses after their discontinuation in the Italian adult patients affected by pITP who have been referred to the GIMEMA Centers.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: December 2018
• Est. primary completion date: December 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Signed written informed consent according to ICH/EU/GCP and national local laws;

• Persistent or chronic pITP adult patients who have been treated with TPO-RAs therapy after failure of one or more therapy lines, splenectomy included;

• Age > 18 years.

Exclusion Criteria:

• Active malignancy;

• Active malignancy;

• HCVAb, HIVAb, HBsAg, HBcAb seropositive status;

• Chronic liver disease;

• Treatment with Rituximab less than 8 weeks before TPO-RAs start;

• Recent splenectomy (less than 8 weeks) before TPO-RAs start. Active malignancy;

• HCVAb, HIVAb, HBsAg, HBcAb seropositive status;

• Chronic liver disease;

• Treatment with Rituximab less than 8 weeks before TPO-RAs start;

• Recent splenectomy (less than 8 weeks) before TPO-RAs start. HCVAb, HIVAb, HBsAg, HBcAb seropositive status;

• Chronic liver disease;

• Treatment with Rituximab less than 8 weeks before TPO-RAs start;

• Recent splenectomy (less than 8 weeks) before TPO-RAs start.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Eltrombopag
Investigation of the impact of TPO-RAs on the achievement of sustained and persistent response after discontinuation in adult patients affected by pITP.
• Romiplostin
Investigation of the impact of TPO-RAs on the achievement of sustained and persistent response after discontinuation in adult patients affected by pITP.

Locations

Country	Name	City	State
Italy	S.O.C. di Medicina Interna B - Ospedale - Cardinal Massaia di Asti	Asti
Italy	Istituto di Ematologia "Lorenzo e A. Seragnoli"	Bologna
Italy	Unità di Onco-Ematologia - Azienda Ospedaliera - Garibaldi	Catania
Italy	Unità di Ricerca e di Malattie del sangue - Ematologia San Luca Vecchio Pad. 16 - 1° Piano	Firenze
Italy	Clinica Medica III - Padiglione ex isolamento - Azienda Ospedale Università San Martino	Genova
Italy	ASL Le/1 P.O. Vito Fazzi - U.O. di Ematologia ed UTIE	Lecce
Italy	Azienda Ospedaliera "S.Gerardo"	Monza
Italy	Ematologia - Ospedale San Carlo	Potenza
Italy	Unità Operativa Complessa di Ematologia - Arcispedale S. Maria Nuova	Reggio Emilia
Italy	Università Cattolica del Sacro Cuore - Policlinico A. Gemelli	Roma
Italy	U.O.C. Ematologia - Ospedale S. Eugenio	Rome
Italy	Università degli studi "Sapienza" di Roma	Rome
Italy	Ematologia - Dipartimento di Medicina Clinica e Sperimentale	Sassari

Sponsors (1)

Lead Sponsor	Collaborator
Gruppo Italiano Malattie EMatologiche dell'Adulto

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Estimation of sustained response rate after discontinuation of treatment with TPO-RAs in terms of patients still alive in Complete Response or R at more than 4 weeks after discontinuation.		One year from start of recruitment.
Secondary	Frequency of the type of response in patients treated with TPO-RAs in absence of concomitant or rescue medication, in order to estimate the rate of Response (R) and Complete Response (CR).		One year from start of recruitment.
Secondary	Estimation of the suspension criteria in terms of frequency of discontinuation causes for each center.		One year from start of recruitment.
Secondary	Estimation of the duration of sustained response in terms of patients alive in CR or R at 6 and 12 months after sustained response achievement.		One year from start of recruitment.
Secondary	Estimation of incidence of relapse and loss of response at 6 and 12 months after sustained response achievement.		One year from start of recruitment.
Secondary	Estimation of treatment duration starting from the date of first dose of TPO-RAs.		One year from start of recruitment.
Secondary	Description of Platelet levels and TPO-RAs dosage at stop.		One year from start of recruitment.
Secondary	Description of patients' characteristics in term of: biological and clinical features, disease duration at the beginning of TPO-RAs, number and type of therapy lines preceding TPO-RAs treatment, platelet levels at TPO-RAs start.		One year from start of recruitment.
Secondary	Assessment of an association between previous splenectomy and sustained response after discontinuation of treatment with TPO-RAs.		One year from start of recruitment.
Secondary	Description of concomitant therapies since TPO-RAs start in terms of type and duration of therapies.		One year from start of recruitment.

External Links

•   GIMEMA Foundation website