A Retrospective, Observational Study on the Response to Caplacizumab Treatment in aTTP Patients: the Italian Experience (ROSCAPLI)

NCT05785468 | Unknown DMC

• 1 other identifier: 4305
• Study Type: observational
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

Thrombotic thrombocytopenic purpura (TTP) is a rare disease with a mortality rate of over 90% if left untreated \[1\]. TTP is a prototype of the thrombotic microangiopathies (TMAs), and it is characterized by disseminated formation of platelet-rich thrombi in arterioles and capillaries resulting in microangiopathic hemolytic anemia (MAHA), thrombocytopenia, and potential end-organ injury mainly involving the brain, heart, and kidneys leading to significant morbidity/mortality

Conditions

Thrombotic Thrombocytopenic Purpura

Outcome Measures

Primary Outcomes (2)

• description and quantification of clinical response in terms of platelet count recovery in patients with aTTP treated t with caplacizumab

  The primary objective in this study is the description of clinical response in terms of platelet count recovery in patients with aTTP treated with caplacizumab , in addition to PEX and immunosuppression in the real-world setting.

  18 months

• The primary objective in this study is the quantification of clinical response in terms of platelet count recovery in patients with aTTP treated t with caplacizumab

  The primary objective in this study is the quantification of clinical response in terms of platelet count recovery in patients with aTTP treated with caplacizumab , in addition to PEX and immunosuppression in the real-world setting.

  18 months

Secondary Outcomes (5)

• evaluation of number of exacerbations,rate of relapse and TTP-related mortality

  18 months

• evaluation of number of exacerbations,rate of relapse and TTP-related mortality

  18 months

• evaluation of number of exacerbations,rate of relapse and TTP-related mortality

  18 months

• evaluation of number of exacerbations,rate of relapse and TTP-related mortality

  18 months

• evaluation of number of exacerbations,rate of relapse and TTP-related mortality

  18 months

Interventions

Caplacizumab DRUG

description and quantification of clinical response in terms of platelet count recovery in patients with aTTP treated t with caplacizumab

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

This study is a multicenter, retrospective, observational study in patients with aTTP treated with PEX in association with caplacizumab, and immunosuppression between Q4-2019 and 28 February2021.The incidence of aTTP has been estimated at 3-11 cases per million people per year \[1-3\]. Considering that the adult population of Italy is about 45 million, the number of aTTP cases in Italy is approximately 300 per year. Assuming that 15% of the patients are treated at about 20 sites participating in the study and are eligible to participate, approximately 50 patients can be expected to be included in an 18-month period.The clinical signs will be parametrized by the PLASMIC score and the Glasgow score, where calculated in the various centers. The PLASMIC score and the Glasgow score will be evaluated according to the obtained values (0-4, 5-6, and 7; \<13 and 13-15, respectively). All the clinical and laboratory parameters will be taken in each center and reported in the CRF.

You may qualify if:

• The patients included in this study should have received caplacizumab for treatment in the period between Q4-2019 and the end of February 2021 while the end of follow up observation is scheduled for Q1-2021 (to observe at least one month of post treatment follow up).
• The diagnosis should be based on either clinical/laboratory parameters inclusive of measurement of ADAMTS13 level \<10%) or the PLASMIC score (platelets, lysis, active cancer, stem cell or solid organ transplant, MCV, INR, and creatinine) with intermediate and high risk (sore\>5) already computed or retrospectively calculated as previously detailed \[19\] for centers that did not measure the ADAMTS13 level.

Sponsors & Collaborators

• Fondazione Policlinico Universitario Agostino Gemelli IRCCS: lead

Study Sites (1)

FPG

Roma, 00168, Italy

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Interventions

caplacizumab

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Central Study Contacts

Raimondo De Cristofaro, MD

CONTACT

0630156329; raimondo.decristofaro@policlinicogemelli.it

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof. Raimondo De Cristofaro

Study Record Dates

• First Submitted: February 27, 2023
• First Posted: March 27, 2023
• Study Start: October 21, 2021
• Primary Completion: March 30, 2023
• Study Completion: March 30, 2023
• Last Updated: March 27, 2023

Record last verified: 2023-03

Locations

IT (1)