NCT05262881: ROSCAPLI

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

Status unknown Last updated 2 March 2022

What this trial tests

trial in Thrombotic Thrombocytopenic Purpura, Acquired in 50 participants. Status unknown.

Timeline

1 March 2022

Primary endpoint
30 September 2022

30 October 2022

Quick facts

Lead sponsor	Catholic University of the Sacred Heart
Status	Status unknown
Study type	OBSERVATIONAL
Enrollment	50
Start date	1 March 2022
Primary completion	30 September 2022
Estimated completion	30 October 2022
Sites	1 location across Italy

Conditions studied

• Thrombotic Thrombocytopenic Purpura, Acquired — all drugs for Thrombotic Thrombocytopenic Purpura, Acquired →

Sponsor

Catholic University of the Sacred Heart

Who can join

18 and older, any sex, with Thrombotic Thrombocytopenic Purpura, Acquired. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study is a multicenter, retrospective, observational study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) treated with plasma exchange (PEX) in association with caplacizumab, and immunosuppression between Q4-2019 and 28 February2021. The retrospective study will measure: Age, sex, BMI, blood pressure at diagnosis, Platelet count at diagnosis and at the follow up visits, Hb level at diagnosis at the follow up visits, White blood cell count at diagnosis at the follow up visits, Creatinine at diagnosis at the follow up visits, schistocytes count at diagnosis at the follow up visits, LDH at diagnosis at the follow up visits, Coombs' assay at diagnosis, alanine-leucine-amino-transferase (ALT) at diagnosis at the follow up visits, total bilirubin at diagnosis at the follow up visits, Troponin above ULN at any point, ADAMTS13 activity (where measured) at diagnosis at the follow up visits, Anti-ADAMTS13 antibodies (where measured) at diagnosis at the follow up visits.

The primary objective in this study is the description and quantification of clinical response in terms of platelet count recovery in patients with aTTP treated t with caplacizumab , in addition to PEX and immunosuppression in the real-world setting. The secondary objectives include: number of exacerbations, defined as recurrent thrombocytopenia within 30 days after the end of therapy; rate of relapse, defined as a TTP event occurring more than 30 days after the end of daily plasma exchange; refractoriness; defined by the lack of a doubling of platelet count after 4 days of treatment and a lactate dehydrogenase level that remained above the upper limit of the normal range, TTP-related mortality and evaluation of adverse events.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Nanobodies: Robust miniprotein binders in biomedicine.
   Yong Joon Kim J, Sang Z, Xiang Y, Shen Z, et al · · 2023 · cited 46× · PMID 36754285 · DOI 10.1016/j.addr.2023.114726
2. Real-World Data on Effectiveness and Safety of First-Line Use of Caplacizumab in Italian Centers for the Treatment of Thrombotic Thrombocytopenic Purpura: The Roscapli Study.
   Fianchi L, Bonanni M, Borchiellini A, Valeri F, et al · · 2024 · PMID 39518700 · DOI 10.3390/jcm13216561