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

February 21, 2022 updated by: Raimondo De Cristofaro, Catholic University of the Sacred Heart

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.

Study Overview

Status

Not yet recruiting

Conditions

Detailed 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.

Study design

The retrospective study will measure the following vital, clinical chemistry, hematological, and hemostatic parameters, according to the consensus guidelines of aTTP diagnosis and as a part of routine diagnostic work-up:

  • 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 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.

OBJECTIVES 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.

Data collection and management A customized eCRF (RedCap) will be created for the study and only the principal investigator and collaborating investigators of the center will have access to patient medical records during the study.

STATISTICAL CONSIDERATIONS Qualitative variables will be expressed by absolute and percentage frequency. Quantitative variables will be previously assessed for their distribution by the Shapiro-Wilk test. Normally distributed data will be described by mean and standard deviation (SD), whilst non-normally distributed variable by median and interquartile range (IQR).

INFORMED CONSENT Informed consent form will be collected for all except for all patients whom death is ascertained

Study Type

Observational

Enrollment (Anticipated)

50

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Rome, Italy, 00168
        • Fondazione Policlinico Universitario Agostino Gemelli, IRCCS

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The study population consists of patients treated for acute aTTP episodes in the hematology, internal medicine, and critical care centers between Q4-2019 and Q1-2021 in Italy.

Description

Inclusion Criteria:

  • 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 for centers that did not measure the ADAMTS13 level.

Exclusion Criteria:

  • Patients treated with uncertain aTTP diagnosis according to the above inclusion criteria and manifesting clinical signs like aTTP but characterized by a different pathogenesis (e.g. cancer, sepsis)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets
Time Frame: 18 months
Platelet response was defined as recovery of platelets ≥ 150,000/µL.
18 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
study of critical parameters
Time Frame: 18 months
  1. Number and percentage of subjects with TTP-Related Death
  2. Number and percentage of subjects with relapse of TTP, defined as a TTP event occurring more than 30 days after the end of daily plasma exchanges
  3. assessment of all SAEs of thromboembolic events
  4. Number and percentage of subjects with exacerbations, defined as recurrent thrombocytopenia within 30 days after the end of daily plasma exchanges that required reinitiation of daily exchanges
  5. Number and percentage of subjects with refractory TTP (); and the time to normalization (i.e., to a level below the defined upper limit of the normal range) of three organ-damage markers (LDH, cardiac troponin I, and serum creatinine).
18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Catholic University of the Sacred Heart

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

March 1, 2022

Primary Completion (Anticipated)

September 30, 2022

Study Completion (Anticipated)

October 30, 2022

Study Registration Dates

First Submitted

February 21, 2022

First Submitted That Met QC Criteria

February 21, 2022

First Posted (Actual)

March 2, 2022

Study Record Updates

Last Update Posted (Actual)

March 2, 2022

Last Update Submitted That Met QC Criteria

February 21, 2022

Last Verified

February 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 4305

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Thrombotic Thrombocytopenic Purpura, Acquired

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Mali

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe