CompletedPhase 2/3

A Trial of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Japan21 participantsStarted 2019-10-21

Plain-language summary

Primary Objective: To evaluate the effect of caplacizumab on prevention of recurrence of aTTP (proportion of participants with a recurrence of aTTP) during the overall study period. Secondary Objectives: * To evaluate effect of caplacizumab on * prevention of recurrence of TTP (the number of recurrences of TTP) during overall study period. * a composite endpoint consisting of aTTP-related mortality, recurrence of aTTP and major thromboembolic events during study drug treatment * restoring platelet counts as a measure of prevention of further microvascular thrombosis * refractory disease * biomarkers of organ damage: LDH, cardiac troponin I, serum creatinine * plasma exchange (PE) parameters (days of PE and volume of plasma used), days in intensive care unit, days in hospital * cognitive status of Japanese patients * To evaluate safety profile of caplacizumab in Japanese patients * To evaluate effect of caplacizumab on pharmacodynamic (PD) markers in Japanese patients * To evaluate pharmacokinetic (PK) profile of caplacizumab in Japanese patients * To evaluate immunogenicity of caplacizumab in Japanese patients

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria : * Japanese participant must be 18 years or older at the time of signing the informed consent. * Participants who have a clinical diagnosis of aTTP (initial or recurrent), which includes thrombocytopenia (defined as platelet count \<100,000/µL), microangiopathic hemolytic anemia as evidenced by red blood cell fragmentation (eg, presence of schistocytes), and increased levels of LDH * Participants who require initiation of daily PE treatment and have received a maximum of 1 PE treatment prior to enrollment in the study * Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies * Capable of giving signed informed consent Exclusion criteria: * Platelet count ≥100,000/µL, * Serum creatinine level \> 2.3mg/dL in case platelet count is \> 30,000µL * Known other causes of thrombocytopenia * Congenital TTP * Clinically significant active bleeding or high risk of bleeding * Malignant arterial hypertension * Known chronic treatment with anticoagulant treatment that cannot be stopped * Participants who were previously enrolled in a clinical study with caplacizumab and received caplacizumab or for whom the assigned treatment arm is unknown * Participants currently or less than 28 days prior to enrollment in this study, enrolled in a clinical study with another investigational drug or device * Clinical condition other than that associated with TTP, with life expec…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Proportion of participants with a recurrence of acquired thrombotic thrombocytopenic purpura (aTTP)

Timeframe: Approximately 2 months up to approximately 6 months

Trial details

NCT IDNCT04074187

SponsorSanofi

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-05-19

View on ClinicalTrials.gov More Thrombotic Thrombocytopenic Purpura trials