A Phase 2b study of TAK-755 (rADAMTS13) with minimal to no PEX in the treatment of patients with iTT
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StatusAccepting Candidates
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Age18 Years - N/A
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SexesAll
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网红黑料y VolunteersNo
Objective
This is a study of TAK-755 in adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP). The main aim of this study is to determine the percentage of participants with a clinical (Part 1) or platelet (Part 2) response without plasma exchange during the study. Participants who have an acute attack of iTTP will receive TAK-755 and immunosuppressive therapy during their stay at the hospital until they achieve a clinical response in Part 1 or platelet response in Part 2. Participants will also be treated with TAK-755 for an additional time of up to 6 weeks after the acute phase. In total, participants will stay in the study for approximately 3 months.
Description
This study consists of 2-parts. Part 1 is a double-blind, randomized study in which participants were randomized 1:1, in a blinded fashion, into 2 TAK-755 dose groups. Part 1, randomization was stratified based on whether the participant had received pre-study PEX and on the participant's Glasgow Coma Scale. Part 2 is an open-label study in which participants with iTTP experiencing an acute iTTP episode will be enrolled and assigned to a single-arm treatment.
Details
Full study title | A Phase 2b, multicenter, randomized, double-blind study of safety and efficacy of TAK-755 (rADAMTS13) with minimal to no plasma exchange (PEX) in the treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP) |
Protocol number | OCR43938 |
ClinicalTrials.gov ID | NCT05714969 |
Phase | Phase 2 |
Eligibility
Key Inclusion Criteria (Part 1 and Part 2)
Participant must provide a signed informed consent form. A fully recognized proxy may be used per local laws for participants unable to provide consent.
Participant is 18 years or older at time of screening.
Participant has been diagnosed with de novo or relapsed iTTP.
Participant must be willing to fully comply with study procedures and requirements.
Female participants of childbearing potential must present with a negative pregnancy test and agree to employ highly effective birth control measures for duration of study. Sexually active male participants must agree to use an effective method of contraception for the duration of the study.
Key Exclusion Criteria (Part 1 and Part 2)
Participant has received more than 2 pre-study PEX prior to randomization in Part 1 or first dose of investigational product in Part 2.
Participant has been diagnosed with cTTP or another cause of thrombotic microangiopathy (TMA).
Participant has been exposed to another investigational product within 30 days prior to enrollment or is scheduled to participate in another clinical study involving investigational product or investigational device during the course of the study.
Participant has received caplacizumab within 30 days prior to study enrollment.
Participant has had a previous iTTP event within the past 30 days.
Participant is positive for human immunodeficiency virus (HIV) with unstable disease or cluster of differentiation (CD)4+ count 鈮200 cells/mm^3 within 3 months of screening.
Participant has condition of severe immunodeficiency.
Participant has a severe systemic acute infection.
Participant has another underlying progressive fatal disease and/or life expectancy
Lead researcher
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Marc S Zumberg, MDCancer Specialist (Oncologist), Hematologist (Blood Disease Specialist)
Participate in a study
Here are some general steps to consider when participating in a research study:
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Step1
Contact the research team
Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.
Primary contact
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Step2
Get screened to confirm eligibility
You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.
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Step3
Provide your consent to participate
If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.
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Step4
Participate
If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.