A Phase 2b study of TAK-755 (rADAMTS13) with minimal to no PEX in the treatment of patients with iTTP
- Conditions
- immune-mediated thrombotic thrombocytopenic purpura (iTTP)MedDRA version: 20.0Level: SOCClassification code 10005329Term: Blood and lymphatic system disordersSystem Organ Class: 10005329 - Blood and lymphatic system disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2022-001940-36-GR
- Lead Sponsor
- Takeda Development Center Americas, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 40
1. Subject must provide a signed informed consent form. A fully recognized proxy may be used per local laws for subjects unable to provide consent.
2. Subject is 18 years or older at time of screening.
3. Subject has been diagnosed with de novo or relapsed iTTP based on the following criteria:
a. Thrombocytopenia [platelet count <100,000/µL].
b. Microangiopathic hemolytic anemia [elevation of LDH >2×ULN or by the presence or an increase of schistocytes in peripheral blood smear].
4. Subject must be willing to fully comply with study procedures and requirements.
5. Female subjects 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 subjects must agree to use an effective method of contraception for the duration of the study.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 35
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5
1. Subject has received more than 2 pre-study PEX prior to randomization.
2. Subject has been diagnosed with cTTP or another cause of TMA, including: disseminated intravascular coagulation (DIC), disseminated malignancy, malignant hypertension, solid organ and hematopoietic stem cell transplantation, shiga toxin–related and atypical hemolytic uremic syndrome, drug toxicity (eg, gemcitabine, mitomycin C, clopidogrel) and pregnancy-related thrombocytopenia syndromes (eg, hemolysis, elevated liver enzymes and low platelets [HELLP]; eclampsia), serum creatinine >2.25 mg/dL, active cancer within the last year.
3. Subject 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.
4. Subject has received caplacizumab within 30 days prior to study enrollment.
5. Subject has had a previous iTTP event within the past 30 days.
6. Subject is positive for human immunodeficiency virus (HIV) with unstable disease or CD4+ count =200 cells/mm3 within 3 months of screening.
7. Subject has condition of severe immunodeficiency.
8. Subject has a severe systemic acute infection.
9. Subject has another underlying progressive fatal disease and/or life expectancy <3 months.
10. Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
11. Subject suffers from a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude. However, a fully recognized medical proxy will be permitted to provide consent, under the circumstances that the subject is unable to provide consent due to the effects of iTTP.
12. Subject is pregnant or lactating.
13. Subject is a family member or employee of the sponsor or investigator.
14. Subject has any condition in which methylprednisolone or other streroide equivalent is contraindicated as per prescribing information.
15.Subject has known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule ADAMTS13, CHO cell proteins, or other constituents of TAK-755.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Assess the safety of TAK-755 in the treatment of iTTP;Secondary Objective: 1. Assess the efficacy of TAK-755 in achieving clinical response with and without on-study PEX following an acute TTP event.<br>2. Assess treatment failures.<br>3. Assess the efficacy of TAK-755 in prevention of iTTP recurrence, exacerbation, and relapse.<br>4. Assess changes in organ damage biomarkers.<br>5. Evaluate the durability of clinical response.<br>6. Evaluate the ADAMTS13 antigen and activity over time.<br>7. Evaluate VWF antigen and activity over time.<br>;Primary end point(s): Incidence of AEs, SAEs, and AESIs after receiving any dose of IP;Timepoint(s) of evaluation of this end point: Variable time point
- Secondary Outcome Measures
Name Time Method