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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-507787-39-00 | EU Trial (CTIS) Number |
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| Name | Class |
|---|---|
| Takeda Development Center Americas, Inc. | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part 1: TAK-755 Dose 1 in Acute Phase and Dose 2 in Post-acute Phase | Experimental | TAK-755 Dose 1, IV infusion, in the acute phase until clinical response is achieved. All participants achieving clinical response will receive TAK-755 at Dose 2, for up to 6 weeks during the post-acute phase. |
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| Part 1: TAK-755 Dose 2 in Both Acute and Post-Acute Phase | Experimental | TAK-755 Dose 2, IV infusion, in the acute phase until clinical response is achieved. All participants achieving clinical response will receive TAK-755 at Dose 2, for up to 6 weeks during the post-acute phase. |
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| Part 2: TAK-755 Dose 3 in Acute Phase and Dose 2 in Post-acute Phase | Experimental | TAK-755 Dose 3, IV infusion, in the acute phase until 48-hour platelet response is achieved. All participants achieving platelet response will receive TAK-755 at Dose 2, 4 times per week for Week 1 and 3 times per week for Week 2 and 3 during the post-acute phase based on investigator judgement as high-risk for developing iTTP recurrence. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TAK-755 | Biological | TAK-755 IV infusion |
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs) After Receiving any Dose of Investigational Product (IP) | An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (e.g., a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. SAE: Signs, symptoms or outcomes which results in death, requires inpatient hospitalization or prolongation of hospitalization, results in persistent or significant disability/incapacity, results in a congenital abnormality/birth defect, or is an important medical event. Adverse events of special interest include major thrombotic events and treatment-related bleeding events. | Through study completion, approximately 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Achievement of Clinical Response Without On-Study Plasma Exchange (PEX) | Clinical response is defined as normalization of platelets and no clinical evidence of new or progressive ischemic organ injury. Normalization of platelets: First occurrence of normal platelet count (greater than or equal to [>=]150,000/microliter [mcL]) that is followed by a confirmatory platelet count of >=150,000/mcL and a lactate dehydrogenase (LDH) <1.5×upper limit of normal (ULN) at 48±12 hours after the first occurrence. |
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Key Inclusion Criteria (Part 1 and Part 2)
Key Exclusion Criteria (Part 1 and Part 2)
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida - Shands | Gainesville | Florida | 32610 | United States | ||
| Massachusetts General Hospital |
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| Label | URL |
|---|---|
| Click here for more information about this trial in easy-to-understand language, including a Plain Language Summary of the results if the trial has been completed. | View source |
| Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language. | View source |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
NOTE: IPD Sharing Time Frame has not been entered.
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement
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Part 1 is double-blind, randomization and Part 2 is open-label, single-arm.
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| Through study completion, approximately 12 weeks |
| Part 2: Achievement of Platelet Response Without On-Study Plasma Exchange (PEX) | Platelet response is defined as first occurrence of normal platelet count (>=150,000/mcL) that is followed by a confirmatory platelet count of >=150,000/mcL at 48±12 hours after the first occurrence. | Through study completion, approximately 12 weeks |
| Part 1: Achievement of Clinical Response With Zero or Minimal on-Study PEX | The number of PEX administered considered Zero when no PEX is administered and considered Minimal when 1 to 3 PEX are administered. | Through study completion, approximately 12 weeks |
| Part 2: Achievement of Platelet Response With Zero or Minimal on-Study PEX | The number of PEX administered considered Zero when no PEX is administered and considered Minimal when 1 to 3 PEX are administered. | Through study completion, approximately 12 weeks |
| Part 1: Achievement of Clinical Response Overall | Overall indicates clinical response regardless of whether on-study PEX is administered, or the number of PEX administered. | Through study completion, approximately 12 weeks |
| Part 2: Achievement of Platelet Response Overall | Overall indicates platelet response regardless of whether on-study PEX is administered, or the number of PEX administered. | Through study completion, approximately 12 weeks |
| Part 1: Time to Clinical Response (Acute Phase) | Through study completion, approximately 12 weeks |
| Part 2: Time to Platelet Response (Acute Phase) | Through study completion, approximately 12 weeks |
| Part 1: Occurrence of Refractoriness (Acute Phase) | Through study completion, approximately 12 weeks |
| Part 1: Time to First On-Study PEX in Participants who Achieved Clinical Response | Through study completion, approximately 12 weeks |
| Part 2: Time to First On-Study PEX in Participants who Achieved Platelet Response | Through study completion, approximately 12 weeks |
| Part 1: Number of Days of On-study PEX in Participants to Achieve Clinical Response (Acute Phase) | Through study completion, approximately 12 weeks |
| Part 2: Number of Days of On-study PEX in Participants to Achieve Platelet Response (Acute Phase) | Through study completion, approximately 12 weeks |
| Part 1: Total Volume of Plasma Administered (Acute Phase) to Achieve Clinical Response | Through study completion, approximately 12 weeks |
| Part 2: Total Volume of Plasma Administered (Acute Phase) to Achieve Platelet Response | Through study completion, approximately 12 weeks |
| Part 1: Occurrence of Treatment Failure | Treatment failure is defined as failure to achieve clinical response, or experience iTTP recurrence. | Through study completion, approximately 12 weeks |
| Part 2: Occurrence of Treatment Failure | Treatment failure is defined as failure to achieve platelet response, or experience iTTP recurrence. | Through study completion, approximately 12 weeks |
| Part 1: Occurrence of Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP) Recurrence (Following Clinical Response), Exacerbation, or Relapse (Post-acute Phase) | iTTP recurrence comprised of exacerbation or relapse. Clinical exacerbation: Occurs <30 days after achieving initial clinical response (i.e., before clinical remission) and recurrent thrombocytopenia (platelet levels <150,000/μL), with or without clinical evidence of new or progressive ischemic organ damage, requiring daily PEX or rescue therapy. Clinical relapses: Occurs >=30 days after achieving initial clinical response (i.e., after clinical remission) and recurrent thrombocytopenia (platelet levels <150,000/μL), with or without clinical evidence of new or progressive ischemic organ damage, requiring daily PEX or rescue therapy. | Through study completion, approximately 12 weeks |
| Part 2: Occurrence of iTTP Recurrence (Following Platelet Response), Exacerbation, or Relapse (Post-acute Phase) | iTTP recurrence comprised of exacerbation or relapse. Clinical exacerbation: Occurs <30 days after achieving initial platelet response (i.e., before clinical remission) and recurrent thrombocytopenia (platelet levels <150,000/μL), with or without clinical evidence of new or progressive ischemic organ damage, requiring daily PEX or rescue therapy. Clinical relapses: Occurs >=30 days after achieving initial platelet response (i.e., after clinical remission) and recurrent thrombocytopenia (platelet levels <150,000/μL), with or without clinical evidence of new or progressive ischemic organ damage, requiring daily PEX or rescue therapy. | Through study completion, approximately 12 weeks |
| Part 1: Time to iTTP Recurrence (Following Clinical Response), Exacerbation, or Relapse | Through study completion, approximately 12 weeks |
| Part 2: Time to iTTP Recurrence (Following Platelet Response), Exacerbation, or Relapse | Through study completion, approximately 12 weeks |
| Part 1: Occurrence of Any One of the Following Events: Clinical Recurrence (Following Clinical Response), iTTP-Related Death, or Major Thrombotic Event From Time of First IP Administration Through Study Completion | Through study completion, approximately 12 weeks |
| Part 2: Occurrence of Any One of the Following Events: Clinical Recurrence (Following Platelet Response), iTTP-Related Death, or Major Thrombotic Event From Time of First IP Administration Through Study Completion | Through study completion, approximately 12 weeks |
| Part 1: Time to Occurrence of Any One of the Following Events: Clinical Recurrence (Following Clinical Response), iTTP-Related Death, or Major Thrombotic Event From Time of First IP Administration Through Study Completion | Through study completion, approximately 12 weeks |
| Part 2: Time to Occurrence of Any One of the Following Events: Clinical Recurrence (Following Platelet Response), iTTP-Related Death, or Major Thrombotic Event From Time of First IP Administration Through Study Completion | Through study completion, approximately 12 weeks |
| Part 1: Change From Baseline in Lactate Dehydrogenase [LDH] Levels at Clinical Response and Study Completion | Through study completion, approximately 12 weeks |
| Part 2: Change From Baseline in LDH Levels at Platelet Response and Study Completion | Through study completion, approximately 12 weeks |
| Part 1: Change From Baseline in Troponin Levels at Clinical Response and Study Completion | Through study completion, approximately 12 weeks |
| Part 2: Change From Baseline in Troponin Levels at Platelet Response and Study Completion | Through study completion, approximately 12 weeks |
| Part 1: Achievement of Clinical Remission | Clinical remission is defined as achieving clinical response and no recurrence for >=30 days. | Through study completion, approximately 12 weeks |
| Part 2: Achievement of Clinical Remission | Clinical remission is defined as achieving platelet response and no recurrence for >=30 days. | Through study completion, approximately 12 weeks |
| Part 1 and 2: A Disintegrin and Metalloproteinase With Thrombospondin Motifs 13 (ADAMTS13) Antigen Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases | Through study completion, approximately 12 weeks |
| Part 1 and 2: ADAMTS13 Activity Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases | Through study completion, approximately 12 weeks |
| Part 1: Von Willebrand Factor (VWF) Antigen Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases | Through study completion, approximately 12 weeks |
| Part 1: VWF Activity Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases | Through study completion, approximately 12 weeks |
| Boston |
| Massachusetts |
| 02114 |
| United States |
| Brigham and Women's Hospital | Boston | Massachusetts | 02115 | United States |
| Beth Israel Deaconess Medical Center | Boston | Massachusetts | 02215 | United States |
| University of Minnesota Clinical Research Unit | Minneapolis | Minnesota | 55455 | United States |
| Rutgers University | New Brunswick | New Jersey | 08901 | United States |
| Weill Cornell | New York | New York | 10021 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Leo Jenkins Cancer Center/ECU School of Medicine | Greenville | North Carolina | 27858 | United States |
| Ohio State University | Columbus | Ohio | 43210 | United States |
| University of Utah Health Sciences Center | Salt Lake City | Utah | 84132 | United States |
| Versiti Wisconsin, Inc. | Milwaukee | Wisconsin | 53226 | United States |
| AKH - Medizinische Universitat Wien | Vienna | 1090 | Austria |
| General Hospital of Thessaloniki "G. Papanikolaou" | Thessaloniki | 57010 | Greece |
| Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino | Torino | 10126 | Italy |
| Hospital Universitario Virgen del Rocio | Seville | Sevilla | 41010 | Spain |
| Hospital de Cruces | Barakaldo | Vizcaya | 48903 | Spain |
| Hospital General Universitario Gregorio Maranon | Madrid | 28007 | Spain |
| Hospital Universitari i Politecnic La Fe | Valencia | 46026 | Spain |
| University College London Hospital | London | Greater London | NW1 2PG | United Kingdom |
| Royal Liverpool University Hospital | Liverpool | Merseyside | L7 8XP | United Kingdom |
| ID | Term |
|---|---|
| D011697 | Purpura, Thrombotic Thrombocytopenic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D019851 | Thrombophilia |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
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| ID | Term |
|---|---|
| C099604 | ADAMTS13 protein, human |
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