A Study of TAK-755 (rADAMTS13) With Little to No Plasma Exchange (PEX) Treatment in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

NCT05714969 | Completed Phase 2 DMC FDA Drug

• 2 other identifiers: TAK-755-20012023-507787-39-00
• Study Type: interventional
• Target: 29
• Locations: 6 countries
• Sites: 22

Brief Summary

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.

Conditions

Thrombotic Thrombocytopenic Purpura (TTP)

Keywords

Drug Therapy; Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (35)

• Part 1: Achievement of Clinical Response Without On-Study Plasma Exchange (PEX)

  Through study completion, approximately 12 weeks

• Part 2: Achievement of Platelet Response Without On-Study Plasma Exchange (PEX)

  Through study completion, approximately 12 weeks

• Part 1: Achievement of Clinical Response With Zero or Minimal on-Study PEX

  Through study completion, approximately 12 weeks

• Part 2: Achievement of Platelet Response With Zero or Minimal on-Study PEX

  Through study completion, approximately 12 weeks

• Part 1: Achievement of Clinical Response Overall

  Through study completion, approximately 12 weeks

Study Arms (3)

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.

Biological: TAK-755

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.

Biological: TAK-755

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.

Biological: TAK-755

Interventions

TAK-755 BIOLOGICAL

TAK-755 IV infusion

Also known as: rADAMTS13, recombinant ADAMTS13, SHP-655, BAX 930

Part 1: TAK-755 Dose 1 in Acute Phase and Dose 2 in Post-acute Phase; Part 1: TAK-755 Dose 2 in Both Acute and Post-Acute Phase; Part 2: TAK-755 Dose 3 in Acute Phase and Dose 2 in Post-acute Phase

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 ≤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 \<3 months.
• Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures.
• Participant is pregnant or lactating.
• Participant has any condition in which methylprednisolone or other steroid equivalent is contraindicated as per prescribing information.
• Participant has known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule ADAMTS13, Chinese hamster ovary (CHO) cell proteins, or other constituents of TAK-755.

Sponsors & Collaborators

• Takeda: lead
• Takeda Development Center Americas, Inc.: collaborator

Study Sites (22)

University of Florida - Shands

Gainesville, Florida, 32610, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

University of Minnesota Clinical Research Unit

Minneapolis, Minnesota, 55455, United States

Location

Rutgers University

New Brunswick, New Jersey, 08901, United States

Location

Weill Cornell

New York, New York, 10021, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Leo Jenkins Cancer Center/ECU School of Medicine

Greenville, North Carolina, 27858, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

University of Utah Health Sciences Center

Salt Lake City, Utah, 84132, United States

Location

Versiti Wisconsin, Inc.

Milwaukee, Wisconsin, 53226, United States

Location

AKH - Medizinische Universitat Wien

Vienna, 1090, Austria

Location

General Hospital of Thessaloniki "G. Papanikolaou"

Thessaloniki, 57010, Greece

Location

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino

Torino, 10126, Italy

Location

Hospital Universitario Virgen del Rocio

Seville, Sevilla, 41010, Spain

Location

Hospital de Cruces

Barakaldo, Vizcaya, 48903, Spain

Location

Hospital General Universitario Gregorio Maranon

Madrid, 28007, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, 46026, Spain

Location

University College London Hospital

London, Greater London, NW1 2PG, United Kingdom

Location

Royal Liverpool University Hospital

Liverpool, Merseyside, L7 8XP, United Kingdom

Location

Related Links

• 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.
• 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.

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Interventions

ADAMTS13 protein, human

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Part 1 is double-blind, randomization and Part 2 is open-label, single-arm.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 24, 2023
• First Posted: February 6, 2023
• Study Start: March 21, 2023
• Primary Completion: April 16, 2026
• Study Completion: April 16, 2026
• Last Updated: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: NOTE: IPD Sharing Time Frame has not been entered.
• Access Criteria: 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

Locations

AU (1); GR (1); GB (2); IT (2); US (12); ES (4)