AzTrials
AzTrials

Thrombotic Thrombocytopenic Purpura (TTP)Phase 2NCT05714969

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

A Phase 2 study testing TAK-755 in people with Thrombotic Thrombocytopenic Purpura (TTP), run by Takeda at 22 sites in 6 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
TAK-755
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
29 participants
Where
22 sitesin 6 countries
Study dates
21 Jul 2023to 16 Apr 2026
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 13Show fewer
  • 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.

Trial locations and contacts

22 sites in 6 countries.

22 sites in 6 countries

United States12 sites

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

  • Ohio State University

    Columbus, Ohio, 43210

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • University of Florida - Shands

    Gainesville, Florida, 32610

  • Leo Jenkins Cancer Center/ECU School of Medicine

    Greenville, North Carolina, 27858

  • Versiti Wisconsin, Inc.

    Milwaukee, Wisconsin, 53226

  • University of Minnesota Clinical Research Unit

    Minneapolis, Minnesota, 55455

  • Rutgers University

    New Brunswick, New Jersey, 08901

  • Weill Cornell

    New York, New York, 10021

  • University of Utah Health Sciences Center

    Salt Lake City, Utah, 84132

Other trials in United States

Spain4 sites

  • Hospital de Cruces

    Barakaldo, Vizcaya, 48903

  • Hospital General Universitario Gregorio Maranon

    Madrid, 28007

  • Hospital Universitario Virgen del Rocio

    Seville, Sevilla, 41010

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026

Other trials in Spain

Italy2 sites

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milan, 20122

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

    Torino, 10126

Other trials in Italy

United Kingdom2 sites

  • Royal Liverpool University Hospital

    Liverpool, Merseyside, L7 8XP

  • University College London Hospital

    London, Greater London, NW1 2PG

Other trials in United Kingdom

Austria1 site

  • AKH - Medizinische Universitat Wien

    Vienna, 1090

Other trials in Austria

Greece1 site

  • General Hospital of Thessaloniki "G. Papanikolaou"

    Thessaloniki, 57010

Other trials in Greece

About this study

Official 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)

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.

Read the detailed 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.

Being tested
TAK-755
Keywords
Drug Therapy, Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
Medical subject headings
Purpura, Thrombotic Thrombocytopenic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TAK-755Biological

    Also called rADAMTS13, recombinant ADAMTS13, SHP-655, BAX 930

    TAK-755 IV infusion

Study groups in NCT05714969
Study groupRoleWhat they receive
Part 1: TAK-755 Dose 1 in Acute Phase and Dose 2 in Post-acute PhaseExperimentalTAK-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.
Receives: Biological: TAK-755
Part 1: TAK-755 Dose 2 in Both Acute and Post-Acute PhaseExperimentalTAK-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.
Receives: Biological: TAK-755
Part 2: TAK-755 Dose 3 in Acute Phase and Dose 2 in Post-acute PhaseExperimentalTAK-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.
Receives: Biological: TAK-755

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Through study completion, approximately 12 weeks

Secondary outcomes (35)

  1. 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.

    Measured: Through study completion, approximately 12 weeks

  2. 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.

    Measured: Through study completion, approximately 12 weeks

  3. 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.

    Measured: Through study completion, approximately 12 weeks

  4. 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.

    Measured: Through study completion, approximately 12 weeks

  5. 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.

    Measured: Through study completion, approximately 12 weeks

  6. 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.

    Measured: Through study completion, approximately 12 weeks

  7. Part 1: Time to Clinical Response (Acute Phase)

    Measured: Through study completion, approximately 12 weeks

  8. Part 2: Time to Platelet Response (Acute Phase)

    Measured: Through study completion, approximately 12 weeks

  9. Part 1: Occurrence of Refractoriness (Acute Phase)

    Measured: Through study completion, approximately 12 weeks

  10. Part 1: Time to First On-Study PEX in Participants who Achieved Clinical Response

    Measured: Through study completion, approximately 12 weeks

  11. Part 2: Time to First On-Study PEX in Participants who Achieved Platelet Response

    Measured: Through study completion, approximately 12 weeks

  12. Part 1: Number of Days of On-study PEX in Participants to Achieve Clinical Response (Acute Phase)

    Measured: Through study completion, approximately 12 weeks

  13. Part 2: Number of Days of On-study PEX in Participants to Achieve Platelet Response (Acute Phase)

    Measured: Through study completion, approximately 12 weeks

  14. Part 1: Total Volume of Plasma Administered (Acute Phase) to Achieve Clinical Response

    Measured: Through study completion, approximately 12 weeks

  15. Part 2: Total Volume of Plasma Administered (Acute Phase) to Achieve Platelet Response

    Measured: Through study completion, approximately 12 weeks

  16. Part 1: Occurrence of Treatment Failure

    Treatment failure is defined as failure to achieve clinical response, or experience iTTP recurrence.

    Measured: Through study completion, approximately 12 weeks

  17. Part 2: Occurrence of Treatment Failure

    Treatment failure is defined as failure to achieve platelet response, or experience iTTP recurrence.

    Measured: Through study completion, approximately 12 weeks

  18. 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.

    Measured: Through study completion, approximately 12 weeks

  19. 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.

    Measured: Through study completion, approximately 12 weeks

  20. Part 1: Time to iTTP Recurrence (Following Clinical Response), Exacerbation, or Relapse

    Measured: Through study completion, approximately 12 weeks

  21. Part 2: Time to iTTP Recurrence (Following Platelet Response), Exacerbation, or Relapse

    Measured: Through study completion, approximately 12 weeks

  22. 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

    Measured: Through study completion, approximately 12 weeks

  23. 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

    Measured: Through study completion, approximately 12 weeks

  24. 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

    Measured: Through study completion, approximately 12 weeks

  25. 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

    Measured: Through study completion, approximately 12 weeks

  26. Part 1: Change From Baseline in Lactate Dehydrogenase [LDH] Levels at Clinical Response and Study Completion

    Measured: Through study completion, approximately 12 weeks

  27. Part 2: Change From Baseline in LDH Levels at Platelet Response and Study Completion

    Measured: Through study completion, approximately 12 weeks

  28. Part 1: Change From Baseline in Troponin Levels at Clinical Response and Study Completion

    Measured: Through study completion, approximately 12 weeks

  29. Part 2: Change From Baseline in Troponin Levels at Platelet Response and Study Completion

    Measured: Through study completion, approximately 12 weeks

  30. Part 1: Achievement of Clinical Remission

    Clinical remission is defined as achieving clinical response and no recurrence for >=30 days.

    Measured: Through study completion, approximately 12 weeks

  31. Part 2: Achievement of Clinical Remission

    Clinical remission is defined as achieving platelet response and no recurrence for >=30 days.

    Measured: Through study completion, approximately 12 weeks

  32. 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

    Measured: Through study completion, approximately 12 weeks

  33. Part 1 and 2: ADAMTS13 Activity Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases

    Measured: Through study completion, approximately 12 weeks

  34. Part 1: Von Willebrand Factor (VWF) Antigen Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases

    Measured: Through study completion, approximately 12 weeks

  35. Part 1: VWF Activity Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases

    Measured: Through study completion, approximately 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Feb 2023
  2. Study start21 Jul 2023
  3. Primary completion16 Apr 2026
  4. Study completion16 Apr 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
29 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05714969
Sponsor's protocol ID
TAK-755-2001
Other IDs
2023-507787-39-00, 2023
Status last verified
Jun 2026
First submitted
24 Jan 2023
Last updated
17 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing TAK-755 in people with Thrombotic Thrombocytopenic Purpura (TTP), run by Takeda at 22 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in United States, Spain, Italy, United Kingdom, Austria and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs) After Receiving any Dose of Investigational Product (IP) (time frame: Through study completion, approximately 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05714969.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jun 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in