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

Takeda

Status and phase: Active, not recruiting; Phase 2

Conditions: Thrombotic Thrombocytopenic Purpura (TTP)

Treatments: Biological: TAK-755

Study type: Interventional

Funder types: Industry

Identifiers

NCT05714969

2022-001940-36 (EudraCT Number)

2023-507787-39-00 (EU Trial (CTIS) Number)

TAK-755-2001

Details and patient eligibility

About

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 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. 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.

Enrollment: 40 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria

1. Participant must provide a signed informed consent form. A fully recognized proxy may be used per local laws for participants unable to provide consent.
2. Participant is 18 years or older at time of screening.
3. Participant has been diagnosed with de novo or relapsed iTTP.
4. Participant must be willing to fully comply with study procedures and requirements.
5. 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

1. Participant has received more than 2 pre-study PEX prior to randomization.
2. Participant has been diagnosed with cTTP or another cause of thrombotic microangiopathy (TMA).
3. 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.
4. Participant has received caplacizumab within 30 days prior to study enrollment.
5. Participant has had a previous iTTP event within the past 30 days.
6. 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.
7. Participant has condition of severe immunodeficiency.
8. Participant has a severe systemic acute infection.
9. Participant has another underlying progressive fatal disease and/or life expectancy <3 months.
10. Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures.
11. Participant is pregnant or lactating.
12. Participant has any condition in which methylprednisolone or other steroid equivalent is contraindicated as per prescribing information.
13. 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 design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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40 participants in 2 patient groups

TAK-755 Dose 1 in Acute Phase and Dose 2 in Post-acute Phase

Experimental group

Description:

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.

Treatment:

Biological: TAK-755

TAK-755 Dose 2 in Both Acute and Post-Acute Phase

Experimental group

Description:

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.

Treatment:

Biological: TAK-755