What side effects are associated with this type of intervention?

Common treatments for Thrombotic Thrombocytopenic Purpura (TTP) include plasma exchange (PEX) and immunosuppressive therapies. PEX can cause side effects such as allergic reactions, infections, and blood clotting issues. Immunosuppressive therapies, like corticosteroids and rituximab, may lead to increased susceptibility to infections, hypertension, and gastrointestinal disturbances. For recombinant ADAMTS13 enzyme replacement therapy, such as TAK-755, potential side effects could include infusion-related reactions, headache, and nausea. It is important to discuss these potential side effects with a healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Thrombotic Thrombocytopenic Purpura (TTP), including plasma exchange (PEX) and immunosuppressive therapies such as rituximab. Caplacizumab, an anti-von Willebrand factor (vWF) nanobody, has also been approved for the treatment of acquired TTP. These treatments aim to reduce the formation of microthrombi by targeting different aspects of the disease's pathophysiology. TAK-755, a recombinant ADAMTS13 enzyme replacement therapy, is being studied as a potential treatment to directly address the deficiency of ADAMTS13 enzyme in TTP patients, which is a novel approach compared to existing therapies.

What other types of research exist?

Promising novel alternatives to TAK-755 for TTP patients include caplacizumab, an anti-von Willebrand factor (vWF) nanobody that inhibits the interaction between vWF and platelets, and rituximab, an anti-CD20 monoclonal antibody used to reduce the production of autoantibodies against ADAMTS13. Both therapies have shown efficacy in clinical trials and are considered effective in managing TTP.

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Virus Therapy

TAK-755 for Thrombotic Thrombocytopenic Purpura

Q: What is the mechanism of action of this treatment?

Thrombotic Thrombocytopenic Purpura (TTP) is commonly treated with plasma exchange (PEX) and immunosuppressive therapy. Plasma exchange removes the patient's plasma, which contains the autoantibodies against ADAMTS13, and replaces it with donor plasma, providing functional ADAMTS13 enzyme. Immunosuppressive therapy, such as corticosteroids, reduces the production of these autoantibodies. TAK-755, a recombinant ADAMTS13 enzyme replacement therapy, directly supplements the deficient enzyme, addressing the root cause of TTP. This is crucial for TTP patients as it helps to prevent the formation of microthrombi, which can lead to severe organ damage and other complications.

Phase 2

Recruiting

Research Sponsored by Takeda

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participant has been diagnosed with de novo or relapsed iTTP.

Participant is 18 years or older at time of screening.

Must not have

Participant has another underlying progressive fatal disease and/or life expectancy <3 months.

Participant has a severe systemic acute infection.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through study completion, approximately 12 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing TAK-755, a medication, in adults with a rare blood disorder called iTTP. The goal is to see if TAK-755 can help manage their symptoms without needing additional treatments. Participants will receive the medication during an acute attack and for a period afterward.

Who is the study for?

Adults diagnosed with iTTP who haven't had more than two plasma exchanges before the study can join. They must be willing to use effective birth control, follow study rules, and give consent. Those with severe allergies to TAK-755 components, recent other investigational treatments, or certain health conditions like unstable HIV cannot participate.

What is being tested?

The trial tests TAK-755 in adults with iTTP to see if they can respond clinically without needing plasma exchange. Participants will receive TAK-755 along with immunosuppressive therapy during an acute attack and for up to six weeks after, totaling about three months of study involvement.

What are the potential side effects?

Potential side effects of TAK-755 may include allergic reactions such as anaphylaxis due to its ingredients or related proteins from Chinese hamster ovary cells used in its production.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with iTTP for the first time or it has come back.

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I am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a life-threatening illness with a life expectancy of less than 3 months.

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I am currently suffering from a severe infection.

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I have been diagnosed with a condition that causes blood clots in small vessels.

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I cannot take methylprednisolone or similar steroids due to a health condition.

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I have had more than 2 plasma exchange treatments before joining the study.

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I have taken caplacizumab within the last 30 days.

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I have a severe immune system deficiency.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through study completion, approximately 12 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through study completion, approximately 12 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion, approximately 12 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs) After Receiving any Dose of Investigational Product (IP)

Secondary study objectives

Achievement of Clinical Remission

Achievement of Clinical Response Overall

Achievement of Clinical Response With Zero or Minimal on-Study PEX

+3 more

Side effects data

From 2022 Phase 1 trial • 19 Patients • NCT03997760

25%

Muscle spasms

25%

Sickle cell anaemia with crisis

25%

Abdominal pain

25%

Fibrin D dimer increased

100%

80%

60%

40%

20%

Study treatment Arm

TAK-755: 160 IU/kg

TAK-755: 40 IU/kg

TAK-755: 80 IU/kg

Placebo

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: TAK-755 Dose 2 in Both Acute and Post-Acute PhaseExperimental Treatment1 Intervention

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.

Group II: TAK-755 Dose 1 in Acute Phase and Dose 2 in Post-acute PhaseExperimental Treatment1 Intervention

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

First Studied

Drug Approval Stage

How many patients have taken this drug

TAK-755

2019

Completed Phase 1

~20

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Thrombotic Thrombocytopenic Purpura (TTP) is commonly treated with plasma exchange (PEX) and immunosuppressive therapy. Plasma exchange removes the patient's plasma, which contains the autoantibodies against ADAMTS13, and replaces it with donor plasma, providing functional ADAMTS13 enzyme. Immunosuppressive therapy, such as corticosteroids, reduces the production of these autoantibodies. TAK-755, a recombinant ADAMTS13 enzyme replacement therapy, directly supplements the deficient enzyme, addressing the root cause of TTP. This is crucial for TTP patients as it helps to prevent the formation of microthrombi, which can lead to severe organ damage and other complications.