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Enzyme Replacement Therapy

TAK-755 for Purpura

Phase 3
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 severe congenital ADAMTS-13 deficiency.
Participants must have a specific Karnofsky or Lansky score
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at trough (for acute events) and within 72 hours pre-infusion and 65 minutes post-infusion (for both acute and subacute) at interval study visits every 12 weeks (up to 6 years)
Awards & highlights

Study Summary

This trial will look at the long-term safety and effectiveness of TAK-755 in people with thrombotic thrombocytopenic purpura.

Who is the study for?
This trial is for individuals aged 0-70 with severe congenital ADAMTS-13 deficiency, a condition leading to excessive blood clotting and bleeding issues. Participants must be in good health otherwise, not pregnant, agree to use birth control, and have no history of drug/alcohol abuse or immune deficiencies. They cannot have other TTP-like disorders or life-threatening reactions to similar treatments.Check my eligibility
What is being tested?
TAK-755 is being tested as a replacement therapy for the ADAMTS13 enzyme in patients with congenital thrombotic thrombocytopenic purpura (TTP). The study has two parts: one where TAK-755 is given regularly to prevent flare-ups ('prophylactic' cohort) and another where it's given during acute flare-ups ('on-demand' cohort).See study design
What are the potential side effects?
While specific side effects are not listed here, participants will be closely monitored for any adverse reactions due to long-term treatment with TAK-755. This could include allergic reactions or other responses related to the body's tolerance of this new medication.

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 severe congenital ADAMTS-13 deficiency.
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My doctor says I am mostly active and can care for myself.
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I am not pregnant and use birth control.
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I don't have severe blood clotting issues or very high LDH levels.
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I am over 16 and can care for myself, or I am under 16 and mostly active.
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I am between 0 and 70 years old.
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I have been diagnosed with severe congenital ADAMTS-13 deficiency.
Select...
I use effective birth control methods.
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I am between 0 and 70 years old.
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I have signs of TTP (a rare blood disorder).

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at trough (for acute events) and within 72 hours pre-infusion and 65 minutes post-infusion (for both acute and subacute) at interval study visits every 12 weeks (up to 6 years)
This trial's timeline: 3 weeks for screening, Varies for treatment, and at trough (for acute events) and within 72 hours pre-infusion and 65 minutes post-infusion (for both acute and subacute) at interval study visits every 12 weeks (up to 6 years) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of Related Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Secondary outcome measures
Assessment of Trough and Postdose ADAMTS13 Activity: Antigen Levels (Activity:Ag)
Assessment of VWF:Ag in Relation to ADAMTS13 Activity Levels
Assessment of VWF:RCo in Relation to ADAMTS13 Activity Levels
+35 more

Side effects data

From 2022 Phase 1 trial • 19 Patients • NCT03997760
25%
Sickle cell anaemia with crisis
25%
Alanine aminotransferase increased
25%
Aspartate aminotransferase increased
25%
Blood lactate dehydrogenase increased
25%
Dizziness
25%
Foot fracture
25%
Hypokalaemia
25%
Nasal congestion
25%
Nausea
25%
Pain
25%
Pyrexia
25%
Vomiting
25%
Musculoskeletal chest pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
TAK-755: 40 IU/kg
TAK-755: 80 IU/kg
TAK-755: 160 IU/kg

Trial Design

2Treatment groups
Experimental Treatment
Group I: Prophylactic Cohort: TAK-755Experimental Treatment1 Intervention
All participants will receive prophylactic treatment with 40 IU/kg TAK-755 intravenous (IV) infusions once every week or once every other week for the duration of the study. Participants who are naïve will receive an initial IV dose of 40 IU/kg TAK-755 to allow measurement of the pharmacokinetics of TAK-755, followed by prophylactic treatment with 40 IU/kg TAK-755 by IV infusion once every week or once every other week for the duration of the study.
Group II: On-Demand Cohort: TAK-755Experimental Treatment1 Intervention
Participants will receive daily IV infusions of TAK-755 when experiencing an acute thrombotic thrombocytopenic purpura (TTP) event until 2 days after the acute TTP event is resolved. Participants will receive 40 IU/kg TAK-755 on the first day, followed by 20 IU/kg on Day 2, and then 15 IU/kg daily until 2 days after the acute TTP event has resolved. Upon resolution of the acute TTP event, participants may choose to move to the prophylactic cohort of the study or discontinue entirely from the study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
TAK-755
2019
Completed Phase 1
~20

Find a Location

Who is running the clinical trial?

TakedaLead Sponsor
1,203 Previous Clinical Trials
4,177,948 Total Patients Enrolled
Takeda Development Center Americas, Inc.Industry Sponsor
56 Previous Clinical Trials
11,957 Total Patients Enrolled
ShireIndustry Sponsor
456 Previous Clinical Trials
97,215 Total Patients Enrolled

Media Library

TAK-755 (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04683003 — Phase 3
TAK-755 (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04683003 — Phase 3
Thrombotic Thrombocytopenic Purpura Research Study Groups: Prophylactic Cohort: TAK-755, On-Demand Cohort: TAK-755
Thrombotic Thrombocytopenic Purpura Clinical Trial 2023: TAK-755 Highlights & Side Effects. Trial Name: NCT04683003 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the goals of this particular clinical trial?

"The primary outcome of this six-year study will be the incidence of treatment-emergent adverse events related to the medication. The secondary outcomes include the impact of the intervention on quality of life, number of acute thrombotic thrombocytopenic purpura events resolved, and incidence of manifestations in those receiving prophylactic treatment."

Answered by AI

What other medical research studies have included TAK-755?

"TAK-755 was first researched at Kyushu University Hospital in 2017. So far, there is only 1 completed study but 2 more are underway with many taking place in Atlanta, Georgia."

Answered by AI

How many different medical clinics are participating in this clinical trial?

"This clinical trial has 5 locations: Childrens Healthcare of Atlanta in Atlanta, Georgia, Mid Ohio Heart Clinic Inc in Dublin, Ohio, Duke University Medical Center in Durham, North carolina as well as 4 other undisclosed locations."

Answered by AI

Does this research break new ground in the medical field?

"TAK-755 has been under investigation since 2017 by Takeda Development Center Americas, Inc. After the first trial in 57 people, it was approved for Phase 3 clinical trials. Now, there are 2 active studies being conducted in 20 cities across 10 countries."

Answered by AI

Are there any short- or long-term risks associated with taking TAK-755?

"TAK-755 has efficacy data from Phase 3 trials, as well as multiple rounds of safety data, so it received a score of 3."

Answered by AI

Are there any unfilled patient slots in this clinical trial?

"This trial, which was originally advertised on April 14th 2021, is looking for participants according to information found on clinicaltrials.gov. The listing was last updated on October 3rd, 2022."

Answered by AI

How many people are enrolled in this experiment?

"Seventy-seven patients that fit the bill are needed to enroll in this study. These individuals can come from various locations, such as Childrens Healthcare of Atlanta or Mid Ohio Heart Clinic Inc."

Answered by AI

Who else is applying?

What state do they live in?
New York
Texas
North Carolina
Other
What portion of applicants met pre-screening criteria?
Met criteria
Did not meet criteria
How many prior treatments have patients received?
3+
0
What site did they apply to?
Duke University Medical Center
University of Oklahoma
Recent research and studies
clinicaltrials.govStudy
A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
2021. "A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04683003.
~34 spots leftby Aug 2026
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