Thrombotic Thrombocytopenic Purpura (TTP)NCT06441578
A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
An observational study of Thrombotic Thrombocytopenic Purpura (TTP), run by Takeda at 1 site in Japan.
Trial summary
This study is a survey in Japan of recombinant ADAMTS13 used to treat or to prevent participants with congenital thrombotic thrombocytopenic purpura (cTTP). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura (TTP)
- What is being tested?
- Recombinant ADAMTS13
- Type of treatment
- Injection or infusion
- How many people
- 40 participants
- Where
- 1 siteJapan
- Study dates
- 30 May 2024to 30 Sep 2032
- Phase
- Observational
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The population of this survey are all participants who meet the inclusion/exclusion criteria.
Who may be able to join
- All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13
Who cannot join
- None
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)
This study is a survey in Japan of recombinant ADAMTS13 used to treat or to prevent participants with congenital thrombotic thrombocytopenic purpura (cTTP). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study.
The main aim of the study is to check for side effects related from recombinant ADAMTS13 and to check if recombinant ADAMTS13 improves or prevents cTTP.
During the study, participants with cTTP will take recombinant ADAMTS13 intravenous injection according to their clinic's standard practice. The study doctors will check for side effects from recombinant ADAMTS13 for 18 months.
- Being tested
- Recombinant ADAMTS13
- 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.
Recombinant ADAMTS13Drug
Also called ADZYNMA Intravenous 1500, TAK-755
Recombinant ADAMTS13, Intravenous injection
| Study group | Role | What they receive |
|---|---|---|
| Recombinant ADAMTS13 | Participants will receive recombinant ADAMTS13 intravenous injection. Receives: Drug: Recombinant ADAMTS13 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)
Measured: Up to 18 Months
Secondary outcomes (4)
Percent Change in Observed Platelet Count from Baseline at the End of Treatment
Percent change in platelet count from baseline at the end of treatment will be reported.
Measured: Up to 18 Months
Percent Change in Observed ADAMTS13 Activity from Baseline at the End of Treatment
Percent change in ADAMTS13 activity from baseline at the end of treatment will be reported.
Measured: Up to 18 Months
Percent Change in Observed ADAMTS13 Inhibitor from Baseline at the End of Treatment
Percent change in ADAMTS13 inhibitor from baseline at the end of treatment will be reported.
Measured: Up to 18 Months
Number of Participants with Thrombotic Thrombocytopenic Purpura (TTP) Events on Periodic Replacement Therapy
Number of participants with TTP events on periodic replacement therapy will be reported.
Measured: Up to 18 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2024
- Study start30 May 2024
- Primary completion30 Sep 2032 (estimated)
- Study completion30 Sep 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombotic Thrombocytopenic Purpura (TTP)Studies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06441578
- Sponsor's protocol ID
- TAK-755-4005
- Other IDs
- jRCT2031240130
- Status last verified
- Oct 2026
- First submitted
- 29 May 2024
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- 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.