Thrombotic Thrombocytopenic Purpura (TTP)NCT05770219
Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
A study testing TAK-755 in people with Thrombotic Thrombocytopenic Purpura (TTP), run by Takeda.
Trial summary
The expanded access program allows people to gain access to unlicensed treatment on compassionate grounds. TAK-755 also known as rADAMTS13, is a medicine that treats people born with severe congenital or hereditary thrombotic thrombocytopenic purpura (cTTP). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura (TTP)
- What is being tested?
- TAK-755
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participant or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (signed by legal representative if participants is <18 years of age).
- Participant has a documented diagnosis of severe congenital ADAMTS13 deficiency, defined by:
- Confirmed molecular genetic testing, or
- ADAMTS13 activity and a negative test for ADAMTS13 antibodies.
- Participant is currently on a prophylactic regimen or has a documented history of at least 1 TTP event.
- Standard of Care (SoC) is considered inadequate, or the participant is intolerant to SoC*.
- SoC are plasma-based therapies, e.g., fresh frozen plasma or S/D treated plasma
Who cannot join
- Participant is currently eligible to enter into an interventional clinical trial for cTTP.
- There is a suitable alternative commercially available treatment for the treatment of cTTP.
- Participant has been diagnosed with any other thrombotic microangiopathies (TMAs) like disorder (microangiopathic hemolytic anemia), including acquired TTP.
- Participant has a known hypersensitivity/allergies/intolerance or contraindications to TAK-755 or its excipients, such as hamster proteins.
- Participant has a medical history or presence of a functional ADAMTS13 inhibitor.
- Participant has a medical history of genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including Participants who are human immunodeficiency virus (HIV)-positive with an absolute cluster of differentiation 4 (CD4) count <200 per millimeter cube (<200/mm^3) or who are receiving chronic immunosuppressive drugs
About this study
Official title: Expanded Access Program: TAK-755 (rADAMTS13) for the Prophylaxis and Treatment of Severe Congenital or Hereditary Thrombotic Thrombocytopenic Purpura
The expanded access program allows people to gain access to unlicensed treatment on compassionate grounds. TAK-755 also known as rADAMTS13, is a medicine that treats people born with severe congenital or hereditary thrombotic thrombocytopenic purpura (cTTP). This expanded access program enables continued access to those participants who have no other treatment options available for cTTP.
Read the detailed description
This is an expanded access program in which the drug being given is called TAK-755. This study will provide access to TAK-755 prior to marketing authorization for eligible participants with severe congenital or hereditary TTP who cannot adequately be treated via current standard of care and who cannot enter a clinical trial.
All participants will receive TAK-755 as intravenous (IV) injection based on their weight as measured at each dosing visit.
This is a multi-center, international program. Participants will continue treatment until benefit is no longer derived from the treatment (or treatment is no longer tolerable), sponsor decision, the participant chooses to discontinue the treatment, or TAK-755 becomes commercially available.
- Being tested
- TAK-755
- Keywords
- Drug Therapy
- 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-755Drug
Also called recombinant ADAMTS13 (rADAMTS13)
TAK-755 as IV injection at dose calculated based on the participant's body weight as measured at each dosing visit. For most participants, the final volume of infusion will be between 5 and 14 mL.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2023
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
- Expanded access
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
- NCT05770219
- Sponsor's protocol ID
- TAK-755 EAP
- Status last verified
- Feb 2025
- First submitted
- 3 Mar 2023
- Last updated
- 24 Feb 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Program: TAK-755 (rADAMTS13) for the Prophylaxis and Treatment of Severe Congenital or Hereditary Thrombotic Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT05770219
- Acronym / sponsor ID
- TAK-755 EAP
- Keywords
- Drug Therapy
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- First posted
- 15 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The expanded access program allows people to gain access to unlicensed treatment on compassionate grounds. TAK-755 also known as rADAMTS13, is a medicine that treats people born with severe congenital or hereditary thrombotic thrombocytopenic purpura (cTTP). This expanded access program enables continued access to those participants who have no other treatment options available for cTTP.
Detailed description
This is an expanded access program in which the drug being given is called TAK-755. This study will provide access to TAK-755 prior to marketing authorization for eligible participants with severe congenital or hereditary TTP who cannot adequately be treated via current standard of care and who cannot enter a clinical trial.
All participants will receive TAK-755 as intravenous (IV) injection based on their weight as measured at each dosing visit.
This is a multi-center, international program. Participants will continue treatment until benefit is no longer derived from the treatment (or treatment is no longer tolerable), sponsor decision, the participant chooses to discontinue the treatment, or TAK-755 becomes commercially available.
Study design
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Participant or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (signed by legal representative if participants is <18 years of age).
- Participant has a documented diagnosis of severe congenital ADAMTS13 deficiency, defined by:
- Participant is currently on a prophylactic regimen or has a documented history of at least 1 TTP event.
- Standard of Care (SoC) is considered inadequate, or the participant is intolerant to SoC*.
Key exclusion criteria (6 listed)
- Participant is currently eligible to enter into an interventional clinical trial for cTTP.
- There is a suitable alternative commercially available treatment for the treatment of cTTP.
- Participant has been diagnosed with any other thrombotic microangiopathies (TMAs) like disorder (microangiopathic hemolytic anemia), including acquired TTP.
- Participant has a known hypersensitivity/allergies/intolerance or contraindications to TAK-755 or its excipients, such as hamster proteins.
- Participant has a medical history or presence of a functional ADAMTS13 inhibitor.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TAK-755Also: recombinant ADAMTS13 (rADAMTS13)TAK-755 as IV injection at dose calculated based on the participant's body weight as measured at each dosing visit. For most participants, the final volume of infusion will be between 5 and 14 mL. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2025.
- To obtain more information on the study, click here on this linkLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
Questions about this trial
What is this clinical trial studying?
A study testing TAK-755 in people with Thrombotic Thrombocytopenic Purpura (TTP), run by Takeda.
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 7 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 24 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
Results are not available in the source record.
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.
Where can I read the original study information?
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 24 Feb 2025.