- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05770219
Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
Expanded Access Program: TAK-755 (rADAMTS13) for the Prophylaxis and Treatment of Severe Congenital or Hereditary Thrombotic Thrombocytopenic Purpura
Study Overview
Status
Conditions
Intervention / Treatment
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 Type
Expanded Access Type
- Treatment IND/Protocol
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Study Plan
How is the study designed?
Collaborators and Investigators
Sponsor
Publications and helpful links
Study record dates
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- TAK-755 EAP
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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