search
Back to results

Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

Primary Purpose

Thrombotic Thrombocytopenic Purpura (TTP)

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
TAK-755
Sponsored by
Takeda
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Thrombotic Thrombocytopenic Purpura (TTP) focused on measuring Drug Therapy

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    March 3, 2023
    Last Updated
    April 4, 2023
    Sponsor
    Takeda
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT05770219
    Brief Title
    Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
    Official Title
    Expanded Access Program: TAK-755 (rADAMTS13) for the Prophylaxis and Treatment of Severe Congenital or Hereditary Thrombotic Thrombocytopenic Purpura
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    April 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Takeda

    4. Oversight

    5. Study Description

    Brief 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). 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Thrombotic Thrombocytopenic Purpura (TTP)
    Keywords
    Drug Therapy

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    TAK-755
    Other Intervention Name(s)
    recombinant ADAMTS13 (rADAMTS13)
    Intervention Description
    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.

    10. Eligibility

    Sex
    All
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Takeda Contact
    Phone
    +1-877-825-3327
    Email
    medinfoUS@takeda.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Study Director
    Organizational Affiliation
    Takeda
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Links:
    URL
    https://clinicaltrials.takeda.com/study-detail/4e6348797ec74b0a?idFilter=%5B%22TAK-755+EAP%22%5D
    Description
    To obtain more information on the study, click here/on this link

    Learn more about this trial

    Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

    We'll reach out to this number within 24 hrs