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

NCT05770219 | Unknown

• 1 other identifier: TAK-755 EAP
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Thrombotic Thrombocytopenic Purpura (TTP)

Keywords

Drug Therapy

Interventions

TAK-755 DRUG

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.

Also known as: recombinant ADAMTS13 (rADAMTS13)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Takeda: lead

Related Links

• To obtain more information on the study, click here on this link

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 3, 2023
• First Posted: March 15, 2023
• Last Updated: February 24, 2025

Record last verified: 2025-02