A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

  • STATUS
    Recruiting
  • End date
    Aug 27, 2026
  • participants needed
    77
  • sponsor
    Takeda
Updated on 12 August 2021

Summary

The main aim of the study is to check for side effects of TAK-755. Participants will either receive TAK-755 every week or every other week to prevent bleeds (prophylactic arm) or they will receive TAK-755 to treat an acute bleed (on-demand arm).

Participants in the prophylactic arm will receive treatment in the clinic or at home for 3 years. They will visit the clinic at least every 12 weeks.

Participants in the on-demand arm will receive treatment for the acute bleed until the bleed has stopped. They will have a follow-up visit at the clinic 4 weeks later.

Description

This is a follow-up study to the Phase 3 pivotal study (281102 [NCT03393975]) and will comprise of two treatment cohorts (Prophylactic and On-demand) consisting of TAK-755 nave and non-nave participants with a total duration of 6 years. A total of 77 participants will be enrolled in this study (up to 57 participants who have completed study 281102 [NCT03393975] study [non-nave participants] and at least 20 TAK-755-nave participants (participants who are nave to TAK-755). In addition, participants from Expanded Access Programs or participants from study 281102 (NCT03393975) who had an allergic reaction to standard of care prophylactic treatment will also be eligible for enrollment in this continuation study. Participants on prophylactic regimen will be able to opt for treatment in a home setting by caregiver or self-infusion.

Details
Condition THROMBOTIC THROMBOCYTOPENIC PURPURA
Treatment TAK-755
Clinical Study IdentifierNCT04683003
SponsorTakeda
Last Modified on12 August 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Participants who have completed TAK-755 Phase 3 pivotal Study 281102
(NCT03393975) (includes prophylactic cohort) and who meet all of the following
criteria are eligible for this study
Participants or legally authorized representative has provided signed informed consent >=18 years of age and/or assent form <18 years of age
Participant 0 to 70 years of age at the time of screening of the 281102 (NCT03393975) study
Participant has been diagnosed with severe congenital ADAMTS-13 deficiency
Participant does not display any severe thrombotic thrombocytopenic purpura (TTP) signs (platelet count <100,000/ microliter (mcL) and elevation of lactate dehydrogenase (LDH) greater than (>) 2 ULN at screening (prophylactic cohort only)
Participants >=16 years of age must have a Karnofsky score >= 70% and participants <16 years of age must have a Lansky score >=80%
If female of childbearing potential, participant presents with a negative serum or urine pregnancy test confirmed not more than 7 days before the first IP administration and agrees to employ adequate birth control measures for the duration of the study and to undergo quarterly pregnancy testing
Sexually active males must use an accepted and effective method of contraception during the treatment and until a minimum of 16 days after the last dose administered
Participant is willing and able to comply with the requirements of the protocol
TAK-755 nave participants and non-nave on-demand cohort participants
TAK-755 nave participants can only be enrolled in this continuation after
enrollment of the adult participants in the prophylactic arm of TAK-755 Phase
pivotal study 281102 (NCT03393975) has been completed. TAK-755 naive
pediatric participants can be enrolled after enrollment of the respective age
cohort into the pivotal Phase 3 study 281102 (NCT03393975) has been
completed.TAK-755 nave participants and participants who were enrolled into
the on-demand cohort of theTAK-755 Phase 3 pivotal study 281102 (NCT03393975)
who meet ALL of the following criteria are eligible for this study
Participant is nave to TAK-755 or was enrolled into the on-demand cohort of the TAK-755 Phase 3 pivotal study 281102 (NCT03393975) for treatment of an acute event but did not receive prophylactic treatment
Participant or legally authorized representative has provided signed informed consent (>=18 years of age) and/or assent form (<18 years of age)
Participant is 0 to 70 years of age at the time of screening
Participant has been diagnosed with severe congenital ADAMTS-13 deficiency defined as
Confirmed by molecular genetic testing, documented in participant history or at screening, and
ADAMTS-13 activity <10% as measured by the fluorescence resonance energy transfer (FRETS)-VWF73 assay, documented in participant history or at screening. Participants currently receiving standard of care prophylactic therapy may exceed 10% ADAMTS-13 activity at screening
Participants currently receiving prophylactic therapy will be screened immediately prior to their usual prophylactic infusion
Participant does not display any severe TTP signs (platelet count <100,000/microliter (mcL) and elevation of LDH >2ULN) at screening (prophylactic cohort only)
Participants >=16 years of age must have a Karnofsky score >=70% and participants <16 years of age must have a Lansky score >=80%
Participants is hepatitis C virus negative (HCV-) as confirmed by antibody or polymerase chain reaction testing OR HCV positive (HCV+) if their disease is chronic but stable
If female of childbearing potential, participant presents with a negative serum or urine pregnancy test confirmed not more than 7 days before the first IP administration and agrees to employ adequate birth control measures for the duration of the study and to undergo quarterly pregnancy testing
Sexually active males must use an accepted and effective method of contraception during treatment and until a minimum of 16 days after the last dose administered
Participant is willing and able to comply with the requirements of the protocol
Participants from an Expanded Access Program or participants in Study 281102
(NCT03393975) who had an allergic reaction to standard of care prophylactic
treatment
Participants from an expanded access program as well as participants who
participated in Study 281102(NCT03393975) who had an allergic reaction to
standard-of-care prophylactic treatment are eligible for enrollment in the
continuation study if they meet ALL of the following criteria
Participants or legally authorized representative has provided signed informed consent (>=18 years of age) and/or assent (<18 years of age)
Participants is 0 to 70 years of age at the time of screening
Participants has been diagnosed with severe congenital ADAMTS-13 deficiency defined
as
Confirmed by molecular genetic testing, documented in participant history or at screening, and
ADAMTS-13 activity <10% as measured by the fluorescence resonance energy transfer (FRETS)- VWF 73 assay, documented in participant history or at screening. Participants currently receiving standard of care prophylactic therapy may exceed 10% ADAMTS 13 activity at screening
Participant does not display any severe TTP signs (platelet count <100,000/mcL and elevation of LDH >2ULN) at screening (prophylactic cohort only)
Participants >=16 years of age must have a Karnofsky score >=70% and participants <16 years of age must have a Lansky score >=80%
If female of childbearing potential, participant presents with a negative serum or urine pregnancy test confirmed not more than 7 days before the first IP administration and agrees to employ highly effective birth control measures for the duration of the study and to undergo quarterly pregnancy testing
Sexually active males must use an accepted and effective method of contraception during treatment and until a minimum of 16 days after the last dose administered
Participant is willing and able to comply with the requirements of the protocol

Exclusion Criteria

Participants who have completed TAK-755 Phase 3 pivotal study (281102)
(NCT03393975) and TAK 755 nave participants and non-nave on-demand cohort
participants and participants from an Expanded Access Program or participants
in Study 281102 (NCT03393975) who had an allergic reaction to standard-of-care
prophylactic treatment
Participant has been diagnosed with any other TTP-like disorder (microangiopathic hemolytic anemia), including immune-mediated TTP
Known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule ADAMTS-13, hamster protein, or other constituents of TAK-755
Participant has a presence of a functional ADAMTS-13 inhibitor at screening
Participant has a medical history of a genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including participants who are human immunodeficiency virus-positive with an absolute cluster of differentiation 4 (CD4) count < 200/ cubic millimeter (mm^3) or who are receiving chronic immunosuppressive drugs
Participant has a history of significant neurological events, such as major stroke, indicating that a relapse might have severe consequences, as judged by the investigator
Participant has been diagnosed with severe cardiovascular disease (New York Heart Association classes 3 to 4)
Participant with end stage renal disease requiring chronic dialysis
Participant has been diagnosed with hepatic dysfunction, as evidenced by, but not limited to, any of the following
Serum alanine aminotransferase >= 2ULN
Severe hypoalbuminemia <24 gram per liter (g/L)
Portal vein hypertension (e.g., presence of otherwise unexplained splenomegaly, history of esophageal varices)
In the opinion of the investigator, the participant has another clinically significant concomitant disease that may pose additional risks for the participant
Participant has been treated with an immunomodulatory drug, excluding topical treatment (e.g., ointments, nasal sprays), within 30 days prior to enrollment. Use of corticosteroids in conjunction with administration of fresh frozen plasma to prevent allergic manifestations is permitted
Participant has an acute illness (e.g., influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, bronchial asthma) at the time of screening (prophylactic cohort only)
Participant is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment
Participant has a history of drug and/or alcohol abuse within the last 2 years
Participant has a progressive fatal disease and/or life expectancy of <= 3 months
Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures
Participant suffers from a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude
Participant is a family member or employee of the sponsor or investigator
If female, participant is pregnant or lactating at the time of enrollment
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