2024-513839-24-00招募中3 期
A phase 3b, prospective, open-label, multicenter, single treatment arm, continuation study of the safety and efficacy of TAK-755 (rADAMTS-13, also know as BAX930/SHP655) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP; Upshaw-Schulman Syndrome, or hereditary thrombotic thrombocytopenic purpura)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 13
- 主要终点
- There is no primary efficacy endpoint for this study, as the primary objective of the study is long-term safety.
研究概览
简要总结
To evaluate the long-term safety and tolerability of TAK-755 (rADAMTS13) in terms of related treatment-emergent adverse events (TEAEs) and related serious adverse events (SAEs) in both the prophylactic and the on-demand cohorts.
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Prospective, open-label, multicenter, single treatment arm continuation study
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 65+ years（65+ Years, 0-17 Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Applies to Subjects who have completed the TAK-755 Phase 3 pivotal study (Study 281102) in the prophylactic cohort Subjects who have completed TAK-755 Study 281102 (in the prophylactic cohort who meet ALL of the following criteria are eligible for this study:
- •Subject or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (<18 years of age).
- •Subject is 0 to 70 years of age at the time of screening of the 281102 study.
- •Subject has been diagnosed with severe congenital ADAMTS-13 deficiency.
- •Subject does not display any severe TTP signs (platelet count <100,000/μL and elevation of LDH >2 × upper limit of normal [ULN]) at screening (prophylactic cohort only).
- •Subjects ≥16 years of age must have a Karnofsky score ≥70% and subjects <16 years of age must have a Lansky score ≥80%.
- •If female of childbearing potential, subject 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 the treatment and until a minimum of 16 days after the last dose administered.
- •Subject is willing and able to comply with the requirements of the protocol.
- •Applies to naïve subjects and non-naïve on-demand cohort subjects Naïve subjects can only be enrolled in this continuation study after enrollment of the adult subjects in the prophylactic arm of the TAK-755 Phase 3 pivotal study (Study 281102) has been completed. Naïve pediatric subjects can be enrolled after enrollment of the respective age cohort into Study 281102 has been completed. The following criteria also applies to subjects who completed study 281101, but did not participate in
- •The following criteria do not apply to subjects from the Expanded AccessProgram or subjects from 281102 who had an allergic reaction to SoC. See separate criteria below for study eligibility of EAP subjects and subjects from study 281102 who had an allergic reaction to SoC. Naïve subjects and subjects who were enrolled into the on-demand cohort of the TAK-755 Phase 3 pivotal study (281102) who meet ALL of the following criteria are eligible for this study:
- •Subject is naïve or was enrolled into the on-demand cohort of the TAK-755 Study 281102 for treatment of an acute TTP event but did not receive prophylactic treatment.
- •Subject or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (<18 years of age).
- •Subject is 0 to 70 years of age at the time of screening.
- •Subject has been diagnosed with severe congenital ADAMTS-13 deficiency defined as: a. Confirmed by molecular genetic testing, documented in subject history or at screening, and b. ADAMTS-13 activity <10% as measured by the fluorescence resonance energy transfer (FRETS)-Von Willebrand factor (VWF)73 assay, documented in subject history or at screening. Subjects currently receiving standard of care prophylactic therapy may exceed 10% ADAMTS-13 activity at screening.
- •Subjects currently receiving prophylactic therapy will be screened immediately prior to their usual prophylactic infusion.
- •Subject does not display any severe TTP signs (platelet count <100,000/μL and elevation of LDH >2 × ULN) at screening (prophylactic cohort only).
- •Subjects ≥16 years of age must have a Karnofsky score ≥70% and subjects <16 years of age must have a Lansky score ≥80%.
- •Subject 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, subject 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.
- •Subject is willing and able to comply with the requirements of the protocol.
- •Subjects from an Expanded Access Program or subjects in Study 281102 who had an allergic reaction to standard of care prophylactic treatment must meet ALL of the following criteria.
- •Subject or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (<18 years of age), as applicable.
- •Subject is 0 to 70 years of age at the time of screening. For more inclusion criteria please refer to the Protocol.
排除标准
- •The subject will be excluded from the study if any of the following exclusion criteria are met. Applies to subjects who have completed TAK-755 Phase 3 pivotal study (Study 281102):
- •Known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule ADAMTS-13, hamster protein, or other constituents of TAK-
- •Subject has presence of a functional ADAMTS-13 inhibitor at screening.
- •In the opinion of the investigator, the subject has another clinically significant concomitant disease that may pose additional risks for the subject.
- •Subject is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment.
- •Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
- •Subject 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.
- •Subject is a family member or employee of the sponsor or investigator
- •The following criteria do not apply to subjects from the Expanded Access Program or subjects from 281102 who had an allergic reaction to SoC. The following criteria also applies to subjects who completed study 281101, but did not participate in
- •Subject 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-
- •Subject has presence of a functional ADAMTS-13 inhibitor at screening.
- •Subject has a medical history of a genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including subjects who are human immunodeficiency virus-positive with an absolute cluster of differentiation 4 (CD4) count <200/mm3 or who are receiving chronic immunosuppressive drugs.
- •Subject has a history of significant neurological events, such as major stroke, indicating that a relapse might have severe consequences, as judged by the investigator.
- •Subject has been diagnosed with severe cardiovascular disease (New York Heart Association classes 3 to 4).
- •Subject with end stage renal disease requiring chronic dialysis.
- •Subject has been diagnosed with hepatic dysfunction, as evidenced by, but not limited to, any of the following: a. Serum alanine aminotransferase ≥2 × ULN b. Severe hypoalbuminemia <24 g/L c. Portal vein hypertension (e.g., presence of otherwise unexplained splenomegaly, history of esophageal varices).
- •In the opinion of the investigator, the subject has another clinically significant concomitant disease that may pose additional risks for the subject.
- •Subject 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 reactions is permitted.
- •Subject has an acute illness (e.g., influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, bronchial asthma) at the time of screening (prophylactic cohort only).
- •Subject is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment.
- •Subject has a history of drug and/or alcohol abuse within the last 2 years.
- •Subject has a progressive fatal disease and/or life expectancy of ≤3 months.
- •Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
- •Subject 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.
- •Subject is a family member or employee of the sponsor or investigator.
- •If female, subject is pregnant or lactating at the time of enrollment.
- •Study 281102 who had an allergic reaction to standard of care prophylactic treatment:
- •Subject 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-
- •Subject has presence of a functional ADAMTS-13 inhibitor at screening.
- •Subject has a medical history of a genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including subjects who are human immunodeficiency virus-positive with an absolute cluster of differentiation 4 (CD4) count <200/mm3 or who are receiving chronic immunosuppressive drugs.
结局指标
主要结局
There is no primary efficacy endpoint for this study, as the primary objective of the study is long-term safety.
There is no primary efficacy endpoint for this study, as the primary objective of the study is long-term safety.
次要结局
- The key secondary efficacy outcome measure is the incidence of acute TTP events among subjects receiving TAK755 prophylactically. Analyses will be conducted using FAS for the prophylactic cohort. The number and incidence rate of acute TTP events will be summarized by enrollment status (""Naïve"" or having completed the Phase 3 pivotal study [Study 281102]) and overall.
研究者
Parth Patwari
Scientific
Baxalta Innovations GmbH
研究点 (13)
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