A Phase 3b, prospective, open-label, multicenter, single treatment arm, continuation study of the safety and efficacy of TAK-755 (rADAMTS-13, also known as BAX 930/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)

JRCT ID: jRCT2031220348

Registered date:27/09/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: congenital thrombotic thrombocytopenic purpura
• Date of first enrollment: 29/09/2021
• Target sample size: 5
• Countries of recruitment: United States,Japan,Austria,Japan,France,Japan,Germany,Japan,Italy,Japan,Poland,Japan,Spain,Japan,Switzerland,Japan,United Kingdom,Japan
• Study type: Interventional

Intervention(s)

Participants in prophylaxis cohort will receive IV infusions of 40 IU/kg TAK-755 during Q1W or Q2W up to 3 years.
On-demand cohort participants will receive an initial IV infusion of 40 IU/kg followed by 20 IU/kg on Day 2 and 15 IU/kg daily until 2 days after the acute event has resolved (approximately one month).

Outcome(s)

Primary Outcome

Incidence of Related Treatment-emergent Adverse Events (TEAEs) and Related Serious Adverse Events (SAEs) [ Time Frame: Throughout the study period of approximately 6 years ]
TEAE: any event emerging or manifesting at or after the initiation of treatment with an investigational product (IP) or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the IP or medicinal product.
SAE: Signs, symptoms or outcomes which results in death, requires inpatient hospitalization or prolongation of hospitalization, results in persistent or significant disability/incapacity, results in a congenital abnormality/birth defect, important medical event, bronchospasm, reviewed and confirmed seroconversion for human immunodeficiency virus (HIV), hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), hepatitis E virus (HEV), or parvovirus B19 (B19V).
Product related AE: event emerging or manifesting at or after the initiation of treatment with an IP or medicinal product or any existing event that worsens. Vital signs, clinical chemistry parameters and hematology parameters will be recorded as AEs.

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: <= 70age old
• Gender: Both

Include criteria

Inclusion Criteria:
Applies to Subjects who have completed TAK-755 Phase 3 pivotal study (Study 281102) Subjects who have completed TAK-755 Study 281102 (includes prophylactic cohort) and who meet ALL of the following criteria are eligible for this study:
1. Subject or legally authorized representative has provided signed informed consent (>=18 years of age) and/or assent form (<18 years of age).
2. Subject is 0 to 70 years of age at the time of screening of the 281102 study.
3. Subject has been diagnosed with severe congenital ADAMTS-13 deficiency.
4. Subject does not display any severe TTP signs (platelet count <100,000/uL and elevation of LDH >2 x upper limit of normal [ULN]) at screening (prophylactic cohort only).
5. Subjects >= 16 years of age must have a Karnofsky score >= 70% and subjects <16 years of age must have a Lansky score >= 80%.
6. 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.
7. 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.

Exclude criteria

Exclusion Criteria
Applies to subjects who have completed TAK-755 Phase 3 pivotal study (Study 281102):
1. Known life-threatening hypersensitivity reaction, including anaphylaxis, to the parent molecule ADAMTS-13, hamster protein, or other constituents of TAK-755.
2. Subject has presence of a functional ADAMTS-13 inhibitor at screening.
3. In the opinion of the investigator, the subject has another clinically significant concomitant disease that may pose additional risks for the subject.
4. Subject is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment.
5. Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
6. 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.
7. Subject is a family member or employee of the sponsor or investigator

Related Information

• Primary Sponsor: Iwasaki Tsubasa