선천성 혈전성 혈소판 감소성 자반증(cTTP) 환자 아동 및 성인을 대상으로 Adzynma로 치료하는 연구
Adzynma로 치료받은 선천성 혈전성 혈소판감소성 자반증(cTTP) 환자에서 실제 임상 환경에서의 안전성을 추가로 평가하기 위한 허가 후 안전성 연구(PASS)
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce a enzyme called ADAMTS13. This enzyme helps to cut down a larger form of protein called von Willebrand Factor (VWF). People with cTTP have low levels of ADAMTS13. Without ADAMTS13, large forms of vWF build up and cause blood clots in small blood vessels. These clots can block blood flow to vital organs, causing serious health problems. Adzynma is a human ADAMTS13 protein made in the laboratory. It works the same way as natural ADAMTS13 does and may provide higher levels of ADAMTS13.
The main aim of this study is to learn more about the risk of children and adults with cTTP treated with Adzynma developing antibodies that prevent Adzynma from working properly (called neutralizing antibodies) within 6 months after the first treatment and to understand the risk of allergic reactions within 7 days of the first treatment with Adzynma. Other aims are to better understand how safe treatment with Adzynma is over a longer period of time (called long-term safety) in children and adults with cTTP and to gather information about pregnancies and babies of women who have received Adzynma while pregnant.
Only data already available in the medical records of the people who received Adzynma for the treatment of cTTP in normal clinical practice will be reviewed and collected during this study.
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
연구 유형
연구 유형
등록 (추정된)
등록
연락처 및 위치
연구 연락처
연구 연락처
- 이름: Takeda Contact
- 전화번호: +1-877-825-3327
- 이메일: medinfoUS@takeda.com
연구 장소
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Bonn, 독일, 53127
- 모병
- Universitatsklinikum Bonn
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연락하다:
- Site Contact
- 이메일: heiko.ruehl@ukbonn.de
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수석 연구원:
- Ruehl Heiko
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Cologne, 독일, 50937
- 모병
- Universitaetsklinikum Koeln
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연락하다:
- Site Contact
- 이메일: lucas.kuehne@uk-koeln.de
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수석 연구원:
- Kuehne Lucas
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Jena, 독일, 7743
- 아직 모집하지 않음
- Universitaetsklinikum Jena
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연락하다:
- Site Contact
- 이메일: karim.kentouche@med.uni-jena.de
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수석 연구원:
- Kentouche Karim
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Leipzig, 독일, 4103
- 모병
- Universitaetsklinikum Leipzig
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연락하다:
- Site Contact
- 이메일: maria.weise@medizin.uni-leipzig.de
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수석 연구원:
- Weise Maria
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München, 독일
- 모병
- LMU Klinikum
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연락하다:
- Site Contact
- 이메일: ulf.schoenermarck@med.uni-muenchen.de
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수석 연구원:
- Schoenermarck Ulf
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München, 독일, 81675
- 모병
- Klinikum Rechts der Isar der Technischen Universitaet Muenchen
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수석 연구원:
- Braunisch Matthias
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연락하다:
- Site Contact
- 이메일: matthiaschristoph.braunisch@mri.tum.de
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Vienna, 오스트리아, 1090
- 모병
- AKH - Medizinische Universitaet Wien
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연락하다:
- Site Contact
- 이메일: johannes.thaler@meduniwien.ac.at
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수석 연구원:
- Johannes Thaler
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
- 어린이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
Pediatric and adult participants who have received Adzynma for the treatment of cTTP, either prophylactically or as on-demand therapy for acute episodes-including those who became pregnant during Adzynma treatment.
설명
Inclusion criteria:
- Is diagnosed with cTTP.
- Had received commercially available Adzynma treatment for cTTP within the eligibility period of which there must be a recorded date and dose of at least one Adzynma administration in their medical chart.
- Provides a signed informed consent form (informed consent form [ICF]; or assent and consent forms, if applicable), in accordance with local ethical and institutional requirements.
Exclusion criteria:
- Has a history or presence of a functional ADAMTS13 inhibitor (that is, neutralizing antibodies to ADAMTS13/rADAMTS13).
- Has concurrent use of an investigational drug or is enrolled in another clinical trial at the time of index Adzynma infusion.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: COHORT
- 시간 관점: RETROSPECTIVE
그룹/코호트 수
코호트 및 개입
그룹/코호트그룹/코호트 |
개입 / 치료개입 / 치료 |
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All Participants
Data of participants who have received Adzynma for the treatment of cTTP will be collected retrospectively from medical records for approximately 5 years. |
This is a non-interventional study. |
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions
기간: Up to 6 months following the index Adzynma infusion
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Index infusion is defined as the first Adzynma infusion date recorded in the medical record using commercially available Adzynma (excluding doses received in clinical studies or early access program). |
Up to 6 months following the index Adzynma infusion
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Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions
기간: Up to 6 months following the index Adzynma infusion
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Number of participants with neutralizing antibodies to ADAMTS13 following the index Adzynma infusions will be reported. |
Up to 6 months following the index Adzynma infusion
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2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion
기간: Up to 6 months of follow-up time from the index Adzynma infusion
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Number of participants with long term safety risk of hypersensitivity reactions following the index Adzynma infusion will be reported. |
Up to 6 months of follow-up time from the index Adzynma infusion
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Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion
기간: Up to 7 days after each administration of Adzynma
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Number of participants with risk of hypersensitivity reactions after each administration of Adzynma following the index infusion will be reported. |
Up to 7 days after each administration of Adzynma
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Number of Participants With Treatment-emergent Adverse Events (TEAEs)
기간: From start of the study drug administration up to 5.5 years
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TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with a study intervention or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the study intervention or medicinal product. |
From start of the study drug administration up to 5.5 years
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Gestational age at the Time of Infant Birth
기간: At birth
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Gestational age at the time of infant birth will be reported. |
At birth
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Number of Participants With Spontaneous Abortion
기간: Up to 5.5 years
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Spontaneous abortion is defined as pregnancy loss before 20 weeks of gestation. |
Up to 5.5 years
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Number of Participants With Stillbirth
기간: Up to 5.5 years
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Stillbirth is defined as a fetal death after 20 weeks of gestation. |
Up to 5.5 years
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Number of Participants With Induced Abortion
기간: Up to 5.5 years
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Number of participants with induced abortion will be reported. |
Up to 5.5 years
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Number of Participants With Live Birth
기간: Up to 5.5 years
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Number of participants with live birth will be reported. |
Up to 5.5 years
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Number of Participants With any Pregnancy Related Complications
기간: Up to 5.5 years
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Number of participants with any pregnancy related complications will be reported. |
Up to 5.5 years
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Number of Participants Categorized According to Gestational age of Greater Than or Equal to (>=) 37 Gestational Weeks and Less Than (<) 37 Gestational Weeks at Birth
기간: At birth
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Number of participants categorized according to gestational age of greater than or equal to (>=) 37 gestational weeks and less than (<) 37 gestational weeks at birth will be reported. |
At birth
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Number of Infants Categorized According to Year of Birth
기간: Up to 5.5 years
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Number of infants categorized according to year of birth will be reported. |
Up to 5.5 years
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Number of Infants With Normal Birth Weight
기간: Up to 5.5 years
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Number of infants with normal birth weight will be reported. |
Up to 5.5 years
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Number of Infants With Small for Gestational Age (SGA)
기간: Up to 5.5 years
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Small for gestational age is defined as birth size (weight, length or head circumference) less than the 10th centile for sex and gestational age. |
Up to 5.5 years
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Number of Infants With Congenital Anomaly Detected at the Time of Birth
기간: At birth
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Number of infants with congenital anomaly detected at the time of birth will be reported. |
At birth
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Number of Participants by Breastfeeding Status While Receiving Adzynma
기간: Up to 5.5 years
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Breastfeeding status during Adzynma treatment will be assessed and categorized based on response (Yes, No, or Unknown). |
Up to 5.5 years
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Number of Infant Categorized by Growth and Development Outcomes
기간: Up to 5.5 years
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Number of infants categorized by growth and development outcomes (normal, abnormal [specify], or unknown) will be reported. |
Up to 5.5 years
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공동 작업자 및 조사자
수사관
수사관
- 연구 책임자: Study Director, Takeda
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- TAK-755-4007
- EUPAS1000000870 (기타 식별자: EU PAS)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
IPD 공유 액세스 기준
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD 공유 지원 정보 유형
- 연구_프로토콜
- 수액
- ICF
- CSR
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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