Study of Biostate® in Children With Von Willebrand Disease
2017년 10월 2일 업데이트: CSL Behring
A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease
This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.
연구 개요
연구 유형
중재적
등록 (실제)
17
단계
- 3단계
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
참여기준
연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.
자격 기준
공부할 수 있는 나이
12년 이하 (어린이)
건강한 자원 봉사자를 받아들입니다
아니
연구 대상 성별
모두
설명
Inclusion Criteria:
- Male and female subjects between 0 and <12 years of age
- Diagnosed with VWD Type 1, 2A, or 3
- Desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available for subject
- von Willebrand factor: ristocetin cofactor (VWF:RCo) is <20% at screening or the subject has a history of VWF:RCo <10%
- Evidence of vaccination against hepatitis A and B or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunization
- Written informed consent given
Exclusion Criteria:
- Active bleeding immediately prior to initial PK period
- Received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first study treatment
- Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
- Known history or suspicion of having VWF or FVIII inhibitors
- Acute or chronic medical condition, other than VWD, which may affect the conduct of the study
- Known or suspected hypersensitivity or previous evidence of severe side effects to other FVIII/VWF concentrates
- Participation in a clinical study or use of an investigational compound in another study in the 3 months preceding study start
- Unwillingness and/or inability to comply with the study requirements
공부 계획
이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: Biostate
|
PK component: Single bolus infusion of 80 IU VWF:RCo/kg administered intravenously on Day 1, and approximately Day 180 in Type 3 VWD subjects only. Efficacy component: Repeated bolus doses over 12 months as required to manage VWD condition. |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
기간 |
|---|---|
|
Half-life of FVIII
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
|
Mean residence time (MRT) of FVIII
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
|
Haemostatic efficacy
기간: From Day 1 until final study visit
|
From Day 1 until final study visit
|
|
Incremental Recovery of VWF
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
|
Incremental Recovery of FVIII
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
|
Half-life of VWF
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
|
Area under the concentration curve (AUC) of VWF
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
|
AUC of FVIII
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
|
Maximum plasma concentration (Cmax) of VWF
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
|
Maximum plasma concentration (Cmax) of FVIII
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
|
Minimum plasma concentration (Cmin) of VWF
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
|
Minimum plasma concentration (Cmin) of FVIII
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
|
Time to maximum concentration (tmax) of VWF
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
|
Time to maximum concentration (tmax) of FVIII
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
|
Mean residence time (MRT) of VWF
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
|
Clearance (CL) of VWF
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
|
Clearance (CL) of FVIII
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
|
Volume of distribution of steady state (Vss) of VWF
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
|
|
Volume of distribution of steady state (Vss) of FVIII
기간: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
2차 결과 측정
결과 측정 |
기간 |
|---|---|
|
Frequency of adverse events (AEs) per infusion
기간: 13 months
|
13 months
|
|
Severity of AEs per infusion
기간: 13 months
|
13 months
|
|
Severity of AEs per subject
기간: 13 months
|
13 months
|
|
Relatedness of AEs per infusion
기간: 13 months
|
13 months
|
|
Relatedness of AEs per subject
기간: 13 months
|
13 months
|
|
Development of VWF inhibitors
기간: Sample taken at baseline, then every 3 months up to 12 months
|
Sample taken at baseline, then every 3 months up to 12 months
|
|
Development of FVIII inhibitors
기간: Sample taken at baseline, then every 3 months up to 12 months
|
Sample taken at baseline, then every 3 months up to 12 months
|
|
Frequency of adverse events (AEs) per subject
기간: 13 months
|
13 months
|
공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
스폰서
협력자
연구 기록 날짜
이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.
연구 주요 날짜
연구 시작
2010년 8월 1일
기본 완료 (실제)
2013년 8월 1일
연구 완료 (실제)
2013년 8월 1일
연구 등록 날짜
최초 제출
2010년 10월 1일
QC 기준을 충족하는 최초 제출
2010년 10월 1일
처음 게시됨 (추정)
2010년 10월 4일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2017년 10월 3일
QC 기준을 충족하는 마지막 업데이트 제출
2017년 10월 2일
마지막으로 확인됨
2017년 10월 1일
추가 정보
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .
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