CSL312_3003 유전성 혈관부종을 가진 2-11세 피험자를 대상으로 한 안전성 및 약동학 연구
2~11세 소아 피험자의 유전성 혈관부종 예방 치료에서 CSL312(가라다시맙)의 안전성, 약동학, 약력학 및 효능을 평가하기 위한 3상 공개 라벨 연구
연구 개요
연구 유형
등록 (실제)
단계
- 3단계
연락처 및 위치
연구 장소
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Berlin, 독일, 12203
- Charité - Universitätsmedizin Berlin
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Frankfurt am Main, 독일, 60590
- Universitätsklinikum Frankfurt
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Hesse
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Frankfurt am Main, Hesse, 독일, 60596
- HZRM Hämophilie Zentrum Rhein Main GmbH
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Arizona
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Litchfield Park, Arizona, 미국, 85340
- Research Solutions of Arizona
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Scottsdale, Arizona, 미국, 85251
- Medical Research of Arizona
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California
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Orange, California, 미국, 92868
- Donald S. Levy M.D.
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Santa Monica, California, 미국, 90404
- Raffi Tachdjian MD, Inc.
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Ohio
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Cincinnati, Ohio, 미국, 45236
- Bernstein Clinical Research
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Pennsylvania
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Hershey, Pennsylvania, 미국, 17033
- PennState Health Milton S. Hershey Medical Center
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Texas
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Dallas, Texas, 미국, 75231
- AARA Research Center
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Ashkelon, 이스라엘, 7830604
- Barzilai University Medical Center
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Ottawa, 캐나다, K1H1E4
- Ottawa Allergy Research Corp
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Campbelltown, 호주, NSW 2560
- Campbelltown Hospital, Western Sydney University
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참여기준
자격 기준
공부할 수 있는 나이
- 어린이
건강한 자원 봉사자를 받아들입니다
설명
포함 기준:
- 남성 또는 여성
- 2~11세, 연령 기준 체중 ≥ 10번째 백분위수
- 임상적으로 확인된 C1-INH HAE로 진단됨
- 스크리닝 전 6개월 동안 2회 이상의 HAE 발작 경험
제외 기준:
- 특발성 또는 후천성 혈관부종, 두드러기와 관련된 재발성 혈관부종 또는 HAE III형과 같은 다른 형태의 혈관부종의 동시 진단
- 임상 연구 동안 사전에 계획된 주요 수술 또는 절차
- HAE 발작에 대한 일상적인 예방을 위한 C1-INH 제품, 안드로겐, 항섬유소 용해제, 승인되었거나 향후 승인될 약물 또는 기타 소분자 약물 사용
- 다른 중재 임상 연구에 참여
공부 계획
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 |
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실험적: CSL312
2~5세 및 6~11세는 특정 피하 투여 일정이 있습니다.
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완전 인간 면역글로불린 G 서브클래스 4/람다 재조합 억제제 단일클론 항체를 피하 투여(SC)
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Number of Participants With Treatment Emergent Adverse Events (TEAE)
기간: Up to Month 12
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Up to Month 12
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Percentage of Participants With TEAE
기간: Up to Month 12
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The percentage of participants was rounded to one place of decimal.
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Up to Month 12
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Number of TEAE
기간: Up to Month 12
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Up to Month 12
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TEAE Rates Per Injection
기간: Up to Month 12
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The TEAE rate per injection was calculated as the number of TEAE/ number of injections.
The number of injections was defined as the total injections a participant received during the Safety Evaluation Period under the dosing regimen to which the TEAE was assigned.
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Up to Month 12
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TEAE Rates Per Participant-Year
기간: Up to Month 12
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The TEAE rate per participant year was calculated as number of TEAEs/ participant years.
Participant-years of exposure were calculated as the sum of each participant's exposure duration (in years) under the specified dosing regimen or overall.
For the time assigned to a dosing regimen, each study day was counted under the corresponding regimen.
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Up to Month 12
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Maximum Concentration (Cmax) of CSL312 at Steady-state
기간: Up to Month 12
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Up to Month 12
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Trough Concentration (Ctrough) of CSL312 at Steady-state
기간: At Months 3, 4, 6, 9, 10, and 12
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At Months 3, 4, 6, 9, 10, and 12
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Time to Maximum Concentration (Tmax) of CSL312 at Steady-State
기간: Up to Month 12
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Up to Month 12
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Time-normalized Number of HAE Attacks Per Month
기간: Up to Month 12
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Time-normalized number of HAE attacks per month during treatment was calculated per participant as: [Number of HAE attacks / Length of participant treatment in days] * 30.4375.
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Up to Month 12
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Time-normalized Number of HAE Attacks Per Year
기간: Up to Month 12
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Time-normalized number of HAE attacks per year during treatment was calculated per participant as: [Number of HAE attacks / Length of participant treatment in days] * 365.25.
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Up to Month 12
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Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Month
기간: Up to Month 12
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The time-normalized number of HAE attacks per month treated with on-demand treatment were calculated as follows: [(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days] ∗ 30.4375. |
Up to Month 12
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Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Year
기간: Up to Month 12
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The time-normalized number of HAE attacks per year treated with on-demand treatment were calculated as follows: [(Number of HAE attacks treated with on - demand treatment during treatment period)/ Length of participant treatment in days] ∗ 365.25. |
Up to Month 12
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Time-normalized Number of Moderate and/or Severe HAE Attacks Per Month
기간: Up to Month 12
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Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: [number of moderate or severe HAE attacks / length of participant treatment in days] * 30.4375.
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Up to Month 12
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Time-normalized Number of Moderate and/or Severe HAE Attacks Per Year
기간: Up to Month 12
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Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: [number of moderate or severe HAE attacks / length of participant treatment in days] * 365.25.
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Up to Month 12
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Percentage Reduction in the Time-normalized Number of HAE Attacks
기간: Up to Month 12
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The percentage reduction in the time-normalized number of HAE attacks was calculated within a participant as follows: 100*[ 1 - (Time-normalized number of HAE attacks per month during treatment period/Time-normalized number of HAE attacks per month from historical data)].
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Up to Month 12
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Number of Participants Experiencing at Least Greater Than or Equal to (>=) 50 Percent (%), >= 70%, >= 90%, or Equal to 100% (Attack-free) Reduction in the Time-normalized Number of HAE Attacks
기간: Up to Month 12
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A participant was classified as a responder if the percentage reduction in the time-normalized number of HAE attacks under treatment compared to the time-normalized number of HAE attacks documented in the medical records was >= 50%.
Percent Reduction = 100 * [1 - (time-normalized number of HAE attacks during corresponding time window / time-normalized number of HAE attacks based on historical data)].
Here number of participants experiencing at least >= 50%, >= 70%, >= 90%, or equal to 100% (Attack-free) reduction in the time-normalized number of HAE attacks are reported.
The number of responders at each reduction category have been reported.
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Up to Month 12
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Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
기간: Up to Month 12
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Up to Month 12
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Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
기간: Up to Month 12
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The percentage of participants was rounded to one decimal place.
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Up to Month 12
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Number of Participants With TEAE by Severity
기간: Up to Month 12
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Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where: Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. |
Up to Month 12
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Percentage of Participants With TEAE by Severity
기간: Up to Month 12
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Severity of AE was assessed by the investigator and categorized as mild, moderate and severe where: Mild: AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. Moderate: AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. The percentage of participants was rounded to one place of decimal. |
Up to Month 12
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Number of Participants With Anti-CSL312 Antibodies
기간: At Day 1, Months 6 and 12
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At Day 1, Months 6 and 12
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Percentage of Participants With Anti-CSL312 Antibodies
기간: At Day 1, Months 6 and 12
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The percentage of participants was rounded to one place of decimal.
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At Day 1, Months 6 and 12
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Number of Participants With Adverse Events of Special Interest (AESI)
기간: Up to Month 12
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AESI included severe hypersensitivity including anaphylaxis.
The AESI reported have been identified by investigators and suggestive events were independently identified for further review with a Standardized MedDRA Query (SMQ).
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Up to Month 12
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Percentage of Participants With AESI
기간: Up to Month 12
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AESI included severe hypersensitivity including anaphylaxis.
The AESI reported have been identified by investigators and suggestive events were independently identified for further review with an SMQ.
The percentage of participants was rounded to one place of decimal.
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Up to Month 12
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FXIIa-mediated Kallikrein Activity
기간: At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10
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At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10
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Percent of Baseline FXIIa-mediated Kallikrein Activity
기간: At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10
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Percent of Baseline at Visit [i] = 100 * (actual value at Visit [i] / Baseline value), where Baseline is defined as the most recent, non-missing value before the first IP administration (including unscheduled visits).
Here unit of measure is Percent (%) of FXIIa-mediated Kallikrein Activity.
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At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10
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Number of Participants With Laboratory Findings Reported as AE
기간: Up to Month 12
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Up to Month 12
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Percentage of Participants With Laboratory Findings Reported as AE
기간: Up to Month 12
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The participant data were rounded to one decimal place.
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Up to Month 12
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공동 작업자 및 조사자
스폰서
수사관
- 연구 책임자: Study Director, CSL Behring
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- CSL312_3003
- 2022-502386-13-00 (씨티스)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
CSL은 체계적 검토 그룹 또는 선의의 연구자로부터 개별 환자 데이터(IPD)를 공유해 달라는 요청을 고려할 것입니다. IPD에 대한 자발적인 데이터 공유 요청을 제출하기 위한 프로세스 및 요구 사항에 대한 정보는 clinicaltrials@cslbehring.com으로 CSL에 문의하십시오.
해당 국가별 개인 정보 보호 및 기타 법률 및 규정을 고려하여 IPD 공유를 방지할 수 있습니다.
요청이 승인되고 연구원이 적절한 데이터 공유 계약을 체결한 경우 적절하게 익명화된 IPD를 사용할 수 있습니다.
IPD 공유 기간
IPD 공유 액세스 기준
IPD 사용 제안이 본질적으로 비상업적이며 내부 검토 위원회의 승인을 받은 체계적 검토 그룹 또는 선의의 연구자만 요청을 할 수 있습니다.
제안된 연구 질문이 CSL의 내부 검토 위원회에서 결정한 바와 같이 중요하고 알려지지 않은 의학 또는 환자 치료 질문에 대한 답변을 추구하지 않는 한 CSL은 IPD 요청을 고려하지 않습니다.
요청 당사자는 IPD를 사용할 수 있게 되기 전에 적절한 데이터 공유 계약을 체결해야 합니다.
IPD 공유 지원 정보 유형
- 연구_프로토콜
- 수액
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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. .