중증 근무력증을 앓고 있는 소아 참가자의 피하 Zilucoplan을 평가하기 위한 연구 (ziMyG)
아세틸콜린 수용체 항체 양성 일반화 중증 근무력증을 가진 2세부터 18세 미만의 소아 연구 참가자를 대상으로 Zilucoplan의 약동학, 약력학, 안전성, 내약성 및 활성을 평가하기 위한 다기관 공개 라벨, 비통제 연구
The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).
연구 개요
연구 유형
등록 (추정된)
단계
- 2 단계
- 3단계
연락처 및 위치
연구 연락처
- 이름: UCB Cares
- 전화번호: 001 844 599 2273
연구 연락처 백업
- 이름: UCB Cares
- 전화번호: +18445992273 (USA)
- 이메일: ucbcares@ucb.com
연구 장소
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Seoul, 대한민국
- 모병
- Mg0014 20220
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Seoul, 대한민국
- 빼는
- Mg0014 20104
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Illinois
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Chicago, Illinois, 미국, 60611
- 빼는
- Mg0014 50168
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Texas
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Denton, Texas, 미국, 76208
- 빼는
- Mg0014 50574
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Glasgow, 영국
- 모병
- Mg0014 40735
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London, 영국
- 모병
- Mg0014 40736
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Milan, 이탈리아
- 모병
- Mg0014 40144
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Katowice, 폴란드
- 모병
- Mg0014 40774
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Warsaw, 폴란드
- 모집하지 않고 적극적으로
- Mg0014 40218
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
United States of America (USA) specific inclusion criterion:
- Participant must be 12 to <18 years of age at the time of signing the Informed consent/assent according to local regulation
Rest of world (ROW) specific inclusion criterion:
- Participant must be 2 to <18 years of age at the time of signing the Informed consent/assent according to local regulation
Global inclusion criteria:
- Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening
- Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening
- Participants with gMG, including:
- An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to <18 years of age at Screening
- Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to <12 years of age at Screening (does not apply to US)
- Documented vaccination against meningococcal infections within 3 years prior to study start. If not fully vaccinated, participants must receive appropriate prophylactic antibiotic treatment until at least 2 weeks after the initial dose of vaccine(s)
Exclusion Criteria:
- Participant has known positive serology for muscle-specific kinase
- Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study
- Participant has had a thymectomy within 6 months prior to Baseline
- Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator
- Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline
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: Zilucoplan Arm
Study participants will receive zilucoplan in pre-defined dose based on their weight. |
Zilucoplan will be administered subcutaneously to pediatric study participants.
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
기간: Week 4 (Day 29)
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Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose. |
Week 4 (Day 29)
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Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
기간: Week 4 (Day 29)
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Samples for measurement of sRBC lysis will be collected on Day 29 predose. |
Week 4 (Day 29)
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Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
기간: Week 4 (Day 29)
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Samples for measurement of C5 will be collected on Day 29 predose. |
Week 4 (Day 29)
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
기간: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. |
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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Occurrence of treatment-emergent serious adverse events (TESAEs)
기간: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: Results in death Is life-threatening Requires inpatient hospitalization or prolongation of existing hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical events |
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
기간: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication. |
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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Occurrence of treatment-emergent infections
기간: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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Percentage of participants who experienced treatment-emergent infections as adverse events. An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. |
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)
기간: Week 4 (Day 29)
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ADA and anti-PEG antibodies will be evaluated in serum samples. |
Week 4 (Day 29)
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Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).
기간: Week 4 (Day 29)
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The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability. |
Week 4 (Day 29)
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Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)
기간: Week 4 (Day 29)
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QMG score is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The QMG total score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity. |
Week 4 (Day 29)
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Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)
기간: Week 4 (Day 29)
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The MGFA-PIS is a physician-determined assessment of clinical symptoms of MG after initiation of MG specific therapy. For the purpose of the current study, Minimal Manifestation will be determined at each scheduled time point after treatment initiation (rather than after 1 year). Change in status (improved, unchanged, worse, exacerbation, or died of MG) will also be determined. |
Week 4 (Day 29)
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Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)
기간: Week 4 (Day 29)
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The PedsQoL generic core scale (Version 4) is a validated instrument that is suitable for use with pediatric populations. PedsQoL generic core scales assess Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. The scale has 23 items with a score range of 0 to 4. Following transformation, the score range of each domain as well as the total score is 0-100 with higher scores indicating higher HRQoL. |
Week 4 (Day 29)
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공동 작업자 및 조사자
수사관
- 연구 책임자: UCB Cares, 001 844 599 2273
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (추정된)
연구 완료 (추정된)
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
- MG0014
- U1111-1290-3349 (기타 식별자: World Health Organization (WHO))
- 2022-502072-23 (레지스트리 식별자: EU Clinical Trials)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
IPD 공유 기간
Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
IPD 공유 액세스 기준
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed.All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
IPD 공유 지원 정보 유형
- 연구_프로토콜
- 수액
- CSR
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
미국에서 제조되어 미국에서 수출되는 제품
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