안구 중증 근무력증 성인 참가자를 대상으로 Rozanoloxizumab의 효능과 안전성을 평가하는 연구 (MyVision)
안구 중증 근무력증을 가진 성인 참가자를 대상으로 로잔올록시주맙의 유효성 및 안전성을 평가하기 위한 3상 무작위 배정 이중 맹검 위약 대조 연구
The purpose of the study is to demonstrate the efficacy, safety and tolerability of rozanolixizumab compared with placebo in the treatment of adult study participants with Ocular Myasthenia Gravis.
연구 개요
연구 유형
등록 (추정된)
단계
- 3단계
연락처 및 위치
연구 연락처
- 이름: UCB Cares
- 전화번호: +18445992273
- 이메일: ucbcares@ucb.com
연구 연락처 백업
- 이름: UCB Cares
- 전화번호: 00184459922733 (UCB)
- 이메일: ucbcares@ucb.com
연구 장소
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Sarajishvili, 그루지야
- 모병
- Mg0038 21403
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Tbilisi, 그루지야
- 모병
- Mg0038 21402
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Rüdersdorf, 독일
- 모병
- Mg0038 20405
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Arizona
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Scottsdale, Arizona, 미국, 85251
- 모병
- Mg0038 10104
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Illinois
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O'Fallon, Illinois, 미국, 62269
- 모병
- Mg0038 10105
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New York
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Amherst, New York, 미국, 14226
- 모병
- Mg0038 10106
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Ohio
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Cincinnati, Ohio, 미국, 45219
- 모병
- Mg0038 10102
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Columbus, Ohio, 미국, 43210
- 모병
- Mg0038 10103
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Wisconsin
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Greenfield, Wisconsin, 미국, 53228
- 모병
- Mg0038 10108
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Alicante, 스페인
- 모병
- Mg0038 20605
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Barcelona, 스페인
- 모병
- Mg0038 20604
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Barcelona, 스페인
- 모병
- Mg0038 20607
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Madrid, 스페인
- 모병
- Mg0038 20611
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Murcia, 스페인
- 모병
- Mg0038 20602
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Málaga, 스페인
- 모병
- Mg0038 20606
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Seville, 스페인
- 모병
- Mg0038 20603
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Kobe, 일본
- 모병
- Mg0038 31802
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Minatoku, 일본
- 모병
- Mg0038 31804
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Sendai, 일본
- 모병
- Mg0038 31805
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Beijing, 중국
- 모병
- Mg0038 31901
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Beijing, 중국
- 모병
- Mg0038 31912
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Beijing, 중국
- 모병
- Mg0038 31911
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Chengdu, 중국
- 모병
- Mg0038 31906
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Chongqing, 중국
- 모병
- Mg0038 31908
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Nanchang, 중국
- 모병
- Mg0038 31907
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Suzhou, 중국
- 모병
- Mg0038 31902
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Montreal, 캐나다
- 모병
- Mg0038 10201
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Katowice, 폴란드
- 모병
- Mg0038 21204
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Krakow, 폴란드
- 모병
- Mg0038 21203
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Lublin, 폴란드
- 모병
- Mg0038 21206
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Warsaw, 폴란드
- 모병
- Mg0038 21202
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Dijon, 프랑스
- 모병
- Mg0038 20303
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Nantes, 프랑스
- 모병
- Mg0038 20304
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Nice, 프랑스
- 모병
- Mg0038 20301
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Paris, 프랑스
- 모병
- Mg0038 20302
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Paris, 프랑스
- 모병
- Mg0038 20305
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
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Participant must be a minimum of 18 years of age inclusive at the time of signing the informed consent form (ICF)
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Participant has Myasthenia Gravis Foundation of America (MGFA) Class I with any ocular weakness at Screening through Baseline. The participant may have weakness in muscles of eye (ie, extraocular muscles that move the eyeball, including the medial rectus, lateral rectus, superior rectus, inferior rectus, superior oblique, and inferior oblique, orbicularis oculi muscles, and levator palpebrae superioris) but must have normal strength in all other facial, bulbar, and limb muscles.
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Study participant has been diagnosed with Ocular Myasthenia Gravis (oMG) with consistent ocular clinical features at Screening and supported by:
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Documented presence of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK), OR
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Documented absence of autoantibodies against AChR or MuSK; in this case, documented abnormal repetitive nerve stimulation (RNS) or single fiber electromyography (SFEMG) (as defined in the adjudication manual) and at least 1 of the following should be met:
- Documented positive ice test (Ptosis recovers with the ice test [applied 2 minutes (min) to the ptotic lid] with >2mm improvement)
- History of positive edrophonium chloride (Tensilon) test (or equivalent tests used to establish oMG diagnostic as per current practice)
- Demonstrated objective improvement in oMG signs with acetylcholinesterase inhibitor (AChEIs), plasma exchange (PLEX), intravenous immunoglobulin (IVIg), subcutaneous immunoglobulin (SCIg) or corticosteroids (CSs)
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Participant has an Myasthenia Gravis Impairment Index (MGII) ocular score (Patient-Reported Outcome (PRO) part) ≥6 with at least 2 ocular items with a score of ≥2 at both Screening and Baseline visits.
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Participant reported ocular symptom(s) onset <3 years before Screening or ≥3 years provided they have demonstrated response (ie, improvement in ptosis or diploplia) to treatment (IVIg, PLEX, SCIg, pyridostigmine, and/or CSs) in the past year.
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Participant has no pupillary abnormality except those resulting from prior localized eye disease or surgical intervention.
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Participant who:
- is currently receiving background treatment for oMG symptoms at the time of Screening and has been receiving treatment for oMG with a stable dose for at least 30 days prior to Screening, OR
- is not receiving any background treatment at the time of Screening
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Participant has a body weight ≥35kg at Baseline.
Exclusion Criteria:
- Participant has any clinically significant medical or psychiatric condition (including an alcohol or drug use disorder), recent major surgery (including thymectomy [within 3 months of Screening], solid organ, stem cell or marrow transplant), planned major surgery (including thymectomy) during study participation, and/or significant laboratory abnormality that, in the opinion of the investigator, could jeopardize or would compromise the study participant's ability to participate in this study.
- Participant has been diagnosed with other diseases that lead to eyelid dropping, peripheral muscle weakness, or diplopia, including other autoimmune diseases that would interfere with an accurate assessment of the oMG clinical symptoms or other neurological diseases, such as congenital myasthenic syndromes, mitochondrial diseases, and muscular dystrophies.
- Participant has a known hypersensitivity to other anti-Fc receptor (FcRn) medications, to any components of the study medication (including the excipient polysorbate 80) or has a known history of hyperprolinemia, since L-proline is a constituent of the rozanolixizumab formulation.
- Participant has active neoplastic disease or has received treatment for neoplastic disease within 5 years of study entry (except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the uterine cervix, carcinoma in situ of the breast, or incidental histological findings of prostate cancer [Tumor, Node, Metastasis (TNM) stage T1a or T1b] that has been definitely treated with standard of care approaches).
- Participant has a clinically relevant active infection (eg, tuberculosis (TB) infection) or a history of serious infection (resulting in hospitalization or requiring intravenous (iv) antibiotic treatment) within 6 weeks before the Baseline Visit.
- Participant has renal impairment, defined as glomerular filtration rate less than 30milliliter/min/1.73m2 at Screening.
- Participant has been previously treated with FcRn inhibitors.
- Participant has been treated with any of the immunosuppressive medications, biologics, or other therapies, in the specified prohibitive timeframe.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 삼루타
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: Rozanolixizumab
After the Screening Period, participants will enter the study Intervention Period and will be administered study drug for up to 6 weeks. Prior to entering the Intervention Period, study participants will be randomized at a 1:1 ratio and will receive rozanolixizumab. At the End of Treatment Visit, participants will enter an Observation Period of 4 to 7 weeks with an opportunity to transition to an open-label extension study. |
Rozanolixizumab will be administered by subcutaneous infusion.
다른 이름들:
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위약 비교기: Placebo
After the Screening Period, participants will enter the study Intervention Period and will be administered study drug for up to 6 weeks. Prior to entering the Intervention Period, study participants will be randomized at a 1:1 ratio and will receive placebo. At the End of Treatment Visit, participants will enter an Observation Period of 4 to 7 weeks with an opportunity to transition to an open-label extension study. |
Placebo will be administered by subcutaneous infusion. |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Change from Baseline at Day 43 in (Myasthenia Gravis Impairment Index) MGII ocular score (Patient-Reported Outcome (PRO) part)
기간: At Day 43
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MGII is a measure of disease severity based on the signs and symptoms of MG patients. The MGII has 22 patient-reported items and 6 examination items and scores are presented as a sum of all items for a total score but also as an ocular and generalized sub-score. The scoring range is 0 to 84, 0-23 for the ocular score, and 0-61 for the generalized score, where higher scores are indicative of more severe symptoms. The recall period is "the past week" ie, the last 7 days. |
At Day 43
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Change from Baseline at Day 43 in Myasthenia Gravis Symptoms Patient-Reported Outcome (MGSPRO) ocular muscle weakness scale score
기간: At Day 43
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The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants will be asked to choose response option that best describes how frequently they experienced muscle weakness fatigability (items 34-42) over the past 7 days using a 5-point Likert scale (1="none of the time" to 5="all of the time") for each item. |
At Day 43
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Change from Baseline at Day 43 in Myasthenia Gravis Quality of Life 15-item Scale (revised version) (MG-QoL15r) total score
기간: At Day 43
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The MG-QoL15r is a brief survey, completed by the study participant, that is designed to assess some aspects of "quality of life" related to MG. The recall period that will be used is "past 7 days". When completing the 15-item MG-QoL15r, MG study participant should consider only how their MG affects these items. |
At Day 43
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Change from Baseline at Day 43 in Myasthenia Gravis Activities of Daily Living (MG-ADL) ocular subdomain score
기간: At Day 43
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The total MG-ADL score is obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement. |
At Day 43
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Change from Baseline at Day 43 in Myasthenia Gravis Impairment Index (MGII) ocular score (Physical Examination part)
기간: At Day 43
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MGII is a measure of disease severity based on the signs and symptoms of MG patients. The MGII has 22 patient-reported items and 6 examination items and scores are presented as a sum of all items for a total score but also as an ocular and generalized sub-score. The scoring range is 0 to 84, 0-23 for the ocular score, and 0-61 for the generalized score, where higher scores are indicative of more severe symptoms. The recall period is "the past week" ie, the last 7 days. |
At Day 43
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Incidence of treatment-emergent adverse events (TEAEs)
기간: Up to Week 13
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Treatment Emergent Adverse Events (TEAEs) are any untoward medical incidence in a participant after the administration of study treatment, whether or not these events are related to study treatment. |
Up to Week 13
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Incidence of treatment-emergent serious adverse events (TESAEs)
기간: Up to Week 13
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An SAE is defined as any untoward medical occurrence that, at any dose, meets 1 or more of the criteria listed:
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Up to Week 13
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Incidence of TEAEs leading to permanent withdrawal of study treatment
기간: Up to Week 13
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Treatment Emergent Adverse Events (TEAEs) are any untoward medical incidence in a participant after the administration of study treatment, whether or not these events are related to study treatment. This measure considers any TEAE leading to permanent withdrawal from study. |
Up to Week 13
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공동 작업자 및 조사자
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
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- MG0038
- 2025-524756-54-00 (씨티스)
- CTR20262520 (레지스트리 식별자: ChinaDrugTrials.org.cn)
- U1111-1334-3276 (기타 식별자: Universal Trial Number)
- 2025 (미국 NIH 보조금/계약)
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 pre-specified 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 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.
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
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