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Pegcetacoplan 장기 안전성 및 효능 확장 연구

2026년 10월 16일 업데이트: Apellis Pharmaceuticals, Inc.

발작성 야간 혈색소뇨증(PNH) 치료에서 페그세타코플란의 장기적 안전성과 효능을 평가하기 위한 공개, 비무작위, 다기관 확장 연구

This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.

연구 개요

상태

완전한

정황

개입 / 치료

연구 유형

중재적

등록 (실제)

137

단계

  • 3단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 장소

  • 대한민국
    • Daejeon
      • Junggu, Daejeon, 대한민국, 35015
        • Chungnam National University Hospital
  • 독일
      • Hamburg, 독일, 20246
        • University Medical Center Hamburg-Eppendorf
    • Baden-Wurttemberg
      • Ulm, Baden-Wurttemberg, 독일, 89081
        • Universitatsklinikum Ulm
    • North Rhine-Westphalia
      • Aachen, North Rhine-Westphalia, 독일, 52074
        • Uniklinik RWTH Aachen
      • Essen, North Rhine-Westphalia, 독일, 45147
        • Essen University Hospital Department of Hematology
  • 러시아 제국
      • Saint Petersburg, 러시아 제국, 197022
        • Pavlov First Saint Petersburg State Medical University
      • Tyumen, 러시아 제국, 625023
        • Therapeutic hospital of regional clinical hospital #1
  • 말레이시아
    • Kuala Lumpur
      • Lembah Pantai, Kuala Lumpur, 말레이시아, 59100
        • University Malaya Medical Centre
    • Selangor
      • Ampang, Selangor, 말레이시아, 68000
        • Hospital Ampang
  • 멕시코
      • Monterrey, 멕시코, 64460
        • Hospital Universitario "José Eleuterio González", UANL
  • 미국
    • California
      • Los Angeles, California, 미국, 90033
        • University of Southern California
    • Colorado
      • Denver, Colorado, 미국, 80204
        • Denver Health Medical Center
    • Florida
      • Jacksonville, Florida, 미국, 32257
        • Cancer Specialists of North Florida
      • Miami, Florida, 미국, 33014
        • Lakes Research
    • Illinois
      • Chicago, Illinois, 미국, 60611
        • Northwestern University
    • Indiana
      • Indianapolis, Indiana, 미국, 46260
        • Investigative Clinical Research of Indiana
    • North Carolina
      • Durham, North Carolina, 미국, 27710
        • Duke University Medical Center
    • Ohio
      • Cleveland, Ohio, 미국, 44195
        • Cleveland Clinic
    • Tennessee
      • Memphis, Tennessee, 미국, 38120
        • Baptist Cancer Center
  • 벨기에
      • Brussels, 벨기에, 1200
        • Cliniques Universitaires Saint-luc
      • Roeselare, 벨기에, 8800
        • AZ Delta
  • 불가리아
      • Sofia, 불가리아, 1407
        • Acibadem City Clinic Tokuda Hospital
      • Sofia, 불가리아, 1756
        • National Specialized Hospital for Active Treatment of Haematologic Diseases
  • 세르비아
      • Belgrade, 세르비아, 11000
        • Clinical Center of Serbia
  • 스페인
      • Valencia, 스페인, 46026
        • Hospital Universitario Politecnico La Fe
    • Canary Islands
      • Las Palmas de Gran Canaria, Canary Islands, 스페인, 35019
        • Hospital Univ. de Gran Canaria Dr.Negrin
  • 싱가포르
      • Singapore, 싱가포르, 169608
        • Singapore General Hospital
  • 영국
      • Leeds, 영국, LS9 7TF
        • Leeds Teaching Hospitals NHS Trust
  • 일본
      • Okayama, 일본, 700-8558
        • Okayama University Hospital
      • Tokyo, 일본, 141-0022
        • NTT Medical Center Tokyo
    • Aichi-ken
      • Showa-ku, Aichi-ken, 일본, 486-8650
        • Japanese Red Cross Nagoya Daini Hospital
    • Nagano
      • Matsumoto, Nagano, 일본, 390-8621
        • Shinshu University Hospital
    • Tokyo
      • Bunkyō-Ku, Tokyo, 일본, 113-8421
        • Juntendo University Hospital
    • Wakayama
      • Tanabe, Wakayama, 일본, 646-8588
        • Kinan Hospital
  • 캐나다
    • Alberta
      • Calgary, Alberta, 캐나다, T2N 4Z6
        • University of Calgary
      • Edmonton, Alberta, 캐나다, T6G 2V2
        • Alberta Health Services
    • Ontario
      • Toronto, Ontario, 캐나다, M5G 2C4
        • Toronto General Hospital
  • 콜롬비아
    • Valle del Cauca Department
      • Cali, Valle del Cauca Department, 콜롬비아, 760035
        • IPS Centro Medico Julian Coronel SAS
  • 태국
      • Bangkok, 태국, 10400
        • Phramongkutklao Hospital
      • Bangkok, 태국, 10400
        • Ramathibodi Hospital
      • Bangkok, 태국, 10400
        • Siriraj Hospital
      • Chiang Mai, 태국, 50200
        • Maharaj Nakorn Chiang Mai Hospital
      • Khon Kaen, 태국, 40002
        • Srinagarind Hospital
      • Pathum Thani, 태국, 12120
        • Thammasat University Hospital
      • Songkhla, 태국, 90110
        • Songklanagarind Hospital
  • 페루
    • Lima
      • Jesús María, Lima, 페루, 15072
        • Hospital Edgardo Rebagliatti
    • Lima region
      • Lima Cercado, Lima region, 페루, 15003
        • Hospital Nacional Dos De Mayo - Centro de Investigación
      • San Isidro, Lima region, 페루, 15047
        • Centro de Investigacion en Hematologia Clinica del Centro Medico Corpac
  • 프랑스
      • Chalon-sur-Saône, 프랑스, 71100
        • Hospital Center Chalon Sur Saone William Morey
      • Lille, 프랑스, 59037
        • CHRU de Lille
      • Paris, 프랑스, 75010
        • Hopital Saint-Louis
      • Pierre-Bénite, 프랑스, 69495
        • Lyon Sud Hospital Center
      • Pringy, 프랑스, 74374
        • Centre Hospitalier Annecy Genevois - Site Annecy
      • Saint-Quentin, 프랑스, 02321
        • Centre Hospitalier de Saint-Quentin
  • 필리핀 제도
    • National Capital Region
      • Cebu, National Capital Region, 필리핀 제도, 6000
        • Perpetual Succour Hospital
      • Lipa City, National Capital Region, 필리핀 제도, 4217
        • Mary Mediatrix Medical Centre
      • Makati City, National Capital Region, 필리핀 제도, 1229
        • Makati Medical Centre
      • Pasig, National Capital Region, 필리핀 제도, 1604
        • The Medical City
      • Quezon City, National Capital Region, 필리핀 제도, 1112
        • St. Lukes Medical Centre
  • 호주
    • Victoria
      • Melbourne, Victoria, 호주, 3000
        • Royal Melbourne Hospital
  • 홍콩
      • Hong Kong, 홍콩
        • Queen Mary Hospital
      • Hong Kong, 홍콩
        • Prince of Wales Hospital

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

18년 이상 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

Inclusion criteria:

  1. Subjects at least 18 years of age with PNH who have participated in a pegcetacoplan clinical trial. Subjects who received treatment with pegcetacoplan must have experienced clinical benefit and adequate tolerability in the opinion of the investigator.

    Note: Subjects with PNH who completed a pegcetacoplan clinical trial without receiving pegcetacoplan (or without receiving pegcetacoplan for long enough to demonstrate clinical benefit) may be enrolled in this study if, in the opinion of the Investigator, the subject is expected to demonstrate clinical benefit upon the initiation or continuation of pegcetacoplan therapy.

  2. Vaccination against Neisseria meningitidis types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing of this study, or within 14 days after starting treatment with pegcetacoplan. Vaccination is mandatory unless documented evidence exists that subjects are nonresponders to vaccination as evidenced by titers or display titer levels within acceptable local limits. Immunization status checks will be performed to determine whether subjects require primary or booster vaccinations.

  3. Willing and able to give written informed consent.

  4. Willing and able to self-administer pegcetacoplan (administration by caregiver will be allowed)

  5. Women of childbearing potential (WOCBP) defined as any females who have experienced menarche and who are NOT permanently sterile or postmenopausal must have a negative pregnancy test and must agree to continue to use an approved method of contraception for the duration of the study and 90 days after their last dose of study drug. Note: Postmenopausal is defined as 12 consecutive months with no menses without an alternative medical cause.

  6. Males must agree to continue to use an approved method of contraception and must agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug.

Exclusion Criteria:

  1. Subjects who have withdrawn from a pegcetacoplan clinical study.
  2. Any condition that could increase the subject's risk by participating in the study.
  3. Any comorbidity or condition (such as malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
  4. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
  5. Known infection with hepatitis B, C, or HIV.
  6. Hereditary complement deficiency.
  7. History of bone marrow transplant.
  8. Concurrent severe aplastic anemia (SAA), defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or anti-thymocyte globulin.
  9. History of meningococcal disease.
  10. Concomitant treatment with any complement inhibitor (eg, eculizumab, ravulizumab).
  11. Pregnancy, breastfeeding, or positive pregnancy test.

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: 1,080 mg pegcetacoplan administered subcutaneously

1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.
의약품: Pegcetacoplan

Complement (C3) Inhibitor

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
기간: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)

An adverse event (AE) is any untoward medical occurrence associated with use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign, including a clinically significant abnormal laboratory finding, symptom, or disease temporally associated with the use of a study drug, whether or not considered related to the study drug. TEAEs were defined as those AEs that occurred or worsened in severity after the first dose of pegcetacoplan.
From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
Number of Participants With Thromboembolic Events
기간: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
Change From Baseline in Hematology Parameters (Erythrocytes)
기간: Baseline, Weeks 168 and 264
Baseline, Weeks 168 and 264
Change From Baseline in Hematology Parameters (Leukocytes, Platelets and Reticulocytes)
기간: Baseline, Weeks 168 and 264
Baseline, Weeks 168 and 264
Change From Baseline in Hematology Parameters (Hematocrit)
기간: Baseline, Weeks 168 and 264
Baseline, Weeks 168 and 264
Change From Baseline in Hematology Parameters (Hemoglobin)
기간: Baseline, Weeks 168 and 264
Baseline, Weeks 168 and 264
Change From Baseline in Serum Chemistry Parameters [Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Lactate Dehydrogenase (LDH)]
기간: Baseline, Weeks 168 and 264
Baseline, Weeks 168 and 264
Change From Baseline in Serum Chemistry Parameters (Bilirubin)
기간: Baseline, Weeks 168 and 264
Baseline, Weeks 168 and 264
Number of Participants With Post-Baseline Potentially Clinically Significant Abnormal Coagulation Parameters
기간: Baseline, Week 264

Coagulation parameters included Activated Partial Thromboplastin Time (APTT), D-Dimer, Fibrinogen, Prothrombin Intl. Normalized Ratio and Prothrombin Time.
Baseline, Week 264
Number of Participants With Post-Baseline Potentially Clinically Significant Abnormal Urinalysis Parameters
기간: Baseline, Week 264

Urinalysis parameters included ketones, leukocyte esterase, nitrite, occult blood, pH, protein, specific gravity and urobilinogen.
Baseline, Week 264
Number of Participants With Shift From Baseline in Clinically Significant Abnormalities in Electrocardiograms (ECG)
기간: Baseline, Week 324

ECG measurements included heart rate, PR, QT, QRS, QTcB and QTcF.
Baseline, Week 324
Change From Baseline in Hemoglobin Concentration
기간: Baseline, Week 324
Baseline, Week 324
Change From Baseline in Lactate Dehydrogenase (LDH) Concentration
기간: Baseline, Week 324
Baseline, Week 324
Change From Baseline in Indirect Bilirubin Level
기간: Baseline, Week 324
Baseline, Week 324
Change From Baseline in Absolute Reticulocyte Count (ARC)
기간: Baseline, Week 324
Baseline, Week 324
Number of Participants Who Received Packed Red Blood Cell Transfusions (PRBCs)
기간: From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)

Transfusion included whole blood, PRBCs, leukocyte-depleted red blood cells (LDPRCs), and all types of red blood cell (RBC) transfusions.
From first dose of study drug up to 8 weeks from last dose (up to approximately 4 years or until pegcetacoplan development in participants with PNH was terminated)
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score
기간: Baseline, Week 324

The FACIT-Fatigue scale consists of 13 questions that assess fatigue and its impact on daily activities over the past 7 days. Each question is scored on a 5-point scale from 0 ("not at all") to 4 ("very much"). For items where a higher response indicates greater fatigue, the score is reversed so that all items are scored in the same direction. The scores from the 13 items are then added together to calculate the FACIT-Fatigue total score. The total score ranges from 0 to 52, with higher scores indicating less fatigue and better functioning.
Baseline, Week 324

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Apellis Pharmaceuticals, Inc.

간행물 및 유용한 링크

연구에 대한 정보 입력을 담당하는 사람이 자발적으로 이러한 간행물을 제공합니다. 이것은 연구와 관련된 모든 것에 관한 것일 수 있습니다.

일반 간행물

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 (실제)

2018년 8월 27일

기본 완료 (실제)

2025년 9월 4일

연구 완료 (실제)

2026년 5월 19일

연구 등록 날짜

최초 제출

2018년 5월 9일

QC 기준을 충족하는 최초 제출

2018년 5월 9일

처음 게시됨 (실제)

2018년 5월 21일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 11월 10일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 10월 16일

마지막으로 확인됨

2026년 10월 1일

추가 정보

이 연구와 관련된 용어

추가 관련 MeSH 약관

기타 연구 ID 번호

  • APL2-307

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

미국에서 제조되어 미국에서 수출되는 제품

아니

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. .

구독하다