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Active (not recruiting) Phase 3 NCT03531255

Pegcetacoplan Long Term Safety and Efficacy Extension Study

Phase 3 – large-scale trial before approval
Conditions: PNH

Sponsor: Apellis Pharmaceuticals, Inc.

trial.available_in: БГ
Overview
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
Who can participate
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 Subjects who have withdrawn from a pegcetacoplan clinical study. 2.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.
Locations 21
Australia (1)
Royal Melbourne Hospital
Melbourne , Victoria
Belgium (1)
Cliniques Universitaires Saint-luc
Brussels
Bulgaria (1)
Acibadem City Clinic Tokuda Hospital
Sofia
Canada (1)
University of Calgary
Calgary , Alberta
Colombia (1)
IPS Centro Medico Julian Coronel SAS
Cali , Valle del Cauca Department
France (1)
Hospital Center Chalon Sur Saone William Morey
Chalon-sur-Saône
Germany (1)
Universitatsklinikum Ulm
Ulm , Baden-Wurttemberg
Hong Kong (1)
Prince of Wales Hospital
Hong Kong
Japan (1)
Japanese Red Cross Nagoya Daini Hospital
Showa-ku , Aichi-ken
Malaysia (1)
University Malaya Medical Centre
Lembah Pantai , Kuala Lumpur
Mexico (1)
Hospital Universitario "José Eleuterio González", UANL
Monterrey
Peru (1)
Hospital Nacional Dos De Mayo - Centro de Investigación
Lima Cercado , Lima region
Philippines (1)
Perpetual Succour Hospital
Cebu , National Capital Region
Russia (1)
Pavlov First Saint Petersburg State Medical University
Saint Petersburg
Serbia (1)
Clinical Center of Serbia
Belgrade
Singapore (1)
Singapore General Hospital
Singapore
South Korea (1)
Chungnam National University Hospital
Junggu , Daejeon
Spain (1)
Hospital Univ. de Gran Canaria Dr.Negrin
Las Palmas de Gran Canaria , Canary Islands
Thailand (1)
Phramongkutklao Hospital
Bangkok
United Kingdom (1)
Leeds Teaching Hospitals NHS Trust
Leeds
United States (1)
University of Southern California
Los Angeles , California
Technical details
Status
Active (not recruiting)
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male and female
Minimum age
18 Years
Healthy volunteers
No
Start date
27.08.2018
Completion date
01.07.2026
Registry ID
NCT03531255
Source
anzctr
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