Pegcetacoplan Long Term Safety and Efficacy Extension Study

Phase 3

Active, not recruiting

• Conditions: PNH
• Interventions: Drug: Pegcetacoplan

• Registration Number: NCT03531255
• Lead Sponsor: Apellis Pharmaceuticals, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-05-09
• Study First Submitted QC: 2018-05-09
• Study First Posted: 2018-05-21
• Study Start: 2018-08-27
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-27
• Last Update Posted: 2025-03-28
• Primary Completion: 2025-06
• Study Completion: 2025-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 160

Inclusion Criteria

Not provided

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 Subjects who have withdrawn from a pegcetacoplan clinical study.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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1,080 mg pegcetacoplan administered subcutaneously	Pegcetacoplan	1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.

Primary Outcome Measures

Name	Time	Method
Incidence and severity of treatment-emergent adverse events	Baseline to 2 Years

Trial Locations

Locations (62)

University of Southern California; 🇺🇸 Los Angeles, California, United States

Denver Health Medical Center; 🇺🇸 Denver, Colorado, United States

Cancer Specialists of North Florida; 🇺🇸 Jacksonville, Florida, United States

Lakes Research; 🇺🇸 Miami, Florida, United States

Northwestern University; 🇺🇸 Chicago, Illinois, United States

Investigative Clinical Research of Indiana; 🇺🇸 Indianapolis, Indiana, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

Baptist Cancer Center; 🇺🇸 Memphis, Tennessee, United States

Royal Melbourne Hospital; 🇦🇺 Melbourne, Victoria, Australia

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