A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH
- Sponsor
- Apellis Pharmaceuticals, Inc.
- Study ID
- NCT03593200
- Phase
- PHASE2
- Status
- Completed
Conditions
- PNH
Eligibility Criteria
- Sex
- ALL
- Age
- 18 Years - N/A
- Healthy Volunteers
- Not accepted
Interventions
- Pegcetacoplan — DRUGComplement (C3) Inhibitor
Study Details
This is a Phase IIa, open-label, multiple dose, study in patients with PNH who have not received eculizumab (Soliris ®) in the past. A single cohort of subjects is planned for evaluation.
Key Dates
- Start date
- Aug 16, 2018
- Status verified
- Dec 2020
- Primary completion
- Oct 22, 2019
- Completion
- Oct 22, 2019
Study Design
- Enrollment
- 4 participants (actual)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: Experimental: Cohort 1270 mg/day (up to 360 mg/day from Day 29) from Day 1 to Day 364\*
Primary Outcome Measure
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity [ Time Frame: From Day 1 to 30 days after the last dose (approximately 56 weeks) ]