NCT02264639

A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH

An Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Apellis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study will be the initial exploration of pegcetacoplan in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of pegcetacoplan will guide decisions to further develop the drug.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

Type	Name	Description
DRUG	Pegcetacoplan	Complement (C3) Inhibitor

Timeline

• Start date: 2015-02-23
• Primary completion: 2018-10-22
• Completion: 2018-10-22
• First posted: 2014-10-15
• Last updated: 2021-01-08
• Results posted: 2021-01-08

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study