Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.

Sponsor
Apellis Pharmaceuticals, Inc.
Study ID
NCT02588833
Phase
PHASE1
Status
Completed

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

Study Details

The objectives of the study are to assess the safety, tolerability, preliminary efficacy and PK of multiple subcutaneous (SC) doses of pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have not received treatment with eculizumab in the past. An exploratory objective of the study is to assess the pharmacodynamics (PD) of multiple SC doses of pegcetacoplan when administered to PNH patients.

Key Dates

Start date
Dec 1, 2015
Status verified
Dec 2020
Primary completion
Aug 26, 2019
Completion
Aug 26, 2019

Study Design

Enrollment
23 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
    180 mg pegcetacoplan/day
  • Experimental: Cohort 2
    270 mg pegcetacoplan/day

Primary Outcome Measure

Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity [ Time Frame: From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. ]

Related Studies