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

Part of paid clinical trials in Los Angeles, California.

Sponsor
Apellis Pharmaceuticals, Inc.
Study ID
NCT02264639
Phase
PHASE1
Status
Completed

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eligibility Criteria

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

Interventions

Study Details

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.

Key Dates

Start date
Feb 23, 2015
Status verified
Dec 2020
Primary completion
Oct 22, 2018
Completion
Oct 22, 2018

Study Design

Enrollment
9 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
    First Dose 25mg, Repeated Dose 5 mg/day
  • Experimental: Cohort 2
    First Dose 50 mg, Repeated Dose 30 mg/day
  • Experimental: Cohort 3
    Repeated Dose 180 mg/day
  • Experimental: Cohort 4
    Repeated Dose 270 mg/day

Primary Outcome Measure

Number of Subjects With Treatment-Emergent Adverse Events (TEAEs), Including by Severity, During Single-dose Phase [ Time Frame: From single dose of study drug (Day 1) up to 30 days ]

Locations (7)

FacilityCityStateZIPSite coordinators
University of Southern California Norris Comprehensive Cancer CenterLos AngelesCalifornia90033-
Lakes ResearchMiami LakesFlorida33014-
University of LousivilleLouisvilleKentucky40202-
John Hopkins HospitalBaltimoreMaryland21231-
Cure 4 The Kids FoundationLas VegasNevada89135-
Duke University Medical CenterDurhamNorth Carolina27710-
Cleveland ClinicClevelandOhio44195-

Find similar trials in Los Angeles, CA

By research site
University of Southern California Norris Comprehensive Cancer Center· Los Angeles, CALakes Research· Miami Lakes, FLUniversity of Lousiville· Louisville, KYJohn Hopkins Hospital· Baltimore, MDCure 4 The Kids Foundation· Las Vegas, NVDuke University Medical Center· Durham, NC

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