Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)

Part of paid clinical trials in Los Angeles, California.

Sponsor
Alexion Pharmaceuticals, Inc.
Study ID
NCT04469465
Phase
PHASE3
Status
Completed

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

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

Interventions

  • Danicopan — DRUG
    Oral tablet
  • Placebo — DRUG
    Oral tablet
  • C5 Inhibitor — DRUG
    Participants will continue to receive their ongoing C5 inhibitor (eculizumab or ravulizumab) therapy according to their usual dose and schedule.

Study Details

The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.

Key Dates

Start date
Dec 16, 2020
Status verified
Apr 2025
Primary completion
Jun 29, 2022
Completion
Jan 16, 2024

Study Design

Enrollment
86 participants (actual)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Danicopan + C5 Inhibitor
    Participants will receive danicopan, in addition to their C5 inhibitor therapy, for 24 weeks (12 weeks in Treatment Period 1, followed by 12 weeks in Treatment Period 2).
  • Placebo Comparator: Placebo + C5 Inhibitor
    Participants will receive placebo, in addition to their C5 inhibitor therapy, for 12 weeks during Treatment Period 1. At Week 12, participants randomized to receive placebo will be switched to danicopan for an additional 12 weeks (Treatment Period 2).

Primary Outcome Measure

Change From Baseline in Hgb at Week 12 [ Time Frame: Baseline, Week 12 ]

Locations (7)

FacilityCityStateZIPSite coordinators
Research SiteLos AngelesCalifornia90089-
Research SiteWestonFlorida33331-
Research SiteChicagoIllinois60612-
Research SiteKalamazooMichigan49007-
Research SiteNew YorkNew York10065-
Research SiteDallasTexas75390-
Research SiteMilwaukeeWisconsin53212-

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