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Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy

NCT04170023 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2024-11-20

Study results available
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Summary

The study will evaluate the efficacy and safety of the oral Factor D (FD) inhibitor ALXN2050 (ACH-0145228) monotherapy in patients with PNH that are treatment naïve, or patients currently treated with eculizumab who still experience anemia and reticulocytosis, or patients currently treated with ALXN2040 (danicopan) as monotherapy. After signing consent, participants will have periodic visits through Week 12, at which time the primary endpoint and key secondary assessments will be analyzed. Participants will continue on treatment past 12 weeks into a long-term extension portion of the trial.

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

DRUG

ALXN2050

Oral FD inhibitor

Sponsors & Collaborators

  • Alexion Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-12-16
Primary Completion
2023-04-27
Completion
2024-03-20
FDA Drug
Yes

Countries

  • Canada
  • Italy
  • New Zealand
  • South Korea
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04170023 on ClinicalTrials.gov