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Factor D Inhibitor

ALXN2050 for Paroxysmal Nocturnal Hemoglobinuria

Phase 2
Waitlist Available
Research Sponsored by Alexion Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female, ≥ 18 years of age
Stable background regimen of at least 24 weeks for eculizumab without change in dose or interval for at least the past 8 weeks (for Group 2)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up long-term extension period
Awards & highlights

Study Summary

This trial will test the efficacy and safety of the oral Factor D inhibitor ALXN2050 in patients with PNH who are treatment naïve or currently treated with eculizumab or ALXN2040.

Who is the study for?
This trial is for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who either haven't been treated, still have anemia after eculizumab treatment, or are on danicopan. They must have certain levels of blood cells and enzymes indicating PNH. People can't join if they've had organ transplants, other bone marrow issues not related to PNH, or severe kidney problems.Check my eligibility
What is being tested?
The study tests ALXN2050 as a solo treatment for PNH. It checks how well it works and its safety in new patients, those previously on eculizumab with ongoing anemia, and those taking danicopan alone. Participants will be checked regularly up to Week 12 then continue into a long-term part of the study.See study design
What are the potential side effects?
Possible side effects aren't specified here but may include reactions typical of oral medications targeting the immune system such as gastrointestinal symptoms, potential liver enzyme changes, and increased risk of infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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I have been on a stable eculizumab dose for at least 24 weeks.
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I have been diagnosed with PNH.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 12
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 12 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change In HgB Relative To Baseline
Secondary outcome measures
Change From Baseline In Absolute Reticulocyte Count
Change From Baseline In C3 Complement Protein Fragment Deposition On PNH RBCs
Change From Baseline In Direct Bilirubin
+9 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Open-label ALXN2050 MonotherapyExperimental Treatment1 Intervention
Experimental: Open-label ALXN2050 Monotherapy ALXN2050 orally administered Group 1: Patients with PNH who are treatment naïve Group 2: Patient with PNH who have received complement component 5 (C5) inhibition with eculizumab for at least 6 months, who continue to experience anemia and reticulocytes above the upper limit of normal (ULN) Group 3: Patients with PNH who have received danicopan monotherapy during study ACH471-103
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
ALXN2050
2021
Completed Phase 1
~250

Find a Location

Who is running the clinical trial?

Alexion Pharmaceuticals, Inc.Lead Sponsor
252 Previous Clinical Trials
41,223 Total Patients Enrolled
AlexionLead Sponsor
246 Previous Clinical Trials
39,348 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,494 Total Patients Enrolled

Media Library

ALXN2050 (Factor D Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04170023 — Phase 2
Paroxysmal Nocturnal Hemoglobinuria Research Study Groups: Open-label ALXN2050 Monotherapy
Paroxysmal Nocturnal Hemoglobinuria Clinical Trial 2023: ALXN2050 Highlights & Side Effects. Trial Name: NCT04170023 — Phase 2
ALXN2050 (Factor D Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04170023 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is there still time to enroll in this experiment?

"This clinical trial is currently seeking participants. The necessary information was first made available on December 16th 2019, and the most recent update occurred October 20th 2022."

Answered by AI

What is the aggregate figure of individuals participating in this clinical research?

"Affirmative. Evidence on clinicaltrials.gov indicates that this research project, which was initially posted on December 16th 2019, is actively recruiting participants. 26 individuals must be recruited from 2 different medical centres."

Answered by AI

What is the regulatory status of ALXN2050?

"The safety of ALXN2050 has been assigned a score of 2, since there is evidence supporting its safety profile but no data confirming efficacy."

Answered by AI
~6 spots leftby Mar 2025