Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
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- STATUS
- Recruiting
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- End date
- Jul 31, 2025
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- participants needed
- 26
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- sponsor
- Alexion Pharmaceuticals
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.
Description
Experimental: Open-label ALXN2050 Monotherapy orally
Group 1: Patients with PNH who are treatment naïve
Group 2: Patients 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) who will switch to ALXN2050 monotherapy
Group 3: Patients with PNH receiving danicopan monotherapy in study ACH471-103 will switch to ALXN2050 monotherapy
After signing the informed consent form, participants will enter the screening period. During the Screening Period, eligibility and screening assessments will be performed. Screening assessments may be spread over more than one visit if necessary. At the baseline visit, screened participants who continue to meet eligibility criteria will enter the Treatment Period.
The treatment phase will be followed by a long-term extension phase, where ALXN2050 will continue to be administered.
Blood will be collected to assess the efficacy endpoints, such as, change in hemoglobin (Hgb), lactate dehydrogenase (LDH), and other measures of hemolysis. Safety and transfusion requirements will also be assessed.
Participants will continue on treatment past 12 weeks in a long-term extension portion of the trial.
Details
| Condition | Paroxysmal Nocturnal Hemoglobinuria (PNH) |
|---|---|
| Treatment | ALXN2050, C5 inhibitor background therapy |
| Clinical Study Identifier | NCT04170023 |
| Sponsor | Alexion Pharmaceuticals |
| Last Modified on | 17 June 2022 |
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
Learn moreIf you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Learn moreComplete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.
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