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Factor D Inhibitor
ALXN2050 for Paroxysmal Nocturnal Hemoglobinuria
Phase 2
Waitlist Available
Research Sponsored by Alexion
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Diagnosis of PNH
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 12, week 160
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 belowSelect...
I have been diagnosed with PNH.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ week 12
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 12
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 moreTrial 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
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Paroxysmal Nocturnal Hemoglobinuria (PNH) is commonly treated with complement inhibitors such as eculizumab and ravulizumab, which block the complement protein C5, preventing the formation of the membrane attack complex that leads to red blood cell destruction. Factor D inhibitors, like the investigational drug ALXN2050, target an earlier step in the complement pathway by inhibiting Factor D, a crucial enzyme in the alternative pathway activation.
This inhibition prevents the amplification of the complement cascade, reducing hemolysis and thrombosis risk. These treatments are vital for PNH patients as they address the underlying cause of hemolysis, improving quality of life and reducing complications associated with the disease.
[The Outcome of Severe Aplastic Anemia Children Treated with Reduced Dose of Cyclophosphamide Combined Cyclosporine A].
[The Outcome of Severe Aplastic Anemia Children Treated with Reduced Dose of Cyclophosphamide Combined Cyclosporine A].
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Who is running the clinical trial?
AlexionLead Sponsor
246 Previous Clinical Trials
39,246 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,392 Total Patients Enrolled
Alexion Pharmaceuticals, Inc.Lead Sponsor
254 Previous Clinical Trials
41,206 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have a specific type of blood cell clone that is larger than 10%.I am 18 years old or older.Your LDH levels in the blood are higher than 1.5 times the normal limit.I have a bleeding disorder or condition causing anemia not related to PNH.I have had a major organ or bone marrow transplant.I have a bone marrow condition needing a stem cell transplant or have been on immunosuppressants for less than 6 months.I have been treated with a complement inhibitor before.Your blood has low levels of hemoglobin (Hgb <10.5 g/dL).Your absolute reticulocyte count is 100 billion per liter or more.My kidney function is very low or I am on dialysis.You need to have at least 30,000 platelets in a small drop of your blood.You have taken danicopan during Study ACH471-103 in Group 3.Your body has enough infection-fighting white blood cells.I have been on a stable eculizumab dose for at least 24 weeks.I have been diagnosed with PNH.
Research Study Groups:
This trial has the following groups:- Group 1: Open-label ALXN2050 Monotherapy
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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