Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
Study Identifier:
ACH228-110
ClinicalTrials.gov Identifier:
EudraCT Identifier:
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other
Available Documents
Study Details
Medical Condition
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study Drug
- Drug: ALXN2050
Date
Dec 2019 - Apr 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Diagnosis of PNH.
- Male or female, ≥ 18 years of age
- Eligibility Criteria:
- Eligibility Criteria Specific for Group 1:
- PNH Patients who have no history of treatment with any complement inhibitor at any dose.
- PNH Type III erythrocyte or granulocyte clone size ≥10%
- Absolute reticulocyte count ≥100×10\^9/liter \[L\].
- Anemia (Hgb \<10.5 grams/deciliter \[g/dL\]).
- LDH ≥1.5× upper limit of normal.
- Platelet count ≥30,000/microliter (µL)
- Absolute neutrophil count (ANC) ≥750/ µL.
- Eligibility Criteria Specific for Group 2:
- Stable background regimen of at least 24 weeks for eculizumab without change in dose or interval for at least the past 8 weeks
- Anemia (Hgb \<10 g/dL)
- Absolute reticulocyte count ≥100×10\^9/L
- Platelet count ≥30,000/µL
- Absolute neurophil count (ANC) ≥750/ µL
- Eligibility Criteria Specific for Group 3:
- 1\. Patient received danicopan during Study ACH471-103
Exclusion Criteria
- History of a major organ transplant or hematopoietic stem cell/marrow transplant .
- Known aplastic anemia or other bone marrow failure that requires HSCT, or if these patients are on immunosuppressive agents for less than 24 weeks.
- Known underlying bleeding disorders or any other conditions leading to anemia not primarily associated with PNH.
- Estimated glomerular filtration rate \<30 milliliters/minute/1.73 meters squared and/or are on dialysis.
Sex
Female & Male
Age
18+ years
Study Details
Medical Condition
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study Drug
- Drug: ALXN2050
Date
Dec 2019 - Apr 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Diagnosis of PNH.
- Male or female, ≥ 18 years of age
- Eligibility Criteria:
- Eligibility Criteria Specific for Group 1:
- PNH Patients who have no history of treatment with any complement inhibitor at any dose.
- PNH Type III erythrocyte or granulocyte clone size ≥10%
- Absolute reticulocyte count ≥100×10\^9/liter \[L\].
- Anemia (Hgb \<10.5 grams/deciliter \[g/dL\]).
- LDH ≥1.5× upper limit of normal.
- Platelet count ≥30,000/microliter (µL)
- Absolute neutrophil count (ANC) ≥750/ µL.
- Eligibility Criteria Specific for Group 2:
- Stable background regimen of at least 24 weeks for eculizumab without change in dose or interval for at least the past 8 weeks
- Anemia (Hgb \<10 g/dL)
- Absolute reticulocyte count ≥100×10\^9/L
- Platelet count ≥30,000/µL
- Absolute neurophil count (ANC) ≥750/ µL
- Eligibility Criteria Specific for Group 3:
- 1\. Patient received danicopan during Study ACH471-103
Exclusion Criteria
- History of a major organ transplant or hematopoietic stem cell/marrow transplant .
- Known aplastic anemia or other bone marrow failure that requires HSCT, or if these patients are on immunosuppressive agents for less than 24 weeks.
- Known underlying bleeding disorders or any other conditions leading to anemia not primarily associated with PNH.
- Estimated glomerular filtration rate \<30 milliliters/minute/1.73 meters squared and/or are on dialysis.
Protocol 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.
Trial Locations
Location
Status
Location
Research Site
Toronto, Ontario, Canada, M5G2C4
Status
N/A
Location
Research Site
Lévis, Quebec, Canada, G6V 3Z1
Status
N/A
Location
Research Site
Avellino, Italy, 83100
Status
N/A
Location
Research Site
Florence, Italy, 50134
Status
N/A
Location
Research Site
Christchurch, New Zealand, 8011
Status
N/A
Location
Research Site
Grafton, New Zealand, 1010
Status
N/A
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