Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
Study Identifier:
ALXN1210-PNH-401
ClinicalTrials.gov Identifier:
EudraCT Identifier:
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete
Available Documents
Study Details
Medical Condition
- Paroxysmal Nocturnal Hemoglobinuria
Study Drug
- Biological: Eculizumab
- Biological: Ravulizumab
Date
Apr 2021 - Dec 2022
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Documented diagnosis of PNH, confirmed by high-sensitivity flow cytometry evaluation of red blood cells and white blood cells, with granulocyte or monocyte clone size of ≥ 5%.
- Received 1200 mg eculizumab every 12 to 16 days (every 2 weeks) for at least 3 months prior to Screening.
- LDH ≤ 2 x upper limit of normal (ULN) according to central laboratory, at Screening.
- To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infections within 3 years prior to initiating study drug.
- Body weight ≥ 40 kilograms.
Exclusion Criteria
- History of major adverse vascular events within 6 months of Day 1.
- History of bone marrow transplantation.
- Lymphoma, leukemia, myelodysplastic syndrome, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.
- Concomitant use of anticoagulants is prohibited if not on a stable regimen for at least 2 weeks prior to Day 1.
- Concomitant use of any of the following medications and not on a stable regimen (as judged by the Investigator) for the time period indicated prior to Screening:
- Erythropoietin or immunosuppressants for at least 8 weeks
- Systemic corticosteroids for at least 4 weeks
- Vitamin K antagonists (for example, warfarin) with a stable international normalized ratio level for at least 4 weeks
- Iron supplements or folic acid for 4 weeks
- Live vaccine(s) within 1 month prior to Screening or plans to receive such vaccines during the study.
- More than 1 LDH value \> 2 × ULN within the 6 months prior to Day 1.
- Platelet count \< 30,000/cubic millimeter (30 × 10\^9/Liter \[L\]) at Screening.
- Absolute neutrophil count \< 500/microliter (0.5 × 10\^9/L) at Screening.
Sex
Female & Male
Age
N/A
Study Details
Medical Condition
- Paroxysmal Nocturnal Hemoglobinuria
Study Drug
- Biological: Eculizumab
- Biological: Ravulizumab
Date
Apr 2021 - Dec 2022
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Documented diagnosis of PNH, confirmed by high-sensitivity flow cytometry evaluation of red blood cells and white blood cells, with granulocyte or monocyte clone size of ≥ 5%.
- Received 1200 mg eculizumab every 12 to 16 days (every 2 weeks) for at least 3 months prior to Screening.
- LDH ≤ 2 x upper limit of normal (ULN) according to central laboratory, at Screening.
- To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infections within 3 years prior to initiating study drug.
- Body weight ≥ 40 kilograms.
Exclusion Criteria
- History of major adverse vascular events within 6 months of Day 1.
- History of bone marrow transplantation.
- Lymphoma, leukemia, myelodysplastic syndrome, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.
- Concomitant use of anticoagulants is prohibited if not on a stable regimen for at least 2 weeks prior to Day 1.
- Concomitant use of any of the following medications and not on a stable regimen (as judged by the Investigator) for the time period indicated prior to Screening:
- Erythropoietin or immunosuppressants for at least 8 weeks
- Systemic corticosteroids for at least 4 weeks
- Vitamin K antagonists (for example, warfarin) with a stable international normalized ratio level for at least 4 weeks
- Iron supplements or folic acid for 4 weeks
- Live vaccine(s) within 1 month prior to Screening or plans to receive such vaccines during the study.
- More than 1 LDH value \> 2 × ULN within the 6 months prior to Day 1.
- Platelet count \< 30,000/cubic millimeter (30 × 10\^9/Liter \[L\]) at Screening.
- Absolute neutrophil count \< 500/microliter (0.5 × 10\^9/L) at Screening.
Protocol Summary
The primary purpose of this study is to assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab in participants who are prescribed and are receiving a higher than approved dose of eculizumab to treat paroxysmal nocturnal hemoglobinuria (PNH).
Trial Locations
Location
Status
Location
Clinical Study Site
Leeds, United Kingdom, LS9 7TF
Status
N/A
Location
Research Site
Leeds, United Kingdom, LS9 7TF
Status
N/A
Location
Clinical Study Site
London, United Kingdom, SE5 9RS
Status
N/A
Location
Research Site
London, United Kingdom, SE5 9RS
Status
N/A