Status:
COMPLETED
Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
Phase:
PHASE4
Brief 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 d...
Eligibility Criteria
Inclusion
- Key
- 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.
- Key
Exclusion
- 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.
Key Trial Info
Start Date :
April 14 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 20 2022
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT04320602
Start Date
April 14 2021
End Date
December 20 2022
Last Update
September 5 2024
Active Locations (4)
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1
Clinical Study Site
Leeds, United Kingdom, LS9 7TF
2
Research Site
Leeds, United Kingdom, LS9 7TF
3
Clinical Study Site
London, United Kingdom, SE5 9RS
4
Research Site
London, United Kingdom, SE5 9RS