Status:
COMPLETED
A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
Up to 17 years
Phase:
PHASE3
Brief Summary
The purpose of this study was to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ravulizumab in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH).
Detailed Description
The study consists of a 4-week Screening Period, a 26-week Primary Evaluation Period, and an Extension Period of up to 4 years (with the exception of any country-specific mandates), whichever occurs f...
Eligibility Criteria
Inclusion
- Participants from birth up to \<18 years of age and weighing ≥ 5 kilograms at the time of consent.
- PNH diagnosis confirmed by documented high-sensitivity flow cytometry.
- Presence of 1 or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia, history of a major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction; or history of packed red blood cell transfusion due to PNH.
- Lactate dehydrogenase (LDH) level ≥ 1.5 × upper limit of normal (ULN) for participants not being treated with eculizumab at screening and LDH level ≤ 1.5 × ULN for participants taking eculizumab.
- Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae.
- Female participants of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
Exclusion
- History of bone marrow transplantation.
- History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation.
- Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH).
- Females who are pregnant or breastfeeding or who have a positive pregnancy test at screening or Day 1.
- Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater.
Key Trial Info
Start Date :
February 22 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 25 2022
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT03406507
Start Date
February 22 2018
End Date
August 25 2022
Last Update
March 27 2023
Active Locations (9)
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1
Clinical Trial Site
Atlanta, Georgia, United States, 30329
2
Clinical Trial Site
Milwaukee, Wisconsin, United States, 53226
3
Clinical Trial Site
Paris, France
4
Clinical Trial Site
Utrecht, Netherlands