Phase 2
N=26
Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria · PNH
Bottom Line
View on ClinicalTrials.gov: NCT02605993 ↗Enrolled (actual)
26
Serious AEs
42.3%
Results posted
Feb 2019
Primary outcome: Primary: Percent Change In LDH Levels From Baseline To Day 253 And Day 281 — -72.85; -78.12; -84.96; -87.63 Percent Change — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ravulizumab (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Feb 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change In LDH Levels From Baseline To Day 253 And Day 281 |
-72.85; -78.12; -84.96; -87.63; -89.89 | <0.0001 sig |
| SECONDARY Percent Change In Free Hemoglobin Levels From Baseline To Day 253 And Day 281 |
14.07; -12.32; -22.14; -40.80; -45.64 | 0.0214 sig |
| SECONDARY Percent Change In Haptoglobin Levels From Baseline To Day 253 And Day 281 |
21.67; 81.67; 4.29; 34.29; 27.14 | 0.0625 |
| SECONDARY Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 253 And Day 281 |
-1.27; -5.05; 10.46; 14.66; -4.66 | 0.4871 |
| SECONDARY Percent Change In PNH RBC Types II And III Clone Size From Baseline To Day 253 |
6.65; 5.47; 54.14; 88.21 | 0.0023 sig |
| SECONDARY Percent Change In D-dimer From Baseline To Day 253 And Day 281 |
-24.80; -29.47; -10.90; -16.08; -27.05 | 0.0029 sig |
| SECONDARY Change In Clinical Manifestations Of PNH From Baseline To Day 253 And Day 281 |
4; 2; 3; 3; 0; 0 | — |
Summary
The primary purpose of this study is to evaluate the safety, tolerability, and efficacy of multiple intravenous (IV) doses of ravulizumab administered to complement inhibitor treatment-naïve participants with PNH.
Eligibility Criteria
Inclusion Criteria
- Male or female ≥18 years of age
- PNH diagnosis confirmed by documented high-sensitivity flow cytometry
- Documented meningococcal vaccination not more than 3 years prior to dosing
- Female participants of childbearing potential were to use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
- Willing and able to give written informed consent and comply with the study visit schedule
Exclusion Criteria
- Treatment with a complement inhibitor at any time
- Female participants who are planning to become pregnant, or are pregnant, breastfeeding or who had a positive pregnancy test at screening or Day 1
- Participation in an interventional clinical study within 30 days before initiation of dosing on Day 1, or use of any experimental therapy within 30 days prior to dosing on Day 1, or within 5 half-lives of the investigational product, whichever was greater
- History of allergy to any drug, allergen, excipients of ravulizumab or known allergy to Chinese hamster ovary cell proteins
- Inability to comply with study requirements
- History of any clinically significant cardiac, hepatic, immunologic, pulmonary, or rheumatoid disease that, in the Investigator's judgment, would preclude participation
- Other unspecified reasons that, in the opinion of the Investigator or Sponsor, make the participant unsuitable for enrollment
Data sourced from ClinicalTrials.gov (NCT02605993). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.