Paroxysmal Nocturnal Hemoglobinuria Clinical Trial
Official title:
A Phase 2, Open-label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria
Verified date | December 2022 |
Source | Alexion Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 26 |
Est. completion date | January 12, 2022 |
Est. primary completion date | February 23, 2017 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: 1. Male or female =18 years of age 2. PNH diagnosis confirmed by documented high-sensitivity flow cytometry 3. Documented meningococcal vaccination not more than 3 years prior to dosing 4. 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. 5. Willing and able to give written informed consent and comply with the study visit schedule Exclusion Criteria: 1. Treatment with a complement inhibitor at any time 2. Female participants who are planning to become pregnant, or are pregnant, breastfeeding or who had a positive pregnancy test at screening or Day 1 3. 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 4. History of allergy to any drug, allergen, excipients of ravulizumab or known allergy to Chinese hamster ovary cell proteins 5. Inability to comply with study requirements 6. History of any clinically significant cardiac, hepatic, immunologic, pulmonary, or rheumatoid disease that, in the Investigator's judgment, would preclude participation 7. Other unspecified reasons that, in the opinion of the Investigator or Sponsor, make the participant unsuitable for enrollment |
Country | Name | City | State |
---|---|---|---|
Canada | Clinical Trial Site | Toronto | Ontario |
France | Clinical Trial Site | Lille | |
France | Clinical Trial Site | Lyon | Pierre-Bénite |
France | Clinical Trial Site | Paris | |
Germany | Clinical Trial Site | Aachen | Nordrhein Westfalen |
Germany | Clinical Trial Site | Essen | Nordrhein Westfalen |
Germany | Clinical Trial Site | Ulm | Baden Wuerttemberg |
Korea, Republic of | Clinical Trial Site | Seoul | |
Korea, Republic of | Clinical Trial Site | Seoul | |
Spain | Clinical Trial Site | Badalona | Barcelona |
Spain | Clinical Trial Site | Barcelona | |
Spain | Clinical Trial Site | Madrid | |
Spain | Clinical Trial Site | Majadahonda | Madrid |
Taiwan | Clinical Trial Site | Taipei City | |
United Kingdom | Clinical Trial Site | Leeds | West Yorkshire |
United Kingdom | Clinical Trial Site | London |
Lead Sponsor | Collaborator |
---|---|
Alexion Pharmaceuticals |
Canada, France, Germany, Korea, Republic of, Spain, Taiwan, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percent Change In LDH Levels From Baseline To Day 253 And Day 281 | The percent change in LDH levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only. | Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4) | |
Secondary | Percent Change In Free Hemoglobin Levels From Baseline To Day 253 And Day 281 | The percent change in free hemoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only. | Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4) | |
Secondary | Percent Change In Haptoglobin Levels From Baseline To Day 253 And Day 281 | The percent change in haptoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only. | Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4) | |
Secondary | Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 253 And Day 281 | The percent change in reticulocyte/erythrocyte count levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only. | Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4) | |
Secondary | Percent Change In PNH RBC Types II And III Clone Size From Baseline To Day 253 | The percent change in paroxysmal nocturnal hemoglobinuria (PNH) red blood cell (RBC), summed types II and III, clone size levels were assessed from Baseline to Day 253 for Cohorts 1 to 4. | Baseline, Day 253 (Cohorts 1 to 4) | |
Secondary | Percent Change In D-dimer From Baseline To Day 253 And Day 281 | The percent change in D-dimer levels were assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only. | Baseline to Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4) | |
Secondary | Change In Clinical Manifestations Of PNH From Baseline To Day 253 And Day 281 | Clinical manifestations were assessed from Baseline to Day 253 for Cohorts 1 to 3 and from Baseline to Day 281 for Cohort 4 only. Clinical manifestations were defined as fatigue, abdominal pain, dyspnea, dysphagia, chest pain, and erectile dysfunction (male participants only). Improvement was defined as present at Baseline and absent at Day endpoint. Worsening was defined as absent at Baseline and present at Day endpoint. No Change was defined as no change from Baseline and time point of endpoint. | Baseline, Day 253 (Cohorts 1 to 3) and Day 281 (Cohort 4) |
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