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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02605993
Other study ID # ALXN1210-PNH-201
Secondary ID 2015-002674-20
Status Completed
Phase Phase 2
First received
Last updated
Start date January 4, 2016
Est. completion date January 12, 2022

Study information

Verified date December 2022
Source Alexion Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Description:

The study consisted of a screening period of up to 30 days and a Treatment Period of up to 253 days for Cohorts 1-3 and 281 days for Cohort 4. After completion of the Treatment Period, all participants had the opportunity to enter the Extension Period, wherein participants continue to receive ravulizumab for up to 5 years. The first dose in the Extension Period occurred on Day 253 for Cohorts 1-3 and on Day 281 for Cohort 4. The data presented includes the Primary Completion date of the study for the Treatment Period. The results for the Extension Period will be reported after study completion.


Recruitment information / eligibility

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

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
Ravulizumab
All treatments were given as IV infusions.

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

Canada,  France,  Germany,  Korea, Republic of,  Spain,  Taiwan,  United Kingdom, 

Outcome

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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