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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02598583
Other study ID # ALXN1210-PNH-103
Secondary ID
Status Completed
Phase Phase 1/Phase 2
First received
Last updated
Start date November 12, 2015
Est. completion date March 11, 2021

Study information

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

Clinical Trial Summary

This study evaluated the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of multiple intravenous (IV) doses of ALXN1210 administered to participants with PNH who have not previously been treated with complement inhibitor.


Description:

The data presented is up to the Primary Completion date of the study and is for the 24-week Primary Evaluation period. The study also includes an Extension Period of up to 5 years.


Recruitment information / eligibility

Status Completed
Enrollment 13
Est. completion date March 11, 2021
Est. primary completion date July 14, 2016
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 used highly effective contraception starting at screening and continuing until at least 24-weeks after the last dose of ALXN1210 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. Females who were 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 product, whichever is greater 4. History of allergy to excipients of ALXN1210 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, made the participant unsuitable for enrollment

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
ALXN1210
Participants were administered ravulizumab as an IV infusion every 4 weeks.

Locations

Country Name City State
Australia Clinical Trial Site Liverpool New South Wales
Australia Clinical Trial Site Woolloongabba Queensland
Korea, Republic of Clinical Trial Site Seoul
Korea, Republic of Clinical Trial Site Seoul
Korea, Republic of Clinical Trial Site Seoul
Korea, Republic of Clinical Trial Site Seoul
Korea, Republic of Clinical Trial Site Seoul
Korea, Republic of Clinical Trial Site Ulsan

Sponsors (1)

Lead Sponsor Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

Australia,  Korea, Republic of, 

References & Publications (1)

Röth A, Rottinghaus ST, Hill A, Bachman ES, Kim JS, Schrezenmeier H, Terriou L, Urbano-Ispizua Á, Wells RA, Jang JH, Kulasekararaj AG, Szer J, Aguzzi R, Damokosh AI, Shafner L, Lee JW. Ravulizumab (ALXN1210) in patients with paroxysmal nocturnal hemoglobinuria: results of 2 phase 1b/2 studies. Blood Adv. 2018 Sep 11;2(17):2176-2185. doi: 10.1182/bloodadvances.2018020644. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary Percent Change In Lactate Dehydrogenase (LDH) Levels From Baseline To Day 169 Baseline was defined as the average of all available assessments prior to first ALXN1210 infusion. Baseline, Day 169
Secondary Percent Change In Free Hemoglobin Levels From Baseline To Day 169 And Day 1821 Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion. Baseline, Day 169, Day 1821
Secondary Percent Change In Haptoglobin Levels From Baseline To Day 169 And Day 1821 Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion. Baseline, Day 169, Day 1821
Secondary Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 169 And Day 1821 Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion. Baseline, Day 169, Day 1821
Secondary Percent Change In Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clones From Baseline To Day 169 And Day 1933 Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion. Baseline, Day 169, Day 1933
Secondary Percent Change In D-dimer Levels From Baseline To Day 169 And Day 1821 Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion. Baseline, Day 169, Day 1821
Secondary Change In Clinical Manifestations Of PNH From Baseline To Day 169 And Day 1821 Clinical manifestations are defined as fatigue, abdominal pain, dyspnea, dysphagia, chest pain, and erectile dysfunction (ED) by cohort. Improvement is defined as present at baseline and absent at Day 169 endpoint. Worsening is defined as absent at Baseline and present at Day 169 endpoint. Baseline, Day 169, Day 1821
Secondary Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) At Day 1 AUCt reported in hours*microgram/milliliter (h*ug/mL). Day 1
Secondary AUCt/ Dose-normalized (D) At Day 1 Day 1
Secondary Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The End Of The Dosing Interval (AUCtau) At Day 141 Day 141
Secondary AUCtau/D At Day 141 Day 141
Secondary Maximum Observed Serum Concentration (Cmax) At Day 1 And Day 141 Day 1 and Day 141
Secondary Cmax/D At Day 1 And Day 141 Day 1 and Day 141
Secondary Concentration At The End Of The Dosage Interval (Ctrough) At Day 1 And At Day 141 Day 1 and Day 141
Secondary Time To Maximum Observed Serum Concentration (Tmax) At Day 1 And Day 141 Day 1 and Day 141
Secondary Percent Change In Chicken Red Blood Cell (cRBC) Hemolysis From Baseline To Day 1709 Baseline, Day 1709
Secondary Percent Change In Free Complement Component 5 (C5) Concentration From Baseline To Day 1709 Baseline, Day 1709
Secondary Percent Change In Total C5 Concentration From Baseline To Day 1709 Baseline, Day 1709
Secondary Participants Experiencing Antidrug Antibodies (ADAs) Day 1821
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