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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02598583
Registration number
NCT02598583
Ethics application status
Date submitted
2/11/2015
Date registered
6/11/2015
Date last updated
16/05/2022
Titles & IDs
Public title
Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Scientific title
An Open-Label, Intrapatient, Dose-Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria
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Secondary ID [1]
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ALXN1210-PNH-103
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
PNH
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Condition category
Condition code
Blood
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Haematological diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - ALXN1210
Experimental: Cohort 1 - Participants were administered ALXN1210 900 mg.
In the Extension period participants continued at the same dose and frequency as the Primary Evaluation Period.
Experimental: Cohort 2 - Participants were administered ALXN1210 1800 mg.
In the Extension period participants continued at the same dose and frequency as the Primary Evaluation Period.
Other interventions: ALXN1210
Participants were administered ravulizumab as an IV infusion every 4 weeks.
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percent Change In Lactate Dehydrogenase (LDH) Levels From Baseline To Day 169
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Assessment method [1]
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Baseline was defined as the average of all available assessments prior to first ALXN1210 infusion.
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Timepoint [1]
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Baseline, Day 169
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Secondary outcome [1]
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Percent Change In Free Hemoglobin Levels From Baseline To Day 169 And Day 1821
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Assessment method [1]
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Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
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Timepoint [1]
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Baseline, Day 169, Day 1821
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Secondary outcome [2]
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Percent Change In Haptoglobin Levels From Baseline To Day 169 And Day 1821
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Assessment method [2]
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Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
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Timepoint [2]
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Baseline, Day 169, Day 1821
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Secondary outcome [3]
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Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 169 And Day 1821
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Assessment method [3]
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Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
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Timepoint [3]
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Baseline, Day 169, Day 1821
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Secondary outcome [4]
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Percent Change In Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clones From Baseline To Day 169 And Day 1933
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Assessment method [4]
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Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
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Timepoint [4]
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Baseline, Day 169, Day 1933
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Secondary outcome [5]
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Percent Change In D-dimer Levels From Baseline To Day 169 And Day 1821
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Assessment method [5]
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Baseline was defined as the last non-missing assessment value prior to the first ALXN1210 infusion.
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Timepoint [5]
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Baseline, Day 169, Day 1821
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Secondary outcome [6]
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Change In Clinical Manifestations Of PNH From Baseline To Day 169 And Day 1821
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Assessment method [6]
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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.
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Timepoint [6]
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Baseline, Day 169, Day 1821
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Secondary outcome [7]
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Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) At Day 1
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Assessment method [7]
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AUCt reported in hours*microgram/milliliter (h*ug/mL).
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Timepoint [7]
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Day 1
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Secondary outcome [8]
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AUCt/ Dose-normalized (D) At Day 1
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Assessment method [8]
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Timepoint [8]
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Day 1
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Secondary outcome [9]
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Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The End Of The Dosing Interval (AUCtau) At Day 141
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Assessment method [9]
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Timepoint [9]
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Day 141
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Secondary outcome [10]
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AUCtau/D At Day 141
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Assessment method [10]
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Timepoint [10]
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Day 141
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Secondary outcome [11]
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Maximum Observed Serum Concentration (Cmax) At Day 1 And Day 141
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Assessment method [11]
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Timepoint [11]
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Day 1 and Day 141
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Secondary outcome [12]
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Cmax/D At Day 1 And Day 141
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Assessment method [12]
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Timepoint [12]
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Day 1 and Day 141
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Secondary outcome [13]
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Concentration At The End Of The Dosage Interval (Ctrough) At Day 1 And At Day 141
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Assessment method [13]
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Timepoint [13]
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Day 1 and Day 141
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Secondary outcome [14]
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Time To Maximum Observed Serum Concentration (Tmax) At Day 1 And Day 141
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Assessment method [14]
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Timepoint [14]
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Day 1 and Day 141
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Secondary outcome [15]
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Percent Change In Chicken Red Blood Cell (cRBC) Hemolysis From Baseline To Day 1709
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Assessment method [15]
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Timepoint [15]
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Baseline, Day 1709
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Secondary outcome [16]
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Percent Change In Free Complement Component 5 (C5) Concentration From Baseline To Day 1709
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Assessment method [16]
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Timepoint [16]
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Baseline, Day 1709
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Secondary outcome [17]
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Percent Change In Total C5 Concentration From Baseline To Day 1709
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Assessment method [17]
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Timepoint [17]
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Baseline, Day 1709
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Secondary outcome [18]
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Participants Experiencing Antidrug Antibodies (ADAs)
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Assessment method [18]
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Timepoint [18]
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Day 1821
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Eligibility
Key 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
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1/Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
12/11/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
11/03/2021
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Sample size
Target
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Accrual to date
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Final
13
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Recruitment in Australia
Recruitment state(s)
NSW,QLD
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Recruitment hospital [1]
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Clinical Trial Site - Liverpool
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Recruitment hospital [2]
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Clinical Trial Site - Woolloongabba
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Recruitment postcode(s) [1]
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2170 - Liverpool
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Recruitment postcode(s) [2]
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4102 - Woolloongabba
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Recruitment outside Australia
Country [1]
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Korea, Republic of
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State/province [1]
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Seoul
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Country [2]
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Korea, Republic of
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State/province [2]
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Ulsan
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alexion Pharmaceuticals, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief 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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02598583
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Trial related presentations / publications
Roth A, Rottinghaus ST, Hill A, Bachman ES, Kim JS, Schrezenmeier H, Terriou L, Urbano-Ispizua A, 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.
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Public notes
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Contacts
Principal investigator
Name
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Alexion Pharmaceuticals, Inc.
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Address
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Alexion Pharmaceuticals, Inc.
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02598583
Download to PDF