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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02598583




Registration number
NCT02598583
Ethics application status
Date submitted
2/11/2015
Date registered
6/11/2015

Titles & IDs
Public title
Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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
Secondary ID [1] 0 0
ALXN1210-PNH-103
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
PNH 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - 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.


Treatment: Other: ALXN1210
Participants were administered ravulizumab as an IV infusion every 4 weeks.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Change In Lactate Dehydrogenase (LDH) Levels From Baseline To Day 169
Timepoint [1] 0 0
Baseline, Day 169
Secondary outcome [1] 0 0
Percent Change In Free Hemoglobin Levels From Baseline To Day 169 And Day 1821
Timepoint [1] 0 0
Baseline, Day 169, Day 1821
Secondary outcome [2] 0 0
Percent Change In Haptoglobin Levels From Baseline To Day 169 And Day 1821
Timepoint [2] 0 0
Baseline, Day 169, Day 1821
Secondary outcome [3] 0 0
Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 169 And Day 1821
Timepoint [3] 0 0
Baseline, Day 169, Day 1821
Secondary outcome [4] 0 0
Percent Change In Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clones From Baseline To Day 169 And Day 1933
Timepoint [4] 0 0
Baseline, Day 169, Day 1933
Secondary outcome [5] 0 0
Percent Change In D-dimer Levels From Baseline To Day 169 And Day 1821
Timepoint [5] 0 0
Baseline, Day 169, Day 1821
Secondary outcome [6] 0 0
Change In Clinical Manifestations Of PNH From Baseline To Day 169 And Day 1821
Timepoint [6] 0 0
Baseline, Day 169, Day 1821
Secondary outcome [7] 0 0
Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The Last Quantifiable Concentration (AUCt) At Day 1
Timepoint [7] 0 0
Day 1
Secondary outcome [8] 0 0
AUCt/ Dose-normalized (D) At Day 1
Timepoint [8] 0 0
Day 1
Secondary outcome [9] 0 0
Area Under The Serum Concentration-versus-time-curve From Time 0 (Dosing) To The End Of The Dosing Interval (AUCtau) At Day 141
Timepoint [9] 0 0
Day 141
Secondary outcome [10] 0 0
AUCtau/D At Day 141
Timepoint [10] 0 0
Day 141
Secondary outcome [11] 0 0
Maximum Observed Serum Concentration (Cmax) At Day 1 And Day 141
Timepoint [11] 0 0
Day 1 and Day 141
Secondary outcome [12] 0 0
Cmax/D At Day 1 And Day 141
Timepoint [12] 0 0
Day 1 and Day 141
Secondary outcome [13] 0 0
Concentration At The End Of The Dosage Interval (Ctrough) At Day 1 And At Day 141
Timepoint [13] 0 0
Day 1 and Day 141
Secondary outcome [14] 0 0
Time To Maximum Observed Serum Concentration (Tmax) At Day 1 And Day 141
Timepoint [14] 0 0
Day 1 and Day 141
Secondary outcome [15] 0 0
Percent Change In Chicken Red Blood Cell (cRBC) Hemolysis From Baseline To Day 1709
Timepoint [15] 0 0
Baseline, Day 1709
Secondary outcome [16] 0 0
Percent Change In Free Complement Component 5 (C5) Concentration From Baseline To Day 1709
Timepoint [16] 0 0
Baseline, Day 1709
Secondary outcome [17] 0 0
Percent Change In Total C5 Concentration From Baseline To Day 1709
Timepoint [17] 0 0
Baseline, Day 1709
Secondary outcome [18] 0 0
Participants Experiencing Antidrug Antibodies (ADAs)
Timepoint [18] 0 0
Day 1821

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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/11/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
11/03/2021
Sample size
Target
Accrual to date
Final
13
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Recruitment hospital [1] 0 0
Clinical Trial Site - Liverpool
Recruitment hospital [2] 0 0
Clinical Trial Site - Woolloongabba
Recruitment postcode(s) [1] 0 0
2170 - Liverpool
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment outside Australia
Country [1] 0 0
Korea, Republic of
State/province [1] 0 0
Seoul
Country [2] 0 0
Korea, Republic of
State/province [2] 0 0
Ulsan

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Alexion Pharmaceuticals, Inc.
Address 0 0
Alexion Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Supporting document/s available: Study protocol, Clinical study report (CSR)
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02598583