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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03078582




Registration number
NCT03078582
Ethics application status
Date submitted
8/03/2017
Date registered
13/03/2017

Titles & IDs
Public title
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
Scientific title
Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
Secondary ID [1] 0 0
2016-003522-16
Secondary ID [2] 0 0
RA101495-01.201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria (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: Drugs - Zilucoplan (RA101495)

Experimental: Zilucoplan (RA101495) treatment naive - 0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC (treatment naïve)

Experimental: Zilucoplan (RA101495) previously on eculizumab - 0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC (previously on eculizumab)


Treatment: Drugs: Zilucoplan (RA101495)
0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level.
Timepoint [1] 0 0
Through Week 12 of the study
Secondary outcome [1] 0 0
Changes From Baseline in Bilirubin Values
Timepoint [1] 0 0
Through Week 12 of the study
Secondary outcome [2] 0 0
Total Hemoglobin
Timepoint [2] 0 0
Through Week 12 of the Study
Secondary outcome [3] 0 0
Changes From Baseline in Free Hemoglobin Values
Timepoint [3] 0 0
Through Week 12 of the study
Secondary outcome [4] 0 0
Haptoglobin Values
Timepoint [4] 0 0
Through Week 12 of the Study
Secondary outcome [5] 0 0
Reticulocyte Values
Timepoint [5] 0 0
Through Week 12 of the Study
Secondary outcome [6] 0 0
Hemoglobinuria Values
Timepoint [6] 0 0
Through Week 12 of the Study

Eligibility
Key inclusion criteria
* Diagnosis of PNH by flow cytometry
* For treatment naive patients: subjects must not have received treatment with eculizumab prior to or during the Screening Period and must have a lactate dehydrogenase (LDH) level =2 times the upper limit of normal (xULN) during Screening
* For patients who previously received eculizumab: subjects must have received treatment with eculizumab for at least 6 months prior to Screening
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of meningococcal disease
* Current systemic infection or suspicion of active bacterial infection

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Parallel
Other design features
Phase
Phase 2
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
8/03/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
28/03/2018
Sample size
Target
Accrual to date
Final
26
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Investigative Site - Gosford
Recruitment hospital [2] 0 0
Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
- Gosford
Recruitment postcode(s) [2] 0 0
- Parkville
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Ontario
Country [2] 0 0
Denmark
State/province [2] 0 0
Copenhagen
Country [3] 0 0
Finland
State/province [3] 0 0
Helsinki
Country [4] 0 0
Germany
State/province [4] 0 0
Essen
Country [5] 0 0
Germany
State/province [5] 0 0
Ulm
Country [6] 0 0
Hungary
State/province [6] 0 0
Budapest
Country [7] 0 0
New Zealand
State/province [7] 0 0
Christchurch
Country [8] 0 0
New Zealand
State/province [8] 0 0
Hamilton
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Leeds
Country [10] 0 0
United Kingdom
State/province [10] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ra Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Dr. Anita Hill
Address 0 0
St James' Institute of Oncology
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)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03078582