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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
Date last updated
27/07/2022

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
The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). There will be two groups of patients in the study: the first group will include patients who have never received eculizumab for treatment of PNH. The second group will include patients who have received treatment with eculizumab for at least 6 months prior to the study. Patients will be treated with RA101495 for 12 weeks.
Trial website
https://clinicaltrials.gov/study/NCT03078582
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03078582