Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 2NCT03078582
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
A Phase 2 study testing Zilucoplan (RA101495) in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Ra Pharmaceuticals at 12 sites in 8 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- What is being tested?
- Zilucoplan (RA101495)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 12 sitesin 8 countries
- Study dates
- 8 Mar 2017to 28 Mar 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- History of meningococcal disease
- Current systemic infection or suspicion of active bacterial infection
Trial locations and contacts
12 sites in 8 countries.
12 sites in 8 countries
Australia2 sites
Investigative Site
Gosford, New South Wales
Investigative Site
Parkville
New Zealand2 sites
Investigative Site
Christchurch
Investigative Site
Hamilton
United Kingdom2 sites
Investigative Site
Leeds
Investigative Site
London
About this study
Official 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
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.
- Being tested
- Zilucoplan (RA101495)
- Keywords
- PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Zilucoplan (RA101495)Drug
0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
| Study group | Role | What they receive |
|---|---|---|
| Zilucoplan (RA101495) treatment naive | Experimental | 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) Receives: Drug: Zilucoplan (RA101495) |
| Zilucoplan (RA101495) previously on eculizumab | Experimental | 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) Receives: Drug: Zilucoplan (RA101495) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level.
The primary efficacy endpoint is the change-from-baseline in serum LDH levels during this period, defined as the mean LDH values of Weeks 6, 8, 10, and 12 minus the baseline value of LDH.
Measured: Through Week 12 of the study
Secondary outcomes (6)
Changes From Baseline in Bilirubin Values
Changes from baseline at each of the scheduled post-baseline time-points
Measured: Through Week 12 of the study
Total Hemoglobin
Changes from baseline at each of the scheduled post-baseline time-points
Measured: Through Week 12 of the Study
Changes From Baseline in Free Hemoglobin Values
Changes from baseline at each of the scheduled post-baseline time-points
Measured: Through Week 12 of the study
Haptoglobin Values
Changes from baseline at each of the scheduled post-baseline time-points
Measured: Through Week 12 of the Study
Reticulocyte Values
Changes from baseline at each of the scheduled post-baseline time-points
Measured: Through Week 12 of the Study
Hemoglobinuria Values
Changes from baseline at each of the scheduled post-baseline time-points; Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10. Where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria.
Measured: Through Week 12 of the Study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2017
- Study start8 Mar 2017
- Primary completion28 Mar 2018
- Study completion28 Mar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ra Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dr. Anita Hill, Principal investigator, St James' Institute of Oncology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03078582
- Sponsor's protocol ID
- RA101495-01.201
- Other IDs
- 2016-003522-16, 2016
- Status last verified
- Jul 2022
- First submitted
- 8 Mar 2017
- Last updated
- 27 Jul 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT03078582
- Acronym / sponsor ID
- RA101495-01.201
- Keywords
- PNH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Ra PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 8 Mar 2017 (actual)
- Primary completion
- 28 Mar 2018 (actual)
- Study completion
- 28 Mar 2018 (actual)
- First posted
- 13 Mar 2017 (actual)
- Results
- Posted 10 Mar 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Zilucoplan (RA101495) treatment naive | Experimental | Drug: 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 (treatment naïve) |
| Zilucoplan (RA101495) previously on eculizumab | Experimental | Drug: 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 (previously on eculizumab) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- History of meningococcal disease
- Current systemic infection or suspicion of active bacterial infection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Drug | Zilucoplan (RA101495) previously on eculizumab, Zilucoplan (RA101495) treatment naive |
Primary outcome
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level.
Time frame: Through Week 12 of the study
The primary efficacy endpoint is the change-from-baseline in serum LDH levels during this period, defined as the mean LDH values of Weeks 6, 8, 10, and 12 minus the baseline value of LDH.
6 secondary outcomes
Changes From Baseline in Bilirubin Values
Time frame: Through Week 12 of the study
Total Hemoglobin
Time frame: Through Week 12 of the Study
Changes From Baseline in Free Hemoglobin Values
Time frame: Through Week 12 of the study
Haptoglobin Values
Time frame: Through Week 12 of the Study
Reticulocyte Values
Time frame: Through Week 12 of the Study
Hemoglobinuria Values
Time frame: Through Week 12 of the Study
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dr. Anita Hill principal investigator
St James' Institute of Oncology
12 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Zilucoplan (RA101495) in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Ra Pharmaceuticals at 12 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Australia, Germany, New Zealand, United Kingdom, Canada and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level. (time frame: Through Week 12 of the study). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2022.