NCT03078582

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 Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
26

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Mar 2017

Shorter than P25 for phase_2

Geographic Reach
8 countries

12 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 8, 2017

Completed
Same day until next milestone

Study Start

First participant enrolled

March 8, 2017

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 13, 2017

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 28, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 28, 2018

Completed
2 years until next milestone

Results Posted

Study results publicly available

March 10, 2020

Completed
Last Updated

July 27, 2022

Status Verified

July 1, 2022

Enrollment Period

1.1 years

First QC Date

March 8, 2017

Results QC Date

June 19, 2019

Last Update Submit

July 26, 2022

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Keywords

PNH

Outcome Measures

Primary Outcomes (1)

  • 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.

    Through Week 12 of the study

Secondary Outcomes (6)

  • Changes From Baseline in Bilirubin Values

    Through Week 12 of the study

  • Total Hemoglobin

    Through Week 12 of the Study

  • Changes From Baseline in Free Hemoglobin Values

    Through Week 12 of the study

  • Haptoglobin Values

    Through Week 12 of the Study

  • Reticulocyte Values

    Through Week 12 of the Study

  • +1 more secondary outcomes

Study Arms (2)

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)

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)

Drug: Zilucoplan (RA101495)

Interventions

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

Zilucoplan (RA101495) previously on eculizumabZilucoplan (RA101495) treatment naive

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • History of meningococcal disease
  • Current systemic infection or suspicion of active bacterial infection

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Investigative Site

Gosford, New South Wales, Australia

Location

Investigative Site

Parkville, Australia

Location

Investigative Site

Toronto, Ontario, Canada

Location

Investigative Site

Copenhagen, Denmark

Location

Investigative Site

Helsinki, Finland

Location

Investigative Site

Essen, Germany

Location

Investigative Site

Ulm, Germany

Location

Investigative Site

Budapest, Hungary

Location

Investigative Site

Christchurch, New Zealand

Location

Investigative Site

Hamilton, New Zealand

Location

Investigative Site

Leeds, United Kingdom

Location

Investigative Site

London, United Kingdom

Location

Related Links

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

zilucoplan

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Results Point of Contact

Title
Sponsor Ra Pharmaceuticals, Inc.
Organization
Ra Pharmaceuticals, Inc
trials@rapharma.com
+1 617 401 4060

Study Officials

  • Dr. Anita Hill

    St James' Institute of Oncology

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 8, 2017

First Posted

March 13, 2017

Study Start

March 8, 2017

Primary Completion

March 28, 2018

Study Completion

March 28, 2018

Last Updated

July 27, 2022

Results First Posted

March 10, 2020

Record last verified: 2022-07

Data Sharing

IPD Sharing
Will not share

Locations

GB(2)HU(1)CA(1)AU(2)FI(1)NZ(2)DK(1)DE(2)