NCT03030183

Brief Summary

The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
3

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Apr 2017

Shorter than P25 for phase_2

Geographic Reach
1 country

6 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

January 20, 2017

Completed
4 days until next milestone

First Posted

Study publicly available on registry

January 24, 2017

Completed
3 months until next milestone

Study Start

First participant enrolled

April 17, 2017

Completed
12 months 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
1.9 years until next milestone

Results Posted

Study results publicly available

February 26, 2020

Completed
Last Updated

July 27, 2022

Status Verified

July 1, 2022

Enrollment Period

12 months

First QC Date

January 20, 2017

Results QC Date

August 13, 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) Levels.

    Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels

    Through Week 12 of the study

Secondary Outcomes (6)

  • Change-from-baseline Bilirubin Values

    Through week 12

  • Change-from-baseline Total Hemoglobin Values

    Through week 12

  • Change-from-baseline Free Hemoglobin Values

    Through week 12

  • Change-from-baseline Haptoglobin Values

    Through week 12

  • Change-from-baseline Reticulocyte Values

    Through week 12

  • +1 more secondary outcomes

Study Arms (1)

Zilucoplan (RA101495)

EXPERIMENTAL

Subjects will receive RA101495 at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC

Drug: Zilucoplan (RA101495)

Interventions

Zilucoplan (RA101495)DRUG

0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC

Zilucoplan (RA101495)

Eligibility Criteria

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

You may qualify if:

  • Diagnosis of PNH by flow cytometry
  • Inadequate response to eculizumab defined as having received eculizumab for at least 6 months plus a documented LDH level ≥ 1.5 x the upper limit of normal (ULN) and/or the presence of a known C5 mutation conferring resistance to eculizumab

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 (6)

Investigative Site

Duarte, California, 91010, United States

Location

Investigative Site

Los Angeles, California, 90033, United States

Location

Investigative Site

Chicago, Illinois, 60612, United States

Location

Investigative Site

Manhasset, New York, 11030, United States

Location

Investigative Site

Durham, North Carolina, 27710, United States

Location

Investigative Site

Dallas, Texas, 75390, United States

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 Pharmaceutical, Inc
Organization
Ra Pharmaceutical, Inc
trials@rapharma.com
1 617 401 4060

Study Officials

  • Dr. Anita Hill

    St James' Institute of Oncology

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 20, 2017

First Posted

January 24, 2017

Study Start

April 17, 2017

Primary Completion

March 28, 2018

Study Completion

March 28, 2018

Last Updated

July 27, 2022

Results First Posted

February 26, 2020

Record last verified: 2022-07

Locations

US(6)