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Phase 2 Safety and Efficacy Study of RA101495 to Treat PNH Patients

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ClinicalTrials.gov Identifier: NCT03078582
Recruitment Status : Active, not recruiting
First Posted : March 13, 2017
Last Update Posted : February 28, 2018
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals

Brief Summary:
The purpose of the study is to evaluate the safety and efficacy of 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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: RA101495 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : March 8, 2017
Estimated Primary Completion Date : February 2018
Estimated Study Completion Date : February 2018


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




Primary Outcome Measures :
  1. Change-from-baseline in serum lactate dehydrogenase (LDH) level. [ Time Frame: Through Week 12 of the study ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Exclusion Criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03078582


Locations
Australia
Investigative Site
North Gosford, New South Wales, Australia
Investigative Site
Parkville, Australia
Canada, Ontario
Investigative Site
Toronto, Ontario, Canada
Denmark
Investigative Site
Copenhagen, Denmark
Finland
Investigative Site
Helsinki, Finland
Germany
Investigative Site
Essen, Germany
Investigative Site
Ulm, Germany
Hungary
Investigative Site
Budapest, Hungary
New Zealand
Investigative Site
Christchurch, New Zealand
Investigative Site
Hamilton, New Zealand
United Kingdom
Investigative Site
Leeds, United Kingdom
Investigative Site
London, United Kingdom
Sponsors and Collaborators
Ra Pharmaceuticals

Responsible Party: Ra Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03078582     History of Changes
Other Study ID Numbers: RA101495-01.201
2016-003522-16 ( EudraCT Number )
First Posted: March 13, 2017    Key Record Dates
Last Update Posted: February 28, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Ra Pharmaceuticals:
PNH

Additional relevant MeSH terms:
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases