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Trial record 2 of 9 for:    paroxysmal nocturnal hemoglobinuria | Recruiting, Not yet recruiting, Available Studies | United States

Phase 2 Safety and Efficacy Study of RA101495 to Treat PNH Patients Who Have an Inadequate Response to Eculizumab

This study is currently recruiting participants.
See Contacts and Locations
Verified August 2017 by Ra Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT03030183
First received: January 20, 2017
Last updated: August 29, 2017
Last verified: August 2017
  Purpose
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.

Condition Intervention Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: RA101495 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have an Inadequate Response to Eculizumab

Resource links provided by NLM:


Further study details as provided by Ra Pharmaceuticals:

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

Estimated Enrollment: 8
Actual Study Start Date: April 17, 2017
Estimated Study Completion Date: February 2018
Estimated Primary Completion Date: February 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RA101495
Subjects will receive RA101495 at the dose of 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

  Eligibility

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

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • History of meningococcal disease
  • Current systemic infection or suspicion of active bacterial infection
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03030183

Contacts
Contact: Sponsor: Ra Pharmaceuticals, Inc. +1 617 401 4060 trials@rapharma.com

Locations
United States, California
Investigative Site Recruiting
Duarte, California, United States, 91010
Investigative Site Recruiting
Los Angeles, California, United States, 90033
United States, Illinois
Investigative Site Recruiting
Chicago, Illinois, United States, 60612
United States, New York
Investigative Site Recruiting
Manhasset, New York, United States, 11030
United States, North Carolina
Investigative Site Recruiting
Durham, North Carolina, United States, 27710
United States, Texas
Investigative Site Not yet recruiting
Dallas, Texas, United States, 75390
Sponsors and Collaborators
Ra Pharmaceuticals
  More Information

Responsible Party: Ra Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03030183     History of Changes
Other Study ID Numbers: RA101495-01.203
Study First Received: January 20, 2017
Last Updated: August 29, 2017

Studies a U.S. FDA-regulated Drug Product: Yes
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

ClinicalTrials.gov processed this record on September 19, 2017