A Phase 2 Safety and Efficacy Study of RA101495 to Treat PNH Patients Who Have an Inadequate Response to Eculizumab | Aplastic Anemia and MDS International Foundation

ClinicalTrials.gov Identifier:



RA Pharma

Contact Info

Tammy Nguyen



(617) 674-9869


Start: February 2017
End: February 2018

Official Title

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


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. Note: Patients on the study 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


If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.

Associated Drug(s): 
Phase 2
Age Group: 
18 years and older
Accepts Healthy Volunteers: 
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

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