CompletedPhase 2

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

United States3 participantsStarted 2017-04-17

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.

Timeframe: Through Week 12 of the study

Trial details

NCT IDNCT03030183

SponsorRa Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-03-28

Results submitted2019-08-13

View on ClinicalTrials.gov More Paroxysmal Nocturnal Hemoglobinuria (PNH) trials