Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000012950
Conditions
Interventions
Product Name: RA101495
Product Code: RA101495
Pharmaceutical Form: Solution for injection
INN or Proposed INN: RA101495 sodium
CAS Number: 1841136-73-9
Current Sponsor code: RA101495
Other descriptive
Sponsors
Ra Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: To be eligible for this study, subjects must meet ALL of the following inclusion criteria: 1. Male or female =18 years 2. Completed informed consent procedures, including signing and dating the informed consent form (ICF) 3. Diagnosis of PNH by flow cytometry 4. Cohort A (Naïve) 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 5. Cohort B (Switch) subjects must have received treatment with eculizumab for at least 6 months prior to Screening 8. Finland specific inclusion criterion: - All Cohort A (Naïve) subjects who have not been previously vaccinated against Neisseria meningitidis prior to study entry must be vaccinated and must receive ciprofloxacin for 14 days starting with the first dose of RA101495 at the Day 1 Visit. - All Cohort B (Switch) subjects must have documentation of prior Neisseria meningitidis vaccination (and booster if appropriate) prior to study entry. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 16 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 4
Exclusion criteria
Exclusion criteria: 1. Platelet count <30,000/µL or absolute neutrophil count (ANC) <500 cells/µL at Screening 2. Calculated glomerular filtration rate of <30 mL/min/1.73m2 based on modification of diet in renal disease (MDRD) equation at Screening 3. History of meningococcal disease 4. Current systemic infection or suspicion of active bacterial infection
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To assess the safety and tolerability of RA101495 in subjects with PNH • To assess preliminary efficacy of RA101495 in subjects with PNH • To assess PK and PD of RA101495 in subjects with PNH;Secondary Objective: ;Primary end point(s): Change-from-baseline in serum lactate dehydrogenase (LDH) levels through Week 12 of study.;Timepoint(s) of evaluation of this end point: The timepoints of evaluation for the primary endpoint are baseline and Weeks 6, 8, 10, and 12. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary efficacy: Changes from baseline in LDH, total bilirubin, total hemoglobin, free hemoglobin, haptoglobin, reticulocytes, and hemoglobinuria, at each of the scheduled postbaseline assessment time-points.;Timepoint(s) of evaluation of this end point: The timepoints for the evaluation of the secondary endpoints are baseline and Weeks 1, 2, 3, 4, 6, 8, 10 and 12. | — |
Countries
Australia, Canada, Denmark, Finland, Germany, Hungary, New Zealand, United Kingdom
Outcome results
None listed