Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 1

• Conditions: Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]; Paroxysmal Nocturnal Hemoglobinuria (PNH); MedDRA version: 20.0Level: LLTClassification code 10055629Term: Paroxysmal nocturnal hemoglobinuriaSystem Organ Class: 100000012950

• Registration Number: EUCTR2016-003522-16-FI
• Lead Sponsor: Ra Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-12-27
• Last Update Posted: 7 May 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified