Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)

Phase 1

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

Registration Number: EUCTR2016-003522-16-DK

Lead Sponsor: Ra Pharmaceuticals, Inc.

Brief Summary: Not available

Detailed Description: Not available

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
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
5. History of meningococcal disease
6. Current systemic infection or suspicion of active bacterial infection

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: • To assess the safety and tolerability of RA101495 in subjects with PNH<br>• To assess preliminary efficacy of RA101495 in subjects with PNH<br>• To assess PK and PD of RA101495 in subjects with PNH;Secondary Objective: N/A;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 is baseline and Weeks 6, 8, 10, and 12.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary efficacy:<br>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.