Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Register: EUCTR
• Last refreshed on: 7 May 2018
• Main ID: EUCTR2016-003522-16-FI
• Date of registration: 27/12/2016
• Prospective Registration: Yes
• Primary sponsor: Ra Pharmaceuticals, Inc.
• Public title: Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Scientific title: A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
• Date of first enrolment: 20/06/2017
• Target sample size: 20
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003522-16
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Canada, Denmark, Finland, Germany, Hungary, New Zealand, United Kingdom

Contacts

• Affiliation: Ra Pharmaceuticals, Inc.

• Affiliation: Ra Pharmaceuticals, Inc.

Key inclusion & exclusion 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:
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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 20.0 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000012950

Intervention(s)

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 name: Synthetic 15-amino-acid macrocyclic peptide
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-

Primary Outcome(s)

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 Outcome(s)

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.

Secondary ID(s)

RA101495-01.201

Source(s) of Monetary Support

Ra Pharmaceuticals, Inc.