Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)

• Register: EUCTR
• Last refreshed on: 8 November 2021
• Main ID: EUCTR2016-003523-34-HU
• Date of registration: 31/01/2017
• Prospective Registration: Yes
• Primary sponsor: Ra Pharmaceuticals, Inc.
• Public title: Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)
• Scientific title: A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study
• Date of first enrolment: 17/03/2017
• Target sample size: 28
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003523-34
• 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; Number of treatment arms in the trial: 1
• 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, Denmark, Finland, Germany, Hungary, New Zealand, United Kingdom, United States

Contacts

• Name: xx
• Affiliation: Ra Pharmaceuticals, Inc.

• Name: xx
• Affiliation: Ra Pharmaceuticals, Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
Male or female > 18 years having completed a qualifying RA101495 clinical study and met predefined criteria for extended treatment with RA101495
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 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8

Exclusion criteria:
1. Platelet count <30,000/µL or absolute neutrophil count (ANC) <500 cells/µL at Screening
2. Caluculated 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
7. Pregnant, planning to become pregnant, or nursing female subjects



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 Haemoglobinuria (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 acylated with a ethyleneglycol24
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-

Primary Outcome(s)

Primary end point(s): Adverse Events and Serious Adverse Events profile including major adverse vascular event (MAVE criteria), clinical laboratory tests, ECGs, vital signs, physical examinations, and determination of anti-drug antibodies (ADA).

Secondary Objective: N/A

Main Objective: • To provide access to RA101495 for subjects with PNH who have who have completed a Ra Pharmaceuticals sponsored study, have demonstrated clinical benefit, and who wish to continue receiving RA101495 for treatment of PNH
• To evaluate the long-term safety and tolerability of RA101495 administered to subjects with PNH who have completed a Ra Pharmaceuticals sponsored RA101495 clinical study
• To evaluate the long-term preliminary efficacy of RA101495 administered to subjects with PNH who have completed a Ra Pharmaceuticals sponsored RA101495 clinical study
• To obtain periodic PK and PD data to confirm long-term maintenance of steady-state RA101495 plasma levels and sustained inhibition of hemolysis and complement

Timepoint(s) of evaluation of this end point: At each study visit (Month 1, 2, 3, 6, 9, 12, then every 3 months).

Secondary Outcome(s)

Secondary end point(s): Not applicable

Timepoint(s) of evaluation of this end point: Not applicable

Secondary ID(s)

2016-003523-34-FI

RA101495-01.202

Source(s) of Monetary Support

Ra Pharmaceuticals, Inc.

Ethics review

Status: Approved
Approval date: 06/03/2017
Contact: