Extension Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-003523-34-FI

Enrollment

Registered

2016-12-27

Start date

2017-04-06

Completion date

Unknown

Last updated

2021-11-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857

Interventions

Product Name: Zilucoplan Product Code: RA101495 Pharmaceutical Form: Solution for injection INN or Proposed INN: Zilucoplan CAS Number: 1841136-73-9 Current Sponsor code: RA101495 Other descriptive na

Eligibility

Sex/Gender

All

Inclusion 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

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 5. Pregnant, planning to become pregnant, or nursing female subjects

Design outcomes

Primary

Measure	Time frame
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;Secondary Objective: N/A;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), serum LDH levels, total bilirubin, total hemoglobin, free hemoglobin, haptoglobin, reticulocytes, and hemoglobinuria, at each of the scheduled assessment time-points.;Timepoint(s) of evaluation of this end point: At each study visit (Month 1, 2, 3, 6, 9, 12, then every 3 months).	—

Measure

Time frame

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;Secondary Objective: N/A;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), serum LDH levels, total bilirubin, total hemoglobin, free hemoglobin, haptoglobin, reticulocytes, and hemoglobinuria, at each of the scheduled assessment time-points.;Timepoint(s) of evaluation of this end point: At each study visit (Month 1, 2, 3, 6, 9, 12, then every 3 months).

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Secondary

Measure	Time frame
Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable	—

Countries

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

Contacts

Public ContactClin Trial Reg & Results Disclosure

UCB BIOSCIENCES GmbH

clinicaltrials@ucb.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026