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Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT03439839 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2024-06-12

Study results available
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Summary

This was a Phase 2, open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of iptacopan when administered in addition to Standard of care (SoC) in patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis.

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis

Interventions

DRUG

iptacopan

iptacopan bid orally administered

COMBINATION_PRODUCT

Standard of Care

Standard of Care (SoC) is defined as an antibody with anti C5 activity. At the time of study start, eculizumab was the only available SoC; eculizumab will be hereafter referred to as SoC.

Sponsors & Collaborators

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-04-09
Primary Completion
2020-04-22
Completion
2022-02-28

Countries

  • France
  • Germany
  • Italy

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03439839 on ClinicalTrials.gov