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

Conditions

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

Eligibility Criteria

Sex

ALL

Age

18 Years - 80 Years

Healthy Volunteers

Not accepted

Interventions

• iptacopan — DRUG
  iptacopan bid orally administered
• Standard of Care — COMBINATION_PRODUCT
  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.

Study Details

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.

Key Dates

Start date

Apr 9, 2018

Status verified

Jun 2024

Primary completion

Apr 22, 2020

Completion

Feb 28, 2022

Study Design

Enrollment

16 participants (actual)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1: LNP023 200mg bid + SoC
  Orally administered iptacopan 200 mg b.i.d. in Part 1 and Part 2
• Experimental: Cohort 2: LNP023 50mg/200mg bid + SoC
  Orally administered iptacopan 50 mg b.i.d. for a minimum of 2 weeks in addition to SoC; this could be increased to iptacopan 200 mg b.i.d. at study day 15 or at any time later in the study if LDH was not within limit of normal or reduced by at least 60% as compared to baseline values.

Primary Outcome Measure

Percent Change From Baseline in Lactate Dehydrogenase (LDH) Level at Day 92 [ Time Frame: Baseline and Day 92 ]