Trial intelligence / Profile preview

Ruxoprubart in Treatment-Naïve PNH

NCT05646524Phase 2

Key facts

Status: Not yet recruiting
Start: Apr 2026
Primary completion: Aug 2027
Sponsor: NovelMed Therapeutics
Modality: Monoclonal Antibodies

Drugs: ruxoprubart
Conditions: paroxysmal nocturnal hemoglobinuria (PNH)

Overview

Phase 2, open-label trial of ruxoprubart in paroxysmal nocturnal hemoglobinuria (PNH) (2 cohorts), sponsored by NovelMed Therapeutics. Primary endpoints: Monitoring of Adverse Events (AEs) and Serious Adverse Events (SAEs), Number of Participants with Antidrug Antibodies (ADAs) to NM8074 and 5 more. Planned start Apr 2026; status: not yet recruiting.

Design

2 arms

Study design: Non-randomizedOpen-label

Cohort 1
NM8074 20 mg/kg q2w
Cohort 2
NM8074 10 mg/kg weekly x4 then 20 mg/kg q2w

Who it enrols

Treatment-naïve adult PNH patients with hemolytic anemia (LDH >= 1.5xULN, Hb <= 10.5 g/dL), PNH clone size >= 10%, and history of transfusions.

Endpoints

Primary

Monitoring of Adverse Events (AEs) and Serious Adverse Events (SAEs)Up to Study Day 105
Number of Participants with Antidrug Antibodies (ADAs) to NM8074Up to Study Day 105
Change from Baseline or Percent Change from Baseline in Hemoglobin (Hgb) LevelsUp to Study Day 105
Change from Baseline or Percent Change from Baseline in Lactate Dehydrogenase (LDH) LevelsUp to Study Day 105
Change from Baseline or Percent Change from Baseline in Number of Packed Red Blood Cell (pRBC) TransfusionsUp to Study Day 105

Secondary

Change from Baseline or Percent Change from Baseline in Reticulocyte CountUp to Study Day 105
Change from Baseline or Percent Change from Baseline in Bilirubin LevelsUp to Study Day 105
Change from Baseline or Percent Change from Baseline in Quality of Life (QoL) Survey Assessed via the FACIT-Fatigue ScaleUp to Study Day 105
Change from Baseline or Percent Change from Baseline in Quality of Life (QoL) Survey Assessed via the EORTC QLQ-C30 ScaleUp to Study Day 105
Changes in plasma concentration, Cmax, tmax, AUC0-t of NM8074Up to Study Day 105

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