NCT03157635

Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria

An Adaptive Phase I/II Study to Assess Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Crovalimab in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 59 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Accepted

Summary

This is a Phase I/II, first-in-human study consisting of four sequential parts and an open-label extension (OLE). The safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single doses of crovalimab will be evaluated in healthy volunteers (HV) during part 1. The safety, tolerability, PK and PD of multiple doses of crovalimab will be evaluated in participants with paroxysmal nocturnal hemoglobinuria (PNH) in parts 2, 3, 4, and OLE of the study. Efficacy of crovalimab will be evaluated in Parts 2, 3, and 4.

Conditions

• Paroxysmal Hemoglobinuria, Nocturnal

Interventions

Type	Name	Description
DRUG	Crovalimab	Crovalimab will be administered as per schedule described in individual arm.
DRUG	Placebo	Placebo will be administered as per schedule described in Part 1 placebo arm.

Timeline

• Start date: 2016-11-14
• Primary completion: 2026-09-30
• Completion: 2026-09-30
• First posted: 2017-05-17
• Last updated: 2026-02-17

Locations

14 sites across 7 countries: France, Germany, Hungary, Italy, Japan, Netherlands, South Korea