Danicopan for Pediatric PNH Patients

We are studying whether adding danicopan to existing treatments helps children with Paroxysmal Nocturnal Hemoglobinuria improve their hemoglobin levels and quality of life.

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Danicopan
Danicopan is a substance that blocks part of the immune system to reduce red blood cell destruction in paroxysmal nocturnal hemoglobinuria.

What experimental substances are being tested?

Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.

Alxn 2040

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

Loading map...
Robert Debre University Hospital
Pediatric hemato- immunology Department
Le Pré-Saint-Gervais, France
Sponsor: Alexion Pharmaceuticals Inc.
Last updated: Nov 27, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.