Pegcetacoplan for Adolescents with Paroxysmal Nocturnal Hemoglobinuria

We are studying the safety and biological activity of pegcetacoplan in patients aged 12-17 with PNH. This trial will help us understand how well the treatment works and its effects on health-related 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 medicines are being studied?

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

Aspaveli
Aspaveli is a medicine used to treat a rare blood disorder called paroxysmal nocturnal hemoglobinuria that causes red blood cell breakdown.

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.

Pegcetacoplan
Pegcetacoplan is a substance that blocks part of the immune system to reduce red blood cell destruction and slow certain age-related vision loss.

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.

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Universitair Medisch Centrum Utrecht
Pediatric Hematology
De Bilt, Netherlands
Radboud universitair medisch centrum / RADBOUDUMC
Pediatrics Department
Nijmegen, Netherlands
Hospital Universitari Vall D Hebron
Department of pediatrics hematology
Barcelona, Spain

Sponsor: Apellis Pharmaceuticals Inc.
Last updated: Nov 26, 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.