New drug pegcetacoplan tested in teens with rare blood disorder

NCT ID NCT04901936

Recruiting now Disease control Sponsor: Apellis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called pegcetacoplan in 12 to 17-year-olds with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug aims to control the disease by blocking part of the immune system. The trial will check safety, how the drug works in the body, and its effects on blood counts over 16 weeks.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: pegcetacoplan
What this could lead to: If successful, this could provide a new treatment option for teenagers with PNH, helping control red blood cell destruction and reduce symptoms.
What could go wrong: This is a small, early-phase study with only 12 participants, so results may not apply to all patients. Risks include infections and other side effects from the drug.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal hemolytic anemia paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PAROXYSMAL HEMOGLOBINURIA PAROXYSMAL NOCTURNAL HEMOGLOBINURIA, PNH

Contacts and locations

Locations

Children's Hospital of Atlanta
RECRUITING

Atlanta, Georgia, 30329, United States
Hospital Ampang
RECRUITING

Ampang, 68000, Malaysia
Maharaj Nakorn Chiang Mai hospital
RECRUITING

Chiang Mai, 50200, Thailand
Motol University Hospital
COMPLETED

Prague, 150 06, Czechia
Phramongkutklao Hospital and College of Medicine
COMPLETED

Bangkok, 10400, Thailand
Radboud University Hospital Nijmegen
COMPLETED

Nijmegen, 6525 GA, Netherlands
Robert-Debré Hospital Paris
NOT_YET_RECRUITING

Paris, 75009, France
St. Mary's Hospital
COMPLETED

London, W2 1NY, United Kingdom
University Children's Hospital
RECRUITING

Belgrade, 11000, Serbia
University Hospital 12 de Octubre
RECRUITING

Madrid, E-28041, Spain
University Hospital Vall d'Hebron
RECRUITING

Barcelona, E-08035, Spain
University Medical Center Utrecht
RECRUITING

Utrecht, 3508 GA, Netherlands

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