New drug pegcetacoplan tested in teens with rare blood disorder
NCT ID NCT04901936
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States
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Hospital Ampang
RECRUITINGAmpang, 68000, Malaysia
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Maharaj Nakorn Chiang Mai hospital
RECRUITINGChiang Mai, 50200, Thailand
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Motol University Hospital
COMPLETEDPrague, 150 06, Czechia
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Phramongkutklao Hospital and College of Medicine
COMPLETEDBangkok, 10400, Thailand
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Radboud University Hospital Nijmegen
COMPLETEDNijmegen, 6525 GA, Netherlands
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Robert-Debré Hospital Paris
NOT_YET_RECRUITINGParis, 75009, France
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St. Mary's Hospital
COMPLETEDLondon, W2 1NY, United Kingdom
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University Children's Hospital
RECRUITINGBelgrade, 11000, Serbia
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University Hospital 12 de Octubre
RECRUITINGMadrid, E-28041, Spain
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University Hospital Vall d'Hebron
RECRUITINGBarcelona, E-08035, Spain
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University Medical Center Utrecht
RECRUITINGUtrecht, 3508 GA, Netherlands