New drug shows promise for rare blood disorder
NCT ID NCT05096403
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a drug called pegcetacoplan in 24 adults with cold agglutinin disease, a rare condition where the immune system destroys red blood cells. Participants received either the drug or a placebo twice a week for 24 weeks. The goal was to see if the drug could raise hemoglobin levels and reduce the need for blood transfusions.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Pegcetacoplan (a drug injected under the skin twice a week)
What this could lead to
If it works, this could offer a new treatment option for people with cold agglutinin disease to improve red blood cell levels and reduce the need for blood transfusions.
What could go wrong
This is a small Phase 3 trial with only 24 participants, so results may not apply to everyone. The drug requires lifelong use and carries infection risks due to required vaccinations.
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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Locations
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"LTD Medinvest Institute of Hematology and Transfusiology "
Tbilisi, Georgia
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"FOND IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, Italy
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A.O.R.N. S.G. Moscati di Avellino
Avellino, 83100, Italy
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AOU Maggiore della Carità SCDU Ematologia
Novara, Italy
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ASST degli Spedali Civili di Brescia_Presidio Ospedaliero di Brescia_U.O. Ematologia
Brescia, 25123, Italy
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Aichi Medical University Hospital
Nagakute, 480-1195, Japan
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Algemeen Ziekenhuis Klina
Brasschaat, 2930, Belgium
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Amsterdam UMC
Amsterdam, Netherlands
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Azienda Ospedaliera Ospedali Riuniti ""Villa Sofia-Cervello
Palermo, 90146, Italy
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CHU de Liège
Liège, 4000, Belgium
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Cancer Clinical Trials Unit, Haematology -University College London -
London, United Kingdom
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Churchill Hospital
Oxford, United Kingdom
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East Carolina University Division of Hematology/ Oncology
Greenville, North Carolina, 27858, United States
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Fukushima Medical University Hospital
Fukushima, Japan
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Grande Ospedale Metropolitano ""Bianchi - Melacrino - Morellii
Reggio Calabria, 89133, Italy
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Haukeland University Hospital
Bergen, Norway
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Helsinki University Hospital - Comprehensive Cancer Center
Helsinki, Finland
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Hospital Clinic de Barcelona
Barcelona, Spain
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Hospital Clínico Universitario de Salamanca
Madrid, Spain
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Hospital Universitario Infanta Leonor
Madrid, Spain
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Hospital Universitario La Paz
Madrid, Spain
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Hospital Universitario Virgen del Rocio
Seville, Spain
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Hospital Universitario de Gran Canaria Dr. Negrin
Las Palmas de Gran Canaria, Spain
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Hospital Universitario y Politecnico La Fe
Valencia, Spain
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Institut f. Transfusionsmedizin - Universität Ulm
Ulm, Germany
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Ishikawa Prefectural Central Hospital
Kanazawa, 9208530, Japan
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Lakes Research
Miami Lakes, Florida, 33014, United States
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Ltd M. Zodelava Hematology Centre
Tbilisi, Georgia
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Medical University
Vienna, Austria
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Osaka University Hospital
Osaka, 5650871, Japan
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Royal London Hospital
London, E1 2ES, United Kingdom
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Russell Centre for Clinical Haematology
Nottingham, NG5 1PB, United Kingdom
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Semmelweis Egyetem
Budapest, Hungary
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Shinshu University Hospital
Nagano, Japan
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St James' University Hospital
Leeds, LS9 7TF, United Kingdom
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St Olavs Hospital, Avdeling for blodsykdommer
Trondheim, Norway
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St. Michael's Hospital
Toronto, Canada
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Sykehuset Østfold Kalnes
Grålum, Norway
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The Oncology Institute of Hope and Innovation
Whittier, California, 90603, United States
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UZ Gasthuisberg
Leuven, 3000, Belgium
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University of Iowa Hospitals & Clinics - The Hemophilia Treatment Center (HTC)
Iowa City, Iowa, 52242, United States
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University of Tsukuba Hospital
Ibaraki, Japan
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Universitätsklinikum Essen Klinik f. Hämatologie - Westdeutsches Tumorzentrum
Essen, 45147, Germany
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Weill Cornell Medicine / NewYork Presbyterian Hospital
New York, New York, 10021, United States