New drug hopes to ease transfusion burden for kids with blood disorder
NCT ID NCT04143724
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a drug called luspatercept in children aged 6 to 18 with beta-thalassemia, a blood disorder that often requires regular transfusions. The goal is to see if the drug is safe and can reduce the need for transfusions or raise hemoglobin levels. The trial is still recruiting and will follow participants for up to 5 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
luspatercept (ACE-536)
What this could lead to
If successful, this could lead to a treatment that reduces the need for blood transfusions and improves hemoglobin levels in children with beta-thalassemia.
What could go wrong
This is an early-phase (Phase 2a) study with a small number of participants, so results may not apply broadly. The drug may not be effective or could cause unexpected side effects in children.
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: •••••@•••••
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Contact
Locations
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AOU dell'Universita degli Studi della Campania Luigi Vanvitelli
RECRUITINGNaples, 80131, Italy
Contact Phone: •••-•••-••••
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Azienda Ospedaliero Universitaria S. Luigi Gonzaga
RECRUITINGOrbassano, 10043, Italy
Contact
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Christian Medical College & Hospital
RECRUITINGVellore, 632004, India
Contact
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Chulalongkorn University Faculty of Medicine - King Chulalongkorn Memorial Hospital
RECRUITINGBangkok, 10330, Thailand
Contact
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Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite
RECRUITINGGenoa, 16128, Italy
Contact Phone: •••-•••-••••
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General Children's Hospital "Agia Sophia"
RECRUITINGAthens, 115 27, Greece
Contact Phone: •••-•••-••••
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Kamala Hospital and Research Center
RECRUITINGHyderabad, Andhra Pradesh, 500052, India
Contact
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Kingsway Hospitals
RECRUITINGNagpur, Maharashtra, 440001, India
Contact
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Local Institution - 401
COMPLETEDIzmir, 35100, Turkey (Türkiye)
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Local Institution - 601
COMPLETEDLos Angeles, California, 90027, United States
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Local Institution - 700
WITHDRAWNBeirut, 0, Lebanon
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Local Institution - 803
WITHDRAWNKolkata, 700094, India
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MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre, Borivali (E)
RECRUITINGMumbai, Maharashtra, 400022, India
Contact
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New York Presbyterian Hospital
RECRUITINGNew York, New York, 10065-4870, United States
Contact Phone: •••-•••-••••
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Ospedale Pediatrico Bambino Gesù IRCCS
RECRUITINGRome, Roma, 00165, Italy
Contact Phone: •••-•••-••••
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People's Liberation Army The 923rd Hospital
RECRUITINGNanning, GX, 530021, China
Contact Phone: •••-•••-••••
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Post Graduate Institute of Child Health
RECRUITINGNoida, Uttar Pradesh, 201303, India
Contact Phone: •••-•••-••••
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Ramathibodi Hospital, Mahidol University
RECRUITINGPhyathai, 10400, Thailand
Contact
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Shenzhen Second People's Hospital
RECRUITINGShenzhen, Guangdong, 518028, China
Contact Phone: •••-•••-••••
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Siriraj Hospital Mahidol University
RECRUITINGBangkok, 10700, Thailand
Contact Phone: •••-•••-••••
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Southern Medical University Nanfang Hospital
RECRUITINGGuangzhou, Guangdong, 510515, China
Contact
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Sun Yat-sen Memorial Hospital, Sun Yat-Sen University
RECRUITINGGuangzhou, 510120, China
Contact Phone: •••-•••-••••
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The First Affiliated Hospital of Guangxi Medical University
RECRUITINGNanning, 530021, China
Contact Phone: •••-•••-••••
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Universitatsklinikum Ulm
RECRUITINGUlm, 89081, Germany
Contact Phone: •••-•••-••••
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Universitätsklinikum Essen
RECRUITINGEssen, 45147, Germany
Contact
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West China Hospital - Sichuan University
RECRUITINGChengdu, Sichuan, 610041, China
Contact