New drug hopes to ease transfusion burden for kids with blood disorder

NCT ID NCT04143724

Recruiting now Disease control Sponsor: Celgene Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jul 10, 2026 · Updated 2 times

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.

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Conditions

The condition(s) this trial relates to.

beta thalassemia hemoglobin E-beta-thalassemia syndrome

As listed by the trial registrant

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

BETA-THALASSEMIA

Contacts and locations

Locations

AOU dell'Universita degli Studi della Campania Luigi Vanvitelli
RECRUITING

Naples, 80131, Italy
Azienda Ospedaliero Universitaria S. Luigi Gonzaga
RECRUITING

Orbassano, 10043, Italy
Christian Medical College & Hospital
RECRUITING

Vellore, 632004, India
Chulalongkorn University Faculty of Medicine - King Chulalongkorn Memorial Hospital
RECRUITING

Bangkok, 10330, Thailand
Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite
RECRUITING

Genoa, 16128, Italy
General Children's Hospital "Agia Sophia"
RECRUITING

Athens, 115 27, Greece
Kamala Hospital and Research Center
RECRUITING

Hyderabad, Andhra Pradesh, 500052, India
Kingsway Hospitals
RECRUITING

Nagpur, Maharashtra, 440001, India
Local Institution - 401
COMPLETED

Izmir, 35100, Turkey (Türkiye)
Local Institution - 601
COMPLETED

Los Angeles, California, 90027, United States
Local Institution - 700
WITHDRAWN

Beirut, 0, Lebanon
Local Institution - 803
WITHDRAWN

Kolkata, 700094, India
MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre, Borivali (E)
RECRUITING

Mumbai, Maharashtra, 400022, India
New York Presbyterian Hospital
RECRUITING

New York, New York, 10065-4870, United States
Ospedale Pediatrico Bambino Gesù IRCCS
RECRUITING

Rome, Roma, 00165, Italy
People's Liberation Army The 923rd Hospital
RECRUITING

Nanning, GX, 530021, China
Post Graduate Institute of Child Health
RECRUITING

Noida, Uttar Pradesh, 201303, India
Ramathibodi Hospital, Mahidol University
RECRUITING

Phyathai, 10400, Thailand
Shenzhen Second People's Hospital
RECRUITING

Shenzhen, Guangdong, 518028, China
Siriraj Hospital Mahidol University
RECRUITING

Bangkok, 10700, Thailand
Southern Medical University Nanfang Hospital
RECRUITING

Guangzhou, Guangdong, 510515, China
Sun Yat-sen Memorial Hospital, Sun Yat-Sen University
RECRUITING

Guangzhou, 510120, China
The First Affiliated Hospital of Guangxi Medical University
RECRUITING

Nanning, 530021, China
Universitatsklinikum Ulm
RECRUITING

Ulm, 89081, Germany
Universitätsklinikum Essen
RECRUITING

Essen, 45147, Germany
West China Hospital - Sichuan University
RECRUITING

Chengdu, Sichuan, 610041, China

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