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NCT04143724 · ClinicalTrials.gov registry record · Phase 2

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

A Phase 2 study of Beta-Thalassemia, sponsored by Celgene.

Recruiting
Registry status
Phase 2
Development phase
99
Enrollment target
20
Study locations

NCT04143724: Recruiting Phase 2 study of Beta-Thalassemia, sponsored by Celgene.

NCT04143724 is a Phase 2 study of Beta-Thalassemia that is actively recruiting participants, run by Celgene. The registered enrollment target is 99 participants, below the 174-participant average among 14 other Beta-Thalassemia trials with a reported enrollment target (43% lower). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT04143724, a Phase 2 study of Beta-Thalassemia, is actively recruiting participants, sponsored by Celgene.

RECRUITING
Registry status
Phase 2
Development phase
99 participants
Enrollment target
20
Study locations

Study Summary

This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants aged 12 to \<18 years with two dose escalation cohorts, followed by a dose expansion cohorts. NTD Part A will be conducted in the same age group participants as TD Part A with dose confirmation and expansion cohorts. After Part A TD participants have completed at least one year of treatment, all available safety data from Part A adolescent participants will be evaluated before initiating TD and NTD Part B in the age group from 6 to \<12 years old. Part B will consist of two dose escalation cohorts for TD and two dose escalation cohorts for NTD. Upon completion of the Treatment Period, participants of any cohort who are benefiting from the study treatment, will be offered the opportunity to continue luspatercept treatment in the Long-term Treatment Period for up to 5 years from their first dose. Participants who discontinue study treatment at any time will continue in the Posttreatment Follow-up Period for at least 5 years from their first dose of luspatercept, or 3 years from their last dose, whichever occurs later, or until they withdraw consent/assent, are lost to follow-up, or the End of Trial, whichever occurs first.

Primary Outcome

Determine the recommended dose of luspatercept that is safe and tolerable in pediatric participants with transfusion-dependent B-thalassemia or non-transfusion-dependent β-thalassemia

Conditions Studied

Beta-Thalassemia

Interventions

  • DRUG ACE-536

Study Locations (20)

Other

  • Sun Yat-sen Memorial Hospital, Sun Yat-Sen University - Guangzhou
  • The First Affiliated Hospital of Guangxi Medical University - Nanning
  • Universitätsklinikum Essen - Essen
  • Universitatsklinikum Ulm - Ulm
  • General Children's Hospital "Agia Sophia" - Athens
  • Local Institution - 803 - Kolkata
  • Christian Medical College & Hospital - Vellore
  • Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite - Genoa
  • AOU dell'Universita degli Studi della Campania Luigi Vanvitelli - Naples

Guangdong

  • Southern Medical University Nanfang Hospital - Guangzhou
  • Shenzhen Second People's Hospital - Shenzhen

Maharashtra

  • MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre, Borivali (E) - Mumbai
  • Kingsway Hospitals - Nagpur

California

  • Local Institution - 601 - Los Angeles

New York

  • New York Presbyterian Hospital - New York

GX

  • People's Liberation Army The 923rd Hospital - Nanning

Sichuan

  • West China Hospital - Sichuan University - Chengdu

Andhra Pradesh

  • Kamala Hospital and Research Center - Hyderabad

Trial Details

FieldValue
Enrollment Target 99 participants
Start Date 2019-11-07
Est. Completion 2035-06-11
Phase Phase 2
Celgene

308 total trials

What NCT04143724 shows while recruiting

NCT04143724 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 99 participants, a relatively small participant target, below the 174-participant average among 14 other Beta-Thalassemia trials with a reported enrollment target (43% lower).

The record links to 1 condition, with Beta-Thalassemia appearing as the primary indexed condition, and to 1 intervention - of which ACE-536 is the first listed.

NCT04143724 names 20 study sites across 10 states, led by Other, Guangdong, Maharashtra.

Frequently Asked Questions

What is clinical trial NCT04143724 about?

NCT04143724 is a clinical study titled "Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia". This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in ado...

What is the current status of trial NCT04143724?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 99 participants. The study started on 2019-11-07. Estimated completion is 2035-06-11.

What conditions does trial NCT04143724 study?

This clinical trial studies the following conditions: Beta-Thalassemia.

What interventions are being tested in trial NCT04143724?

The interventions under investigation include: ACE-536 (DRUG).

Who is sponsoring clinical trial NCT04143724?

This trial is sponsored by Celgene, which has 308 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04143724 being conducted?

This trial has 20 study locations across California, New York, Guangdong, GX, Sichuan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Learn More About Clinical Trials

Similar trials for Beta-Thalassemia

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT04143724's enrollment target sits among peer trials

99 5th of 14 higher than 10 of 14 other Beta-Thalassemia trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Beta-Thalassemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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More recruiting context for NCT04143724

Source: ClinicalTrials.gov NCT04143724, the US trial registry maintained by the National Library of Medicine. NCT04143724 (small enrollment · wide site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.