Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

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 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.

View on ClinicalTrials.gov ↗

View your shortlist →

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.

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

Sponsor

Celgene

308 total trials

Interested in This Trial?

Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT04143724

The ClinicalTrials.gov registry entry for NCT04143724 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 99 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 174-participant average among 14 other Beta-Thalassemia trials with a reported enrollment target (43% lower). The listed sponsor is Celgene, which has 308 total studies on file at ClinicalTrials.gov.

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 reports 20 study locations spanning 10 distinct geographic areas - top geographies include 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.

Explore more on PlainTrial

Beta-Thalassemia

All registered trials for this condition

California

All trials with a study site in California

Recruiting now

Find trials open to new participants near you

Rankings

Top sponsors, conditions, and phases

Read our methodology - how this data is sourced, computed, and verified.

Related

Browse all → | Rankings →
Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.