Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
NCT04143724 · View on ClinicalTrials.gov ↗
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
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
| Field | Value |
|---|---|
| Enrollment Target | 99 participants |
| Start Date | 2019-11-07 |
| Est. Completion | 2035-06-11 |
| Phase | Phase 2 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical 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. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 99 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Celgene, which has 73 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04143724 reports 20 study locations spanning 10 distinct geographic areas — top geographies include Other, Guangdong, Maharashtra. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04143724?
The interventions under investigation include: ACE-536 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04143724?
This trial is sponsored by Celgene, which has 73 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.