NCT04143724 · Celgene
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
What this study is about
This is a Phase 2a study to evaluate the safety and how the drug moves through the body (PK) of luspatercept in pediatric participants with β-thalassemia.
View original scientific description
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.
Interventions
DRUG
ACE-536
Specified dose on specified days
Primary outcome measures
Determination of the Recommended Dose (RD
Time frame: Cycle 1 up to the day before Cycle 2 Day 1 or Study Day 22 if not receiving the second treatment cycle
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
Pharmacokinetics - Cmax
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Maximum serum concentration of drug
Pharmacokinetics - AUC
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Area under the curve
Pharmacokinetics (PK) - t1/2
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Half-life
Pharmacokinetics (PK) - CL/F
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Apparent oral clearance
Pharmacokinetics (PK) - Vd/F
Time frame: Time from Cycle 1 Day 1 of Treatment Period up to a maximum of 1 year
Apparent volume of distribution
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Participants must be 6 years to \< 18 years of age at the time of signing the informed consent form (ICF)/informed assent form (IAF).
- Participants (and when applicable, parent/legal representative) must understand and voluntarily sign an ICF/IAF prior to conducting any study-related assessments/procedures.
- Participants (and when applicable, parent/legal representative) is willing and able to adhere to the study visit schedule and other protocol requirements.
- Participants must have documented diagnosis of β-thalassemia or Hemoglobin E/β-thalassemia.
- Transfusion dependence (TD): a. TD participant i. Participant is regularly transfused, defined as: ≥ 4 RBC transfusion events in the 24 weeks prior to enrollment with no transfusion-free period ≥ 42 days during that period. Note: For the purpose of the study, transfusions administered over 2 or 3 consecutive days are considered as part of a single transfusion event. Participant must have a history of regular
Where
- Los Angeles, California
- New York, New York
Collaborators
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 10, 2026 · Source of record for eligibility and locations