A phase 2a study to evaluate the safety and pharmacokinetics of Luspatercept (ACE-536) in paediatric subjects who required regular red blood cell transfusions due to beta thalassemia
- Conditions
- Beta-ThalassemiaMedDRA version: 20.1Level: LLTClassification code 10054660Term: Thalassemia betaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2019-000208-13-IT
- Lead Sponsor
- CELGENE CORPORATIO
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 48
6 months to < 18 years of age at the time of signing the informed consent form (ICF)/informed assent form (IAF).
Documented diagnosis of ß-thalassemia or Hemoglobin E/ß-thalassemia
Regularly transfused, defined as: >= 4 RBC transfusions 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.
Karnofsky (age >=16 years) or Lansky (age < 16 years) performance status score >= 50 at screening
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Subject has a diagnosis of Hemoglobin S/ß-thalassemia or alpha (a)-thalassemia (eg, Hemoglobin H); ß-thalassemia combined with a-thalassemia is allowed.
Subject has chronic anticoagulant therapy <= 28 days prior to enrollment
Subject has used an erythropoiesis-stimulating agent (ESA) <= 24 weeks prior to enrollment.
Subject use of iron chelation therapy, if initiated <= 8 weeks prior to enrollment (allowed if initiated > 8 weeks before or during treatment).
Subject use of hydroxyurea treatment <= 24 weeks prior to enrollment.
Subject use of chronic systemic glucocorticoids <= 12 weeks prior to enrollment
Subject use of cytotoxic agents, immunosuppressants <= 28 days prior to enrollment
Subject has treatment with another investigational drug or device <= 28 days prior to enrollment
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of the study is:<br>- To determine the recommended dose (RD) of luspatercept that is safe and tolerable in pediatric subjects with transfusion-dependent (TD) ß-thalassemia<br>- To examine the pharmacokinetic (PK) profile of luspatercept in pediatric subjects with TD ß-thalassemia;Secondary Objective: The secondary objectives are to evaluate:<br>- The mean change in red blood cell (RBC) transfusion burden<br>- The hemoglobin levels over the course of the study<br>- The immunogenicity of luspatercept<br>- Safety of luspatercept in pediatric subjects;Primary end point(s): Determination of the Recommended Dose<br>Development of a PK model that describes the PK exposure data of luspatercept and associated variability;Timepoint(s) of evaluation of this end point: Cycle 1 Day 1 through Cycle 1 Day 22<br>Cycle 1 Day 1 through EOT following the Treatment Period
- Secondary Outcome Measures
Name Time Method Secondary end point(s): To evaluate the change in RBC transfusion burden<br>To evaluate the change in hemoglobin levels over the course of the study<br>Safety<br>Frequency of antidrug antibodies and its effect on safety<br>Exposure-response relationships for measures of activities and toxicity;Timepoint(s) of evaluation of this end point: 12 weeks prior to enrollment; 12 weeks of the Treatment Period through to EOT<br>Cycle 1 Day 1 through 12 weeks post-last dose<br>Cycle 1 Day 1 through 12 weeks post-last dose; if positive, every 24 weeks for up to 2 years from Cycle 1 Day 1 or until negative or stabilized<br>Cycle 1 Day 1 through EOT following the Treatment Period