Phase 2a Study of Luspatercept in Pediatric Participants With Beta (ß)-Thalassemia
- Conditions
- Beta (ß) ThalassemiaMedDRA version: 20.1Level: LLTClassification code: 10054660Term: Thalassemia beta Class: 10010331Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2022-502499-22-00
- Lead Sponsor
- Celgene Corp.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 76
Participants must satisfy the following criteria to be enrolled into the study: 1) Participant must be 6 years to < 18 years of age the time of signing the informed consent form (ICF)/informed assent form (IAF)., Male participants, as appropriate to age and the discretion of the study physician: - Must practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a synthetic or latex condom during sexual contact with a pregnant female or a FCCBP/FCBP while participating in the study, during dose interruptions and for at least 12 weeks (approximately 5 times the mean terminal t1/2 of luspatercept based on multiple-dose PK data) following IP discontinuation, even if he has undergone a successful vasectomy., Participant (and when applicable, parent/legal representative) must understand and voluntarily sign an ICF/IAF prior to conducting any study-related assessments/procedures., Participant (and when applicable, parent/legal representative) is willing and able to adhere to the study visit schedule and other protocol requirements., Participant must have documented diagnosis of ß-thalassemia or HbE/ß-thalassemia., Transfusion dependence: 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 transfusions for at least 2 years. b) NTD participant (ex-US sites only) i) Participant must have received < 4 RBC transfusion events in the 24 weeks prior to enrollment. ii) Participant must not be on a regular transfusion program and must be RBC transfusion-free for at least 8 weeks prior to enrollment. iii) Participant must have mean baseline hemoglobin = 10 g/dL, based on a minimum of 2 measurements = 1 week apart within 4 weeks prior to enrollment; hemoglobin values within 21 days post-transfusion will be excluded., Participant has Karnofsky (age =16 years) or Lansky (age < 16 years) performance status score = 50 at screening, Female children of childbearing potential (FCCBP), females of childbearing potential (FCBP), and male participants that have reached puberty (and when applicable, parent/legal representative) must agree to undergo physician-approved reproductive education and discuss the side effects of the study therapy on reproduction., Female children of childbearing potential, defined as females who have achieved menarche and/or breast development in Tanner Stage 2 or greater and have not undergone a hysterectomy or bilateral oophorectomy and FCBP defined as a sexually mature woman who has achieved menarche at some point, has not undergone a hysterectomy or bilateral oophorectomy and has not been naturally postmenopausal for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) must meet the following conditions below (Note: Secondary amenorrhea from any cause does not rule out childbearing potential): - Medically supervised serum pregnancy tests with a sensitivity of at least 25 mIU/mL must be conducted in FCCBP/FCBP, including those who commit to complete abstinence*. Female children of childbearing potential/FCBP must have 2 negative pregnancy tests as verified by the Investigator prior to starting study therapy (one of these tests should be
The presence of any of the following will exclude a participant from enrollment into the study: 1) Participant has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the participant from participating in the study., Participant has platelet count > 1000 x 109/L., Participant has poorly controlled diabetes mellitus within 24 weeks prior to enrollment as defined by short term (eg, hyperosmolar or ketoacidotic crisis) and/or history of diabetic cardiovascular complications (eg, stroke or myocardial infarction)., Participant has treatment with another investigational drug or device = 28 days prior to enrollment., Participant has prior exposure to sotatercept (ACE-011) or luspatercept (ACE-536)., Participant underwent or is scheduled for HSCT or gene therapy., Participant has used an erythropoiesis-stimulating agent (ESA) = 24 weeks prior to enrollment., Participant use of iron chelation therapy (ICT), if initiated = 8 weeks prior to enrollment (allowed if initiated > 8 weeks before or during treatment)., Participant use of hydroxyurea treatment = 24 weeks prior to enrollment., Participant is pregnant or breastfeeding female., Participant has uncontrolled hypertension. Controlled hypertension for this protocol is considered = Grade 1 according to NCI CTCAE version 5.0., Participant has any condition including the presence of laboratory abnormalities, which places the participant at unacceptable risk if he/she were to participate in the study., Participant has major organ damage, including: a) Symptomatic splenomegaly b) Liver disease with alanine aminotransferase (ALT)/aspartate aminotransferase (AST) > 3× the upper limit of normal (ULN) for age c) Heart disease, heart failure as classified by the New York Heart Association (NYHA) classification 3 or higher, or significant arrhythmia requiring treatment, or recent myocardial infarction within 6 months of enrollment d) Lung disease, including pulmonary fibrosis or pulmonary hypertension which are clinically significant e) Renal insufficiency, Participant has proteinuria = Grade 3 according to NCI CTCAE version 5.0. (which is equivalent to a urine protein/creatinine ratio > 215 mg/mmol of creatinine), or a urine albumin/creatinine ratio > 129 mg/mmol of creatinine., Participant use of chronic systemic glucocorticoids = 12 weeks prior to enrollment (physiologic replacement therapy for adrenal insufficiency is allowed). Single day glucocorticoid treatment (eg, for prevention or treatment of transfusion reactions) is allowed., Participant has major surgery = 12 weeks prior to enrollment (participants must have completely recovered from any previous surgery prior to enrollment)., Participant has history of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the IP (refer to the IB)., Participant use of cytotoxic agents, immunosuppressants = 28 days prior to enrollment (ie, antithymocite globulin [ATG] or cyclosporine)., Participant has history of malignancy with the exception of: a) Curatively resected nonmelanoma skin cancer. b) Curatively treated cervical carcinoma in situ. c) Other solid tumor with no known active disease in the opinion of the Investigator., Participant who has EMH complications or requires treatment to control the growth of EMH masse(s) during the screening period., Participant has any condition that confounds the ability to interpret data from the study., Participant has a dia
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method