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Tests treatment safety and results for Beta-Thalassemia
Official title Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
ClinicalTrials.gov ID: NCT04143724
What this study is testing
What is ACE-536?
ACE-536 is an investigational medicine, being studied as a potential treatment for beta-thalassemia.
Also referred to as Luspatercept, BMS-986346.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 5 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 17
You may be able to join if
- 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...
- Participants (and when applicable, parent/legal representative) is willing and able to adhere to the study visit schedule and other protocol...
- Participants must have documented diagnosis of β-thalassemia or Hemoglobin E/β-thalassemia.
- Transfusion dependence (TD):
You likely can't join if
- Participant has a diagnosis of Hemoglobin S/β-thalassemia or alpha (α)-thalassemia (eg, Hemoglobin H); β-thalassemia combined with α-thalassemia is...
- Participant has of active hepatitis C (HCV) infection, as demonstrated by a positive HCF-ribonucleic acid (RNS) test of sufficient sensitivity, or...
- Participant has deep vein thrombosis (DVT), stroke, or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical...
- Participant has platelet count \> 1000 x 109/L.
- 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).
See the full eligibility criteria
Who can join
- 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):
- 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...
- 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...
- Participants have Karnofsky (age ≥16 years) or Lansky (age \< 16 years) performance status score ≥ 50 at screening.
- Female children of childbearing potential (FCCBP), individuals of childbearing potential (IOCBP), and male (as assigned at birth) participants that have reached puberty (and when applicable, parent/legal representative)...
- Female children of childbearing potential, defined as females who have achieved menarche with or without breast development in Tanner Stage 2 or greater and have not undergone a hysterectomy or bilateral oophorectomy...
- Medically supervised serum pregnancy tests with a sensitivity of at least 25 mIU/mL must be conducted in Female children of childbearing potential (FCCBP)/ individuals of childbearing potential (IOCBP), including those...
- Female participants must, as appropriate to age and at the discretion of the site Investigator, either commit to true abstinence\ from heterosexual contact (which must be reviewed on a monthly basis) or agree to use...
- Male (as assigned at birth) 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 Female children of childbearing potential (FCCBP)/...
- True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the participant. [Periodic abstinence (eg, calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not...
What rules you out
- Participant has a diagnosis of Hemoglobin S/β-thalassemia or alpha (α)-thalassemia (eg, Hemoglobin H); β-thalassemia combined with α-thalassemia is allowed.
- Participant has of active hepatitis C (HCV) infection, as demonstrated by a positive HCF-ribonucleic acid (RNS) test of sufficient sensitivity, or active infectious hepatitis B (as demonstrated by the presence of...
- Participant has deep vein thrombosis (DVT), stroke, or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical intervention ≤ 24 weeks prior to enrollment.
- Participant has platelet count \> 1000 x 109/L.
- 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 (candidates for HSCT or gene therapy with anticipated waiting period of ≥ 12 months are eligible).
- Participant use of iron chelation therapy (ICT), if initiated ≤ 8 weeks prior to enrollment (allowed if initiated \> 8 weeks before or during treatment).
- Participant received treatment with hydroxyurea immunomodulatory drugs IMiDs (such as thalidomide), other fetal Hb (HbF) inducers or erythropoiesis-stimulating agents (ESAs) ≤ 12 weeks prior to enrollment for NTD...
- Participant is pregnant or breastfeeding female or plan to get pregnant during the study.
- Participant has uncontrolled hypertension. Controlled hypertension for this protocol is considered: blood pressure value corresponding to ≤ Grade 1 according to NCI CTCAE version 5.0 with or without pharmacological...
- Participant has major organ damage, including:
- Symptomatic splenomegaly
- Liver disease with alanine aminotransferase (ALT)/aspartate aminotransferase (AST) \> 3X the upper limit of normal (ULN) for age
- 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
- Lung disease, including pulmonary fibrosis or pulmonary hypertension of Grade ≥ 3 according to NCI-CTCAE version 5.0.
- Renal insufficiency defined as:
- A serum creatinine based on age/gender based on threshold derived from Schwartz formula for estimating GFR utilizing child length and stature data published by the Centers for Disease Control.
- 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...
- Participant use high dose long-term therapy with systemic glucocorticoids ≤ 12 weeks prior to enrollment (physiologic replacement therapy for adrenal insufficiency is allowed). Low-dose long-term (defined as ≤ 0.2...
- 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:
- Curatively resected nonmelanoma skin cancer.
- Curatively treated carcinoma in situ.
- Other solid tumor with no known active disease in the opinion of the Investigator.
- Participant who has extramedullary hematopoiesis (EMH) complications or requires treatment to control the growth of EMH masse(s) during the screening period.
- Any medical or psychiatric condition that in the opinion of the investigator would put the participant at unacceptable risk of participating in the study or may impact interpretation of the study results.
- Use of herbs or food supplements (eg, Chinese traditional medicine), if, per investigator's judgment, likely to impact the safety and how well it works assessment, for 24 weeks before initiating the study treatment for...
- Other protocol-defined Inclusion/Exclusion criteria apply.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- New York, New York, United States
- Guangzhou, Guangdong, China
- Shenzhen, Guangdong, China
- Nanning, GX, China
- Chengdu, Sichuan, China
- Guangzhou, China
- Nanning, China
- Essen, Germany
- Ulm, Germany
- Athens, Greece
- Hyderabad, Andhra Pradesh, India
- Mumbai, Maharashtra, India
- Nagpur, Maharashtra, India
- Noida, Uttar Pradesh, India
- Kolkata, India
- Vellore, India
- Rome, Roma, Italy
- Genoa, Italy
- Naples, Italy
+ 5 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 5 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; New York, New York, United States; Guangzhou, Guangdong, China; Shenzhen, Guangdong, China; Nanning, GX, China; Chengdu, Sichuan, China and 19 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.