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Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participant must be 6 years to < 18 years of age at the time of signing the informed consent form (ICF)/informed assent form (IAF).
History of regular transfusions for at least 2 years
Must not have
Proteinuria ≥ Grade 3 or urine albumin/creatinine ratio > 129 mg/mmol of creatinine
Use of hydroxyurea treatment ≤ 24 weeks prior to enrollment
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 weeks prior to enrollment; treatment period and up to end of treatment including long-term treatment period - up to 5 years
Awards & highlights
Summary
This trial is testing a drug called luspatercept in children with β-thalassemia to see if it is safe and how it works in their bodies. The study includes children who need regular blood transfusions and those who do not. Luspatercept helps the body make more red blood cells, which can lessen the need for transfusions. Luspatercept is a newly approved treatment for reducing the need for blood transfusions in adults.
Who is the study for?
This trial is for children and adolescents aged 6 to less than 18 with β-thalassemia who need regular blood transfusions. They should have a performance status score ≥50, be on transfusion treatment for at least 2 years, and agree to contraception if applicable. Excluded are those with recent major surgery, certain other health conditions or treatments, or prior exposure to similar drugs.
What is being tested?
The study tests the safety and how the body processes luspatercept (ACE-536) in young patients requiring regular red blood cell transfusions due to β-thalassemia. It has two parts: one for ages 12-<18 and another for ages 6-<12, each with dose escalation followed by expansion cohorts.
What are the potential side effects?
While specific side effects of luspatercept in this pediatric population aren't listed here, common ones may include reactions at injection site, headache, muscle pains or cramps; more serious risks could involve blood pressure changes or abnormal liver function.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 6 and 17 years old and can sign the consent form.
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I have been receiving regular blood transfusions for at least 2 years.
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I am between 6 and 17 years old.
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I have been diagnosed with β-thalassemia.
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I have been diagnosed with β-thalassemia or Hemoglobin E/β-thalassemia.
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I have received 4 or more blood transfusions in the last 6 months without a break of 42 days or more.
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I can do most activities but may need help.
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I can do most activities myself, but may need help.
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I have had at least 4 blood transfusions in the last 6 months without a break of more than 6 weeks.
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I agree to use a condom or practice true abstinence.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My protein levels in urine are very high.
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I have used hydroxyurea within the last 24 weeks.
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My high blood pressure is not under control.
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I haven't used any experimental drugs or devices in the last 28 days.
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I have an active hepatitis C or B infection, or I am HIV positive.
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I have previously been treated with sotatercept or luspatercept.
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My diabetes has been poorly controlled in the last 6 months.
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I need treatment to manage growths related to my bone marrow condition.
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I have been diagnosed with Hemoglobin S/β-thalassemia or α-thalassemia.
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I have significant damage to a major organ.
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I have had or am scheduled for a stem cell transplant or gene therapy.
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I have not had major surgery in the last 3 months.
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I have been on blood thinner medication for less than 28 days before joining.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 12 weeks prior to enrollment; treatment period and up to end of treatment including long-term treatment period - up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 weeks prior to enrollment; treatment period and up to end of treatment including long-term treatment period - up to 5 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Determination of the Recommended Dose (RD
Pharmacokinetics (PK) - CL/F
Pharmacokinetics (PK) - Vd/F
+3 moreSecondary outcome measures
Immunogenicity
Mean change from baseline in mean daily dose of iron chelation therapy (ICT)
Mean change from baseline in serum ferritin
+3 moreTrial Design
9Treatment groups
Experimental Treatment
Group I: Cohort 9: NTD 6 to < 12 years - Luspatercept 1.2 mg/kgExperimental Treatment1 Intervention
Group II: Cohort 8: NTD 6 to < 12 years - Luspatercept 1.0 mg/kgExperimental Treatment1 Intervention
Group III: Cohort 7 (Expansion Phase): NTD 12 to < 18 yearsExperimental Treatment1 Intervention
Group IV: Cohort 6 (Dose Confirmation Phase): NTD 12 to < 18 years - Luspatercept 1.0 mg/kgExperimental Treatment1 Intervention
Group V: Cohort 5: 6 to <12 years: Luspatercept 1.2 mg/kgExperimental Treatment1 Intervention
Luspatercept 1.2 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Group VI: Cohort 4: 6 to < 12 years: Luspatercept 1.0 mg/kgExperimental Treatment1 Intervention
Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Group VII: Cohort 3 (Expansion Cohort): 12 to <18 years Luspatercept 1.0 mg/kgExperimental Treatment1 Intervention
Group VIII: Cohort 2: 12 to < 18 years: Luspatercept 1.0 mg/kg,Experimental Treatment1 Intervention
Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Group IX: Cohort 1: 12 to < 18 years - Luspatercept 0.75 mg/kgExperimental Treatment1 Intervention
Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
ACE-536
2011
Completed Phase 1
~40
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Beta Thalassemia treatments primarily aim to address ineffective erythropoiesis and anemia. Luspatercept, an erythroid maturation agent, works by promoting the maturation of red blood cells, thereby increasing hemoglobin levels and reducing the need for blood transfusions.
This is crucial for Beta Thalassemia patients as it helps manage chronic anemia and reduces complications associated with frequent transfusions, such as iron overload. Other common treatments include regular blood transfusions to maintain adequate hemoglobin levels and iron chelation therapy to manage iron overload.
These treatments collectively improve the quality of life and overall prognosis for Beta Thalassemia patients.
Colla corii asini might upregulate ZNF471 and THOC5 by KRAB domain-containing zinc-finger protein pathway and THO complex subunit 5 pathway to improve anemia of pregnant women with β-thalassemia.
Colla corii asini might upregulate ZNF471 and THOC5 by KRAB domain-containing zinc-finger protein pathway and THO complex subunit 5 pathway to improve anemia of pregnant women with β-thalassemia.
Find a Location
Who is running the clinical trial?
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USAIndustry Sponsor
32 Previous Clinical Trials
4,168 Total Patients Enrolled
CelgeneLead Sponsor
645 Previous Clinical Trials
130,450 Total Patients Enrolled
Acceleron Pharma Inc. (a wholly owned subsidiary of Merck Sharp and Dohme, a subsidiary of Merck & Co., Inc.)Industry Sponsor
32 Previous Clinical Trials
4,168 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am between 6 and 17 years old and can sign the consent form.My kidneys do not work well.If you are a female who can have children or a male who has reached puberty, you must talk to a doctor about the effects of the study treatment on your ability to have children.I am between 6 and 17 years old.I have been diagnosed with β-thalassemia.My protein levels in urine are very high.I received a live COVID-19 vaccine within the last 28 days.I haven't used any experimental drugs or devices in the last 28 days.I agree to learn and discuss how the treatment might affect my ability to have children.I have used hydroxyurea within the last 24 weeks.I have used a drug to increase red blood cells within the last 6 months.You (and your parent/guardian, if applicable) are willing and able to attend all study appointments and follow the study rules.I have been diagnosed with β-thalassemia or Hemoglobin E/β-thalassemia.My high blood pressure is not under control.I have received 4 or more blood transfusions in the last 6 months without a break of 42 days or more.I have an active hepatitis C or B infection, or I am HIV positive.I have been receiving regular blood transfusions for at least 2 years.Female participants who are able to have children must take a pregnancy test before starting the study and agree to take more tests during the study. They must have two negative pregnancy tests before starting the study medication, and agree to ongoing pregnancy testing during the study and after it ends.I can do most activities but may need help.I haven't had a severe infection or unresolved COVID-19 symptoms in the last 28 days.You (and your parent/legal representative, if applicable) need to read and sign a form that explains the study and the procedures involved before you can participate.I have been receiving regular blood transfusions for at least 2 years.I have not used long-term steroids in the last 12 weeks.I have previously been treated with sotatercept or luspatercept.I haven't used any strong immune system drugs in the last 28 days.My diabetes has been poorly controlled in the last 6 months.I am a woman who can follow strict rules for avoiding pregnancy during and after the study.I need treatment to manage growths related to my bone marrow condition.I have been diagnosed with Hemoglobin S/β-thalassemia or α-thalassemia.I have significant damage to a major organ.I have had or am scheduled for a stem cell transplant or gene therapy.I have had cancer before, but it's one of the exceptions.I can do most activities myself, but may need help.I have not had major surgery in the last 3 months.I have been on blood thinner medication for less than 28 days before joining.I have had a blood clot or stroke needing treatment in the last 6 months.I have had at least 4 blood transfusions in the last 6 months without a break of more than 6 weeks.I started iron chelation therapy less than 8 weeks ago.You are currently pregnant or breastfeeding.I agree to use a condom or practice true abstinence.You have a medical condition that is too risky for you to participate in the study.You have a medical condition that could make it difficult to understand the study results.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 2: 12 to < 18 years: Luspatercept 1.0 mg/kg,
- Group 2: Cohort 7 (Expansion Phase): NTD 12 to < 18 years
- Group 3: Cohort 9: NTD 6 to < 12 years - Luspatercept 1.2 mg/kg
- Group 4: Cohort 8: NTD 6 to < 12 years - Luspatercept 1.0 mg/kg
- Group 5: Cohort 3 (Expansion Cohort): 12 to <18 years Luspatercept 1.0 mg/kg
- Group 6: Cohort 4: 6 to < 12 years: Luspatercept 1.0 mg/kg
- Group 7: Cohort 5: 6 to <12 years: Luspatercept 1.2 mg/kg
- Group 8: Cohort 6 (Dose Confirmation Phase): NTD 12 to < 18 years - Luspatercept 1.0 mg/kg
- Group 9: Cohort 1: 12 to < 18 years - Luspatercept 0.75 mg/kg
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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