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AG-946 for Myelodysplastic Syndrome
Phase 2
Recruiting
Research Sponsored by Agios Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
If taking iron chelation therapy, the iron chelation therapy dose must have been stable and started ≥56 days before administration of the first dose of study drug
2 forms of contraception, 1 of which must be considered highly effective, from the time of providing informed consent, throughout the study, and for 28 days after the last dose of study drug for women and 90 days after the last dose of study drug for men. The second form of contraception can be an acceptable barrier method
Must not have
Platelet count <50,000/μL (50 × 109/L) assessed in the absence of platelet transfusions within 28 days before Screening
Currently receiving treatment with luspatercept, EPO, or G-CSF. Treatment with EPO or G-CSF must have been stopped for ≥28 days before administration of the first dose of study drug; treatment with luspatercept must have been stopped for ≥65 days before randomization
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 8 through week 16
Awards & highlights
No Placebo-Only Group
Summary
This trial is testing a new drug called AG-946 to see if it can help people with a blood disorder called Low-Risk Myelodysplastic Syndromes (LR-MDS). The goal is to find out if the drug can improve the production of healthy red blood cells.
Who is the study for?
Adults with low-risk myelodysplastic syndromes (LR-MDS) and anemia can join this trial. They should have hemoglobin levels below 11.0 g/dL, a good performance status, and less than 5% bone marrow blasts. Participants must not be heavily dependent on blood transfusions and agree to use effective contraception if applicable.
What is being tested?
The study is testing AG-946 against a placebo in two phases to see if it helps improve anemia in LR-MDS patients. Phase 2a establishes the concept while Phase 2b compares AG-946's effectiveness at different doses versus placebo.
What are the potential side effects?
Specific side effects of AG-946 are not listed but may include typical reactions seen with other treatments for MDS such as fatigue, nausea, injection site reactions, or potential allergic responses to the drug's components.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been on a stable dose of iron chelation therapy for at least 56 days.
Select...
I am using two forms of birth control, one highly effective, during and after the study.
Select...
My MDS is classified as lower-risk with a score ≤3.5 and less than 5% blasts.
Select...
I have been on a stable dose of iron chelation therapy for at least 56 days.
Select...
I can take care of myself and am up and about more than half of my waking hours.
Select...
I have received fewer than 3 blood transfusions in the last 4 months and none in the last 2 months.
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My MDS is classified as lower-risk with a score of ≤3.5 and less than 5% blasts.
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I am 18 years old or older.
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My MDS is classified as lower-risk with a score ≤3.5 and less than 5% blasts.
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I can take care of myself and am up and about more than half of my waking hours.
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I am able to care for myself and perform daily activities.
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I am 18 years old or older.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My platelet count is below 50,000 without recent transfusions.
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I stopped taking EPO or G-CSF at least 28 days ago, or luspatercept at least 65 days ago.
Select...
My kidney function is reduced, with an eGFR below 45 mL/min.
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I have stopped taking EPO or G-CSF for at least 28 days, or luspatercept for at least 65 days, before starting the study drug.
Select...
My kidney function is reduced, with an eGFR below 45 mL/min.
Select...
My MDS developed after treatment for another disease.
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I haven't had serious heart or lung problems in the last 6 months.
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I am allergic to AG-946 or its ingredients.
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I have been diagnosed with acute myeloid leukemia.
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I have fully recovered from any major surgery I had in the last 12 weeks.
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I have an active hepatitis C infection or tested positive for hepatitis B.
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I am allergic to AG-946 or its ingredients.
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I have fully recovered from any major surgery I had in the last 12 weeks.
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My platelet count is 75,000 or less without recent transfusions.
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I haven't stopped P-gp inhibitors for the required time before starting the study drug.
Select...
I have a history of acute myeloid leukemia (AML).
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline, week 8 through week 16
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 8 through week 16
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Phase 2a: Proportion of Participants With Hemoglobin (Hb) Response
Phase 2a: Proportion of Participants With Transfusion Independence During the Core Period
Phase 2b: Proportion of Participants With Transfusion Independence
Secondary study objectives
Phase 2a: Proportion of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Leading to Discontinuation During the Core Period
Phase 2a: Proportion of Participants With Hb 1.0+ Response
Phase 2b: Duration of Transfusion Independence (TI)
+1 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
4Treatment groups
Experimental Treatment
Group I: Core Period: Phase 2b - Tebapivat Dose Level 3Experimental Treatment1 Intervention
Participants will receive Dose level 3 of tebapivat, orally, once daily for up to 24 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks.
Group II: Core Period: Phase 2b - Tebapivat Dose Level 2Experimental Treatment1 Intervention
Participants will receive Dose level 2 of tebapivat, orally, once daily for up to 24 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks.
Group III: Core Period: Phase 2b - Tebapivat Dose Level 1Experimental Treatment1 Intervention
Participants will receive Dose level 1 of tebapivat, orally, once daily for up to 24 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks.
Group IV: Core Period: Phase 2a - TebapivatExperimental Treatment1 Intervention
Participants will receive tebapivat orally, once daily for up to 16 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks.
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
Common treatments for Myelodysplastic Syndrome (MDS) include erythropoiesis-stimulating agents (ESAs) like epoetin alfa and darbepoetin alfa, which boost red blood cell production by stimulating the bone marrow. Thrombopoietin mimetics, such as romiplostim, increase platelet counts but carry a risk of leukemic transformation.
DNA methyltransferase inhibitors like azacitidine and decitabine work by reactivating silenced genes that control cell growth and differentiation, thereby reducing the proliferation of abnormal cells. These treatments are crucial for MDS patients as they address the hematologic deficiencies characteristic of the disease, improving quality of life and potentially delaying progression to acute myeloid leukemia (AML).
Mutations in myelodysplastic syndromes: Core abnormalities and CHIPping away at the edges.Lenalidomide as a disease-modifying agent in patients with del(5q) myelodysplastic syndromes: linking mechanism of action to clinical outcomes.Combination therapy with DNA methyltransferase inhibitors in hematologic malignancies.
Mutations in myelodysplastic syndromes: Core abnormalities and CHIPping away at the edges.Lenalidomide as a disease-modifying agent in patients with del(5q) myelodysplastic syndromes: linking mechanism of action to clinical outcomes.Combination therapy with DNA methyltransferase inhibitors in hematologic malignancies.
Find a Location
Who is running the clinical trial?
Agios Pharmaceuticals, Inc.Lead Sponsor
53 Previous Clinical Trials
4,099 Total Patients Enrolled
Medical Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have been on a stable dose of iron chelation therapy for at least 56 days.My platelet count is below 50,000 without recent transfusions.I do not have any health conditions that my doctor thinks would make this study unsafe for me.I am using two forms of birth control, one highly effective, during and after the study.I have been on a stable dose of iron chelation therapy for at least 56 days.My MDS is classified as lower-risk with a score ≤3.5 and less than 5% blasts.I have had cancer before, but it was not active or treated in the last 5 years, except for certain skin, cervical, or breast cancers.I am using or willing to use two forms of birth control if I or my partner can become pregnant.I am not currently on antibiotics or have finished them at least 7 days ago.My kidney function is reduced, with an eGFR below 45 mL/min.I have been on a stable dose of iron chelation therapy for at least 56 days.My kidney function is reduced, with an eGFR below 45 mL/min.I haven't had serious heart or lung problems in the last 6 months.I stopped taking EPO or G-CSF at least 28 days ago, or luspatercept at least 65 days ago.I am not engaging in or will use contraception to prevent pregnancy during the trial.I have stopped taking EPO or G-CSF for at least 28 days, or luspatercept for at least 65 days, before starting the study drug.My MDS developed after treatment for another disease.I am not on antibiotics for an infection when I give my consent.I have not been treated with specific drugs for my condition, or if I have, it was only for a short time and long ago.I am allergic to AG-946 or its ingredients.I have been treated for high-risk MDS but not with specific drugs for more than a week in the last 8 weeks.I can take care of myself and am up and about more than half of my waking hours.You understand what the study involves and agree to follow all the study procedures.I am using or willing to use two forms of birth control if I or my partner can become pregnant.I haven't stopped P-gp inhibitors for the required time before joining.I have received fewer than 3 blood transfusions in the last 4 months and none in the last 2 months.My MDS is classified as lower-risk with a score of ≤3.5 and less than 5% blasts.I am 18 years old or older.My MDS is classified as lower-risk with a score ≤3.5 and less than 5% blasts.I have not had blood transfusions, or I have a specific transfusion history.I have had up to 2 treatments with drugs like EPO or luspatercept.I have been diagnosed with acute myeloid leukemia.I have fully recovered from any major surgery I had in the last 12 weeks.I have an active hepatitis C infection or tested positive for hepatitis B.I am allergic to AG-946 or its ingredients.I have fully recovered from any major surgery I had in the last 12 weeks.My trial is in Phase 2b.You are currently pregnant or breastfeeding.Your blood hemoglobin level is less than 11.0 grams per deciliter (g/dL) during the 4 weeks before the study.I can take care of myself and am up and about more than half of my waking hours.My platelet count is 75,000 or less without recent transfusions.I haven't stopped P-gp inhibitors for the required time before starting the study drug.I have a history of acute myeloid leukemia (AML).I have a history of liver or bile duct disorders.I am able to care for myself and perform daily activities.I am 18 years old or older.My trial is in Phase 2a.
Research Study Groups:
This trial has the following groups:- Group 1: Core Period: Phase 2a - Tebapivat
- Group 2: Core Period: Phase 2b - Tebapivat Dose Level 1
- Group 3: Core Period: Phase 2b - Tebapivat Dose Level 2
- Group 4: Core Period: Phase 2b - Tebapivat Dose Level 3
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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