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New treatment option for Myelodysplastic Syndromes
Official title A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
ClinicalTrials.gov ID: NCT04256317
What this study is testing
What is Azacitidine?
Azacitidine is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for myelodysplastic syndromes.
Also referred to as Vidaza™ (powder only).
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
- Study ASTX030-01 is a multi-phase study comprising of Phases 1-3 Monotherapy arms, and Phase 1 and Phase 2 Combination Therapy arms. Phase 1 Monotherapy consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B).
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Phase 2 Monotherapy: 1\. Has Confirmed MDS, CMML, or other MDS/MPN diagnosis who are candidates to receive and benefit from single agent azacitidine...
- Phase 3 Monotherapy:
- Has confirmed MDS or CMML and is a candidate to receive and benefit from single agent azacitidine as applicable according to local country approvals...
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
- Participants with adequate organ function.
You likely can't join if
- All Monotherapy Phases:
- Has an active uncontrolled gastric or duodenal ulcer.
- Has poor medical risk because of other conditions.
- Has known human immunodeficiency virus (HIV) infection.
- Is known to be positive for Hepatitis B or C infection.
- Has a life-threatening illness.
See the full eligibility criteria
Who can join
- Phase 2 Monotherapy: 1\. Has Confirmed MDS, CMML, or other MDS/MPN diagnosis who are candidates to receive and benefit from single agent azacitidine and as applicable according to local country approvals and/or local...
- Phase 3 Monotherapy:
- Has confirmed MDS or CMML and is a candidate to receive and benefit from single agent azacitidine as applicable according to local country approvals and/or local institution standard practice: a) French-American-British...
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
- Participants with adequate organ function.
- For participants with prior allogeneic stem cell transplant, no evidence of graft-versus-host disease (GVHD).
- Participants with no major surgery within 3 weeks before first study treatment.
- Participants with no cytotoxic chemotherapy (excluding hydroxyurea) within 4 weeks before first study treatment.
- Is able to swallow the number of tablets/capsules required for the treatment assignment within a 10-minute period and tolerate 4 hours of fasting.
- Participants with projected life expectancy of at least 12 weeks.
- Phase 1 and Phase 2 Combination Therapy:
- Has histological confirmation of newly diagnosed AML by World Health Organization (WHO) 2022 criteria (Phase 1) or 2016 criteria (Phase 2).
- Participants with projected life expectancy of at least 12 weeks.
- Must be considered ineligible for intensive induction chemotherapy defined by the following: a. Aged 75 years or older, or b. Aged 18 to 74 years with at least one of the following comorbidities: i. Severe cardiac...
- Has an ECOG Performance Status of 0-2 for participants ≥75 years of age or 0-3 for participants 18 to 74 years of age.
What rules you out
- All Monotherapy Phases:
- Has an active uncontrolled gastric or duodenal ulcer.
- Has poor medical risk because of other conditions.
- Has known human immunodeficiency virus (HIV) infection.
- Is known to be positive for Hepatitis B or C infection.
- Has a life-threatening illness.
- Has a history of other malignancies prior to study entry, with the exception of adequately treated in situ carcinoma of the breast or cervix uteri; localized basal cell carcinoma or squamous cell carcinoma of the skin...
- Participants with MDS/MPN including CMML who have clinical extramedullary disease including clinically palpable hepatomegaly or splenomegaly.
- Has previous treatment with more than 1 cycle of decitabine, azacitidine, or guadecitabine (Phases 2 and 3 only).
- Has been treated with any investigational drug or therapy within 2 weeks, or 5 half-lives, whichever is longer, before the protocol-defined first dose of study treatment, or ongoing clinically significant adverse events...
- Has a known or suspected hypersensitivity to cedazuridine or azacitidine or any of their excipients.
- Cannot discontinue treatment with any drugs that delay gastric emptying such as glucagon-like peptide-1 (GLP-1) and/or gastric inhibitory polypeptide (GIP) agonists in Cycles 1 and 2 of the study.
- Has a known or suspected hypersensitivity to cedazuridine or azacitidine or any of their excipients.
- Phase 1 and Phase 2 Combination Therapy:
- Has a history of MPN including myelofibrosis, essential thrombocythemia, polycythemia vera, chronic myeloid leukemia with or without BCR-ABL1 translocation, or AML with BCR-ABL1 translocation.
- Has the following karyotype abnormalities: t(15;17) or other acute promyelocytic leukemia variants that remain sensitive to all-trans retinoic acid (ATRA) therapy [t(8;21) and inv(16) are excluded in Phase 2 only].
- Has known active central nervous system involvement from AML.
- Has known human immunodeficiency virus (HIV) infection.
- Is known to be positive for Hepatitis B or C infection.
- Has severe hepatic impairment
- Has severe renal impairment
- Has a malabsorption syndrome or other condition that precludes enteral route of administration.
- Has a cardiovascular disability status of New York Heart Association Class \>2.
- Has significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular, or pulmonary disease; or any other medical condition that in the opinion of the investigator...
- Has clinically significant uncontrolled systemic infection requiring therapy (viral, bacterial, or fungal).
- Has a history of other malignancies prior to study entry with the exception of adequately treated in situ carcinoma of the breast or cervix uteri; localized basal cell carcinoma or squamous cell carcinoma of the skin...
- Has a WBC count \>25,000/ microliters (μL) (hydroxyurea treatment is permitted to meet this criterion).
- Has received treatment with any of the following:
- A hypomethylating agent (azacitidine or decitabine) or venetoclax, including prior treatment for MDS.
- Chimeric Antigen Receptor (CAR)-T cell therapy.
- Investigational therapies for MDS or AML.
- Cannot discontinue treatment with any of the following:
- Prophylactic antifungal therapy with CYP3A inhibitor activity or other concomitant medications with moderate or strong CYP3A inhibitor activity ≥7 days or 5 halflives, whichever is greater, prior to Cycle 1 Day 1 (C1D1).
- Drugs that are strong CYP3A or P-gp inhibitors ≥7 days or 5 half-lives, whichever is greater, prior to C1D1.
- Cannot avoid concomitant drugs known as moderate or strong CYP3A inducers.
- Cannot discontinue treatment with any drugs that delay gastric emptying such as GLP-1 and/or GIP agonists in Cycles 1 and 2 of the study.
- Is participating in another research study requiring interventions such as drug therapy or study procedures.
- Has a known or suspected hypersensitivity to cedazuridine, azacitidine, venetoclax, or any of their excipients.
- Has known significant mental illness or other conditions such as alcohol or other substance abuse or addictions
- Consumes grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or starfruit ≤7 days prior to C1D1.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Orange, California, United States
- New Haven, Connecticut, United States
- Miami, Florida, United States
- Atlanta, Georgia, United States
- Boston, Massachusetts, United States
- Hackensack, New Jersey, United States
- Buffalo, New York, United States
- Mineola, New York, United States
- New York, New York, United States
- The Bronx, New York, United States
- Durham, North Carolina, United States
- Columbus, Ohio, United States
- Portland, Oregon, United States
- Salem, Oregon, United States
- Charleston, South Carolina, United States
- Nashville, Tennessee, United States
- Dallas, Texas, United States
- Houston, Texas, United States
- Seattle, Washington, United States
+ 42 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 listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Orange, California, United States; New Haven, Connecticut, United States; Miami, Florida, United States; Atlanta, Georgia, United States; Boston, Massachusetts, United States and 56 more location(s). Enter your location above to see the nearest site and check your eligibility.
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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.