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A Randomized, Open-Label, Phase 1-2 Study of ASTX727 Low Dose (ASTX727 LD) Extended Schedule in Subjects with Lower Risk (IPSS Low or Intermediate-1) Myelodysplastic Syndromes (MDS)
EU CTIS ID: 2024-516296-32-00
What this study is testing
Phase 1: - to assess the safety profile of doses and schedules of ASTX727. - to select the ASTX727 dose(s) and schedule(s) to study in Phase 2. Phase 2: - to assess the clinical efficacy of ASTX727 dose(s) and schedule(s) studied.
- Human Pharmacology (Phase I)- Other
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Able to understand and comply with the study procedures, understand the risks involved in the study, and provide legally effective informed consent before the first study-specific procedure.
- Men or women ≥18 years with IPSS low risk or Int-1 MDS (all subjects). Subjects must have had at least 1 of the following disease-related criteria during the 8 weeks before randomization: a) RBC transfusion dependence, defined as 2 or more units of RBC transfusions.* b) Hb of ≤9.0 g/dL in at least 2 blood counts prior to randomization or in 1 blood count if RBC transfusion was received. c) ANC of <0.5×10e9/L in at least 2 blood counts prior to randomization. d) Platelet counts of <50×10^9/L in at least 2 blood counts prior to randomization. *RBC transfusions administered for Hb levels ≤9.0 g/dL are counted
- ECOG performance status of 0 to 2.
- Adequate organ function defined as follows: a) Hepatic: Total or direct bilirubin ≤2 × upper limit of normal (ULN); aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≤5 × ULN. b) Renal: serum creatinine ≤1.5 × ULN or calculated creatinine clearance or glomerular filtration rate ≥50 mL/min.
- Women of childbearing potential (according to recommendations of the Clinical Trial Facilitation Group [CTFG]) must not be pregnant or breastfeeding and must have a negative pregnancy test at screening. Women of childbearing potential and their partners must use at least 1 highly effective birth control method (with a failure rate of <1% per year; preferably with low user dependency), from the start of participation in the study and for 6 months after the last dose of study treatment. Contraceptive measures that are considered highly effective with low user dependency include implantable progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, or azoospermic partner (vasectomized or due to a medical cause).
- Male subjects with female partners of childbearing potential must agree to use a male condom and advise their partners to practice 1 highly effective contraceptive measure of birth control (user dependent or with low user dependency) and must agree not to father a child while receiving study treatment for at least 3 months after completing treatment.
You likely can't join if
- Known active infection with human immunodeficiency virus or hepatitis viruses.
- Treatment with any investigational drug or therapy within 2 weeks before study treatment, or 5 half-lives, whichever is longer, before the first dose of study treatment, or ongoing clinically significant AEs from previous treatment.
- Prior treatment with azacitidine, decitabine or gaudecitabine.
- Treatments for MDS, including erythropoietins, colony-stimulating factors (CSFs), thrombopoietins, chemotherapy, and immunosuppression including calcineurin inhibitors, glucocorticoids, etc., must be concluded 1 month prior to study treatment.
- Diagnosis of chronic myelomonocytic leukemia (CMML).
- Poor medical risk because of other conditions such as uncontrolled systemic diseases or active uncontrolled infections.
See the full eligibility criteria
Who can join
- Able to understand and comply with the study procedures, understand the risks involved in the study, and provide legally effective informed consent before the first study-specific procedure.
- Men or women ≥18 years with IPSS low risk or Int-1 MDS (all subjects). Subjects must have had at least 1 of the following disease-related criteria during the 8 weeks before randomization: a) RBC transfusion dependence, defined as 2 or more units of RBC transfusions.* b) Hb of ≤9.0 g/dL in at least 2 blood counts prior to randomization or in 1 blood count if RBC transfusion was received. c) ANC of <0.5×10e9/L in at least 2 blood counts prior to randomization. d) Platelet counts of <50×10^9/L in at least 2 blood counts prior to randomization. *RBC transfusions administered for Hb levels ≤9.0 g/dL are counted
- ECOG performance status of 0 to 2.
- Adequate organ function defined as follows: a) Hepatic: Total or direct bilirubin ≤2 × upper limit of normal (ULN); aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≤5 × ULN. b) Renal: serum creatinine ≤1.5 × ULN or calculated creatinine clearance or glomerular filtration rate ≥50 mL/min.
- Women of childbearing potential (according to recommendations of the Clinical Trial Facilitation Group [CTFG]) must not be pregnant or breastfeeding and must have a negative pregnancy test at screening. Women of childbearing potential and their partners must use at least 1 highly effective birth control method (with a failure rate of <1% per year; preferably with low user dependency), from the start of participation in the study and for 6 months after the last dose of study treatment. Contraceptive measures that are considered highly effective with low user dependency include implantable progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, or azoospermic partner (vasectomized or due to a medical cause).
- Male subjects with female partners of childbearing potential must agree to use a male condom and advise their partners to practice 1 highly effective contraceptive measure of birth control (user dependent or with low user dependency) and must agree not to father a child while receiving study treatment for at least 3 months after completing treatment.
What rules you out
- Known active infection with human immunodeficiency virus or hepatitis viruses.
- Treatment with any investigational drug or therapy within 2 weeks before study treatment, or 5 half-lives, whichever is longer, before the first dose of study treatment, or ongoing clinically significant AEs from previous treatment.
- Prior treatment with azacitidine, decitabine or gaudecitabine.
- Treatments for MDS, including erythropoietins, colony-stimulating factors (CSFs), thrombopoietins, chemotherapy, and immunosuppression including calcineurin inhibitors, glucocorticoids, etc., must be concluded 1 month prior to study treatment.
- Diagnosis of chronic myelomonocytic leukemia (CMML).
- Poor medical risk because of other conditions such as uncontrolled systemic diseases or active uncontrolled infections.
- Known significant mental illness or other condition, such as active alcohol or other substance abuse or addiction, that in the opinion of the investigator predisposes the subject to high risk of noncompliance with the protocol.
- Life-threatening illness, medical condition or organ system dysfunction, or other reasons including laboratory abnormalities, which, in the investigator's opinion, could compromise the subject's safety, interfere with the absorption or metabolism of ASTX727 LD, or compromise the integrity of the study outcomes.
- Prior malignancy, except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, prostate cancer or breast cancer under control with hormone therapy, or other cancer from which the subject has been disease free for at least 1 year.
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- United States
- United Kingdom
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, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; United States; United Kingdom. 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 EU CTIS; the study team decides eligibility.