A Study of ASTX030 (Cedazuridine in Combination With Azacitidine) in MDS CMML or AML

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Recruiting
participants needed

245
sponsor

Astex Pharmaceuticals, Inc.

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Updated on 19 February 2024

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direct bilirubin

immunosuppression

immunosuppressive agents

myeloid leukemia

graft versus host disease

chronic myeloid leukemia

acute leukemia

anemia

thrombocytopenia

azacitidine

major surgery

dysplasia

leukemia

conjugated bilirubin

bone marrow procedure

cytotoxic chemotherapy

chronic myelomonocytic leukemia

ringed sideroblasts

raeb

neutropenia

myelodysplastic/myeloproliferative neoplasm

acute myeloid leukemia

myelodysplastic syndrome

cedazuridine

myelodysplastic/myeloproliferative disease

Summary

Study ASTX030-01 is designed to move efficiently from Phase 1 to Phase 3. Phase 1 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) of ASTX030. Phase 2 is a randomized open-label crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 is a randomized open-label crossover study comparing the final oral ASTX030 tablet to SC azacitidine. The duration of the study is expected to be approximately 36 months.

Details

Condition	Preleukemia, Acute myeloid leukemia, Acute myeloid leukemia, MYELODYSPLASTIC SYNDROME, Chronic myeloid leukemia, Chronic myeloid leukemia
Age	18-100 years
Treatment	Azacitidine, ASTX030 (cedazuridine + azacitidine), Cedazuridine
Clinical Study Identifier	NCT04256317
Sponsor	Astex Pharmaceuticals, Inc.
Last Modified on	19 February 2024

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Eligibility		Yes	No	Not Sure
Inclusion Criteria
	Confirmed MDS, CMML, MDS/MPN, or AML who are candidates to receive and benefit from single agent azacitidine as follows and as applicable according to local country approvals and/or local institution standard practice
	French-American-British myelodysplastic syndrome subtypes: refractory anemia (RA) or refractory anemia with ringed sideroblasts (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), or MDS with intermediate-2 or high risk MDS according to the International Prognostic Scoring System (IPSS). MDS/MPN patients including CMML according to the WHO 2016 classification are also eligible if they are candidates to receive single agent azacitidine per local institution standards; or
	Previously untreated AML with 20% to 30% blasts present in bone marrow and multi-lineage dysplasia (Phase 2 and 3 only); or
	Previously untreated AML with >30% blasts present in bone marrow, who are not eligible for stem cell transplant and unfit for intensive chemotherapy induction (Phase 2 and 3 only)
	Participants with Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1\
	Participants with adequate organ function defined as
	Hepatic: Total or direct bilirubin 2 upper limit of normal (ULN); aspartate aminotransferase (AST)/serum glutamic-oxaloacetic transaminase (SGOT) and alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase (SGPT) 2.5 ULN
	Renal: Calculated creatinine clearance >50 mL/min/1.73 m2 by Cockcroft-Gault formula or other medically acceptable formulas
	For participants with prior allogeneic stem cell transplant, no evidence of graft-versus-host disease (GVHD) and must be 2 weeks off systemic immunosuppressive therapy before start of study treatment
	Participants with no major surgery within 2 weeks before first study treatment
	Participants with no cytotoxic chemotherapy within 4 weeks before first study treatment
	Able to swallow the number of tablets 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
	Women of child-bearing potential (according to recommendations of the Clinical Trial Facilitation Group) must not be pregnant or breastfeeding and must have a negative pregnancy test at screening
Exclusion Criteria
	Active uncontrolled gastric or duodenal ulcer
	Poor medical risk because of other conditions such as uncontrolled systemic diseases or active uncontrolled bacterial, viral, or fungal infections
	Life-threatening illness (e.g., uncontrolled bleeding and patients at risk for or are experiencing leukostasis [AML]), uncontrolled medical condition or organ system dysfunction, or other reasons, which, in the investigator's opinion, could compromise the participant's safety, interfere with the absorption or metabolism of oral cedazuridine + azacitidine 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 participant has been disease free for at least 2 years
	Participants with MDS/MPN who have clinical extramedullary disease including clinically palpable hepatomegaly or splenomegaly
	Previous treatment with more than 1 cycles of decitabine, azacitidine, or guadecitabine (Phases 2 and 3 only)
	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 from previous treatment with investigational drug or therapy
	Known or suspected hypersensitivity to cedazuridine or azacitidine, or any of their excipients

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bone marrow procedure

cytotoxic chemotherapy

chronic myelomonocytic leukemia

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raeb

neutropenia

myelodysplastic/myeloproliferative neoplasm

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Study Definition

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A Study of ASTX030 (Cedazuridine in Combination With Azacitidine) in MDS CMML or AML