Extension Study for Subjects Who Participated in Prior Clinical Studies ofASTX727

Phase 1

• Conditions: Acute myeloid lymphoma (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML) or solid tumors; MedDRA version: 21.1Level: PTClassification code 10000880Term: Acute myeloid leukaemiaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 27.0Level: PTClassification code 10028533Term: Myelodysplastic syndromeSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 21.1Level: LLTClassification code 10065252Term: Solid tumorSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 21.0Level: PTClassification code 10009018Term: Chronic myelomonocytic leukaemiaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2018-003942-18-LT
• Lead Sponsor: Taiho Oncology, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-09-20
• Last Update Posted: 12 August 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

1) Previous participation in an Astex/Taiho-sponsored ASTX727 clinical trial (including but not limited to studies ASTX727-01, ASTX727-02, ASTX727-04, ASTX727-17 and ASTX727-18 and the food effect substudy of ASTX727-06) in which the subject was treated with ASTX727 and was still on active treatment with ASTX727 at the time of study completion as determined by the Sponsor.
2) Subject is considered to be benefitting from ASTX727 treatment in the opinion of the treating investigator at the time of parent study completion. (Subjects must not be withdrawn from the parent study until eligibility for this study is confirmed.)
3) Subject is able to understand and comply with the study procedures and understands the risks involved in the study.
4) Subject provides legally effective informed consent before undergoing any study-specific procedure.
FE Substudy inclusion criteria:
1) Be 18 years of age or older, at the time of signing the informed
consent.
2) Have MDS, including all French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, CMML), and subjects with MDS IPSS int-1, -2, or highrisk MDS, or AML, as diagnosed according to the 2016 World Health Organization (WHO) guidelines on acute leukemia, of any subtype except M3 (acute promyelocytic leukemia), who are not candidates for intensive chemotherapy, including subjects receiving HMA treatment, who have a confirmed diagnosis and a prior confirmatory bone marrow report. Subjects who are currently receiving HMA treatment must complete the ongoing (at the time of Screening) treatment cycle before enrolling in this study; timing of start of treatment cycle with ASTX727 is at the
principal investigator's discretion.
3) Have Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
4) Have adequate organ function defined as follows:
a. Hepatic: Total bilirubin =1.5× upper limit of normal (ULN); aspartate aminotransferase/serum glutamicoxaloacetic transaminase (AST/SGOT) and alanine aminotransferase/serum glutamic-pyruvic transaminase (ALT/SGPT) =5× ULN.
b. Renal: Calculated creatinine clearance =60 mL/min.
5) Women of childbearing potential must not be pregnant or breastfeeding and must have a negative pregnancy test at screening. Women of childbearing potential must agree to practice 1 highly effective contraceptive measure of birth control (with a failure rate of <1% per year; with low user dependency) during the substudy and for 6
months after the last dose of study treatment, agree not to donate eggs (ova, oocytes) for the purpose of reproduction during this period, and must agree not to become pregnant for 6 months after completing treatment; men with female partners of childbearing potential must agree to use a condom and advise their partners to practice 1 highly effective contraceptive measure of birth control (user dependent or with low user dependency) while receiving treatment with ASTX727 and for
at least 3 months after completing treatment and must agree not to father a child while receiving ASTX727 and for at least 3 months after completing ASTX727 treatment.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 72
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 72

Exclusion Criteria

1) Any subject who, in the opinion of the investigator, may have other conditions or for whom safety data from parent study participation suggests the risks of continuing treatment with ASTX727 may outweigh the benefits.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To provide ongoing long-term treatment with ASTX727 for subjects who were benefitting from ASTX727 treatment in a previous Astex/Taiho-sponsored clinical study.<br>To obtain long-term safety information.;Secondary Objective: Not applicable.;Primary end point(s): Safety as measured by AEs<br>;Timepoint(s) of evaluation of this end point: AE monitoring - Day 1 of each cycle and at Safety Follow-up<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Not applicable