A study to evaluate the efficacy of APG-2575 (Lisaftoclax) plus azacitidine (AZA) vs. placebo plus AZA in newly diagnosed adult subjects with higher risk myelodysplastic syndrome (Higher Risk-MDS).

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ascentage Pharma Group Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

7 Jul 2025 → ongoing

Decision date (initial)

2025-04-24

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Ascentage Pharma Group Inc.

External identifiers

EU CT number

2024-517247-31-00

ClinicalTrials.gov

NCT06641414

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Safety

To evaluate the efficacy of xxx

Secondary objectives 5

1. To compare the effect of xxx
2. To evaluate the safety of xxx
3. To evaluate the population pharmacokinetics (Pop PK) following treatment with xxx
4. To evaluate Patient-Reported Outcome (PRO) measures of xxxx based on EORTC QLQ C30 (V3).
5. To evaluate Health Economics Outcomes Research (HEOR) measures of xxx based on EuroQol 5 Dimension (EQ-5D).

Conditions and MedDRA coding

Newly Diagnosed Higher Risk Myelodysplastic Syndrome

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Aged ≥ 18 years old.
2. Newly Diagnosed MDS is defined according to 2022 World Health Organization classification (5th Edition)
3. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
4. Life expectancy ≥ 3 months.
5. Able to receive oral medication.
6. Adequate organ functions as defined below: - Creatinine clearance ≥ 30 ml/min (calculated with Cockcroft formula, as shown in Annex 3) - Total bilirubin < 1.5 × ULN (except Gilbert's syndrome, hyperbilirubinemia due to regular blood transfusions as assessed by the investigator) - Aspartate aminotransferase (ALT) and alanine aminotransferase (AST) ≤ 2.5 × ULN
7. Negative urine or serum pregnancy test prior to dosing in women of childbearing potential. Women of childbearing potential (postmenopausal women must have been postmenopausal for at least 12 months to be considered of non-childbearing potential) and their partners are willing to use contraception as deemed effective by the investigator during treatment and for at least 6 months after the last dose of study drug.
8. Subjects must have the ability to understand and voluntarily sign a written informed consent form that must be signed prior to performing any trial-specific study procedures.
9. Subjects must be willing to participate in the study and are able to complete study procedures and follow-up examinations.

Exclusion criteria 7

1. Previous diagnosis of xxx
2. Any of the following cardiac abnormalities (as determined by the study physician based on clinical examination assessments): - Any history of myocardial infarction within 6 months - Congestive heart failure (CHF) (New York Heart Association [NYHA] Class III or IV) or left ventricular ejection fraction (LVEF) < 40% - Symptomatic ventricular arrhythmia uncontrolled by medication - Any history of familial long QT syndrome - The mean QT interval calculated from 3 electrocardiogram (ECG) readings (1 to 3 minutes apart) is > 470 (Using Fridercia's correction: QTcF = QT/RR0.33)
3. Second malignancies or previous malignancies with a disease-free interval of less than 1 year at the time of signing the informed consent (except for subjects with adequately resected cutaneous basal cell or squamous cell carcinoma or resected carcinoma in situ).
4. Have history of HSCT.
5. Other clinically significant uncontrolled symptoms, including but not limited to: uncontrolled active systemic infection (virus, bacteria or fungi), known clinically active hepatitis B or C, or HIV infection. (as determined by the study physician based on clinical examination assessments).
6. Have malabsorption syndrome or other conditions and are not suitable for enteral drug administration.
7. Have any other conditions or illnesses which, in the investigator's judgment, makes them unsuitable for participation in this study, , including but not limited to; • Women who are breast feeding or planning to donate eggs within 6 months after the end of the study treatment • History of hypersensitivity to compounds related to lisaftoclax or AZA or to any of their excipients • History of bleeding disorders or active uncontrolled coagulopathy.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. xxx
2. xxx

Secondary endpoints 4

1. To compare the efficacy of xxx
2. To evaluate the safety of xxx
3. To evaluate the population pharmacokinetics (Pop PK) following treatment with xxx
4. To evaluate Health Economics Outcomes Research (HEOR) measures of lisaftoclax xxx based on EuroQol 5 Dimension (EQ-5D).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ascentage Pharma Group Inc.

Sponsor organisation

Ascentage Pharma Group Inc.

Address

700 King Farm Boulevard

City

Rockville

Postcode

20850-5736

Country

United States

Scientific contact point

Organisation

Ascentage Pharma Group Inc.

Contact name

Yifan Zhai

Public contact point

Organisation

Ascentage Pharma Group Inc.

Contact name

Yifan Zhai

Third parties 9

Locations

13 EU/EEA countries · 92 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 115 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

28 submissions — initial application plus substantial / non-substantial modifications