A Study to Evaluate the Safety, Pharmacokinetics and Preliminary Efficacy of APG-5918.

NCT05773586 | Recruiting Phase 1

• 1 other identifier: APG5918AC101
• Study Type: interventional
• Target: 105
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics and efficacy of APG-5918 in Healthy Subjects or Anemic Patients.

Conditions

Anemia

Keywords

APG-5918; Anemia

Outcome Measures

Primary Outcomes (1)

• Treatment-Emergent Adverse Events (TEAEs)

  TEAEs will be assessed via CTCAE version 5.0 based on the frequency of adverse events/serious adverse events (AEs/SAEs), clinically significant laboratory test results, 12-lead ECGs, and vital signs.

  up to 7 days in Part A and 84 days or till EOT in Part B

Secondary Outcomes (2)

• Plasma Concentrations of APG-5918

  Days 1, 2 and 3 in Part A; Days 1, 15 and 28 in Part B

• Measurement of Hemoglobin

  84 days or till EOT in Part B

Other Outcomes (1)

• Expression levels of H3K27me3

  84 days or till EOT in Part B

Study Arms (2)

Single Ascending Dose (SAD) cohorts in Healthy Subjects (Part A)

EXPERIMENTAL

Subjects will be randomized to receive a single dose of APG-5918 or placebo.

Drug: APG-5918; Drug: Placebo

Multiple Ascending Dose (MAD) cohorts in Anemic Patients (Part B)

EXPERIMENTAL

Subjects will receive once daily APG-5918 for 84 days or till EOT.

Drug: APG-5918

Interventions

APG-5918 DRUG

oral tablets 10mg, 50mg, 200mg.

Multiple Ascending Dose (MAD) cohorts in Anemic Patients (Part B); Single Ascending Dose (SAD) cohorts in Healthy Subjects (Part A)

Placebo DRUG

Matching placebo

Single Ascending Dose (SAD) cohorts in Healthy Subjects (Part A)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Healthy Subjects:
• \. Age: 18 to 55 years. 2. Body Mass Index (BMI): 18-28 kg/m² (inclusive). 3. Hemoglobin value: 120 g/L-160 g/L (inclusive). 4. Normal body iron stores. 2. Anemic Subjects:
• Age: ≥ 18 years.
• Including beta-thalassemia and other related anemias, with screening Hb ≤ 100.0 g/L.
• Body weight ≥ 40 kg.
• Serum folate and vitamin B12 levels above the lower limit of normal (LLN).
• ALT, AST ≤ 2×ULN, and direct (unconjugated) total bilirubin (DBIL) ≤ 2×ULN. Higher levels may be accepted after excluding other diseases based on investigator judgment.
• No active or chronic bleeding.
• Eastern Cooperative Oncology Group (ECOG) performance status score of 0-1.
• \. For female subjects of childbearing potential, a negative blood or urine pregnancy test within 7 days prior to the first dose.
• \. Subjects and their partners must voluntarily agree to use effective contraceptive measures as required by the protocol during the treatment period and for at least 3 months after the last dose of study drug.
• \. Ability to understand and voluntarily sign a written informed consent form, which must be signed before any trial-specific procedures are performed.

You may not qualify if:

• \. Healthy Subjects:
• History of any disease or clinical condition that, in the investigator's opinion, may confound the study results or pose additional risk to the subject with administration of the study drug.
• ALT or AST \> 2×ULN, or TBIL \> 1.5×ULN at screening.
• Undergone surgery (excluding minor cosmetic or dental procedures) within 3 months prior to screening.
• Blood donation or blood loss exceeding 400 mL within 3 months prior to screening, or planned donation of blood or blood components during the study period.
• Use of another investigational product within 30 days or 5 half-lives (whichever is longer) prior to dosing, or current participation in a prospective study of an investigational product or medical device.
• History of substance abuse within 6 months prior to screening.
• Positive alcohol breath test.
• \. Anemic Subjects:
• Presence of clinically significant or uncontrolled ongoing autoimmune disease.
• Severe cardiac disease.
• Severe renal disease, defined as estimated glomerular filtration rate (eGFR) \< 30 mL/min/1.73m², or dependence on dialysis.
• Active malignancy, history of cancer, or presence of a known or suspected familial cancer syndrome in linealrelatives.
• A history of persistent hemolysis or hemolytic syndrome due to causes other than the study diseases.
• A history of thrombosis or newly developed thrombus within 4 weeks prior to screening.

Sponsors & Collaborators

• Ascentage Pharma Group Inc.: lead

Study Sites (2)

Sun Yat-sen University Cancer Center

Guangzhou, Guangdong, 510000, China

RECRUITING

Nanfang Hospital of Southern Medical University

Guangzhou, Guangdong, 510515, China

RECRUITING

MeSH Terms

Conditions

Anemia

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Zhongyuan Xu, M.D.,Ph.D.

  Nanfang Hospital, Southern Medical University

  PRINCIPAL INVESTIGATOR

• Yang Liang, M.D.,Ph.D.

  Sun Yat-Sen University Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yifan Zhai, Ph.D.

CONTACT

+86-20-28069260; yzhai@ascentage.com

Zhongyuan Xu

CONTACT

020-62787926; nfyygcp@126.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2023
• First Posted: March 17, 2023
• Study Start: March 13, 2023
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): July 15, 2028
• Last Updated: April 15, 2026

Record last verified: 2026-04

Locations

CN (2)