A Study to Evaluating the Pharmacokinetics, Safety, and Efficacy of ATG 016 Monotherapy in IPSS-R Intermediate Risk and Above Myelodysplastic Syndrome (MDS)

NCT04691141 | Terminated Phase 1

• 1 other identifier: ATG-016-MDS-001
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 6

Brief Summary

This is a Phase Ⅰ/II, Open-label Study to Investigate the Pharmacokinetics, Safety, and Efficacyof ATG 016 Monotherapy in IPSS-R Intermediate Risk and above Myelodysplastic Syndrome (MDS) Patients after Failure of Hypomethylating Agent (HMA)-based Therapy.

Conditions

Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (4)

• MTD in Phase I

  MTD will be evaluated using the NCI-CTCAE, Version 5.0

  16 months

• RP2D in Phase I

  RP2D will be determined under the guidance of the SRC.

  16 months

• AEs/SAEs

  Toxicity will be graded according to the NCI CTCAE, Version 5.0.

  25 months

• ORR in Phase II

  Based on 2006 IWG Response Criteria, evaluated by IRC: ORR (CR + PR + mCR)

  25 months

Secondary Outcomes (4)

• Disease Control Rate (DCR) in Phase I/II

  12 months

• Duration of Response (DOR) in Phase I/II

  12 months

• Progression-Free Survival (PFS) in Phase I/II

  12 months

• Overall Survival (OS)

  12 months

Study Arms (1)

ATG-016

EXPERIMENTAL

5 mg QD×Days 1-5/week will be the initial dose of this study.

Drug: ATG-016

Interventions

ATG-016 DRUG

59 patients enrolled will be treated with ATG-016, orally, each 4 week (28-day) a cycle

ATG-016

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Understand and voluntarily sign an informed consent document prior to any study related assessments/procedures are conducted.
• ≥18 years of age, males or females.
• Eastern Cooperative Oncology Group (ECOG) performance status of ≤2.
• A life expectancy longer than 3 months in the opinion of the investigator at the screening.
• Male subjects (Including those who have received vasectomy) must agree to use condoms during sex with a woman of childbearing age and have no plans to impregnate the woman throughout the study and for 3 months following the last dose after the date of signing the ICF.

You may not qualify if:

• History of central nervous system (CNS) involvement.
• History of human immunodeficiency virus (HIV) infection.
• History of severe bleeding disorder, such as hemophilia A, hemophilia B, and vascular hemophilia.
• History of allogeneic stem-cell transplantation.
• Serious psychiatric or medical conditions that, in the opinion of the investigator, could interfere with treatment, compliance, or the ability to give consent.
• Pregnant or lactating women.

Sponsors & Collaborators

• Shanghai Antengene Corporation Limited: lead

Study Sites (6)

Guangdong Provincal People's Hospital

Guangzhou, Guangdong, 510000, China

Location

Henan Cancer Hospital

Zhengzhou, Henan, 450008, China

Location

The Affiliated Hospital of Qingdao University

Qingdao, Shandong, 266071, China

Location

Shanghai the sixth people's hospital

Shanghai, Shanghai Municipality, 201306, China

Location

Tianjin blood research institute

Tianjin, Tianjin Municipality, 300020, China

Location

The First Affiliated Hospital, Zhejiang University, School of Medicine

Hangzhou, Zhejiang, 310009, China

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Zhijian Xiao, MD

  Tianjin blood research institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 23, 2020
• First Posted: December 31, 2020
• Study Start: February 23, 2021
• Primary Completion: September 19, 2023
• Study Completion: September 19, 2023
• Last Updated: April 17, 2024

Record last verified: 2024-03

Locations

CN (6)