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

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

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.

Study Overview

• Status: Terminated
• Conditions: Myelodysplastic Syndrome
• Intervention / Treatment: Drug: ATG-016

Detailed Description

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. In Phase I: approximately 15 to 21 subjects and Phase II: approximately 44 subjects; approximately 59 to 65 subjects will be enrolled totally in this study.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: doris tao; Phone Number: 021-23566665; Email: doris.tao@antengene.com
• Study Contact Backup: Name: sunny he; Phone Number: 021-23566665; Email: sunny.he@antengene.com

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 510000
      • Guangdong Provincal People's Hospital
  • Henan
    • Zhengzhou, Henan, China, 450008
      • Henan Cancer Hospital
  • Shandong
    • Qingdao, Shandong, China, 266071
      • The Affiliated Hospital of Qingdao University
  • Shanghai
    • Shanghai, Shanghai, China, 201306
      • Shanghai the sixth people's hospital
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Tianjin blood research institute
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310009
      • The First Affiliated Hospital, Zhejiang University, School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Understand and voluntarily sign an informed consent document prior to any study related assessments/procedures are conducted.
2. ≥18 years of age, males or females.
3. Eastern Cooperative Oncology Group (ECOG) performance status of ≤2.
4. A life expectancy longer than 3 months in the opinion of the investigator at the screening.
5. 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.

Exclusion Criteria:

1. History of central nervous system (CNS) involvement.
2. Toxicity from prior antitumor therapy did not return to Grade 1 or baseline (Except for alopecia, neutropenia, anemia, and thrombocytopenia. For neutrophils, hemoglobin, and platelets, please follow Exclusion Criteria No. 5).
3. History of human immunodeficiency virus (HIV) infection.
4. History of severe bleeding disorder, such as hemophilia A, hemophilia B, and vascular hemophilia.
5. History of allogeneic stem-cell transplantation.
6. Serious psychiatric or medical conditions that, in the opinion of the investigator, could interfere with treatment, compliance, or the ability to give consent.
7. Pregnant or lactating women.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ATG-016; 5 mg QD×Days 1-5/week will be the initial dose of this study.	Drug: ATG-016; 59 patients enrolled will be treated with ATG-016, orally, each 4 week (28-day) a cycle

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
ORR in Phase II	Based on 2006 IWG Response Criteria, evaluated by IRC: ORR (CR + PR + mCR)	25 months
AEs/SAEs	Toxicity will be graded according to the NCI CTCAE, Version 5.0.	25 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Duration of Response (DOR) in Phase I/II	To evaluate duration of response	12 months
Overall Survival (OS)	The estimates of Kaplan-Meier	12 months
Disease Control Rate (DCR) in Phase I/II	DCR (CR + PR + mCR + HI + SD)	12 months
Progression-Free Survival (PFS) in Phase I/II	To evaluate progression-free survival	12 months

Collaborators and Investigators

• Sponsor: Shanghai Antengene Corporation Limited
• Investigators: Principal Investigator: Zhijian Xiao, MD, Tianjin blood research institute

Study record dates

Study Major Dates

• Study Start (Actual): February 23, 2021
• Primary Completion (Actual): September 19, 2023
• Study Completion (Actual): September 19, 2023

Study Registration Dates

• First Submitted: December 23, 2020
• First Submitted That Met QC Criteria: December 29, 2020
• First Posted (Actual): December 31, 2020

Study Record Updates

• Last Update Posted (Actual): April 17, 2024
• Last Update Submitted That Met QC Criteria: April 15, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Syndrome; Myelodysplastic Syndromes; Preleukemia
• Other Study ID Numbers: ATG-016-MDS-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No