Clinical trial NCT04691141: A Study to Evaluating the Pharmacokinetics, Safety, and Efficacy of ATG 016 Monotherapy in IPSS-R Intermediate Risk and Above Myelodysplastic Syndrome (MDS)
Official title: 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 type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Myelodysplastic Syndrome
Lead sponsor: Antengene Corporation
Sponsor class: INDUSTRY
First posted: 2020-12-31
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: MTD in Phase I
timeFrame: 16 months
measure: RP2D in Phase I
timeFrame: 16 months
measure: AEs/SAEs
timeFrame: 25 months
measure: ORR in Phase II
timeFrame: 25 months
Secondary outcomes:
measure: Disease Control Rate (DCR) in Phase I/II
timeFrame: 12 months
measure: Duration of Response (DOR) in Phase I/II
timeFrame: 12 months
measure: Progression-Free Survival (PFS) in Phase I/II
timeFrame: 12 months
measure: Overall Survival (OS)
timeFrame: 12 months
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Guangdong Provincal People's Hospital
state: Guangdong
city: Zhengzhou
country: China
facility: Henan Cancer Hospital
state: Henan
city: Qingdao
country: China
facility: The Affiliated Hospital of Qingdao University
state: Shandong
city: Shanghai
country: China
facility: Shanghai the sixth people's hospital
state: Shanghai
city: Tianjin
country: China
facility: Tianjin blood research institute
state: Tianjin
city: Hangzhou
country: China
facility: The First Affiliated Hospital, Zhejiang University, School of Medicine
state: Zhejiang
Study officials:
affiliation: Tianjin blood research institute
name: Zhijian Xiao, MD
role: PRINCIPAL_INVESTIGATOR
References: