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A Study of HYML-122 and Cytarabine in Patients With FLT3 Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)

Phase 2
Recruiting
Conditions
Relapsed or Refractory Acute Myeloid Leukemia (AML)
Interventions
Registration Number
NCT05241093
Lead Sponsor
Tarapeutics Science Inc.
Brief Summary

This is a single-arm, open, multicenter, phase 2 study to evaluate the efficacy, safety and pharmacokinetics of HYLM-122 in combination with cytarabine in Chinese subjects with FLT3 positive relapsed or refractory acute myeloid leukemia.

Detailed Description

This study will have two phases. Phase 1: the escalation phase is to establish the recommended phase 2 dose (RP2D) of HYML-122 given in combination with cytarabine.

Phase 2: the extension phase study will treat patients with FLT3 positive relapsed or refractory AML with HYML-122 in combination with cytarabine at the RP2D, and further evaluate efficacy and safety.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
18
Inclusion Criteria
  • Fully understand the procedures of the clinical study and participate voluntarily with signed and dated written informed consent form, comply with the requirements of the study protocol.
  • Males and/or females at least 18 years old when signing the informed consent form.
  • Histologically confirmed AML (defined using WHO criteria 2016) with one of the following: Refractory to at least 1 cycle of induction chemotherapy. Relapsed after achieving remission with a prior therapy.
  • Subject is positive for FLT3 mutation in bone marrow or blood after completion of the subject's last interventional treatment.
  • Eastern cooperative oncology group performance status (ECOG) ≤2 at screening.
  • Life expectancy of at least 3 months.
  • Women of childbearing potential have a negative pregnancy test at baseline and are willing to employ an effective method of contraception for the entire duration of study treatment and 6 months after the last dose.
Exclusion Criteria
  • Known or suspected allergies to any of the investigational drug composition (HYML-122, lactose, hydroxypropyl cellulose, hyposubstituted hydroxypropyl cellulose, silicon dioxide, magnesium stearate, titanium dioxide and polyethylene glycol).
  • Medical history and surgical history excluded according to the protocol.
  • Any previous medical treatment history exclude from the protocol.
  • Abnormal laboratory results exclude from the protocol.
  • Combination of treatments and/or drugs required during the study period and cannot be discontinued that excluded from the protocol.
  • Alcohol abuse within 6 months prior to screening, defined as long-term drinking history, generally more than 5 years, equivalent to alcohol quantity ≥40g/d for men, ≥20g/d for women, or heavy drinking history within 2 weeks, equivalent to alcohol quantity ≥80g/d. alcohol volume (g) conversion formula=alcohol consumption (mL)*alcohol content (%)*0.8.
  • Abortion less than 30 days prior to screening, pregnant and lactating women (currently breast-feeding or less than one year after delivery although not breast-feeding), women of childbearing potential who are not guaranteed effective contraception during the study, planning pregnancy or donating eggs or sperm within 6 months after the last dose.
  • History of drug abuse or drug addicts.
  • Subjects may not be able to complete the study duo to poor compliance or other reasons, or unsuitable for the study by the investigator's judgment.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
HYML-122 plus cytarabineHYML-122; cytarabineThe first three eligible enrolled patients will be treated with initial dosing of HYML-122 400mg bid daily and cytarabine 100mg/m2 intravenously by using "3+3" escalating design to explore RP2D. the Data Monitoring Committee (DMC) will evaluate the safety, efficacy and PK data of the phase 1 subjects and establish the combined regimen recommended dose. Participants will be treated on continuous cycles until they no longer derive clinical benefit in the judgement of the investigator, have unacceptable toxicity, undergo hematopoietic stem cell transplantation (HSCT), or meet 1 of the discontinuation criteria, whichever occurs first.
Primary Outcome Measures
NameTimeMethod
composite complete remission (CRc) rateup to 24 months.

CRc rate is defined as the rate of all complete and incomplete remission (CRMRD-+CR+CRp+CRi).

ORRup to 24 months

overall remission rate, including complete remission without minimum residual disease (CRMRD-), complete remission (CR), complete remission with incomplete hematologic recovery (CRi), complete remission without platelet recovery (CRp), partial remission (PR).

Secondary Outcome Measures
NameTimeMethod
OSup to 24 months

overall survival, OS is defined as time from the date of enrollment until the date of death from any cause. For a subject who is not known to have died buy the end-of-study follow-up, OS is censored at the date of last contact.

Incidence of treatment-emergent adverse events (TEAEs)up to 24 months

safety and tolerability of investigational product assessed as the number of participants experience adverse events (AEs, CTCAE 5.0) or abnormalities in vital signs, laboratory tests, or electrocardiograms.

Cmax,ssat the end of Cycle 1 (each cycle is 28 days)

Peak plasma concentration at steady state (Cmax,ss)

AUCssat the end of Cycle 1 (each cycle is 28 days)

the area under the plasma concentration at steady-state

RFSup to 24 months

relapse-free survival, for patients achieving a complete remission, defined as the interval from the date of first documentation of a leukemia free state to date of recurrence, treatment failure, death due to any cause or last contact of the end-of-study follow up, which ever occurs first.

EFSup to 24 months

event-free survival, EFS is defined as the time from the date of enrollment until the date of documented relapse from CR, CRp or CRi, treatment failure, death from any cause or last contact of the end-of-study follow-up, whichever occurs first.

DOR-CRup to 24 months

duration of CR remission, DOR-CR is defined as the time from the date of first CR, CRp, CRi until the date of documented relapse.

Cmin,ssat the end of Cycle 1 (each cycle is 28 days)

minimum observed plasma concentration at steady state (Cmin,ss) of drug in blood plasma

Cav,ssat the end of Cycle 1 (each cycle is 28 days)

the average steady-state plasma concentration

Trial Locations

Locations (1)

the First Affiliated Hospital of Soochow University

🇨🇳

Suzhou, Jiangsu, China

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