Acute myeloid leukemia MedDRA version: 20.0 Level: PT Classification code 10000880 Term: Acute myeloid leukaemia System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Product Code: GMI-1271
Pharmaceutical Form: Solution for infusion
CAS Number: 1914993-95-5
Current Sponsor code: GMI-1271
Other descriptive name: SODIUM (1R, 3R, 4R, 5S)-3-({2-N-ACETYLAMINO-2-DEOXY-3
Sponsors
GlycoMimetics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Signed informed consent form (ICF) 2.AML (including secondary AML) diagnosed as per World Health Organization (WHO) criteria (Brunning RD 2001) as per local analysis report. 3.For relapsed/refractory subjects only: a) Subjects age = 18 years b) For subjects with primary refractory AML, = 2 prior induction regimens, at least one containing anthracyclines c) For subjects with relapsed AML, first or second untreated relapse. Secondary refractory disease (e.g. refractory after first or second relapse) may not be included. Subjects with one prior HSCT may be included. Any cytotoxic induction regimen used with the intent to induce remission, regardless of the agents used, will be counted as an induction attempt towards this assessment. Single agent FLT3 inhibitors are not considered cytotoxic chemotherapy for the purposes of this assessment. d) Have not previously received MEC and are medically eligible to receive MEC e) Absolute blast count (ABC) = 20,000/mm3 (ABC = total white blood cells [WBC] x blast %) • Entry ABC will be raised to = 40,000/mm3 after evaluation of first two dose levels 4.For treatment-naïve subjects only: a) Subjects = 60 years of age with newly diagnosed AML b) Medically eligible to receive “7+3” cytarabine/idarubicin c) Absolute blast count (ABC) count = 40,000/mm3 5.Eastern Cooperative Oncology Group (ECOG) performance status 0-2 6.Life expectancy > 4 weeks 7.Hemodynamically stable with adequate organ function a)AST and ALT = 2.5 ULN b)Bilirubin = 1.5 ULN c)Creatinine = 1.5 ULN d)QTcF = 480 ms 8.Willing and able to participate in all required evaluations and procedures in this study protocol Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 53 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 49
Exclusion criteria
Exclusion criteria: 1.Acute promyelocytic leukemia 2.Acute leukemia of ambiguous lineage (biphenotypic leukemia) 3.Active signs or symptoms of CNS involvement by malignancy (LP not required) 4.Prior G-CSF, GM-CSF or plerixafor within 14 days of study drug dosing 5.Known history or evidence of active hepatitis A, B, or C or HIV 6.Uncontrolled acute life threatening bacterial, viral or fungal infection 7.Active graft versus host disease (GVHD) = Grade 2 or extensive chronic GVHD requiring immunosuppressive therapy 8.HSCT = 4 months of dosing 9.Any immunotherapy, radiotherapy or experimental therapy within 4 weeks of dosing; any chemotherapy within 14 days of dosing, with the exception of: hydroxyurea, which may be used to control peripheral blast count prior to initiation of GMI-1271 dosing; and FLT3 inhibitors or TKI inhibitors, which are not considered cytotoxic chemotherapy; to avoid any drug-drug interaction such agents must be discontinued 5 days before protocol treatment begins. 10.Major surgery within 4 weeks before first dose of study drug 11.Significant cardiovascular disease 12.Previous or concurrent malignancy 13.Pregnant or nursing (lactating) women 14.WOMEN of child-bearing potential unless they are using highly effective methods of contraception during the study 15.MEN who are sexually active and not willing to use condoms during the duration of the study unless they have undergone vasectomy for sterilization
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of GMI-1271 in combination with chemotherapy;Secondary Objective: •Characterize the PK profile of GMI-1271 •Evaluate efficacy of GMI-1271 in combination with chemotherapy (relapsed/refractory subjects: MEC; treatment-naïve subjects: “7+3” cytarabine/ idarubicin) as measured by overall response rate •Time to response •Evaluate duration of response •Evaluate the event-free survival •Evaluate 6-month and 12- month overall survival probability ;Primary end point(s): The primary endpoint of this study is the frequency, severity, and relatedness of Adverse Events. ;Timepoint(s) of evaluation of this end point: Safety will be assessed from baseline (Day -1) through to End of Treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Pharmacokinetics: The PK data will be analyzed using Bayesian population methods. Interim PK analysis will be performed to evaluate PK parameters for individual dose levels. Secondary efficacy endpoints: Overall response rate (ORR) is defined as the proportion of subjects who achieve a Complete Response (CR) or Complete response with incomplete blood count recovery (CRi) Time to response (TTR): Time from date of first dose to first documentation of response (CR or CRi). Duration of response (DOR): Time (months) from date of first documented remission (CR or CRi) to the date of relapse or death from any cause, whichever occurs first. Event-free survival (EFS): Time (months) from date of first dose to the date of treatment failure, relapse, or death from any cause, whichever occurs first. Overall survival (OS):Time (months) from date of first dose to the date of death from any cause. 6-month and 12- month OS probability: The probability of survival at 6 and 12-month, respectively, after the date of first dose based on the Kaplan-Meier estimate.;Timepoint(s) of evaluation of this end point: The endpoints will be measured at End of Treatment; Phase I (6 months) and Phase II (12 months) | — |
Countries
Australia, Ireland, United States
Contacts
Public ContactClinical Trial Information Desk
GlycoMimetics Inc.
Outcome results
None listed