Acute myelogenous leukemia or high risk myelodysplastic syndromes in patients who are elderly and have previously untreated disease or who are adult and have relapsed or refractory disease. MedDRA version: 8.1 Level: LLT Classification code 10000886 Term: Acute myeloid leukemia
Conditions
Interventions
Product Name: Not yet Assigned
Product Code: MGCD0103
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Not yet assigned
Current Sponsor code: MGCD0103
Concentration unit: mg milligram(s)
Concen
Sponsors
Pharmion GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients must fulfill all of the following inclusion criteria in order to be admitted into the study: i. Pathologic confirmation of AML or high-risk MDS (bone marrow blast count =10% or presence of peripheral blasts). ii. Prior treatment a. Elderly cohort: no prior chemotherapy treatment for AML/high-risk MDS and are not currently candidates for intensive chemotherapy. Prior treatment with agents such as hydroxyurea or corticosteroids used for peripheral count control will be allowed. Prior treatment for non-high-risk MDS will be allowed. b.Relapsed/refractory cohort: Disease that is relapsed or refractory to prior treatment. iii. Age a. Elderly cohort: 70 years or greater b.Relapsed/refractory cohort: 18 years or greater iv. ECOG performance status of 0 or 1. v. Laboratory requirements: • Total Bilirubin = 1.5 x Upper Limit of Normal (ULN) • Aspartate transaminase (AST /SGOT) and alanine transaminase (ALT / SGPT) =2.5 x ULN • Serum Creatinine = 1.5 x ULN vi. Patients or their legal representative must be able to read, understand, and sign a written informed consent (approved by the institutional review board/Ethics Committee (IRB/EC)). 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 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients fulfilling any of the following criteria will not be admitted into the study: i. Pregnant or lactating women. Women of child-bearing potential (WOCBP) must have a negative serum pregnancy test documented within 7 days prior start of study drug. ii. WOCBP and men whose partners are WOCBP must use an acceptable method of contraception while enrolled on this study, and for a period of 3 months following study drug treatment. Patients unwilling or unable to follow this guideline will be excluded. An example of an acceptable form of contraception is a double barrier method, such as condom with diaphragm. iii. Patients with uncontrolled intercurrent illness, active or uncontrolled infections, or a fever >38.5°C (not due to tumor fever) on the day of scheduled dosing. iv. Patients with serious illnesses, medical conditions, or other medical history, including laboratory results, which, in the investigator’s opinion, would be likely to interfere with a patient’s participation in the study, or with the interpretation of the results. v. Patients who have been treated with any investigational drug within 30 days prior to study initiation (an investigational drug is one for which there is no approved indication), or who are receiving concurrent treatment with other experimental drugs or anti-cancer therapy. vi. Known hypersensitivity to HDAC inhibitors, to any of the components of MG-0103. vii. Known HIV (human immunodeficiency virus) or known active Hepatitis B or C. Testing is not required for patients not suspected of having these conditions. For patients with a history of Hepatitis B or C that is no longer active, the Investigator should contact MethylGene in advance to confirm patient’s eligibility. viii. Any condition (e.g., known or suspected poor compliance, psychological instability, geographical location, etc) that, in the judgment of the investigator, may affect the patient’s ability to sign the informed consent and undergo study procedures. ix. Any condition that will put the patient at undue risk or discomfort as a result of adherence to study procedures. For example, consider requirement to take MG-0103 with a low-pH drink and recommendation to avoid agents that increase gastric-pH.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Estimation of the overall response rate (CR + CRi + PR), complete response + partial response rate in patients in two cohorts: 1. Elderly (=70 years) patients with untreated acute myelogenous leukemia (AML)/high risk myelodysplasia (MDS) 2. Adult patients with relapsed or refractory AML/high-risk MDS ;Secondary Objective: 1. Estimation of progression-free survival (PFS) 2. Estimation of duration of objective response 3. Characterisation of the safety profile of MG-0103 4. Assessment of biomarkers and predictive markers for MG-0103 5. Evaluation of pharmacokinetic parameters of MG-0103 in these populations (for patients at selected centers). ;Primary end point(s): The primary objective will be to estimate the overall response rate (CR + CRi + PR) for treated patients induced by MG-0103 in each of the cohorts, defined as complete response (CR) + morphologic complete reponse with incomplete blood count (CRi) + partial response (PR). | — |
Countries
United Kingdom
Outcome results
None listed