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A randomized phase II multicenter study with a safety run-in to assess the tolerability and efficacy of the addition of oral tosedostat to standard induction therapy in AML and RAEB >= 66 years and very poor risk AML >= 18 years.;A study in the frame of the masterprotocol of parallel randomized phase II studies in elderly AM

Phase 2
Recruiting
Conditions
Acute myeloid leukemia
leukemia
10024324
Registration Number
NL-OMON39280
Lead Sponsor
HOVO
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
Not specified
Target Recruitment
168
Inclusion Criteria

· Patients eligible for standard chemotherapy.
· Patients >= 66 years with a cytopathologically confirmed diagnosis according WHO
classification of
o AML (not APL) or
o refractory anemia with excess of blasts (RAEB) with
an IPSS score >= 1.5
OR
Patients of any age >= 18 years with a cytopathologically confirmed diagnosis
according WHO classification of
o AML with very poor risk AML
· Subjects with secondary AML progressing from antecedent (at least 4 months
duration) myelodysplasia are also eligible.
· SGOT (AST) and SGPT (ALT) <= 1.5 x the upper limit of the normal range (ULN) at
the laboratory where the analyses were performed.
· Total serum bilirubin level <= 1.5 x the ULN at the laboratory where the analysis
was performed.
· Serum creatinine concentration <= 1.5 x the ULN at the laboratory where the
analysis was performed.
· WHO performance status <= 2
· Written informed consent.
· Female patients of childbearing potential must have a negative serum pregnancy
test within 2 weeks prior to enrollment.
· Male and female patients must use an effective contraceptive method during the
study and for a minimum of 6 months after study treatment

Exclusion Criteria

· Acute promyelocytic leukemia
· Patients previously treated for AML (any antileukemic therapy including
investigational agents), a short treatment period (< 2 weeks) with Hydroxyurea is
allowed
· Past or current history (within the last 2 years prior to randomization) of
malignancies except for the indication under this study and curatively treated:
* Basal and squamous cell carcinoma of the skin
* in situ carcinoma of the cervix
· Blast crisis of chronic myeloid leukemia
· Clinically significant (i.e. active) cardiovascular disease, for example
cerebrovascular accidents (<= 6 months prior to randomization), myocardial
infarction (<= 6 months prior to randomization), unstable angina, New York Heart
Association (NYHA) grade II or greater congestive heart failure
· Patients with a history of non-compliance to medical regimens or who are
considered unreliable with respect to compliance
· Patients with any serious concomitant medical condition which could, in the
opinion of the investigator, compromise participation in the study.
· Patients who have senile dementia, mental impairment or any other psychiatric
disorder that prohibits the patient from understanding and giving informed
consent.
· Pregnant or lactating patients.
· Current concomitant chemotherapy, radiation therapy, or immunotherapy other
than as specified in the protocol.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
<p>Part A:<br /><br>DLT of tosedostat at three dose levels (120mg, 180mg and 240mg) added to<br /><br>standard chemotherapy<br /><br>DLT is defined as: Death within 31 days of start cycle I<br /><br>Part B:<br /><br>To assess in a randomized comparison the effect of the in Part A selected dose<br /><br>of tosedostat on the CR<br /><br>rate.</p><br>
Secondary Outcome Measures
NameTimeMethod
<p>Part B<br /><br>- Overall survival (time from registration till the death of the patient.)<br /><br>- Event free survival (i.e., time from registration to induction failure (i.e.<br /><br>no CR on induction), death or<br /><br>relapse whichever occurs first)<br /><br>- Disease free survival (time from CR on protocol treatment until relapse or<br /><br>death, whichever comes first)<br /><br>- Prognostic value of molecular markers and gene expression profiles of the<br /><br>leukemia assessed at<br /><br>diagnosis<br /><br>- Prognostic value of minimal residual disease (MRD) measurements following<br /><br>therapy by standardized<br /><br>sampling of marrow/blood</p><br>
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