ENDURE - Efficacy and Safety of AOP2014 With CML Patients in Remission

Inclusion Criteria

• •1. Signed written informed consent form.
• •2. Capability and willingness to comply with study procedures and ability to self-administration of the study drug.
• •3. Male or female aged ≥ 18 years.
• •4. At least three years of TKI therapy.
• •5. BCR-ABL-positive, chronic phase CML patients with a transcript level according to the international scale (IS) of at least MR4, or better (MR4.5, MR5). MR4 is defined as (i) detectable disease ≤0.01% BCR-ABL IS or (ii) undetectable disease in cDNA with ≥10,000 ABL or ≥24,000 GUS transcripts for at least one year. There have to be at least three consecutive PCR-results with MR4 or better within the last year (+ months) before study entry. The latest of these PCRs must be a confirmatory MR4 measurement prior to randomization by the EUTOS-certified Study Reference Laboratories for PCR (BCR-ABL mRNA). No PCR-results in the last year before randomisation can be worse than MR4. If the last PCR was not done within two months from baseline (day 0) in an EUTOS-certified study Reference Laboratory; the PCR sample must be sent to an EUTOS-certified study Reference Laboratory at screening.
• •6. Patients who had failed to discontinue TKI in a prior discontinuation attempt are eligible for this protocol, if they fulfil criterion 5 after retreatment with TKI. A prior TKI discontinuation failure must be specifically indicated at inclusion and documented.
• •7. Adequate organ function:
• •especially total bilirubin, lactate dehydrogenase \[LDH\], aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\] and coagulation parameters ≤ 2 × upper limit of normal (ULN)
• •8. Adequate hematological parameters:
• •platelet count ≥ 100 × 1000000000/L; white blood cell count ≥ 2.5 × 1000000000/L; lymphocytes ≥ 1.0 × 1000000000/L; hemoglobin ≥ 9.0 g/dL or 5.59 mmol/L.
• •9. Female patients with reproductive potential must agree to maintain highly effective methods of contraception by practicing abstinence or by using at least two methods of birth control from the date of consent through the end of the study. If abstinence could not be practiced, a combination of hormonal contraceptive (oral, injectable, or implants) and a barrier method (condom, diaphragm with a vaginal spermicidal agent) has to be used. Male patients must agree to use condoms during study participation.
• •10. Negative serum pregnancy test in women of childbearing potential.
• •11. Date of diagnosis of CML confirmed by laboratory PCR must be known.