APG101 in Myelodysplastic Syndrome

Inclusion criteria

• Signed informed consent
• Male and female patients with cytologically or histologically established diagnosis of de novo MDS according to the WHO-classification, either previously treated or untreated, presenting with low or intermediate risk features according to WHO prognostic status scale (WPSS)
• Diagnosis of MDS with a medullary blast count of less than 5% has to be established or confirmed by bone marrow morphology
• MDS with 5q deletion only if Lenalidomide is not a treatment option
• Red blood cell transfusion dependency of at least 4 units of packed red blood cells (PRBC) during the last 8 weeks before inclusion. Only PRBC transfusions given for a Hb level ≤ 9g/dl or a haemoglobin level > 9g/dl, if clinically indicated (e.g. coronary heart disease, long distance travel), will count.
• Patients refractory to Erythropoietin-stimulating agents (ESA) (as assessed after at least 8 weeks of treatment) or with a low possibility to respond to ESA treatment
• at least 18 years old, smoking or non-smoking, of any ethnic origin
• ECOG performance status ≤ 2
• Suitable veins or existing port system for intra-venous infusion
• Adequate contraception

Exclusion criteria

• Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form
• MDS with medullary blast count ≥ 5%
• Chronic monomyeloic leucemia (CMML)
• Therapy-related / secondary MDS
• High-risk karyotype according to WPSS
• Patients scheduled for bone marrow or stem cell transplant within the next 6 months
• Parallel treatment with ESA or with other experimental therapy
• Prior chemotherapy (including Vidaza)
• Treatment within the last 6 weeks with histone deacetylase (HDAC) inhibitors or ESAs
• Treatment within any other clinical trial parallel to the treatment phase of the current study or within 30 days before inclusion
• Active uncontrolled infection
• HIV, active hepatitis B virus (HBV) and/or hepatitis C virus (HCV) infection
• Any other condition / treatment or past medical history of diseases with poor prognosis that, in the opinion of the investigator, might interfere with the study
• History of or current drug or substance abuse
• History of other (haemato-) oncological disease (except for non-melanoma skin cancer and adequately treated in situ carcinoma of the cervix)
• Inability to understand the protocol requirements, instructions and study-related restrictions, the nature, scope, and possible consequences of the study
• Unlikely to comply with the protocol requirements, instructions and study-related restrictions; e.g., uncooperative attitude, inability to return for follow-up visits, and improbability of completing the study
• Subject is the investigator, research assistant, pharmacist, study coordinator, other staff or relative thereof directly involved in the conduct of the study.
• Hypersensitivity to recombinant proteins or excipients in the investigational drug
• Pregnancy or breast feeding
• Vulnerable patients (e.g., minors or persons kept in detention)