APG101 in Transfusion-Dependent Patients With Low or Intermediate Risk 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)