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APG101 in Transfusion-Dependent Patients With Low or Intermediate Risk Myelodysplastic Syndrome

Phase 1
18 Years
Open (Enrolling)
Myelodysplastic Syndrome

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Trial Information

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)

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety and tolerability

Outcome Description:

Evaluation of adverse events (AEs) and serious adverse events (SAEs). Evaluation of electrocardiograms (ECGs), abdominal ultrasound, anti-drug antibodies (ADA), changes in lymphocyte subpopulations / activation markers and changes in performance status (ECOG). Any side effects potentially related to the APG101 treatment are evaluated.

Outcome Time Frame:

During the whole study (37 weeks)

Safety Issue:


Principal Investigator

Florian Nolte, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Universitaetsmedizin Mannheim, III. Medizinische Klinik, Hämatologie und Onkologie, Theodor-Kutzer-Ufer 1-3, 68167 Mannheim, Germany


Germany: Paul-Ehrlich-Institut

Study ID:




Start Date:

January 2013

Completion Date:

June 2014

Related Keywords:

  • Myelodysplastic Syndrome
  • MDS
  • Myelodysplastic syndrome
  • Low and intermediate risk
  • Transfusion-dependent patients
  • Transfusion
  • Anemia
  • Myelodysplastic Syndromes
  • Preleukemia