Symptoms and General Pathology

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Neoplasms

Blood and Lymph Conditions

Rare Diseases

Syndrome

Preleukemia

Myelodysplastic Syndromes

Anemia

Bone Marrow Diseases

Hematologic Diseases

Precancerous Conditions

Single arm open label study. Patient receive 100 mg APG101 i.v. weekly over 12 weeks with a 6 monthly follow-up phase

Patients will be treated 12 weeks with 100 mg APG101 intravenous weekly

Treatment with APG101

During the study, bone marrow will be collected 4 times to assess study objectives

Bone marrow collection

During the study, blood will be drawn at different time points to assess study objectives

Blood drawings

Florian Nolte, MD

It has been shown in preclinical experiments with bone marrow from patients with myelodysplastic syndrome that APG101 rescues erythrocytes from premature cell death. This is expected to translate in an improved erythropoiesis and ameliorated anemia in MDS patients.

APG101 might, therefore, be a valuable addition to current treatments of low- or intermediate MDS patients suffering from anaemia.

Transfusion-dependent patients with low or intermediate risk MDS according to WHO Prognostic Scoring Scale (WPSS) can be included in this study.

Treatment consists of 100mg APG101 intravenous as a weekly treatment over 12 weeks + 6 months follow up phase.

Primary objective of the trial is safety and tolerability of APG101; secondary objectives are

* Hematologic, cytologic and cytogenetic response rate using modified International Working Group (IWG) response criteria
* Incidence and time to leukemic progression at 37 weeks
* OS (Overall survival) at 37 weeks

APG101 in Myelodysplastic Syndrome

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

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.

Overall survival (OS) is defined as time from start of study treatment to death from any cause

Changes in transfusion frequency will be evaluated as those are early signs of an improval in erythropoiesis

By assessing different parameters (cytologic, hematologic, cytogenetic), safety as well as efficacy of treatment with APG101 can be evaluated

Changes in Hb level will be evaluated as those are early signs of an improval in erythropoiesis

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
APG101 in Myelodysplastic Syndrome	NCT01736436	Myelodysplastic...	Treatment with ... Bone marrow col... Blood drawings	18 Years -	Apogenix AG
APG101 in Myelodysplastic Syndrome	NCT01736436	Myelodysplastic...	Treatment with ... Bone marrow col... Blood drawings	18 Years -	Apogenix AG

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