NCT01736436

APG101 in Myelodysplastic Syndrome

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Apogenix GmbH · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Conditions

• Myelodysplastic Syndrome

Interventions

Type	Name	Description
DRUG	Treatment with APG101	Patients will be treated 12 weeks with 100 mg APG101 intravenous weekly
PROCEDURE	Bone marrow collection	During the study, bone marrow will be collected 4 times to assess study objectives
PROCEDURE	Blood drawings	During the study, blood will be drawn at different time points to assess study objectives

Timeline

• Start date: 2013-01-01
• Primary completion: 2015-12-01
• Completion: 2015-12-01
• First posted: 2012-11-29
• Last updated: 2016-08-24

Locations

2 sites across 1 country: Germany