NCT01451268

Phase I/II Study With Oral Panobinostat Maintenance Therapy Following Allogeneic Stem Cell Transplantation in Patients With High Risk Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML)

Phase I/II Study With Oral Panobinostat Maintenance Therapy Following Allogeneic Stem Cell Transplantation in Patients With High Risk MDS or AML (PANOBEST)

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 62 (estimated)
• Sponsor: Johann Wolfgang Goethe University Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The study's primary objective is to determine the maximum tolerated dose (MTD) and dose-limiting toxicity (DLT) of Panobinostat when administered within 150 days after hematopoietic stem cell transplantation (HSCT) and given in conjunction with standard immunosuppressive therapy after HSCT for patients with high-risk Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML). Secondary objectives are * To determine safety and tolerability of panobinostat * To determine overall and disease-free survival at 12 months after HSCT * To evaluate immunoregulatory properties of panobinostat * To evaluate patient-reported health-related quality of life (HRQL) The hypothesis of this study is that panobinostat can be an effective drug in preventing relapse of MDS and AML patients with high-risk features after hematopoietic stem cell transplantation with reduced-intensity conditioning (RIC-HSCT) while at the same time reducing graft-versus-host disease (GvHD) with preservation of graft-versus-leukemia (GvL) effect.

Conditions

• Myelodysplastic Syndrome
• Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Panobinostat	10mg upto 40mg Panobinostat dose escalation in consequent cohorts; frequency: three times a week, every week; duration: one year
DRUG	Panobinostat	Start of Arm B after completion of Arm A; initial dose-level: one level below MTD of Arm A; 10mg upto 60mg Panobinostat dose escalation in consequent cohorts; frequency: three times a week, every other week; duration: one year

Timeline

• Start date: 2011-01-01
• Primary completion: 2018-04-01
• Completion: 2018-04-01
• First posted: 2011-10-13
• Last updated: 2018-03-20

Locations

6 sites across 1 country: Germany