(very) poor risk AML or RAEB with IPSS = 1.5 MedDRA version: 20.1 Level: LLT Classification code 10024329 Term: Leukemia System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 21.1 Level: PT Classification code 10028533 Term: Myelodysplastic syndrome System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Product Name: panobinostat
Pharmaceutical Form: Capsule
INN or Proposed INN: PANOBINOSTAT
CAS Number: 404950-80-7
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20
Sponsors
HOVON Foundation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Patients with poor risk or very poor risk AML or RAEB with IPSS = 1.5. • Eligibility for continuation with intensive induction/consolidation chemotherapy • Eligible for allogeneic donor search (related/unrelated) • 18-70 years, inclusive • Written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 130 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 15
Exclusion criteria
Exclusion criteria: • History of active malignancy during the past 2 years with the exception of basal carcinoma of the skin or carcinoma “in situ” of the cervix or breast • Known HIV-positivity • Pregnant or breast-feeding female patients
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: *Phase I part - To asses the safety and feasibility of post-transplant panobinostat combined with decitabine to a regimen of T-cell replete RIC alloHSCT in patient with (very) poor-risk AML/RAEB, and select the recommended dose level for part II of the study *Phase II part - To assess the feasibility and efficacy of addition of post-transplant panobinostat combined with decitabine to a regimen of T-cell replete RIC alloHSCT and DLI in patients with (very) poor-risk AML/RAEB *Phase III part - To assess the feasibility and efficacy of post-transplant panobinostat monotherapy to a regimen of T-cell replete RIC alloHSCT and DLI in patients with (very) poor-risk AML;Secondary Objective: - Assess efficacy in terms of complete remission rate, overall and progression free survival. - Assess toxicity ;Primary end point(s): Phase I part • Feasibility of protocol treatment as defined by the number of DLTs during the first cycle PNB/DAC . Phase II part • Feasibility of protocol treatment as defined by percentage of patients actually receiving treatment according to protocol up to eligibility for the first DLI within 115 days. Phase III part • Feasibility of protocol treatment as defined by percentage of patients actually receiving treatment according to protocol up to eligibility for the first DLI within 115 days. ;Timepoint(s) of evaluation of this end point: Evaluation will take place when the required data are available and evaluated | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Response to first cycle PNB/DAC • Response to second cycle PNB/DAC • Percentage of successful donor searches • Percentage of patients who received alloHSCT • Best response on protocol • Engraftment after alloHSCT • Incidence and severity of acute and chronic GvHD • (Serious) adverse events • Overall survival (OS) from registration and start of protocol treatment • PFS from registration and from start of protocol treatment • NRM rate ;Timepoint(s) of evaluation of this end point: Last patient, last visit, 5 years after registration of the last patient, when the required data are available and evaluated | — |
Countries
Belgium, Netherlands, Switzerland
Contacts
Public ContactHOVON Data Center
HOVON
Outcome results
None listed