Phase I/II Study With Oral Panobinostat Maintenance Thera... | NCT01451268

Studies
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)

NCT01451268

Sponsor

Johann Wolfgang Goethe University Hospital

Status

Unknown status

Last Update Posted

Mar 20, 2018Actual

Enrollment

62Estimated

Phase

Phase 1Phase 2

Conditions

Myelodysplastic Syndrome
Acute Myeloid Leukemia

Interventions

Panobinostat
Panobinostat

Countries

Germany

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Status Module

Record Verification Date

Mar 2018

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Active, not recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 2011Actual

Primary Completion Date

Apr 2018Estimated

Completion Date

Apr 2018Estimated

First Submitted Date

Sep 30, 2011

First Submission Date that Met QC Criteria

Oct 11, 2011

First Posted Date

Oct 13, 2011Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 19, 2018

Last Update Posted Date

Mar 20, 2018Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

62Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Panobinostat Arm A	Experimental		Drug: Panobinostat
Panobinostat Arm B	Experimental		Drug: Panobinostat

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Panobinostat	Drug	10mg upto 40mg Panobinostat dose escalation in consequent cohorts; frequency: three times a week, every week; duration: one year	Panobinostat Arm A

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Maximum tolerated dose (MTD) of panobinostat		after 28 days of administration
Dose-limiting toxicity (MTD) of Panobinostat		after 28 days of administration

Secondary Outcomes

Measure	Description	Time Frame
Cumulative incidence of hematologic relapse and death		one year after HSCT
Reconstitution of the immune system as measured by changes in numbers, ratio, phenotype and activation state of peripheral blood cell populations during panobinostat therapy		patients will be followed for up to 2 years depending on the duration of study participation

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

AML (except acute promyelocytic leukemia, AML M3) with high-risk features defined as one or more of the following criteria:
- refractory to or relapsed after at least one cycle of standard chemotherapy
- > 10% bone marrow blasts at day 15 of the first induction cycle
- adverse risk cytogenetics including complex karyotype (≥ 3 abnormalities or abnormalities of chromosomes 3, 5 or 7) regardless of stage
- secondary to MDS or radio-/chemotherapy or
MDS RAEB according to the WHO classification or intermediate-2 or high-risk according to IPSS or
Chronic myelomonocytic leukemia (CMML) with ≥ 5% bone marrow blasts and
- Allogeneic HSCT with reduced intensity conditioning (see Section 15.1 for definition) performed within 60 - 150 days prior to study entry
- Complete hematologic remission documented by bone marrow aspiration within 28 days prior to study entry

Exclusion Criteria:

Active acute GvHD overall grade 2 - 4
Prior treatment with a deacetylase (DAC) inhibitor
Patients with impaired cardiac function or other concurrent severe and/or uncontrolled medical conditions
Clinical symptoms suggesting central nervous system (CNS) leukemia
Patient has an impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral panobinostat

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
University Hospital Düsseldorf		Düsseldorf		40225	Germany
University Hospital Essen

ID	Term
D009190	Myelodysplastic Syndromes
D015470	Leukemia, Myeloid, Acute

ID	Term
D001855	Bone Marrow Diseases
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D007951	Leukemia, Myeloid
D007938	Leukemia
D009370	Neoplasms by Histologic Type
D009369	Neoplasms

ID	Term
D006877	Hydroxamic Acids
D006898	Hydroxylamines
D000588	Amines
D009930	Organic Chemicals
D006880	Hydroxy Acids
D002264	Carboxylic Acids
D007211	Indoles
D006574	Heterocyclic Compounds, 2-Ring
D000072471	Heterocyclic Compounds, Fused-Ring
D006571	Heterocyclic Compounds