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CompletedNCT02722941Updated Nov 1, 2022Results posted

Panobinostat (LBH589): Multiple Myeloma - Autologous Hematopoietic Cell Transplantation (HCT)

A Phase 2 interventional study of Panobinostat in Multiple Myeloma, sponsored by H. Lee Moffitt Cancer Center and Research Institute. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-11-01.

Sponsored by H. Lee Moffitt Cancer Center and Research Institute · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
30
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
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Study summary

The purpose of this study is to learn more about ways to prevent or delay relapse of multiple myeloma (MM). This study will determine the best dosing schedule of LBH589 maintenance therapy as well as the safety (side effects) and tolerability of LBH589 maintenance therapy after autologous hematopoietic cell transplant (HCT).

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Conditions studied

  • Multiple Myeloma

Keywords

  • autologous hematopoietic cell transplant (HCT)
  • autologous
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In context

Multiple Myeloma

3,632 studies on the registry are indexed under Multiple Myeloma; 745 are open to participants now.

This study's enrollment of 30 is below the median of 41 across 2,907 interventional studies indexed under Multiple Myeloma.

Browse Multiple Myeloma studies →

Lead sponsor

H. Lee Moffitt Cancer Center and Research Institute is the lead sponsor of 533 studies on the registry; 74 are open to participants now.

Of its 99 completed or terminated interventional studies of FDA-regulated products, 57 (58%) have results posted.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Adult patients, age ≥ 18 years old
  • Ability to provide written informed consent obtained prior to participation in the study and any related procedures being performed
  • Histologically confirmed diagnosis of multiple myeloma
  • Meeting the Criteria for Symptomatic Multiple Myeloma (CRAB criteria) before the initiation of systemic chemotherapy
  • Received high-dose melphalan (≥ 140 mg/m\^2) followed by autologous HCT based on the institutional guidelines and within +45 and +180 after autologous HCT at the time of panobinostat maintenance initiation
  • Must have achieved at least partial response (PR) prior to autologous HCT and must not have progressive disease (PD) prior to the initiation of maintenance therapy
  • Must meet the following laboratory criteria (prior to the initiation of panobinostat maintenance): Absolute neutrophil count (ANC) ≥ 1 x 10\^9/L; Hemoglobin ≥ 8 g/dl; Platelets ≥ 50 x 10\^9/L (without transfusion support); Creatinine clearance ≥ 40 ml/min or serum creatinine ≤ 2.5 x upper limit of normal (ULN); aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN; Serum bilirubin ≤ 1.5 x ULN; Albumin > 3.0 g/dl; Clinically euthyroid. Note: Participants are permitted to receive thyroid hormone supplements to treat underlying hypothyroidism.
  • Baseline (pre-HCT) multigated acquisition (MUGA) or echocardiogram (ECHO) must demonstrate left ventricular ejection fraction (LVEF) ≥ the limit of normal (LLN) of the institutional normal
  • Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤ 2 or Karnofsky performance status ≥ 70%
  • Prior histone deacetylase (HDAC), deacetylase (DAC), HSP90 inhibitors or valproic acid for the treatment of cancer is allowed

Exclusion criteria

Exclusion Criteria:

  • Potential participants who have purely non-secretory multiple myeloma (i.e., the absence of a measurable protein in serum by electrophoresis and immunofixation and the absence of Bence-Jones protein in the urine defined by use of electrophoresis and immunofixation)
  • Prior allogeneic HCT
  • Prior solid organ transplant requiring immunosuppressive therapy
  • Potential participants who will need valproic acid for any medical condition during the study or within 5 days prior to first panobinostat treatment
  • Impaired cardiac function or clinically significant cardiac diseases
  • Diarrhea > Common Terminology Criteria for Adverse Events (CTCAE) grade 2
  • Other concurrent severe and/or uncontrolled medical conditions (e.g., uncontrolled diabetes or active or uncontrolled infection) including abnormal laboratory values, that could cause unacceptable safety risks or compromise compliance with the protocol
  • Using medications that have a relative risk of prolonging the QT interval or inducing torsade de pointes if treatment cannot be discontinued or switched to a different medication prior to starting study drug
  • Have received targeted agents within 2 weeks or within 5 half-lives of the agent and active metabolites (whichever is longer) and who have not recovered from side effects of those therapies.
  • Have received either immunotherapy within \< 8 weeks; chemotherapy within \< 4 weeks; or radiation therapy to > 30% of marrow-bearing bone within \< 2 weeks prior to starting study treatment; or who have not yet recovered from side effects of such therapies
  • Have undergone major surgery ≤ 4 weeks prior to starting study drug or have not recovered from side effects of such therapy
  • Women who are pregnant or breast feeding or women of childbearing potential (WOCBP) not using an effective method of birth control. WOCBP must have a negative serum pregnancy test within 24 hours of receiving the first dose of study medication.
  • Male patients whose sexual partners are WOCBP not using effective birth control
  • A prior malignancy with in the last 5 years (except for basal or squamous cell carcinoma, or in situ cancer of the cervix)
  • Known positivity for human immunodeficiency virus (HIV) or hepatitis C; baseline testing for HIV and hepatitis C is not required
  • Any significant history of non-compliance to medical regimens or unwilling or unable to comply with the instructions given to him/her by the study staff
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Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
30 participants (actual)

Study arms

  • Active comparator
    Cohort A: Maintenance Therapy

    Panobinostat (LBH589): 20 mg by mouth three (3) times per week, every other week, of a 28-day schedule.

    Drug: Panobinostat

  • Active comparator
    Cohort B: Maintenance Therapy

    Panobinostat (LBH589): 10 mg by mouth daily for seven (7) days, every other week, of a 28-day schedule.

    Drug: Panobinostat

Interventions

  • DrugPanobinostat

    Maintenance therapy dosing as outlined in Cohorts A and B.

    Also known as: LBH589

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What researchers measure

Primary outcomes

  1. Relative Dose Intensity (RDI) Per Cohort

    Investigators will calculate RDI for each cohort. Relative dose intensity (RDI) represents the ratio of the amount of a drug actually delivered \[actual dose intensity (DI)\] to the amount planned (planned DI). The purpose of calculating RDI is to evaluate whether the planned DI of a chemotherapy treatment was actually achieved which may suggest the feasibility of planned treatment regimen. There are multitude of reports demonstrating a correlation between RDI and survival in cancer treatment. RDI = (total dose received by the patient = mg)/(planned full dose of drug = mg).

    Time frame: Up to 2 years

Secondary outcomes

  1. Complete Response Rate

    Complete Response (CR) rate to panobinostat maintenance therapy after autologous HCT. CR: Negative immunofixation of serum and urine, disappearance of any soft tissue plasmacytomas, and \< 5% plasma cells in bone marrow.

    Time frame: Up to 5 years

  2. Progression Free Survival (PFS)

    Progressive Disease (PD) according to Uniform Response Reporting Criteria for Multiple Myeloma by the International Myeloma Working Group (IMWG). Increase of 25% from lowest response value in any of the following: * Serum M- component (absolute increase must be ≥ 0.5 g/dL) * Urine M-component (absolute increase must be ≥ 200 mg/24 h) * Only in patients without measurable serum and urine M protein levels and without measurable disease by free light chain (FLC) levels, bone marrow plasma cell percentage (absolute percentage must be ≥ 10% ) * Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas * Development of hypercalcemia (corrected serum calcium \> 11.5 mg/dL) that can be attributed solely to the plasma cell proliferative disorder

    Time frame: at 2 years

  3. Overall Survival (OS)

    OS: The length of time from either the date of diagnosis or the start of treatment for a disease, such as cancer, that patients diagnosed with the disease are still alive.

    Time frame: at 2 years

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Results

Posted Aug 9, 2021

Participant flow

Participant flow — Overall Study
MilestoneCohort A: Maintenance TherapyCohort B: Maintenance Therapy
Started1515
Completed1515
Not completed00

Outcome measures

PrimaryRelative Dose Intensity (RDI) Per Cohort

Investigators will calculate RDI for each cohort. Relative dose intensity (RDI) represents the ratio of the amount of a drug actually delivered \[actual dose intensity (DI)\] to the amount planned (planned DI). The purpose of calculating RDI is to evaluate whether the planned DI of a chemotherapy treatment was actually achieved which may suggest the feasibility of planned treatment regimen. There are multitude of reports demonstrating a correlation between RDI and survival in cancer treatment. RDI = (total dose received by the patient = mg)/(planned full dose of drug = mg).

Time frame:
Up to 2 years
Reported as:
Median · percentage of dose
Relative Dose Intensity (RDI) Per Cohort
percentage of doseCohort A: Maintenance TherapyCohort B: Maintenance Therapy
Relative Dose Intensity (RDI) Per Cohort97.9 (77.1 to 100)89.6 (63.1 to 100)
SecondaryComplete Response Rate

Complete Response (CR) rate to panobinostat maintenance therapy after autologous HCT. CR: Negative immunofixation of serum and urine, disappearance of any soft tissue plasmacytomas, and \< 5% plasma cells in bone marrow.

Time frame:
Up to 5 years
Reported as:
Number · percentage
Complete Response Rate
percentageCohort A: Maintenance TherapyCohort B: Maintenance Therapy
Complete Response Rate73.33 (44.9 to 92.2)66.6 (38.4 to 88.2)
SecondaryProgression Free Survival (PFS)

Progressive Disease (PD) according to Uniform Response Reporting Criteria for Multiple Myeloma by the International Myeloma Working Group (IMWG). Increase of 25% from lowest response value in any of the following: * Serum M- component (absolute increase must be ≥ 0.5 g/dL) * Urine M-component (absolute increase must be ≥ 200 mg/24 h) * Only in patients without measurable serum and urine M protein levels and without measurable disease by free light chain (FLC) levels, bone marrow plasma cell percentage (absolute percentage must be ≥ 10% ) * Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas * Development of hypercalcemia (corrected serum calcium \> 11.5 mg/dL) that can be attributed solely to the plasma cell proliferative disorder

Time frame:
at 2 years
Reported as:
Median · percentage of participants
Progression Free Survival (PFS)
percentage of participantsCohort A: Maintenance TherapyCohort B: Maintenance Therapy
Progression Free Survival (PFS)71.8 (41.1 to 88.4)53.3 (26.3 to 74.4)
SecondaryOverall Survival (OS)

OS: The length of time from either the date of diagnosis or the start of treatment for a disease, such as cancer, that patients diagnosed with the disease are still alive.

Time frame:
at 2 years
Reported as:
Median · percentage of participants
Overall Survival (OS)
percentage of participantsCohort A: Maintenance TherapyCohort B: Maintenance Therapy
Overall Survival (OS)100 (NA to NA)100 (NA to NA)

Adverse events

Collected over Adverse events collected from date of consent until off study date, 4 years, 2 months.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Cohort A: Maintenance Therapy0/15 (0%)2/15 (13.3%)10/15 (66.7%)
Cohort B: Maintenance Therapy0/15 (0%)3/15 (20%)11/15 (73.3%)
Most frequent serious events
Most frequent serious events
EventCohort A: Maintenance TherapyCohort B: Maintenance Therapy
Lung infectionInfections and infestations0/152/15
Flu like symptomsGeneral disorders0/151/15
ColitisGastrointestinal disorders1/150/15
HeadacheNervous system disorders1/150/15
FeverGeneral disorders0/151/15
Bone painMusculoskeletal and connective tissue disorders1/150/15
CholecystitisGastrointestinal disorders1/150/15
Most frequent other events
Showing 10 of 46
Most frequent other events
EventCohort A: Maintenance TherapyCohort B: Maintenance Therapy
White blood cell decreasedInvestigations6/153/15
Cholesterol highInvestigations6/150/15
HypertensionVascular disorders5/155/15
HyperglycemiaMetabolism and nutrition disorders5/152/15
AnemiaBlood and lymphatic system disorders5/155/15
FatigueGeneral disorders5/152/15
Platelet count decreasedInvestigations4/154/15
Back PainMusculoskeletal and connective tissue disorders4/153/15
Peripheral motor neuropathyNervous system disorders4/152/15
Lymphocyte count decreasedInvestigations4/151/15

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Cohort A: Maintenance TherapyCohort B: Maintenance TherapyTotal
<=18 years000
Between 18 and 65 years11819
>=65 years4711
Sex: Female, Male
Sex: Female, Male(Participants)Cohort A: Maintenance TherapyCohort B: Maintenance TherapyTotal
Female6511
Male91019
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Cohort A: Maintenance TherapyCohort B: Maintenance TherapyTotal
Hispanic or Latino303
Not Hispanic or Latino121527
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Cohort A: Maintenance TherapyCohort B: Maintenance TherapyTotal
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American224
White131326
More than one race000
Unknown or Not Reported000
Region of Enrollment
Region of Enrollment(participants)Cohort A: Maintenance TherapyCohort B: Maintenance TherapyTotal
United States151530
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Study locations

1 site
  • H. Lee Moffitt Cancer Center and Research Institute
    Tampa, Florida 33612, United States
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Apr 13, 2017

Documents are hosted by the registry — open the source record to download them.

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 1, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT02722941
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Collaborators
Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
Mar 30, 2016
Start date
Jun 10, 2016
Primary completion
Jun 7, 2020
Completion
Jan 18, 2021
Results posted
Aug 9, 2021
Last update
Nov 1, 2022

Study contacts

Taiga Nishihori, M.D.
principal investigator · H. Lee Moffitt Cancer Center and Research Institute

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Oct 2022. You cannot join it, but the record below documents what was studied.

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