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A Trial Evaluating Efficacy & Safety of RVD +/- Panobinostat in Transplant Eligible, Newly Diagnosed Multiple Myeloma (NDMM)

Phase 2
Terminated
Conditions
Multiple Myeloma
Interventions
Registration Number
NCT02720510
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

This was a multicenter, open-label, randomized phase II study which were to enroll 112 newly diagnosed symptomatic multiple myeloma patients in a 1:1 fashion. Patients were to enroll at approximately 20 centers in the United States.

Patients were to undergo stem cell mobilization with plerixafor plus Granulocyte Colony Stimulating Factor (G-CSF), according to investigator discretion, after 4 cycles of induction therapy. Study treatment interruption for stem cell collection were not to exceed 30 days. All patients were to receive one additional cycle of study treatment after stem cell collection and then proceed to autologous transplant using melphalan 200mg/m2(140mg/m2 for patients \> 70 years), as conditioning.

After Autologus Stem Cell Transplant( ASCT), patients still on study were to initiate maintenance therapy within the 60-120 day period following ASCT, provided they have adequate blood count and clinical recovery. Patients in the RVD arm were to initiate maintenance therapy with lenalidomide alone, and patients in RVD-panobinostat arm were to receive lenalidomide + panobinostat maintenance. Lenalidomide were to be dosed orally at 10mg/day continuously in both arms, increasing to 15mg/day after the first 84 day cycle. Panobinostat were to be dosed at 10mg three times a week, every other week. Total planned duration of maintenance therapy were to be 3 years.

Patients were to remain on study treatment until they complete the maintenance phase, or until they experience disease progression, unacceptable toxicity, or at the discretion of the Investigator.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
6
Inclusion Criteria
  • Patient newly diagnosed with multiple myeloma, based on following IMWG 2014 definition (Rajkumar et al 2014):

  • Clonal bone marrow plasma cells ≥ 10% or biopsy-proven bony or extramedullary plasmacytoma and any one or more of the following myeloma defining events:

  • Evidence of end organ damage that can be attributed to the underlying plasma cell proliferative disorder

  • Any one or more of the following biomarkers of malignancy:

    1. Clonal bone marrow plasma cell percentage ≥ 60%
    2. Involved: uninvolved serum free light chain ratio ≥ 100
    3. >1 focal lesions on MRI studies
  • Patient with measurable disease defined by at least 1 of the following conditions present at screening:

  • Serum M-protein by Protein Electrophoresis (PEP) ≥ 1.0 g/dL (≥ 10 g/L).

  • Urine M-protein by PEP ≥ 200 mg/24 hours. Involved serum free light chain level ≥ 10 mg/dL (≥ 100 mg/L), provided that the serum free light chain ratio is abnormal.

  • Patient eligible for autologous stem cell transplantation based on the investigator's clinical judgment.

  • Patient with an Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤ 2

  • Patient's age ≥ 18 and <75 years at time of signing the informed consent

  • Patient provided written informed consent prior to any screening procedures

  • Women of childbearing potential (WOCBP) with a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline

Key

Exclusion Criteria

Patients eligible for this study must not meet any of the following criteria:

  • Any concomitant anti-cancer therapy (other than bortezomib/lenalidomide/dexamethasone; bisphosphonates are permitted only if commenced prior to the start of screening period)

  • Unresolved diarrhea ≥ CTCAE grade 2 or presence of medical condition associated with chronic diarrhea (such as irritable bowel syndrome, inflammatory bowel disease).

  • Allogeneic stem cell transplant recipient presenting with graft versus host disease either active or requiring immunosuppression

  • Patient shown intolerance to bortezomib or to dexamethasone or components of these drugs or has any contraindication to one or the other drug, following locally applicable prescribing information

  • Patient with rade ≥ 2 peripheral neuropathy or grade 1 peripheral neuropathy with pain on clinical examination at screening

  • Patient received prior treatment with DAC inhibitors including Panobinostat

  • Patient needing valproic acid for any medical condition during the study or within 5 days prior to first administration of panobinostat/study treatment.

  • Patient taking any anti-cancer therapy concomitantly (bisphosphonates are permitted only if commenced prior to the start of screening period)

  • Patient who received:

    1. prior anti-myeloma chemotherapy or medication including Immunomodulator (IMiDs) and Dex ≤ 3 weeks prior to start of study.
    2. experimental therapy or biologic immunotherapy including monoclonal antibodies ≤ 4 weeks prior to start of study.
    3. prior radiation therapy ≤ 4 weeks or limited field radiotherapy ≤ 2 weeks prior start of study.
  • Patient has not recovered from all therapy-related toxicities associated with above listed treatments to < grade 2 CTCAE.

  • Patient undergone major surgery ≤ 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy to < grade 2 CTCAE

  • Patients with evidence of mucosal or internal bleeding

  • Clinically significant, uncontrolled heart disease and/or recent cardiac event (within 6 month prior to screening)

  • Inability to determine the Fridericia's Correction Formula (QTc) F interval

  • Patient with an impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of panobinostat (e.g. ulcerative disease, uncontrolled nausea, vomiting, malabsorption syndrome, obstruction, or stomach and/or small bowel resection)

  • Sexually active males unless they use a condom during intercourse while taking the drug during treatment, and for 6 months after stopping treatment

  • Pregnant or nursing (lactating) women.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm 1 - RVD + PanRevlimidRevlimid, Velcade, dexamethasone and Farydak
Arm 1 - RVD + PanVelcadeRevlimid, Velcade, dexamethasone and Farydak
Arm 1 - RVD + PanFarydakRevlimid, Velcade, dexamethasone and Farydak
Arm 2 - RVDRevlimidRevlimid, Velcade and Dexamethasone
Arm 2 - RVDVelcadeRevlimid, Velcade and Dexamethasone
Arm 2 - RVDdexamethasoneRevlimid, Velcade and Dexamethasone
Arm 1 - RVD + PandexamethasoneRevlimid, Velcade, dexamethasone and Farydak
Primary Outcome Measures
NameTimeMethod
Near Complete Response (nCR)/CR Rate of the Combination of Panobinostat With Bortezomib, Lenalidomide and Dexamethasone (P-RVD) vs RVD in Newly Diagnosed Multiple Myeloma Patients84 days
Secondary Outcome Measures
NameTimeMethod
Minimal Residual Disease (MRD) Negativity (mCR) After 4 Cycles of Induction by Next Gen SequencingMonth 3

MRD negativity by Clonal Sequencing (ClonoSEQTM) assay (Adaptive Biotechnologies)

Best Overall Response Rate (ORR) and MRD Negativity After ASCT and MaintenanceMonth 3 up to end of study, approximately 3 years.

ORR (CR + PR) and MRD negativity after ASCT and maintenance

Depth of Response by International Myeloma Working Group (IMWG) CriteriaDay 22 up to end of study, approximately 3 years

Rate of Very Good Partial Response (VGPR), Complete Response (CR) and Stringent Complete Response (sCR)

Duration of ResponseFrom measurable response to the date of first documented progression or date of death from any cause, whichever came first, assessed up to 3 years.
Progression Free Survival3 years after the last patient is enrolled to the study
Overall Survival3 years after the last patient is enrolled to the study

Trial Locations

Locations (5)

David Geffen School of Medicine at UCLA UCLA

🇺🇸

Los Angeles, California, United States

Northside Hospital Central Research Dept.

🇺🇸

Atlanta, Georgia, United States

Oncology Hematology West Nebraska Cancer Specialists dbaNebraska Cancer Specialists

🇺🇸

Omaha, Nebraska, United States

Memorial West Cancer Center Memorial Cancer Institute

🇺🇸

Pembroke Pines, Florida, United States

Brooke Army Medical Center Hematology/Oncology

🇺🇸

San Antonio, Texas, United States

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