A Phase 3, Multicenter, Randomized, Open-Label Study to Compare the Efficacy and Safety of Pomalidomide in Combination With Low-Dose Dexamethasone Versus High-Dose Dexamethasone in Subjects With Refractory Multiple Myeloma or Relapsed and Refractory Multiple Myeloma and Companion Study

NCT ID: NCT01311687

Last Updated: 2018-10-24

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

455 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-03-11

Study Completion Date

2017-08-29

Brief Summary

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The purpose of this study is to compare efficacy and safety of pomalidomide in combination with low-dose dexamethasone versus high-dose dexamethasone in subjects with refractory or relapsed and refractory multiple myeloma.

Detailed Description

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Conditions

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Multiple Myeloma

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Pomalidomide + Low-Dose Dexamethasone

Participants received 4 mg pomalidomide administered by mouth on Days 1 to 21 of each 28-day treatment cycle and 40 mg dexamethasone (or 20 mg for participants \> 75 years of age) administered by mouth once per day on Days 1, 8, 15, and 22 of each 28-day cycle until disease progression.

Group Type EXPERIMENTAL

pomalidomide

Intervention Type DRUG

4 mg pomalidomide capsules administered orally

Dexamethasone

Intervention Type DRUG

40 mg dexamethasone (or 20 mg for participants \> 75 years of age) tablets administered orally

High-Dose Dexamethasone

Participants received 40 mg dexamethasone (or 20 mg for participants \> 75 years of age) administered by mouth once per day on Days 1 to 4, 9 to 12, and 17 to 20 of each 28-day treatment cycle until disease progression.

Group Type ACTIVE_COMPARATOR

Dexamethasone

Intervention Type DRUG

40 mg dexamethasone (or 20 mg for participants \> 75 years of age) tablets administered orally

Interventions

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pomalidomide

4 mg pomalidomide capsules administered orally

Intervention Type DRUG

Dexamethasone

40 mg dexamethasone (or 20 mg for participants \> 75 years of age) tablets administered orally

Intervention Type DRUG

Other Intervention Names

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CC-4047 Pomalyst®

Eligibility Criteria

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Inclusion Criteria

* Must be ≥ 18 years of age
* Subjects must have documented diagnosis of multiple myeloma and have measurable disease
* Subjects must have undergone prior treatment with ≥ 2 treatment lines of anti-myeloma therapy
* Subjects must have either refractory or relapsed and refractory disease defined as documented disease progression during or within 60 days of completing their last myeloma therapy
* All subjects must have received at least 2 consecutive cycles of prior treatment that included lenalidomide and bortezomib
* All subjects must have failed treatment with both lenalidomide and bortezomib in one of the following ways: 1) Documented progressive disease on or within 60 days of completing treatment with lenalidomide and/or bortezomib, or 2) In case of prior response \[≥ partial response (PR)\] to lenalidomide or bortezomib, subjects must have relapsed within 6 months after stopping treatment with lenalidomide and/or bortezomib-containing regimens, or 3) Subjects who have not had a ≥ minimal response (MR) and have developed intolerance/toxicity after a minimum of two cycles of lenalidomide- and/or bortezomib-containing regimen
* Patients must have received adequate prior alkylator therapy
* Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2
* Females of childbearing potential (FCBP) must not become pregnant for 28 days prior to initiation of study drug, during the study, and for 28 days after discontinuation
* Females must agree to abstain from breastfeeding during study participation and 28 days after study drug discontinuation
* Males must agree to use a latex condom during any sexual during the study and for 28 days following discontinuation from this study
* Males must also agree to refrain from donating semen or sperm while on pomalidomide and for 28 days after discontinuation from this study

Exclusion Criteria

* Any of the following laboratory abnormalities:

* Absolute neutrophil count (ANC) \< 1,000/μL
* Platelet count \< 75,000/ μL for subjects in whom \< 50% of bone marrow nucleated cells are plasma cells
* Creatinine clearance \< 45 mL/min
* Corrected serum calcium \> 14 mg/dL
* Hemoglobin ≤ 8 g/dL
* Serum glutamic oxaloacetic transaminase (SGOT)/ aspartate aminotransferase (AST) or transaminase, serum glutamic pyruvic (SGPT)/ alanine aminotransferase (ALT) \> 3.0 x upper limit of normal (ULN)
* Serum total bilirubin \> 2.0 mg/dL
* Previous therapy with pomalidomide
* Hypersensitivity to thalidomide, lenalidomide, or dexamethasone
* Resistance to high-dose dexamethasone used in the last line of therapy
* Peripheral neuropathy ≥ Grade 2
* Subjects who received an allogeneic bone marrow or allogeneic peripheral blood stem cell transplant
* Subjects who are planning for or who are eligible for stem cell transplant
* Subjects with any one of the following: 1) Congestive heart failure, 2) Myocardial infarction within 12 months prior to starting study treatment, 3) Unstable or poorly controlled angina pectoris, including Prinzmetal variant angina pectoris
* Subjects who received any of the following within the last 14 days of initiation of study treatment: 1) Plasmapheresis, 2) Major surgery, 3) Radiation therapy, 4) Use of any anti-myeloma drug therapy
* Use of any investigational agents within 28 days or 5 half-lives (whichever is longer) of treatment
* Subjects with conditions requiring chronic steroid or immunosuppressive treatment
* Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study
* Incidence of gastrointestinal disease that may significantly alter the absorption of pomalidomide
* Subjects unable or unwilling to undergo antithrombotic prophylactic treatment
* Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subjects from signing the informed consent form
* Pregnant or breastfeeding females
* Known human immunodeficiency virus (HIV) positivity or active infectious hepatitis A, B, or C
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Celgene

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Lars Sternas, MD, PhD

Role: STUDY_DIRECTOR

Celgene

Locations

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Mayo Clinic

Rochester, Minnesota, United States

Site Status

Royal Adelaide Hospital

Adelaide, South Australia, Australia

Site Status

Peter MacCallum Cancer Institute

East Melbourne, Victoria, Australia

Site Status

Frankston Hospital

Frankston, Victoria, Australia

Site Status

Princess Alexandra Hospital

Brisbane, , Australia

Site Status

Royal Prince Alfred Hospital

Camperdown, , Australia

Site Status

Alfred hospital

Melbourne, , Australia

Site Status

Sir Charles Gairdner Hospital

Nedlands, , Australia

Site Status

Calvary Mater Hospital

Waratah, , Australia

Site Status

Border Medical Oncology

Wodonga, , Australia

Site Status

Wollongong Hospital

Wollongong, , Australia

Site Status

UZ Gent

Ghent, , Belgium

Site Status

UZ Leuven

Leuven, , Belgium

Site Status

CHU UCL Mont-Godinne-Dinant asbl

Yvoir, , Belgium

Site Status

Tom Baker Cancer Center

Calgary, Alberta, Canada

Site Status

Cross Cancer Institute

Edmonton, Alberta, Canada

Site Status

British Columbia Cancer Agency

Vancouver, British Columbia, Canada

Site Status

James Cancer Hospital

Halifax, Nova Scotia, Canada

Site Status

Juravinski Cancer Centre

Hamilton, Ontario, Canada

Site Status

London Health Sciences Centre

London, Ontario, Canada

Site Status

University Health Network

Toronto, Ontario, Canada

Site Status

Maisonneuve Rosemont

Montreal, Quebec, Canada

Site Status

Royal Victoria Hospital McGill Department of Oncology(RVH)

Montreal, Quebec, Canada

Site Status

Sir Mortimer B. Davis - Jewish Genl

Montreal, Quebec, Canada

Site Status

Charles University General Hospital

Prague, , Czechia

Site Status

Hæmatologisk afd. B Aalborg Sygehus Syd

Aalborg, , Denmark

Site Status

Aarhus University Hospital

Arhus C, , Denmark

Site Status

Odense Universitetshospital

Odense C, , Denmark

Site Status

Vejle Hospital

Vejle, , Denmark

Site Status

CHU d'Angers

Angers, , France

Site Status

Centre Hospitalier de la cote basque

Bayonne, , France

Site Status

Centre Hospitalier Departemental

La Roche-sur-Yon, , France

Site Status

CHRU de Lille FR

Lille, , France

Site Status

Institut Paoli-Calmettes

Marseille, , France

Site Status

CHRU Nantes

Nantes, , France

Site Status

Hopital Saint-Louis

Paris, , France

Site Status

CHU Hôpital St-Antoine

Paris, , France

Site Status

CHRU - Hopital du Haut Leveque

Pessac, , France

Site Status

Centre Hospitalier Lyon Sud

Pierre-Bénite, , France

Site Status

Hopital Bretonneau

Tours, , France

Site Status

Hopital Purpan

Tulouse Cedex 9, , France

Site Status

CHU Nancy

Vandœuvre-lès-Nancy, , France

Site Status

Universitatsklinikum Carl Gustav Carus

Dresden, , Germany

Site Status

Universitatsklinikum Essen

Essen, , Germany

Site Status

Askepios Klinik St. Georg

Hamburg, , Germany

Site Status

Universitatsklinikum Heidelberg

Heidelberg, , Germany

Site Status

Universitatsklinikum Jena

Jena, , Germany

Site Status

Universitatsklinikum Leipzig

Leipzig, , Germany

Site Status

University of Tubingen

Tübingen, , Germany

Site Status

Universitatsklinikum Ulm

Ulm, , Germany

Site Status

Universitatsklinikum Wurzburg

Würzburg, , Germany

Site Status

Alexandra General Hospital of Athens

Athens, , Greece

Site Status

Azienda Ospedaliero-Universitaria di Bologna - Policlinico S.Orsola-Malpighi

Bologna, , Italy

Site Status

Clinica Ematologica- A.O.U. San Martino

Genova, , Italy

Site Status

Oncoematologia, Istituto Nazionale Tumori Fondazione G. Pascale

Napoli, , Italy

Site Status

AOU San Luigi Gonzaga

Orbassano, , Italy

Site Status

Universita degli Studi di Padova

Padua, , Italy

Site Status

Hospital Clinic

Placenza, , Italy

Site Status

Servizio di Ematologia, A.O. - Arcispedale S.Maria Nuova

Reggio Emilia, , Italy

Site Status

Univerita La Sapienza Dipartimento di Biotecnologie Cellulari ed Ematologia

Rome, , Italy

Site Status

Azienda Ospedaliera San Giovanni Battista

Torino, , Italy

Site Status

VU University Medical Center

Amsterdam, , Netherlands

Site Status

Erasmus Medical Center

Rotterdam, , Netherlands

Site Status

University Medical Center Utrecht

Utrecht, , Netherlands

Site Status

State Institution Hematological Research, Centre of Russian Academy of Medical Science

Moscow, , Russia

Site Status

State Institution Moscow Regional Research Clinical Institute

Moscow, , Russia

Site Status

St. Petersburg Research Institute of Hematology and Blood Transfusion

Saint Petersburg, , Russia

Site Status

State Educational Institution, Saint Petersburg State Medical University

Saint Petersburg, , Russia

Site Status

Hospital Universitari Germans Trias i Pujol

Badalona (Barcelona), , Spain

Site Status

Hospital Clinic Provincial de Barcelona

Barcelona, , Spain

Site Status

Hospital de La Princesa

Madrid, , Spain

Site Status

Hospital 12 de Octubre

Madrid, , Spain

Site Status

Hospital Universitario de Salamanca

Salamanca, , Spain

Site Status

Hospital Donostia

San Sebastián (Guipuzcoa), , Spain

Site Status

Hospital Universitario Marques de Valdecilla

Santander, , Spain

Site Status

Hospital de la Fe

Valencia, , Spain

Site Status

Department of Hematology Hematology Centre

Gothenburg, , Sweden

Site Status

University Hospital in Lund

Lund, , Sweden

Site Status

Karolinska University Hospital Huddinge

Stockholm, , Sweden

Site Status

Karolinska University Hospital

Stockholm, , Sweden

Site Status

Overlakare Medocomcentrum, hematologi

Uppsala, , Sweden

Site Status

Medizinische Universitatsklinik

Bern, , Switzerland

Site Status

Hopitaux Universitaires de Geneve-HUG

Geneva, , Switzerland

Site Status

UniversitatSspital Zurich

Zurich, , Switzerland

Site Status

Royal Bournemouth Hospital

Bournemouth, , United Kingdom

Site Status

St James's University Hospital

Leeds, , United Kingdom

Site Status

St.Bartholomew's Hospital

London, , United Kingdom

Site Status

King's College Hospital

London, , United Kingdom

Site Status

Freeman Hospital

Newcastle upon Tyne, , United Kingdom

Site Status

Nottingham City Hospital

Nottingham, , United Kingdom

Site Status

Derriford Hospital

Plymouth, , United Kingdom

Site Status

Royal Hallamshire HospitalSheffield Teaching Hospitals NHS Trust

Sheffield, , United Kingdom

Site Status

The Royal Marsden Hospital

Sutton-Surrey, , United Kingdom

Site Status

The Royal Wolverhampton Hospital NHS Trust

Wolverhampton, , United Kingdom

Site Status

Countries

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United States Australia Belgium Canada Czechia Denmark France Germany Greece Italy Netherlands Russia Spain Sweden Switzerland United Kingdom

References

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Moreau P, Weisel KC, Song KW, Gibson CJ, Saunders O, Sternas LA, Hong K, Zaki MH, Dimopoulos MA. Relationship of response and survival in patients with relapsed and refractory multiple myeloma treated with pomalidomide plus low-dose dexamethasone in the MM-003 trial randomized phase III trial (NIMBUS). Leuk Lymphoma. 2016 Dec;57(12):2839-2847. doi: 10.1080/10428194.2016.1180685. Epub 2016 May 13.

Reference Type RESULT
PMID: 27173785 (View on PubMed)

Miguel JS, Weisel K, Moreau P, Lacy M, Song K, Delforge M, Karlin L, Goldschmidt H, Banos A, Oriol A, Alegre A, Chen C, Cavo M, Garderet L, Ivanova V, Martinez-Lopez J, Belch A, Palumbo A, Schey S, Sonneveld P, Yu X, Sternas L, Jacques C, Zaki M, Dimopoulos M. Pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone alone for patients with relapsed and refractory multiple myeloma (MM-003): a randomised, open-label, phase 3 trial. Lancet Oncol. 2013 Oct;14(11):1055-1066. doi: 10.1016/S1470-2045(13)70380-2. Epub 2013 Sep 3.

Reference Type RESULT
PMID: 24007748 (View on PubMed)

Siegel DS, Weisel KC, Dimopoulos MA, Baz R, Richardson P, Delforge M, Song KW, San Miguel JF, Moreau P, Goldschmidt H, Cavo M, Jagannath S, Yu X, Hong K, Sternas L, Zaki M, Palumbo A. Pomalidomide plus low-dose dexamethasone in patients with relapsed/refractory multiple myeloma and moderate renal impairment: a pooled analysis of three clinical trials. Leuk Lymphoma. 2016 Dec;57(12):2833-2838. doi: 10.1080/10428194.2016.1177181. Epub 2016 Jun 7.

Reference Type DERIVED
PMID: 27267105 (View on PubMed)

Weisel KC, Dimopoulos MA, Moreau P, Lacy MQ, Song KW, Delforge M, Karlin L, Goldschmidt H, Banos A, Oriol A, Alegre A, Chen C, Cavo M, Garderet L, Ivanova V, Martinez-Lopez J, Knop S, Yu X, Hong K, Sternas L, Jacques C, Zaki MH, San Miguel J. Analysis of renal impairment in MM-003, a phase III study of pomalidomide + low - dose dexamethasone versus high - dose dexamethasone in refractory or relapsed and refractory multiple myeloma. Haematologica. 2016 Jul;101(7):872-8. doi: 10.3324/haematol.2015.137083. Epub 2016 Apr 14.

Reference Type DERIVED
PMID: 27081177 (View on PubMed)

Dimopoulos MA, Weisel KC, Song KW, Delforge M, Karlin L, Goldschmidt H, Moreau P, Banos A, Oriol A, Garderet L, Cavo M, Ivanova V, Alegre A, Martinez-Lopez J, Chen C, Spencer A, Knop S, Bahlis NJ, Renner C, Yu X, Hong K, Sternas L, Jacques C, Zaki MH, San Miguel JF. Cytogenetics and long-term survival of patients with refractory or relapsed and refractory multiple myeloma treated with pomalidomide and low-dose dexamethasone. Haematologica. 2015 Oct;100(10):1327-33. doi: 10.3324/haematol.2014.117077. Epub 2015 Aug 6.

Reference Type DERIVED
PMID: 26250580 (View on PubMed)

San Miguel JF, Weisel KC, Song KW, Delforge M, Karlin L, Goldschmidt H, Moreau P, Banos A, Oriol A, Garderet L, Cavo M, Ivanova V, Alegre A, Martinez-Lopez J, Chen C, Renner C, Bahlis NJ, Yu X, Teasdale T, Sternas L, Jacques C, Zaki MH, Dimopoulos MA. Impact of prior treatment and depth of response on survival in MM-003, a randomized phase 3 study comparing pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone in relapsed/refractory multiple myeloma. Haematologica. 2015 Oct;100(10):1334-9. doi: 10.3324/haematol.2015.125864. Epub 2015 Jul 9.

Reference Type DERIVED
PMID: 26160879 (View on PubMed)

Weisel K, Dimopoulos M, Song KW, Moreau P, Palumbo A, Belch A, Schey S, Sonneveld P, Sternas L, Yu X, Amatya R, Gibson CJ, Zaki M, Jacques C, San Miguel J. Pomalidomide and Low-Dose Dexamethasone Improves Health-Related Quality of Life and Prolongs Time to Worsening in Relapsed/Refractory Patients With Multiple Myeloma Enrolled in the MM-003 Randomized Phase III Trial. Clin Lymphoma Myeloma Leuk. 2015 Sep;15(9):519-30. doi: 10.1016/j.clml.2015.05.007. Epub 2015 Jun 6.

Reference Type DERIVED
PMID: 26149712 (View on PubMed)

Song KW, Dimopoulos MA, Weisel KC, Moreau P, Palumbo A, Belch A, Schey S, Sonneveld P, Sternas L, Yu X, Amatya R, Monzini MS, Zaki M, Jacques C, San Miguel J. Health-related quality of life from the MM-003 trial of pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone in relapsed and/or refractory multiple myeloma. Haematologica. 2015 Feb;100(2):e63-7. doi: 10.3324/haematol.2014.112557. Epub 2014 Nov 25. No abstract available.

Reference Type DERIVED
PMID: 25425684 (View on PubMed)

Other Identifiers

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2010-019820-30

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CC-4047-MM-003

Identifier Type: -

Identifier Source: org_study_id

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