AzTrials
AzTrials

AnemiaPhase 3MDS-005NCT01029262

A Study of Lenalidomide Versus Placebo in Subjects With Transfusion Dependent Anemia in Lower Risk Myelodysplastic Syndrome (MDS) Without Del 5q

A Phase 3 study testing Lenalidomide in people with Anemia, run by Celgene at 99 sites in 16 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to investigate whether lenalidomide would reduce the number of red blood cell transfusions (RBC) needed in anemic (RBC transfusion-dependent) participants with low or intermediate-1 risk MDS without a deletion 5q chromosome abnormality. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Anemia
What is being tested?
Lenalidomide
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
239 participants
Where
99 sitesin 16 countries
Study dates
26 Jan 2010to 9 May 2018
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 18 years or older
  • Diagnosis of low or intermediate-1 risk Myelodysplastic (MDS) with any chromosome karyotype except del 5q[31]
  • Anemia that requires red blood cell transfusions
  • Resistant to erythropoiesis stimulating agents (ESAs) or blood erythropoietin level > 500 mU/mL
  • Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) ≤ 2
  • Must agree to follow pregnancy precautions as required by the protocol.
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  • Must agree to receive counseling related to teratogenic and other risks of lenalidomide
  • Must agree not to donate blood or semen
  • Must be willing to consent to two or more bone marrow aspirate procedures to be completed during study

Who cannot join

  • Subjects previously receiving immunomodulating or immunosuppressive agents, or epigenetic or deoxyribonucleic acid (DNA) modulation agents
  • Allergic reaction to thalidomide
  • Renal insufficiency creatinine clearance (CrC1)<40 mL/min by Cockcroft-Gault method)
  • Prior history of cancer, other than MDS, unless the subject has been free of the disease for ≥ 5 years. (Basal cell carcinoma of the skin, carcinoma in situ of the cervix, or stage Tumor (T) 1a or T1b prostate cancer is allowed)
  • Absolute neutrophil count (ANC) < 500/uL
  • Platelets < 50,000/uL
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  • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3X upper limit of normal
  • Uncontrolled hyperthyroidism or hypothyroidism
  • Significant neuropathy
  • Prior stem cell transplantation
  • Anemia due to reasons other than MDS
  • History of deep venous thrombosis (DVT) or pulmonary embolus (PE) within past 3 years
  • Significant active cardiac disease within the past 6 months
  • Known Human Immunodeficiency Virus (HIV) infection; known Hepatitis C infection or active Hepatitis B infection

Trial locations and contacts

99 sites in 16 countries.

99 sites in 16 countries

Italy12 sites

  • Az. Osp. SS.Antonio e Biagio - SC Ematologia

    Alessandria, 15100

  • A.O.U. di Bologna Policlinico S.Orsola-Malpighi

    Bologna, 40138

  • P.O. Ospedale Roberto Binaghi (UNI CA/ASL 8)

    Cagliari, 09100

  • Azienda Ospedaliero-Universitaria di Cagliari

    Cagliari, 09121

  • Azienda Ospedaliero-Universitaria Careggi

    Florence, 50139

  • Azienda Ospedaliera Cardarelli

    Naples, 80131

  • AOU San Luigi Gonzaga

    Orbassano, 10043

  • IRCCS Centro di Riferimento Oncologico di Basilicata di Rionero in Vulture

    Rionero in Vulture

  • Azienda Ospedaliera Universitaria Policlinico Tor Vergata

    Roma, 00133

  • Azienda Policlinico Umberto I, Universita La Sapienzadi Roma

    Roma, 00161

  • Policlinico Agostino Gemelli - Istituto di Ematologia

    Roma, 00168

  • Policlinico Univeristario di Udine

    Udine, 33100

Other trials in Italy

Japan11 sites

  • Hiroshima University Hospital

    Hiroshima

  • Tokai University School of Medicine

    Isehara City, Kanagawa, 259-1193

  • Kameda General Hospital

    Kamogawa, 296-8602

  • Kanazawa University Hospital

    Kanazawa

  • Nagasaki Unversity Hospital

    Nagasaki

  • National Hospital Organization Nagoya Medical Center

    Nagoya, 460-0001

  • Osaka Red Cross Hospital

    Osaka

  • Tohoku University Hospital

    Sendai, 980-8574

  • Japanese Red Cross Medical Center

    Shibuya City, 150-8935

  • Jichi Medical University Hospital

    Shimotsuke

  • Kanto Medical Center NTT EC

    Shinagawa City

Other trials in Japan

Germany9 sites

  • Universitat zu Koln

    Cologne, 50924

  • BAG Freiberg-Richter, Jacobash, Illmer, Wolf

    Dresden, 1307

  • Universitätsklinikum Düsseldorf

    Düesseldorf, 40211

  • Sankt Johannes Hospital Duisburg

    Duisberg, 47166

  • Marien Hospital

    Düsseldorf, 40479

  • Medizinische Hochschule Hannover

    Hanover, 30625

  • Klinikum Mannheim der Universitat Heidelberg

    Heidelberg, 69120

  • Universitatsklinikum Mannheim

    Mannheim, 68135

  • TU München - Klinikum rechts der Isar

    München, 81675

Other trials in Germany

Canada8 sites

  • Tom Baker Cancer Center

    Calgary, Alberta, T2N 4N2

  • University of Alberta Hospital

    Edmonton, Alberta, T6G 2B7

  • Maisonneuve Rosemont

    Montreal, Quebec, H1T 2M4

  • McGill University, Dept. Oncology Clinical Research Program

    Montreal, Quebec, H2W 1S6

  • Hopital du Sacre-Coeur de Montreal

    Montreal, Quebec, H4J 1C5

  • Sunnybrook Regional Cancer Center

    Toronto, Ontario, M4N 3M5

  • Princess Margaret Hospital and University of Toronto

    Toronto, Ontario, M5G 2M9

  • Cancer Care Manitoba

    Winnepeg, Manitoba, R3E 0V9

Other trials in Canada

United States7 sites

  • Southern Illinois Hematology Oncology

    Centralia, Illinois, 62801

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

  • Dartmouth Hitchcock Medical Center Norris Cotton Cancer Center

    Lebanon, New Hampshire, 03756

  • University of California at Los Angeles

    Los Angeles, California, 90095

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Columbia University Medical Center

    New York, New York, 10032

Other trials in United States

Belgium7 sites

  • AZ St-Jan Brugge Oostende AV

    Bruges, 8000

  • Cliniques Universitaires Saint-Luc

    Brussels, 1200

  • Grand Hopital de Charleroi

    Charleroi, 6000

  • Universitair Ziekenhuis Antwerpen

    Edegem, 2650

  • Centre Hospitalier Universitaire de Liege

    Liège, 4000

  • Center Hospitalier Universitaire Ambroise Pare

    Mons, 7000

  • Cliniques Universitaires UCL de Mont-Godine

    Namur, 5530

Other trials in Belgium

Spain7 sites

  • Hospital Clinic Provincial de Barcelona

    Barcelona, 08036

  • Hospital Universitario La Paz

    Madrid, 28046

  • Hospital Universitario Virgen de la Victoria

    Málaga, 29010

  • Hospital Son Llatzer

    Palma de Mallorca, 7198

  • Hospital Universitario de Salamanca

    Salamanca, 37007

  • Hospital Universitario Virgen Del Rocio

    Seville, 41013

  • Hospital Universitario La Fe

    Valencia, 46009

Other trials in Spain

United Kingdom7 sites

  • Royal Bournemouth Hospital

    Bournemouth, BH7 7DW

  • University Hospital of Wales

    Cardiff, CF14 4XN

  • Saint James University Hospital

    Leeds, LS9 7TF

  • Barts Cancer Institute, Queen Mary University of London, Charterhouse Square

    London, EC1A 7BE

  • King's College Hospital

    London, SE5 9RS

  • Christie Hospital

    Manchester, M20 4BX

  • John Radcliffe Hospital

    Oxford, OX3 9DU

Other trials in United Kingdom

France6 sites

  • CHU d'Angers

    Angers, 49033

  • Hopital Avicenne

    Bobigny, 93009

  • Hopital A. Michallon

    La Tronche, 38700

  • CHRU de Lille-Hopital Claude Huriez Service des Maladies du Sang

    Lille, 59037

  • Institut Paoli-Calmettes

    Marseille, 13273

  • Groupe hospitalier Cochin Saint-Vincent de Paul

    Paris, 75679

Other trials in France

Austria5 sites

  • Medizinische Universitat Innsbruck

    Innsbruck, 6020

  • Krankenhaus der Elisabethinen Linz, I Interne Abteilung

    Linz, 4020

  • Universitatsklinik fur Innere Medizin Salzburg

    Salzburg, 5020

  • Wiener Gebietskrankenkasse-Hanusch-Krankenhaus

    Vienna, 1140

  • Klinikum Wels-Grieskirchen GmbH

    Weis, 4600

Other trials in Austria

Czechia4 sites

  • Fakultni nemocnice Brno

    Brno, 625 00

  • Fakultni nemocnice Olomouc

    Olomouc, 77520

  • Ustav hematologie a krevni transfuze

    Prague, 128 20

  • Vseobecna Fakultni Nemocnice v Praze

    Prague, 12808

Other trials in Czechia

Turkey (Türkiye)4 sites

  • Gazi Universitesi Tip Fakltesi

    Ankara, 06500

  • Akdeniz Universitesi Tip Fakultesi

    Antalya, 07503

  • Istanbul Universitesi Istanbul

    Istanbul, 34390

  • Dokuz Eylul Universitesi Tip Faiultesi

    Izimir, 35340

Other trials in Turkey (Türkiye)

Australia3 sites

  • Royal Adelaide Hospital Institute of Medical and Veterinary Science

    Adelaide, South Australia, 5000 SA

  • Wollongong Hospital

    Wollongong, New South Wales, 2500

  • Princess Alexandra Hospital

    Woolloongabba, 4102

Other trials in Australia

Israel3 sites

  • Rabin Medical Center

    Petah Tikva, 49100

  • Tel-Aviv Sourasky Medical Center

    Tel Aviv, 64239

  • The Chaim Sheba Medical Center

    Tel HaShomer, 52621

Other trials in Israel

Poland3 sites

  • Katedra i Klinika Hematologii i Transplantacji Szpiku - SLASKIEGO UNIWERSYTETU MEDYCZNEGO

    Gdansk, 80-119

  • Uniwersytet Medyczny w Lodzi

    Lodz, 93-510

  • Instytut Hematologii i Transfuzjologii, Klinika Hematologii

    Warsaw, 02-776

Other trials in Poland

Portugal3 sites

  • Hospitais da Universidade de Coimbra

    Coimbra, 3000-075

  • Instituto Portugues de Oncologia de Lisboa

    Lisbon, 1090-023

  • Hospital Geral de Santo Antonio

    Porto, 4099-001

Other trials in Portugal

About this study

Official title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study To Compare The Efficacy And Safety of Lenalidomide (Revlimid®) Versus Placebo In Subjects With Transufsion-Dependent Anemia Due to IPSS Low Or Imtermidate-1 Risk Myelodysplastic Syndromes Without Deletion 5Q(31) And Unresponsive Or Refractory To Erthropoiesis-Stimulating Agents

The purpose of this study is to investigate whether lenalidomide would reduce the number of red blood cell transfusions (RBC) needed in anemic (RBC transfusion-dependent) participants with low or intermediate-1 risk MDS without a deletion 5q chromosome abnormality. The study also investigated the safety of lenalidomide use in these participants. Two-thirds of the participants received oral lenalidomide and one-third of the participants received oral placebo.

Conditions
Anemia
Being tested
Lenalidomide, Placebo
Keywords
Myelodysplastic Syndromes, MDS, transfusion dependent anemia, Erythropoiesis stimulating agents, non-del 5q
Medical subject headings
Anemia, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LenalidomideDrug

    Also called Revlimid, CC-5013

    One 10 mg Lenalidomide capsule + 2 placebo capsules or (3 placebo capsules) once daily for subjects with a creatinine clearance ≥ 60 mL/min. Alternatively-one 5 mg Lenalidomide capsule + 2 placebo capsules (or 3 placebo capsules) once daily for subjects with a creatinine clearance between 40 and 60 mL/min. Subjects may take study drug for at least 168 days unless there are intolerable side effects or disease progresses. Subjects may continue study drug beyond 168 days if they have an erythroid response (increase in their hemoglobin levels and fewer transfusions administered than before starting study drug)

  • PlaceboOther

    3 placebo capsules once daily. Subjects may take study drug for at least 168 days unless there are intolerable side effects or disease progresses. Subjects may continue study drug beyond 168 days if they have an erythroid response (increase in their hemoglobin levels and fewer transfusions administered than before starting study drug)

Study groups in NCT01029262
Study groupRoleWhat they receive
Arm #1 - Lenalidomide plus placeboExperimentalLenalidomide 10 mg by mouth (PO) daily plus 2 placebo capsules for participants with a creatinine clearance ≥ 60 mL/min for at least 168 days until disease progression, intolerable side effects or withdrawal of consent. Lenalidomide 5 mg PO daily plus 2 placebo capsules for participants with a creatinine clearance ≥ 40 and < 60 mL/min.
Receives: Drug: Lenalidomide
Arm #2 - placeboPlacebo comparatorThree placebo capsules once daily for at least 168 days until disease progression occurred, intolerable side effects or withdrawal of consent.
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for ≥ 56 Days as Determined by an Independent Review Committee (IRC)

    The percentage of participants who achieved the 56-day RBC transfusion independent (TI) response was defined as the absence of any RBC transfusions during any consecutive "rolling" 56-day interval within the double-blind treatment phase (ie, Days 2 (Day 1 is the first study drug day) to 57, Days 3 to 58, etcetera). The double-blind treatment phase was defined as the period between the 1st dosing up until 28 days after the last study drug dose

    Measured: From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively.

  2. Percentage of Participants With a Erythroid Gene Signature Who Achieved RBC Transfusion Independence for ≥ 56 Days as Determined by an Independent Review Committee (IRC)

    The percentage of participants who achieved the 56-day RBC TI response was defined as the absence of any RBC transfusions during any consecutive "rolling" 56-day interval within the double-blind treatment phase (ie, Days 2 (Day 1 is the first study drug day) to 57, Days 3 to 58, etcetera). A participant who achieved at least a 56-day RBC-transfusion-independent response was considered a 56-day RBC-TI responder.

    Measured: From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively.

Secondary outcomes (26)

  1. Percentage of Participants Who Achieved RBC Transfusion Independence With a Duration of ≥ 24 Weeks (168 Days) as Determined by the Sponsor

    The 168-day RBC-transfusion-independent response was defined as the absence of any RBC transfusion during any consecutive "rolling" 168 days during the treatment period, for example Days 2 (Day 1 is the first study drug day) to 169, Days 3 to 170, Days 4 to 171, etcetera. A responder was defined as a participant who had a ≥ 168 consecutive days of RBC-transfusion-free period after the first dose of study drug in the treatment phase.

    Measured: From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively.

  2. Kaplan Meier Estimates of Duration of 56-day RBC Transfusion Independence Response as Determined by the Sponsor

    The duration of the first 56-day RBC transfusion-independence response was calculated for those who achieved a response and was dependent on whether a subsequent RBC transfusion was given after the transfusion-free period (response):

    • For those who received a subsequent RBC transfusion after the response starts, the duration of response was not censored, and was calculated as response duration = last day of response - first day of response +1 where the last day of response was defined as 1 day before the first RBC transfusion which was given at 56 days or more after the response starts.
    • For those who did not receive a subsequent RBC transfusion after the response started, the end day of the response was censored and duration of the response was calculated as response duration = date of last RBC transfusion assessment - first day of response+ 1. A responder was a participant who had a ≥ 56 consecutive days of RBC-transfusion-free period after the first study drug treatment period

    Measured: Response was assessed up to the end of treatment; up to the data cut-off date of 17 Mar 2014.

  3. Percentage of Participants Who Achieved an Erythroid Response Based on the Modified International Working Group (IWG) 2006 Criteria

    A participant was considered as having achieved an erythroid response if the participant either:

    • had a hemoglobin (Hgb) increase ≥1.5 g/dL compared to baseline and confirmed by another central laboratory hemoglobin value at 4 to 8 weeks after the first Hgb measurement that also increased ≥1.5 g/dL. All Hgb values during this time interval must have had a ≥ 1.5 g/dL increase (ie, no central laboratory Hgb increase during this timeframe could be less <1.5 g/dL) OR - had a 50% reduction in the number of the RBC transfusion units over any consecutive 56 days period compared to the baseline transfusion burden.

    The baseline transfusion burden is the number of units over 112 days by the randomization divided by 2. Only transfusions given for a pre-transfusion Hgb value of 9 g/dL or less were used in this response assessment.

    Measured: From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively.

  4. Time to 56-Day RBC-Transfusion-Independent (TI) Response as Determined by the Sponsor

    The time to the first 56-day RBC-transfusion-independent response was calculated for participants who achieved a response. The day from the first dose of study drug to the date at which RBC-transfusion-independence starts was achieved and calculated using: Start date of the first response period - the date of the first study drug +1. A responder was defined as a participant who had a ≥ 56 consecutive days of RBC-transfusion-free period after the first dose of study drug in the treatment phase.

    Measured: From first dose of study drug until 28 days after the last dose of study drug, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively.

  5. Kaplan Meier Estimates for Progression to Acute Myeloid Leukemia (AML)

    Progression to AML is part of the natural course of MDS and is a manifestation of disease progression. The time to progress to AML was calculated from the day of randomization to the first day when AML was diagnosed. Participants who died without AML were censored at the date of death. The participants who were lost to follow-up were censored at the last known day when participants did not have AML. Participants who did not progress to AML at the last follow-up contact were censored at the day of the last follow-up contact.

    Measured: From randomization to final data cut-off date of 03 Jul 2018; median follow up time for progression to AML was 2.3 years (range = 0 to 5.0 years) in the placebo arm and 2.6 years (range = 0 to 6.4 years) in the lenalidomide arm.

  6. Kaplan Meier Estimate for Overall Survival (OS)

    Overall survival was assessed using the time between randomization and the date of death or date of censoring. Participants who were alive at a data cutoff date and participants who were lost to follow-up were censored at the last date when participants were known to be alive.

    Measured: From randomization to final data cut-off date of 03 July 2018; maximum survival follow up was 6.4 years

  7. Number of Participants With Treatment Emergent Adverse Events (TEAE)

    A TEAE was defined as an AE that begins or worsens in intensity of frequency on or after the first dose of study drug through 28 days after last dose of study drug.

    A serious adverse event (SAE) is any:

    • Death;
    • Life-threatening event;
    • Any inpatient hospitalization or prolongation of existing hospitalization;
    • Persistent or significant disability or incapacity;
    • Congenital anomaly or birth defect;
    • Any other important medical event

    The investigator determined the relationship of an AE to study drug based on the timing of the AE relative to drug administration and whether or not other drugs, therapeutic interventions, or underlying conditions could provide a sufficient explanation for the event. The severity of an AE was evaluated by the investigator according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) (Version 3.0) where Grade 1 = Mild, Grade 2 = Moderate, Grade 3 = Severe, Grade 4 = Life-threatening and Grade 5 = Death.

    Measured: From the first dose of study drug through 28 days after discontinuation from the study treatment; up to the final data cut-off date of 03 July 2018; maximum exposure was 2100 days in the lenalidomide arm and 529 days in the placebo arm.

  8. Compliance Rates Using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) From Baseline to Week 48

    The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). Subscale scores are transformed to a 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. A participant was considered compliant at a visit if at least 15 out of the QLQ-C30 items in the questionnaire were checked.

    Measured: Baseline, Week 12, (±3 days), Week 24, (±3 days), Week 36, (±3 days), and Week 48 (±3 days); up to data cut-off of 17 Mar 2014

  9. Mean Change From Baseline in the EORTC QLQ-C30 Fatigue Domain at Week 12 and 24

    The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).

    Measured: Baseline and Week 12, ±3 days and Week 24, ±3 days

  10. Mean Change From Baseline in the EORTC QLQ-C30 Dyspnea Domain at Week 12 and 24

    The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).

    Measured: Baseline and Week 12, ±3 days and Week 24, ±3 days

  11. Mean Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain at Week 12 and 24

    The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Physical Functioning Scale was scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.

    Measured: Baseline and Week 12, ±3 days and Week 24, ±3 days

  12. Mean Change From Baseline in the EORTC QLQ-C30 Global Health Status/Quality of Life (QOL) Domain at Week 12 and 24

    The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale was scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.

    Measured: Baseline and Week 12, ±3 days and Week 24, ±3 days

  13. Mean Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain at Week 12 and 24

    The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Domain was scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.

    Measured: Baseline and Week 12, ±3 days and Week 24, ±3 days

  14. Mean Change From Baseline in Fatigue Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24

    The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  15. Mean Change From Baseline in the Dyspnea Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24

    The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  16. Mean Change From Baseline in the Physical Functioning Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24

    The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). The EORTC QLQ-C30 Physical Functioning was scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  17. Mean Change From Baseline in the Global Health Status/QoL Domain Associated With the EORTC QLQ-C-30 Scale at Week 12 and Week 24

    The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). The EORTC QLQ-C30 Global Health Status/QOL scale was scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  18. Mean Change From Baseline in the Emotional Functioning Domain Associated With the EORTC QLQ-C30 Scale at Weeks 12 and 24

    The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Scale is scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  19. Percentage of Participants With a Clinically Meaningful Improvement in QOL (EORTC QLQ-C-30 Scale) From Baseline in Fatigue Domain at Weeks 12 and 24

    The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). Subscale scores are transformed to a 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. Improvement means at least 10 points better compared to baseline

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  20. Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline in the Dyspnea Domain at Weeks 12 and 24

    The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom). Improvement means at least 10 points better compared to baseline.

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  21. Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline Within the Physical Functioning Domain at Weeks 12 and 24

    The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). Subscale scores are transformed to a 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. A change of at least 10 points on the standardized domain scores was required for it to be considered clinically meaningful.

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  22. Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline in the Global Health Status/QOL Domain at Weeks 12 and 24

    The European Organization for Research and Treatment of Cancer QOL Questionnaire for Patients with Cancer (EORTC QLQ-C30) was a 30-item oncology-specific questionnaire. The questionnaire was developed to assess the quality of life of cancer patients. It contains 30 questions, 24 of which form 9 multi-item scales representing various aspects of HRQOL: 1 global scale, 5 functional scales (Physical, Role, Emotional, Cognitive and Social), and 3 symptom scales (Fatigue, Pain, and Nausea). The remaining 6 items are intended to be mono-item scales describing relevant cancer-oriented symptoms (dyspnea, insomnia, appetite, constipation, diarrhea, financial difficulties). Subscale scores are transformed to a 0 to 100 scale, with higher scores on functional scales indicating better function and higher score on symptom scales indicating worse symptoms. A change of at least 10 points on the standardized domain scores was required for it to be considered clinically meaningful.

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  23. Percentage of Participants With a Clinically Meaningful Improvement in HRQOL Associated With the EORTC QLQ-C-30 Scale From Baseline in the Emotional Functioning Domain at Weeks 12 and 24

    The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Domain was scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.

    Measured: Baseline, Week 12, ±3 days and Week 24, ±3 days

  24. Healthcare Resource Utilization (HRU): Rate of Inpatient Hospitalizations Related to Adverse Events Per Person Year

    Hospitalizations due to adverse events exclude those for transfusions, elective procedures or protocol-driven procedures. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.

    Measured: From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively.

  25. Healthcare Resource Utilization (HRU): Duration of Hospitalizations Due to Adverse Events

    Hospitalizations due to adverse events exclude those for transfusions, elective procedures or protocol-driven procedures. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient.

    Measured: From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively.

  26. Healthcare Resource Utilization (HRU): Number of Days of Hospitalization Due to Adverse Events Per Person-Years

    Hospitalizations due to adverse events exclude those for transfusions, elective procedures or protocol-driven procedures. HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient

    Measured: From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Dec 2009 (estimated)
  2. Study start26 Jan 2010
  3. Primary completion17 Mar 2013
  4. Study completion9 May 2018

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
239 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01029262
Sponsor's protocol ID
CC-5013-MDS-005
Acronym
MDS-005
Status last verified
Jun 2019
First submitted
8 Dec 2009
Last updated
25 Jun 2019
Data monitoring committee
Yes

Publications

  • Santini V, Almeida A, Giagounidis A, Gropper S, Jonasova A, Vey N, Mufti GJ, Buckstein R, Mittelman M, Platzbecker U, Shpilberg O, Ram R, Del Canizo C, Gattermann N, Ozawa K, Risueno A, MacBeth KJ, Zhong J, Seguy F, Hoenekopp A, Beach CL, Fenaux P. Randomized Phase III Study of Lenalidomide Versus Placebo in RBC Transfusion-Dependent Patients With Lower-Risk Non-del(5q) Myelodysplastic Syndromes and Ineligible for or Refractory to Erythropoiesis-Stimulating Agents. J Clin Oncol. 2016 Sep 1;34(25):2988-96. doi: 10.1200/JCO.2015.66.0118. Epub 2016 Jun 27.

    PubMed 27354480

  • Santini V, Almeida A, Giagounidis A, Skikne B, Beach CL, Weaver J, Tu N, Fenaux P. Achievement of red blood cell transfusion independence in red blood cell transfusion-dependent patients with lower-risk non-del(5q) myelodysplastic syndromes correlates with serum erythropoietin levels. Leuk Lymphoma. 2020 Jun;61(6):1475-1483. doi: 10.1080/10428194.2020.1719088. Epub 2020 Feb 17.

    PubMed 32064987

  • Almeida A, Fenaux P, Garcia-Manero G, Goldberg SL, Gropper S, Jonasova A, Vey N, Castaneda C, Zhong J, Beach CL, Santini V. Safety profile of lenalidomide in patients with lower-risk myelodysplastic syndromes without del(5q): results of a phase 3 trial. Leuk Lymphoma. 2018 Sep;59(9):2135-2143. doi: 10.1080/10428194.2017.1421758. Epub 2018 Jan 11.

    PubMed 29322849

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Lenalidomide in people with Anemia, run by Celgene at 99 sites in 16 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 25 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 99 sites in Italy, Japan, Germany, Canada, United States and 11 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Jun 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for ≥ 56 Days as Determined by an Independent Review Committee (IRC) (time frame: From first dose of study drug until 28 days after the last dose, as of the data cut-off date of 17 March 2014; median (minimum, maximum) duration of treatment was 168 (14, 449) and 164 (7, 1158) days in each treatment group respectively.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01029262, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jun 2019.

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