AzTrials
AzTrials

Primary MyelofibrosisPhase 3RESUMENCT01178281

Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence

A Phase 3 study testing Pomalidomide 0.5 mg and Pomalidomide in people with Primary Myelofibrosis and MPN-associated Myelofibrosis, run by Celgene at 87 sites in 16 countries.

Completed

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

Official record
Search all trials

Trial summary

The objective of this study is to determine whether pomalidomide is safe and effective in reversing red blood cell (RBC)-transfusion-dependence in persons with myeloproliferative neoplasm (MPN)-associated myelofibrosis (global study) and in reversing anemia in Chinese with MPN-associated myelofibrosis and severe… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Pomalidomide 0.5 mg and Pomalidomide
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
267 participants
Where
87 sitesin 16 countries
Study dates
8 Sep 2010to 15 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

  • Age ≥ 18 years
  • Myeloproliferative-neoplasm (MPN)-associated myelofibrosis
  • RBC-transfusion-dependence (global study):
  • Average RBC-transfusion frequency ≥ 2 units/28 days over at least the 84 days immediately prior to randomization. There must be no interval > 42 days without ≥ 1 RBC-transfusion.
  • Only RBC-transfusions given when the hemoglobin ≤ 90 g/L³ are scored in
  • determining eligibility.
Show all 18Show fewer
  • RBC-transfusions due to bleeding are not scored in determining eligibility.
  • RBC-transfusions due to chemotherapy-induced anemia are not scored in determining eligibility.
  • Severe anemia (China-specific extension):
  • ≥ 2 hemoglobin concentrations ≤ 80 g/L for ≥ 84 days immediately before the day of enrollment.
  • No RBC-transfusion within 6 months prior to enrollment.
  • Hemoglobin ≤ 130 g/L at randomization (global study); ≤ 80 g/L at enrollment in the China-specific extension.
  • Bone marrow biopsy within 6 months (global study only).
  • Inappropriate to receive blood cell or bone marrow allotransplant, erythropoietin and androgenic steroids
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
  • Agree to follow pregnancy precautions as required by the protocol.
  • Agree to receive counseling related to teratogenic and other risks of pomalidomide.
  • Agree not to donate blood or semen.

Who cannot join

  • Prior blood cell or bone marrow allotransplant.
  • Use of drugs to treat MPN-associated myelofibrosis ≤ 30 days before starting study drug.
  • Treatment with erythropoietin or androgenic steroids ≤ 84 days before starting study drug.
  • Anemia due to reasons other than MPN-associated myelofibrosis.
  • Pregnant or lactating females.
  • More than 10% blasts by bone marrow examination or more than 10% blasts in blood in consecutive measurements spanning at least 8 weeks
Show all 22Show fewer
  • Prior history of malignancies,other than the disease being studied, unless the subject has been free of the malignancy for ≥ 5 years with the following exceptions:
  • Carcinoma in situ of the cervix
  • Carcinoma in situ of the breast
  • Incidental histologic finding of prostate cancer (T 1a or T 1b using TNM [tumor, nodes, metastasis] clinical staging system)
  • Human immunodeficiency virus (HIV) infection, active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections.
  • Prior treatment with pomalidomide.
  • Allergic reaction or rash after treatment with thalidomide or lenalidomide
  • Any of the following laboratory abnormalities:
  • Neutrophils < 0.5x10^9 /L
  • Platelets < 25 x 10^9 /L
  • Estimated glomerular filtration rate (kidney function) < 30 mL/min/1.73 m²
  • Aspartate aminotransferase (AST) and alanine transaminase (ALT) > 3.0 x upper limit of normal (ULN)
  • Total bilirubin ≥ 4 x ULN;
  • Uncontrolled hyperthyroidism or hypothyroidism.
  • Deep venous thrombosis (DVT) or pulmonary embolus (PE) < 6 months before starting study drug
  • Clinically-important heart disease within the past 6 months

Trial locations and contacts

87 sites in 16 countries.

87 sites in 16 countries

United States17 sites

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan, 48109

  • Mount Sinai School of Medicine Brookdale University Hospital

    Brooklyn, New York, 11212

  • University of Illinois at Chicago

    Chicago, Illinois, 60612

  • Medicine Taussig Cancer Institute

    Cleveland, Ohio, 44195

  • University of Florida Shands Cancer Center

    Gainesville, Florida, 32610

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • Mayo Clinic

    Jacksonville, Florida, 32224

  • UCLA School of Medicine

    Los Angeles, California, 90095

  • Weill Medical College of Cornell University

    New York, New York, 10021

  • Ruttenberg Treatment Center

    New York, New York, 10029

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • University of Utah

    Salt Lake City, Utah, 84132

  • Mayo Clinic

    Scottsdale, Arizona, 85259

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109

  • Avera Hematology and Transplant

    Sioux Falls, South Dakota, 57105

Other trials in United States

France8 sites

  • Hopital Albert Michallon

    La Tronche, 38043

  • Hopital Saint Vincent de Paul

    Lille, 59020

  • CHU Dupuytren

    Limoges, 87042

  • Hopital Saint-Louis

    Paris, 75475

  • CHRU - Hopital du Haut Leveque

    Pessac, 33604

  • Hopitaux Universitaires de Strasbourg, CHU Haute-Pierre

    Strasbourg, 67098

  • Hopital Purpan

    Toulouse, 31059

  • Institut Gustave Roussy

    Villejuif, 94805

Other trials in France

Italy8 sites

  • Azienda Ospedaliera Universitaria Consorziale Policlinico di Bari

    Bari, 70124

  • Ospedali Riuniti di Bergamo

    Bergamo, 24128

  • Azienda Ospedaliera Universitaria Careggi

    Florence, 50134

  • Azienda Ospedaliera Universitaria Federico II di Napoli

    Naples, 80131

  • Azienda Ospedaliera San Luigi Gonzaga

    Orbassano, 10043

  • IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Centro per lo Studio della Mielofibrosi

    Pavia, 27100

  • IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Ematologia

    Pavia, 27100

  • Ospedale di Circolo e Fondazione Macchi Varese

    Varese, 21100

Other trials in Italy

United Kingdom7 sites

  • Belfast City Hospital

    Belfast, BT9 7AB

  • Beatson Oncology Centre

    Glasgow, G12 0YN

  • John Radcliffe Hospital NHS Trust

    Headington, OX3 9DU

  • St. Thomas Hospital

    London, SE1 9RT

  • Hammersmith Hospital

    London, W12 ONN

  • Freeman Hospital

    Newcastle upon Tyne, NE7 7DN

  • Royal Hallamshire Hospital

    Sheffield, S10 2JF

Other trials in United Kingdom

China6 sites

  • Peking University People's Hospital

    Beijing, 100044

  • Peking Union Medical College Hospital

    Beijing, 100730

  • Jiangsu Province Hospital

    Jiangsu, 210029

  • Shanghai Ruijin Hospital

    Shanghai, 200025

  • West China Hospital, Sichuan University

    Sichuan, 610041

  • Blood Disease Hospital Chinese Academy of Medical Sciences

    Tianjin, 300041

Other trials in China

Japan6 sites

  • Juntendo University Hospital

    Bunkyou-ku, 113-8431

  • Kyushu University Hospital

    Fukuoka, 812-8582

  • Tokai University Hospital

    Isehara, 259-1193

  • Kyoto University Hospital

    Kyoto, 606-8507

  • Nagasaki University Hospital

    Nagasaki, 852-8501

  • Tokyo Medical University Hospital

    Shinjuku, 160-0023

Other trials in Japan

Germany5 sites

  • Universitatsklinikum Aachen

    Aachen, 52074

  • Medizinische Hochschule Hannover

    Hanover, 30625

  • Universitatsklinikum Leipzig

    Leipzig, 04103

  • Johannes Wesling Klinikum Minden

    Minden, 32429

  • Universitatsklinikum Ulm

    Ulm, 89081

Other trials in Germany

Australia4 sites

  • Frankston Hospital

    Frankston, Victoria, 3199

  • Gosford Hospital

    Gosford, New South Wales, 2250

  • Royal Melbourne Hospital

    Parkville, Victoria, 3050

  • Royal North Shore Hospital

    St Leonards, New South Wales, 2065

Other trials in Australia

Canada4 sites

  • Cross Cancer Institute

    Edmonton, Alberta, T6G lz2

  • Centre Hospitalier de L'Universite de Montreal

    Montreal

  • Princess Margaret Hospital

    Toronto, Ontario, M5G 2M9

  • Vancouver General Hospital

    Vancouver, British Columbia, V5Z 1M9

Other trials in Canada

Russia4 sites

  • Russian Scientific Haematology Centre

    Moscow, 125167

  • Federal State Institution Russian Scientific-research Institute of Hematology and Transfusiology of Federal Medical-Biological Agency of Russia

    Saint Petersburg, 191024

  • State Pavlov Medical University

    Saint Petersburg, 196022

  • Federal State Institution "Federal Centre of Heart, Blood and Endocrinology of Rosmedtechnologies named after V.A. Almazov"

    Saint Petersburg, 197341

Other trials in Russia

Spain4 sites

  • Hospital Clinic I Provincial de Barcelona

    Barcelona, 08036

  • Hospital Universitario Puerta De Hierro Majadahonda

    Majadahonda, 28222

  • Hospital Clinico de Salamanca

    Salamanca, 37007

  • Hospital Clinico de Valencia

    Valencia, 46010

Other trials in Spain

Austria3 sites

  • Medizinische Universitatklinik Graz

    Graz, A-8036

  • Medizinische Universitat Innsbruck

    Innsbruck, A-6020

  • Medizinische Universitat Wien

    Vienna, A-1190

Other trials in Austria

Belgium3 sites

  • Algemeen Ziekenhuis Sint-Jan

    Bruges, 8000

  • Grand Hopital de Charleroi

    Charleroi, 6000

  • Universitaire Ziekenhuis Leuven Gathuisberg

    Leuven, 3000

Other trials in Belgium

Netherlands3 sites

  • VU University Medical Center

    Amsterdam, 1081 HV

  • Erasmus Medish Centrum

    Rotterdam, 3015 CE

  • University Medical Center Utrecht

    Utrecht, 3584 CX

Other trials in Netherlands

Poland3 sites

  • Wojewodzki Szpital Specjalistyczny im. F.Chopina

    Rzeszów, 35-055

  • Samodzielny Publiczny Szpital Kliniczny Nr 1 PAM

    Szczecin, 71-242

  • Centralny Szpital Kliniczny MSWiA

    Warsaw, 02-507

Other trials in Poland

Sweden2 sites

  • Skane University Hospital

    Lund, 22185

  • Karolinska University Hospital Huddinge

    Stockholm, 14185

Other trials in Sweden

About this study

Official title: A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell-Transfusion-Dependence

The objective of this study is to determine whether pomalidomide is safe and effective in reversing red blood cell (RBC)-transfusion-dependence in persons with myeloproliferative neoplasm (MPN)-associated myelofibrosis (global study) and in reversing anemia in Chinese with MPN-associated myelofibrosis and severe anemia not receiving RBC-transfusions (China extension study only)

Read the detailed description

The multicenter global study was conducted in 15 countries including Australia, Austria, Belgium, Canada, China, France, Germany, Italy, Japan, the Netherlands, Russia, Spain, Sweden, the United Kingdom, and the United States. The global study enrolled participants with myeloproliferative neoplasm (MPN)-associated myelofibrosis and RBC-transfusion-dependence. Participants were randomly assigned to receive pomalidomide or placebo in a blinded fashion.

In most countries participating in the global study, RBC-transfusions are typically given for a hemoglobin level <80-90 g/L. In China, RBC-transfusions are rarely given unless the hemoglobin level is <60 g/L. Consequently, few Chinese with MPN-associated myelofibrosis meet RBC-transfusion-dependence criteria of the global study. A China-specific extension was developed to test the ability of pomalidomide to improve severe anemia (defined as a hemoglobin < 80 g/L for ≥ 84 days in persons not receiving RBC-transfusions).

The China-specific extension study consisted of a single-arm, open-label study in adults with MPN-associated myelofibrosis and severe anemia not receiving RBC transfusions with the objective of describing the frequency of anemia response.

The Global (intent-to-treat [ITT] and safety) population in the main study and the China extension (ITT and safety) population are mutually exclusive.

Being tested
Pomalidomide 0.5 mg, Placebo, Pomalidomide
Keywords
Myelofibrosis, Post-polycythemia vera myelofibrosis, Post-essential thrombocythemia myelofibrosis, RBC-transfusion-dependence, Primary Myelofibrosis
Medical subject headings
Primary Myelofibrosis

What participants receive

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

  • Pomalidomide 0.5 mgDrug

    Also called CC-4047; Pomalyst; Imnovid

    Pomalidomide 0.5 mg capsule taken by mouth once daily. Immunomodulatory agent with demonstrated efficacy in the treatment of subjects with RBC-transfusion-dependence associated with MNP-associated myelofibrosis.

  • PlaceboDrug

    Placebo Comparator to active drug; Placebo capsule taken by mouth once daily

  • PomalidomideDrug

    Also called CC-4047, Pomalyst, Imnovid

    Pomalidomide 0.5 mg capsule taken by mouth once daily.

Study groups in NCT01178281
Study groupRoleWhat they receive
Pomalidomide 0.5 mgExperimentalParticipants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit (defined as a reduction from Baseline of ≥ 50% in RBC-transfusion frequency during the prior 84-day interval) could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.
Receives: Drug: Pomalidomide 0.5 mg
PlaceboPlacebo comparatorParticipants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC- transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.
Receives: Drug: Placebo
China Extension: Pomalidomide 0.5 mgExperimentalParticipants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion. Participants who experienced anemia response could continue treatment until the response was lost or other criteria for treatment discontinuation applied.
Receives: Drug: Pomalidomide

What is being measured

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

  1. Percentage of Participants Who Achieved RBC-Transfusion Independence

    RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval.

    Measured: 168 days

  2. China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days

    A response in the China extension study was defined as an increase in hemoglobin ≥ 15 g/L above baseline value (in the absence of RBC transfusion) for ≥ 84 consecutive days.

    Measured: From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks.

Secondary outcomes (8)

  1. Overall Survival

    The time from randomization to the death or to the latest date when participants are known to be alive. Overall survival was analyzed using Kaplan-Meier method; participants who were alive or lost to follow-up were censored at the latest date they were known to be alive.

    Measured: From first dose of study drug up to end of study; median follow-up time was 19.1 months in the pomalidomide 0.5 mg arm and 17.6 months in the placebo arm.

  2. Duration of RBC-Transfusion Independence

    The duration of RBC-transfusion independence is the time from the date at which the first RBC-transfusion independence started to the date of another RBC-transfusion given at least 84 days after the time the transfusion independence started. The duration of the RBC-transfusion independence was analyzed using the Kaplan-Meier method. Data were censored at the end of the treatment phase for participants who had not received another RBC-transfusion after the start of transfusion independence by the end of treatment phase.

    Measured: From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm.

  3. Time to RBC-Transfusion Independence

    Time to response was measured from first dose of study drug to the start of the first response. The start date of the response was defined as one day after the last date of an RBC-transfusion for participants who received a RBC-transfusion after the first dose, and as the date of the first dose of study drug for participants who received no RBC-transfusions during the 84 days after the first dose of study drug.

    Measured: 168 days

  4. Number of Participants With Treatment-emergent Adverse Events (TEAE)

    A TEAE is an adverse event (AE) that starts on or after the first dose of study drug. The severity of each AE was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE),Version 4.0 and according to the following scale: Grade 1 = Mild (transient or mild discomfort; no limitation in activity; no medical intervention/therapy required); Grade 2 = Moderate (mild to moderate limitation in activity, some assistance may be needed; minimal medical intervention/therapy required); Grade 3 = Severe (marked limitation in activity, assistance usually required; medical intervention/therapy required, hospitalization possible); Grade 4 = Life-threatening (extreme limitation in activity, significant assistance or medical intervention/therapy required, hospitalization or hospice care probable); Grade 5 = Death Drug-related (related) AEs are those suspected by the Investigator as being related to administration of study drug

    Measured: From the first dose of study drug until 28 days after last dose; median treatment duration was 23.7 weeks in the pomalidomide arm, 23.9 weeks in the placebo arm, and 24.0 weeks in the China extension pomalidomide arm.

  5. Healthcare Resource Utilization

    Measured: From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm.

  6. Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score

    EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). For the health state profile participants rate their perceived health state today on 5 dimensions: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression on a Likert-type scale from 1 to 3, where 1 = "no problems," 2 = "some problems," and 3 = "extreme problems." The EQ-5D Health Utility Index (HUI) was generated from the five health state domain scores, and ranges from -0.594 (worst) and 1 (best) imaginable health state, with -0.594 representing an "unconscious" health state.

    Measured: Baseline and Days 85 and 169

  7. Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale

    EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). On the VAS the participant rates his/her health state on a line from 0 (worst imaginable health) to 100 (best imaginable health).

    Measured: Baseline and Days 85 and 169

  8. Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score

    The FACT-An is a 47-item, cancer-specific questionnaire consisting of a core 27-item general questionnaire measuring the four general domains of QoL (physical, social/family, emotional and functional well-being), and an additional 20-item anemia questionnaire (FACT-An Anemia subscale) that measures 13 fatigue-associated items (FACT-F Fatigue subscale) and seven non-fatigue-related items. Each item is scored using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a bit; and 4 = Very much). FACT-An total score is calculated by adding all the FACT-An subscales together. The total score ranges from 0-188 with higher scores representing better QOL.

    Measured: Baseline and Days 85 and 169

Timeline

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

  1. First posted10 Aug 2010 (estimated)
  2. Study start8 Sep 2010
  3. Primary completion1 Jan 2013
  4. Study completion15 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
Double blind
Who is masked
Participant, Investigator
Enrolment
267 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01178281
Sponsor's protocol ID
CC-4047-MF-002
Other IDs
2010-018965-42
Acronym
RESUME
Status last verified
Jun 2019
First submitted
15 Jul 2010
Last updated
17 Jul 2019
Data monitoring committee
Yes

Publications

  • Tefferi A, Al-Ali HK, Barosi G, Devos T, Gisslinger H, Jiang Q, Kiladjian JJ, Mesa R, Passamonti F, McMullin MF, Ribrag V, Schiller G, Vannucchi AM, Zhou D, Reiser D, Zhong J, Gale RP. A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. Leukemia. 2017 Apr;31(4):896-902. doi: 10.1038/leu.2016.300. Epub 2016 Oct 24.

    PubMed 27773929

  • Begna KH, Pardanani A, Mesa R, Litzow MR, Hogan WJ, Hanson CA, Tefferi A. Long-term outcome of pomalidomide therapy in myelofibrosis. Am J Hematol. 2012 Jan;87(1):66-8. doi: 10.1002/ajh.22233. Epub 2011 Nov 12.

    PubMed 22081489

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Pomalidomide 0.5 mg and Pomalidomide in people with Primary Myelofibrosis and MPN-associated Myelofibrosis, run by Celgene at 87 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 18 inclusion and 22 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 17 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 87 sites in United States, France, Italy, United Kingdom, China 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 14 Mar 2014). 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 RBC-Transfusion Independence (time frame: 168 days). 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 NCT01178281, which also lists 2 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 17 Jul 2019.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in