AzTrials
AzTrials

Myelodysplastic SyndromesPhase 2NCT02912208

Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes

A Phase 2 study testing Eltrombopag/Revolade in people with Myelodysplastic Syndromes and Thrombocytopenia, run by Associazione Qol-one at 64 sites in 4 countries.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
Search all trials

Trial summary

Myelodysplastic syndromes (MDS) prevail in older age and are characterized by ineffective erythropoiesis and peripheral cytopenias. Supportive therapy is the main therapeutic option for most patients. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndromes, Thrombocytopenia
What is being tested?
Eltrombopag/Revolade
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
174 participants
Where
64 sitesin 4 countries
Study dates
11 Jun 2011to Oct 2026
Phase
Phase 2
Are there results?
None posted in the registry

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

  • Adult subjects (18 years of age or older) with low or intermediate-1 IPSS risk MDS and stable disease.
  • Subjects must have a platelet count taken within the 4 weeks prior to randomization that is <30 Gi/L.
  • Subjects must be ineligible or relapsed or refractory to receive other treatment options (such as azacitidine or lenalidomide) and must be ineligible to receive intensive chemotherapy or autologous/allogeneic stem cell transplantation.
  • Subjects must have platelet count and platelet transfusion data available over a period of 8 weeks prior to randomization.
  • During the 2 months prior to randomization, subjects must have a baseline Bone Marrow examination which includes cytomorphology and cytogenetics. Histopathology should be performed.
  • Erythropoiesis-stimulating agents (ESAs) in anemic subjects or granulocyte colonystimulating factor (G-CSF) in subjects with severe neutropenia and recurrent infections are allowed during the study as per accepted standards. Subjects who enter the study on ESAs or G-CSF should continue at the same dose schedule until the optimal dose of study medication has been established.
Show all 12Show fewer
  • ECOG (Eastern Cooperative Oncology Group) Performance Status 0-3
  • Subject is able to understand and comply with protocol requirements and instructions.
  • Subject has signed and dated informed consent.
  • Adequate baseline organ function defined by the criteria below:
  • total bilirubin (except for Gilbert's Syndrome) ≤ 1.5 x Upper Limit Normal Alanine aminotransferase and Aspartate aminotransferase ≤ 3 x Upper Limit Normal creatinine ≤ 2 x Upper Limit Normal albumin must not be below the lower limit of normal by more than 20%.
  • Subject is practicing an acceptable method of contraception. Female subjects (or female partners of male subjects) must either be of non-childbearing potential (hysterectomy, bilateral oophorectomy, bilateral tubal ligation or post-menopausal >1 year), or of childbearing potential and use of an highly effective method of contraception from 2 weeks prior to administration of study medication, throughout the study, and 28 days after completion or premature discontinuation from the study.

Who cannot join

  • MDS with intermediate-2 or high IPSS risk.
  • History of treatment for cancer other than MDS with systemic chemotherapy and/or radiotherapy within the last 2 years.
  • History of treatment with romiplostim or other Thrombopoietin receptor agonists.
  • Pre-existing cardiovascular disease (including congestive heart failure, New York Heart Association Grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. persistent atrial fibrillation), or subjects with a QTc >450 msec (QTc >480 msec for subjects with Bundle Branch Block).
  • BM fibrosis that leads to an inability to aspirate marrow for assessment.
  • Peripheral monocytosis > 1000/uL prior to Day 1 of study medication.
Show all 12Show fewer
  • Leukocytosis >=25,000/uL prior to Day 1 of study medication.
  • Female subjects who are nursing or pregnant (positive serum or urine Beta-human chorionic gonadotropin [B-hCG] pregnancy test) at screening or pre-dose on Day 1.
  • Current alcohol or drug abuse.
  • Treatment with an Investigational Product within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.
  • Active and uncontrolled infections.
  • Subjects infected with Hepatitis B, C or Human Immunodeficiency Virus (HIV).

Trial locations and contacts

64 sites in 4 countries.

64 sites in 4 countries

Italy33 sites

  • A.O. SS. Antonio e Biagio e Cesare Arrigo

    Alessandria, AL

  • Ospedale Riuniti

    Ancona, AN

  • Ospedale Cardinal Massaia

    Asti, AT

  • A.O. S. Giovanni Moscati

    Avellino, AV

  • Policlinico Università di Bari

    Bari, BA

  • Ospedale A. Perrino

    Brindisi, BR

  • Ospedale "Roberto Binaghi"

    Cagliari, CA

  • Ospedale Ferrarotto

    Catania, CT

  • Ospedale Garibaldi

    Catania, CT

  • Ospedale L'Annunziata

    Cosenza, CS

  • Azienda Ospedaliera Universitaria Careggi

    Florence, FI

  • Università degli Studi di Genova

    Genova, GE

  • Ospedale Vito Fazzi

    Lecce, LE

  • IRCCS Ospedale Maggiore Policlinico

    Milan, MI

  • Ospedale Niguarda

    Milan, MI

  • Ospedale Civile Spirito Santo

    Pescara, PE

  • Azienda Ospedaliera Bianchi-Melacrino-Morelli

    Reggio Calabria, RC, 89100

  • Arcispedale di Santa Maria Nuova

    Reggio Emilia, RE

  • A.O. San Camillo Forlanini

    Roma, RM

  • Ospedale Sant'Eugenio

    Roma, RM

  • Policlinico Agostino Gemelli

    Roma, RM

  • Università Campus Bio Medico di Roma

    Roma, RM

  • Azienda Ospedaliera Sant'Andrea

    Rome, RM

  • IRCCS Istituto Regina Elena

    Rome, RM

  • Ospedale Nuova Regina Margherita

    Rome, RM

  • Policlinico Umberto I

    Rome, RM

  • Policlinico Universitario Tor Vergata

    Rome, RM

  • A.O.U. San Giovanni di Dio e Ruggì D'Aragona

    Salerno, SA

  • Ospedale Casa Sollievo della Sofferenza

    San Giovanni Rotondo, FG

  • Policlinico Santa Maria alle Scotte

    Siena, SI

  • A.O. Santa Maria

    Terni, TE

  • A.O. Citta' della Salute e della Scienza di Torino

    Torino, TO

  • U.O. Citta' della Salute e della Scienza di Torino

    Torino, TO

Other trials in Italy

France22 sites

  • CHU Amiens

    Amiens

  • Centre d'Avignon

    Avignon

  • Hôpital de la Côte Basque

    Bayonne

  • Centre d'Avicenne, Hôpital d'Avicenne

    Bobigny

  • CHU de Haut-Lévèque

    Bordeaux

  • Centre Hospitalier de Boulogne Sur Mer

    Boulogne-sur-Mer

  • CHU Clémenceau

    Caen

  • Centre Henri Mondor

    Créteil

  • CHU de Grenoble

    Grenoble

  • Centre Le Mans

    Le Mans

  • Hôpital Saint Vincent de Paul

    Lille

  • CHRU de Limoges

    Limoges

  • Centre de Marseille

    Marseille

  • CHU Brabois

    Nancy

  • Centre de Nantes

    Nantes

  • Hopital Archet 1

    Nice

  • Centre Hospitalier Universitaire de Nimes

    Nîmes

  • Centre de St Louis, Hôpital St Louis

    Paris

  • Centre Hospitalier de la Région d'Annecy

    Pringy

  • Centre de Rouen, Centre Henri Becquerel

    Rouen

  • CHU Purpan

    Toulouse

  • CHU de Bretonneau

    Tours

Other trials in France

Slovenia7 sites

  • General Hospital Celje

    Celje

  • Univerzitetni klinini center Ljubljana

    Ljubljana

  • University Medical Centre Maribor

    Maribor

  • General Hospital Murska Sobota

    Murska Sobota

  • General Hospital Nova Gorica

    Nova Gorica

  • General Hospital Novo mesto

    Novo Mesto

  • General Hospital Slovenj Gradec

    Slovenj Gradec

Other trials in Slovenia

Germany2 sites

  • Heinrich-Heine-Universität Düsseldorf

    Düsseldorf

  • Universitätsmedizin Mannheim

    Mannheim

Other trials in Germany

About this study

Official title: Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes (EQoL-MDS)

Myelodysplastic syndromes (MDS) prevail in older age and are characterized by ineffective erythropoiesis and peripheral cytopenias. Supportive therapy is the main therapeutic option for most patients. Quality of Life (QoL) is mainly deteriorated by anemia and by the limitations associated with thrombocytopenia, neutropenia and transfusion dependence. The only available treatment for severe thrombocytopenia, in the presence of bleeding, is platelet transfusion.

Eltrombopag is an orally bioavailable agonist of the thrombopoietin receptor. In adult patients with chronic immune thrombocytopenia (ITP), Eltrombopag rapidly increases platelet counts and significantly reduces bleeding episodes during treatment. Eltrombopag is well tolerated. In 2007, Eltrombopag has received the Orphan Drug Designation for the treatment of ITP (EMEA/OD/031/07), and in 2008 the Food and Drug Association approved Eltrombopag for the treatment of ITP refractory or resistant. It has been shown that in patients affected by MDS and by acute myeloid leukemia, Eltrombopag neither increases the proliferation, nor the clonogenic growth capacity of bone marrow blasts. Furthermore, Eltrombopag induces an increase in the megakaryocytic differentiation and in the formation of normal megakaryocytic colonies. These results provide the rationale for pursuing further research on Eltrombopag for the treatment of thrombocytopenia in case of MDS.

Read more

The study is open to adult patients with myelodysplastic syndrome (MDS) with thrombocytopenia and low- or intermediate-1 IPSS risk (Index Prognostic Score System).

Severe thrombocytopenia associated with MDS may lead to death from hemorrhage, even in low prognostic risk patients. The benefit of platelet transfusion is short-termed. Patients become refractory in the long term. The availability of a treatment that induces the increase of platelet count is extremely important, either in terms of quality of life, and in overall survival.

Being tested
Eltrombopag/Revolade, Placebo
Medical subject headings
Myelodysplastic Syndromes, Thrombocytopenia

What participants receive

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

  • Eltrombopag/RevoladeDrug

    Eltrombopag 50 mg once daily has been selected as the starting dose for this study. Thereafter, dependent on platelet response the dose of study medication can be increased by 50 mg every 2 weeks, up to a maximum dose of 300 mg once daily (150 mg in subjects of East Asian ethnicity).

  • PlaceboOther

    The administration is the same of eltrombopag

Study groups in NCT02912208
Study groupRoleWhat they receive
Arm 1 (Eltrombopag)ExperimentalArm 1 is the active treatment arm
Receives: Drug: Eltrombopag/Revolade
Arm 2 (Placebo)Placebo comparatorArm 2 is the control arm
Receives: Other: Placebo

What is being measured

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

  1. Response rate

    Proportion of patients achieving a complete response (CR) or response (R) during the treatment period

    Measured: Six months

  2. Safety and Tolerability (number of adverse events)

    Safety and tolerability in terms of frequency of adverse events (AE) and serious adverse events (SAE)

    Measured: Six month

  3. Duration of platelet response

    Measured: five years

  4. long-term safety and tolerability (number adverse events in the long term)

    Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 and number of adverse events reporting in accordance with CTCAE v4.0

    Measured: five years

Secondary outcomes (7)

  1. Quality of life (QoL) score

    to evaluate the changes of the quality of life in the two arms

    Measured: six months

  2. number of monthly platelet transfusions

    Measured: six months

  3. duration of transfusion independence

    Measured: six months

  4. time to response

    time to response (time from starting treatment to time of achievement of CR or PR)

    Measured: six months

  5. incidence and severity of bleeding

    incidence and severity of bleeding using the WHO (World Health Organization)Bleeding Scale

    Measured: six months

  6. overall survival

    overall survival (OS) at 2 and at 5 years

    Measured: 2 and 5 years

  7. leukemia-free survival (LFS)

    leukemia-free survival (LFS) at 2 and at 5 years (events for LFS are defined as death and progression to AML);

    Measured: 2 and 5 years

Timeline

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

  1. First posted23 Sep 2016 (estimated)
  2. Study start11 Jun 2011
  3. Primary completion2 Feb 2017
  4. Study completionOct 2026 (estimated)

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Single blind
Who is masked
Participant
Enrolment
174 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02912208
Sponsor's protocol ID
EQoL-MDS
Status last verified
Jan 2022
First submitted
9 Sep 2016
Last updated
28 Jan 2022
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • Oliva EN, Riva M, Niscola P, Santini V, Breccia M, Giai V, Poloni A, Patriarca A, Crisa E, Capodanno I, Salutari P, Reda G, Cascavilla N, Ferrero D, Guarini A, Tripepi G, Ianni G, Russo E, Castelli A, Fattizzo B, Beltrami G, Bocchia M, Molteni A, Fenaux P, Germing U, Ricco A, Palumbo GA, Impera S, Di Renzo N, Rivellini F, Buccisano F, Stamatoullas-Bastard A, Liberati AM, Candoni A, Delfino IM, Arcadi MT, Cufari P, Rizzo L, Bova I, D'Errigo MG, Zini G, Latagliata R. Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS). J Clin Oncol. 2023 Oct 1;41(28):4486-4496. doi: 10.1200/JCO.22.02699. Epub 2023 Jun 9.

    PubMed 37294914

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Eltrombopag/Revolade in people with Myelodysplastic Syndromes and Thrombocytopenia, run by Associazione Qol-one at 64 sites in 4 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 12 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 28 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 64 sites in Italy, France, Slovenia, Germany. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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

Are results available for this study?

Results are not available in the source record.

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?

Response rate (time frame: Six months). The study lists 4 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 NCT02912208, which also lists 1 linked publication.

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 28 Jan 2022.

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