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ThrombocytopaeniaPhase 2ASPIRENCT01440374

A Three-part Study of Eltrombopag in Thrombocytopenic Subjects With Myelodysplastic Syndromes or Acute Myeloid Leukemia

A Phase 2 study testing eltrombopag in people with Thrombocytopaenia, run by Novartis Pharmaceuticals at 120 sites in 22 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

This was a worldwide, three-part (Part 1: open-label, Part 2: randomized, double-blind, Part 3: extension), multi-center study to evaluate the effect of eltrombopag in subjects with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who have thrombocytopenia due to bone marrow insufficiency from their… Read more about this study

Who is it for?
Ages 18 to 110 years
Conditions the study is for
Thrombocytopaenia
What is being tested?
eltrombopag
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
162 participants
Where
120 sitesin 22 countries
Study dates
Sep 2011to Dec 2015
Phase
Phase 2
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 to 110 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Adult subjects (18 years of age or older) with MDS or AML (bone marrow blasts ≤50%) with thrombocytopenia due to bone marrow insufficiency from the disease or prior treatment. Subjects with transient thrombocytopenia due to active treatment with disease modifying agents or chemotherapy (except for hydroxyurea) are excluded.
  • Subjects must have Grade 4 thrombocytopenia (platelet counts <25 Gi/L) due to bone marrow insufficiency (or Grade 4 thrombocytopenia, but platelet count greater than or equal to 25 Gi/L due to platelet transfusion). In addition, subjects must have had at least one of the following during the 4 week screening period: platelet transfusion, or symptomatic bleeding or platelet count <10 Gi/L. Subjects whose thrombocytopenia below 10 Gi/L is due to causes other than bone marrow insufficiency (e.g., fever, infection, autoimmune disease) are not eligible.
  • Subjects must have platelet count, bleeding and platelet transfusion data available over a period of at least 4 weeks prior to randomization.
  • Prior systemic treatment for malignancy, with the exception of hydroxyurea, must have been discontinued prior to entry into the study: at least 4 weeks before Day 1 for the following: chemotherapy, demethylating agents (azacitidine or decitabine), lenalidomide, thalidomide, clofarabine and IL-11(oprelvekin); at least 8 weeks before Day 1 for antithymocyte/antilymphocyte globulin.
  • Subjects with a prior stem cell transplant (SCT) must have relapsed after the SCT.
  • Subjects must be stable and, in the opinion of the investigator, be expected to complete a 12 week treatment period.
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  • ECOG Status 0-2.
  • Subject must be able to understand and comply with protocol requirements and instructions.
  • Subject has signed and dated an informed consent form.
  • Adequate baseline organ function defined by the criteria below: total bilirubin ≤ 1.5xULN except for Gilbert's syndrome or cases clearly not indicative of inadequate liver function (i.e. elevation of indirect (hemolytic) bilirubin in the absence of ALT abnormality), ALT ≤ 3xULN, creatinine ≤ 2.5xULN
  • Women must be either of non-child bearing potential or women with child-bearing potential and men with reproductive potential must be willing to practice acceptable methods of birth control during the study Women of childbearing potential must have a negative serum or urine pregnancy test within 7 days prior to the first dose of study treatment.
  • In France, a subject eligible for inclusion in this study only if either affiliated to, or a beneficiary of, a social security category.

Who cannot join

  • Subjects with MDS and an IPSS of low or intermediate-1 risk at screening.
  • Subjects with a diagnosis of acute promyelocytic or megakaryocytic leukemia or AML secondary to a myeloproliferative neoplasm.
  • History of treatment with romiplostim or other TPO-R agonists.
  • Subjects with a QTc >480 msec (QTc >510 msec for subjects with Bundle Branch Block).
  • Leukocytosis ≥25,000/uL on Day 1 of treatment with study medication.
  • Subjects with known thrombophilic risk factors. Exception: Subjects for whom the potential benefits of participating in the study outweigh the potential risks of thromboembolic events, as determined by the investigator.
Show all 15Show fewer
  • Female subjects who are nursing or pregnant (positive serum or urine β-human chorionic gonadotropin [β-hCG] pregnancy test) at screening or pre-dose on Day 1.
  • Current alcohol or drug abuse.
  • Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.
  • Active and uncontrolled infections (e.g. sepsis, hepatitis B, hepatitis C).
  • Subjects infected with Human Immunodeficiency Virus (HIV).
  • Subjects with liver cirrhosis (as determined by the investigator).
  • Subjects receiving or planned to receive any prohibited medication.
  • Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to eltrombopag or excipient (microcrystalline cellulose, mannitol, polyvinylpyrrolidone, sodium starch glycolate, magnesium stearate, hypromellose, titanium dioxide, polyethylene glycol 400 and polysorbate 80) that contraindicates the subjects' participation.
  • In France, subjects who have participated in any study using an investigational drug during the previous 30 days.

Trial locations and contacts

120 sites in 22 countries.

120 sites in 22 countries

United States18 sites

  • Novartis Investigative Site

    Augusta, Georgia, 30912

  • Novartis Investigative Site

    Baltimore, Maryland, 21201

  • Novartis Investigative Site

    Boston, Massachusetts, 02115

  • Novartis Investigative Site

    Hackensack, New Jersey, 07601

  • Novartis Investigative Site

    Hot Springs, Arkansas, 71913

  • Novartis Investigative Site

    Jacksonville, Florida, 32256

  • Novartis Investigative Site

    Jonesboro, Arkansas, 72401

  • Novartis Investigative Site

    Kansas City, Missouri, 64128

  • Novartis Investigative Site

    Los Angeles, California, 90095

  • Novartis Investigative Site

    Milwaukee, Wisconsin, 53226

  • Novartis Investigative Site

    Orlando, Florida, 32806

  • Novartis Investigative Site

    Philadelphia, Pennsylvania, 19140

  • Novartis Investigative Site

    Phoenix, Arizona, 85016

  • Novartis Investigative Site

    Seattle, Washington, 98108

  • Novartis Investigative Site

    Stanford, California, 94305

  • Novartis Investigative Site

    The Bronx, New York, 10467

  • Novartis Investigative Site

    Voorhees Township, New Jersey, 08043

  • Novartis Investigative Site

    West Palm Beach, Florida, 33401

Other trials in United States

Brazil10 sites

  • Novartis Investigative Site

    Barretos, São Paulo, 14784-400

  • Novartis Investigative Site

    Barretos, São Paulo, 14784-400

  • Novartis Investigative Site

    Curitiba, Paraná, 81520-060

  • Novartis Investigative Site

    Curitiba, Paraná, 81520-060

  • Novartis Investigative Site

    Goiânia - GO, Goiás, 74605-020

  • Novartis Investigative Site

    Goiânia - GO, Goiás, 74605-020

  • Novartis Investigative Site

    Porto Alegre, Rio Grande do Sul, 90035-903

  • Novartis Investigative Site

    Rio de Janeiro, 20211-030

  • Novartis Investigative Site

    São Paulo, São Paulo, 01236030

  • Novartis Investigative Site

    São Paulo, 01236030

Other trials in Brazil

Israel9 sites

  • Novartis Investigative Site

    Beersheba, 84101

  • Novartis Investigative Site

    Haifa, 31048

  • Novartis Investigative Site

    Haifa, 31096

  • Novartis Investigative Site

    Holon, 58100

  • Novartis Investigative Site

    Jerusalem, 91120

  • Novartis Investigative Site

    Petah Tikva, 49100

  • Novartis Investigative Site

    Ramat Gan, 52621

  • Novartis Investigative Site

    Rehovot, 76100

  • Novartis Investigative Site

    Tel Aviv, 64239

Other trials in Israel

Spain9 sites

  • Novartis Investigative Site

    Barcelona, 08036

  • Novartis Investigative Site

    Granada

  • Novartis Investigative Site

    Málaga, 29010

  • Novartis Investigative Site

    Madrid, 28034

  • Novartis Investigative Site

    Málaga, 29010

  • Novartis Investigative Site

    Pozuelo de Alarcón/Madrid, 28223

  • Novartis Investigative Site

    Pozuelo de Alarcón/Madrid, 28223

  • Novartis Investigative Site

    Salamanca, 37007

  • Novartis Investigative Site

    Santander, 39008

Other trials in Spain

Belgium7 sites

  • Novartis Investigative Site

    Antwerp, 2060

  • Novartis Investigative Site

    Brasschaat, 2930

  • Novartis Investigative Site

    Bruges, 8000

  • Novartis Investigative Site

    Brussels, 1000

  • Novartis Investigative Site

    Ghent, 9000

  • Novartis Investigative Site

    Leuven, 3000

  • Novartis Investigative Site

    Yvoir, 5530

Other trials in Belgium

Germany6 sites

  • Novartis Investigative Site

    Cologne, North Rhine-Westphalia, 50937

  • Novartis Investigative Site

    Dresden, Saxony, 01307

  • Novartis Investigative Site

    Düsseldorf, North Rhine-Westphalia, 40225

  • Novartis Investigative Site

    Göttingen, Lower Saxony, 37075

  • Novartis Investigative Site

    Mannheim, Baden-Wurttemberg, 68167

  • Novartis Investigative Site

    Stuttgart, Baden-Wurttemberg, 70199

Other trials in Germany

Ireland6 sites

  • Novartis Investigative Site

    Cork

  • Novartis Investigative Site

    Dublin, 7

  • Novartis Investigative Site

    James Street, 8

  • Novartis Investigative Site

    Limerick

  • Novartis Investigative Site

    Tallaght, Dublin, 24

  • Novartis Investigative Site

    Tullamore

Other trials in Ireland

Italy6 sites

  • Novartis Investigative Site

    Alessandria, Piedmont, 15100

  • Novartis Investigative Site

    Bologna, Emilia-Romagna, 40138

  • Novartis Investigative Site

    Brescia, Lombardy, 25123

  • Novartis Investigative Site

    Florence, Tuscany, 50134

  • Novartis Investigative Site

    Milan, Lombardy, 20122

  • Novartis Investigative Site

    Milan, Lombardy, 20162

Other trials in Italy

Argentina5 sites

  • Novartis Investigative Site

    Buenos Aires, 1405

  • Novartis Investigative Site

    Buenos Aires, C1025ABH

  • Novartis Investigative Site

    Ciudad Autónoma de Buenos Aires, 1405

  • Novartis Investigative Site

    Ciudad Autonoma de Buenos Aires, Buenos Aires, C1431FWO

  • Novartis Investigative Site

    La Plata, Buenos Aires, B1900AXI

Other trials in Argentina

Hungary5 sites

  • Novartis Investigative Site

    Budapest, 1088

  • Novartis Investigative Site

    Debrecen, 4012

  • Novartis Investigative Site

    Kaposvár, 7400

  • Novartis Investigative Site

    Kaposvár, 7400

  • Novartis Investigative Site

    Szeged, 6720

Other trials in Hungary

Mexico5 sites

  • Novartis Investigative Site

    Chihuahua City, 31203

  • Novartis Investigative Site

    Mexico City, CP 14080

  • Novartis Investigative Site

    Monterrey, Nuevo León, 64460

  • Novartis Investigative Site

    Monterrey, Nuevo León, 64460

  • Novartis Investigative Site

    Oaxaca City, 68000

Other trials in Mexico

Russia5 sites

  • Novartis Investigative Site

    Nizhny Novgorod, 603126

  • Novartis Investigative Site

    Petrozavodsk, 185019

  • Novartis Investigative Site

    Saint Petersburg, 197 089

  • Novartis Investigative Site

    St'Petersburg, 191024

  • Novartis Investigative Site

    St'Petersburg, 197341

Other trials in Russia

Thailand5 sites

  • Novartis Investigative Site

    Bangkok, 10330

  • Novartis Investigative Site

    Bangkok, 10400

  • Novartis Investigative Site

    Bangkok, 10700

  • Novartis Investigative Site

    Khon Kaen, 40002

  • Novartis Investigative Site

    Songkhla, 90110

Other trials in Thailand

Czechia4 sites

  • Novartis Investigative Site

    Brno, 625 00

  • Novartis Investigative Site

    Prague, 100 34

  • Novartis Investigative Site

    Prague, 128 08

  • Novartis Investigative Site

    Prague, 128 20

Other trials in Czechia

Greece4 sites

  • Novartis Investigative Site

    Athens, 11527

  • Novartis Investigative Site

    Heraklion, Crete, 71201

  • Novartis Investigative Site

    Ioannina, 45 500

  • Novartis Investigative Site

    Thessaloniki, 57010

Other trials in Greece

Canada3 sites

  • Novartis Investigative Site

    Halifax, Nova Scotia, B3H 2Y9

  • Novartis Investigative Site

    Montreal, Quebec, H3T 1E2

  • Novartis Investigative Site

    Toronto, Ontario, M5G 2M9

Other trials in Canada

Poland3 sites

  • Novartis Investigative Site

    Chorzów, 41-500

  • Novartis Investigative Site

    Torun, 87-100

  • Novartis Investigative Site

    Warsaw, 02-097

Other trials in Poland

South Korea3 sites

  • Novartis Investigative Site

    Hwasun, 519-809

  • Novartis Investigative Site

    Seoul, 110-744

  • Novartis Investigative Site

    Seoul, 120-752

Other trials in South Korea

Taiwan3 sites

  • Novartis Investigative Site

    Kaohsiung City, 83301

  • Novartis Investigative Site

    Tainan County, 736

  • Novartis Investigative Site

    Taoyuan County, 333

Other trials in Taiwan

Hong Kong2 sites

  • Novartis Investigative Site

    Chai Wan

  • Novartis Investigative Site

    Shatin, New Territories

Other trials in Hong Kong

Netherlands1 site

  • Novartis Investigative Site

    Amsterdam, 1081 HV

Other trials in Netherlands

Puerto Rico1 site

  • Novartis Investigative Site

    San Juan, 00927

Other trials in Puerto Rico

About this study

Official title: A Three-part Study of Eltrombopag in Thrombocytopenic Subjects With Myelodysplastic Syndromes or Acute Myeloid Leukemia (Part 1: Open-label, Part 2: Randomized, Double-blind, Part 3: Extension)

This was a worldwide, three-part (Part 1: open-label, Part 2: randomized, double-blind, Part 3: extension), multi-center study to evaluate the effect of eltrombopag in subjects with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who have thrombocytopenia due to bone marrow insufficiency from their underlying disease or prior chemotherapy. This objective was assessed by a composite primary endpoint that consists of the following: the proportion of ≥Grade 3 hemorrhagic adverse events, or platelet counts <10 Gi/L, or platelet transfusions. Patients with MDS or AML and Grade 4 thrombocytopenia due to bone marrow insufficiency from their underlying disease or prior chemotherapy were enrolled in the study. No low or intermediate-1 risk MDS subjects were enrolled in the study. Subjects must have had at least one of the following during the 4 weeks prior to enrolment: platelet count <10 Gi/L, platelet transfusion, or symptomatic hemorrhagic event. Supportive standard of care (SOC), including hydroxyurea, was allowed as indicated by local practice throughout the study. The study had 3 sequential parts. Subjects who were enrolled in Part 1 (open-label) cannot be enrolled in Part 2 of the study (randomized, double-blind); however, subjects who completed the treatment period for Part 1 or Part 2 (8 and 12 weeks, respectively) continued in Part 3 (extension) if the investigator determined that the subject was receiving clinical benefit on treatment.

Being tested
eltrombopag, placebo
Keywords
acute myeloid leukemia (AML), acute myelogenous leukemia (AML), acute non lymphocytic leukemia (ANLL), myeloproliferative disease (MDS), myelodysplastic syndrome ( secondary acute myeloid leukemia), refractory acute myeloid leukemia
Medical subject headings
Thrombocytopenia, Leukemia, Myeloid, Acute, Myeloproliferative Disorders, Myelodysplastic Syndromes

What participants receive

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

  • eltrombopagDrug

    Also called Promacta

    100 mg daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg (150 mg for subjects of East Asian heritage)

  • placeboDrug

    100 mg matching placebo daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg matching placebo (150 mg for subjects of East Asian heritage)

Study groups in NCT01440374
Study groupRoleWhat they receive
Part 1, Open LabelExperimental100 mg daily (50 mg for subjects of East Asian heritage), intrasubject dose escalations to a maximum dose 300 mg (150 mg for subjects of East Asian heritage) are allowed.
Receives: Drug: eltrombopag
Part 2, eltrombopag armExperimental100 mg daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg (150 mg for subjects of East Asian heritage)
Receives: Drug: eltrombopag
Part 2, placebo armExperimental100 mg matching placebo daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg matching placebo (150 mg for subjects of East Asian heritage)
Receives: Drug: placebo
part 3 extensionExperimental100 mg daily (50 mg for subjects of East Asian heritage), intra-subject dose escalations to a maximum dose of 300 mg (150 mg for subjects of East Asian heritage)
Receives: Drug: eltrombopag

What is being measured

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

  1. Number of Participants With Platelet Response up to Week 8 During Part 1

    A participant was considered as a responder if he/she met the following response criteria: a Baseline platelet count <20 Giga cells per liter (Gi/L) and a post-Baseline increased to >20 Gi/L and at least 2 times the Baseline value; or a Baseline platelet count >=20 Gi/L and a post-Baseline absolute platelet count increased to >=50 Gi/L and at least 2 times the Baseline value. The response criteria was evaluated at each visit. Increase in platelet count observed up to 3 days after a platelet transfusion was not considered as a platelet response. The Part 1 Population was comprised of all participants enrolled into Part 1.

    Measured: From Baseline up to Week 8 during Part 1

  2. Clinically Relevant Thrombocytopenic Events (CRTE) From Week 5 up to Week 12 During Part 2

    A participant was considered to have a CRTE at a given assessment if he/she had platelet counts <10 Gi/L, or platelet transfusions, or >=Grade 3 hemorrhagic adverse events. CRTEs during Weeks 5 to 12 were compared between treatments using a generalized linear mixed model. Average of weekly proportion of subjects with CRTE during Week 5 to 12 was estimated for each treatment. Intent to Treat (ITT) Population was comprised of all randomized participants during Part 2.

    Measured: From Week 5 up to Week 12 during Part 2

Secondary outcomes (13)

  1. Plasma Eltrombopag Pharmacokinetic Concentration-Time Data - by Visit (Part 1 Subjects)

    Measured: Day 1 to week 8

  2. Plasma Eltrombopag Pharmacokinetic Concentration-Time Data - by Visit (Part 2 Subjects)

    Measured: Day 1 to week 12

  3. Mean Number of Platelet Transfusions

    Measured: Weeks 5 to 12

  4. Hematologic Improvement

    Definitions of hematologic improvement for platelets, neutrophils, and hemoglobin were based on modified International Working Group (IWG) consensus criteria.

    Measured: Weeks 5 to 12

  5. Change in Mean Platelet Count

    Measured: Baseline to Week 12

  6. Maximum Duration of Platelet Transfusion Independence

    Measured: Weeks 5 to 12

  7. Number of Participants With Maximum Bleeding Grade According to World Health Organization on Bleeding Scale

    Occurrence and severity of bleeding, measured using the WHO Bleeding Scale Grade 0=no bleeding; Grade 1=petechiae; Grade 2=mild blood loss; Grade 3=gross blood loss; Grade 4=debilitating blood loss.

    Measured: Weeks 5 to 12

  8. Independent Reviewer-Assessed Best Response

    Participants were evaluated in accordance with the modified International Working Group (Cheson, 2006). CR: Bone marrow blasts <5%, Hgb ≥11g/dL, Hematologic Improvement - Platelets (Baseline <20Gi/L: >20 Gi/L and 2x baseline; Baseline ≥20 Gi/L: ≥50 Gi/L and 2x baseline), Neutrophils ≥1.0 Gi/L, Peripheral blasts 0%. PR: Bone marrow blasts decreased by ≥50% but >5%, Peripheral blood as in CR. Marrow CR: Bone marrow blasts <5% and decrease by ≥50%, Note any Hematologic Improvements, Stable disease: Failure to achieve PR, but no evidence of progression for >8w, Cytogenetic response: Complete: disappearance of chromosomal abnormality; no new abnormalities Partial: ≥50% reduction of chromosomal abnormality.

    Measured: up to week 12

  9. Independent Reviewer Assessed Disease Progression

    Measured: Up to week 12

  10. Median Overall Survival

    Measured: Up to 13 months

  11. Summary of Health Outcomes

    The number of subject with medical resource utilization (MRU) data are reported in this table.

    MRU included number of emergency room visits, number of home healthcare visits, number of hospitalization days, number of medication or surgery specialist visits, number of procedures inpatient, number of procedures outpatient, number of non-study radiology visits, number of non-study laboratory visits, number of nurse practitioner/physician assistance/nurse visits, number of primary care physician visits, number of telephone consultations.

    Measured: week 12

  12. Functional Assessment of Cancer Therapy (FACT)

    The FACT-Th-18 is the most widely used and accepted tool evaluating health-related quality-of-life outcomes in cancer patients with chronically low platelets (where Th designates thrombocytopenia). The entire FACT-Th-18 was used in this trial, which includes the 18-item thrombocytopenia subscale used to assess the impact of symptoms, signs, and functional consequences of thrombocytopenia in MDS and AML subjects. The FACT-Th-18 is a validated and reliable instrument with known psychometric properties. FACT-ThS is an 18 item questionnaire specific to assessing the impact of symptoms, signs, and functional consequences of Thrombocytopenia. ThS score ranges from 0 to 72 with higher the score, the better the QoL. FACT G total score moves from 0 to 108 where higher the score, the better the HRQL.

    FACT-Th Total Score is calculated by adding the FACT-ThS and FACT-G score. Total score ranges from 0 to 180 and again, higher the score, the better the HRQL.

    Measured: Change from baseline, up to week 12

  13. EQ-5D Utility Score Analysis

    EuroQoL Five Dimensions Questionnaire (EQ-5D) is a standardized generic preference based health related quality of life instrument. It records how one's health is "today" and consists of a descriptive system. The Descriptive system is Comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. Each dimension on the EQ-5D involves a 3-point response scale which indicates the level of impairment (level 1 = no problem; level 2 = some or moderate problem(s) and level 3 = unable, or extreme problems). Level of problem reported in each EQ-5D dimension determines a unique health state which is converted into a weighted health state index by applying scores from EQ-5D preference weights elicited from general population samples. This generates a unique description of the subjects' health status, which is valued between 0 (representing death) and 1 (representing perfect health). Higher the score, the better the quality of life.

    EQ-ED is a score.

    Measured: Change from baseline, up to week 12

Timeline

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

  1. First posted26 Sep 2011 (estimated)
  2. Study startSep 2011
  3. Primary completionMar 2015
  4. Study completionDec 2015

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
Supportive care
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
162 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01440374
Sponsor's protocol ID
114968
Other IDs
2011-000114-19
Acronym
ASPIRE
Status last verified
May 2017
First submitted
15 Sep 2011
Last updated
5 Jun 2017
Data monitoring committee
Yes

Publications

  • Mittelman M, Platzbecker U, Grosicki S, Lawniczek T, Zhu Z, Selleslag D. Long-Term Safety and Efficacy of Eltrombopag for Advanced Myelodysplastic Syndromes or Acute Myeloid Leukemia and Severe Thrombocytopenia: Results of the ASPIRE Extension Study. Acta Haematol. 2023;146(5):373-378. doi: 10.1159/000531146. Epub 2023 May 19.

    PubMed 37231838

  • Mittelman M, Platzbecker U, Afanasyev B, Grosicki S, Wong RSM, Anagnostopoulos A, Brenner B, Denzlinger C, Rossi G, Nagler A, Garcia-Delgado R, Portella MSO, Zhu Z, Selleslag D. Eltrombopag for advanced myelodysplastic syndromes or acute myeloid leukaemia and severe thrombocytopenia (ASPIRE): a randomised, placebo-controlled, phase 2 trial. Lancet Haematol. 2018 Jan;5(1):e34-e43. doi: 10.1016/S2352-3026(17)30228-4. Epub 2017 Dec 11.

    PubMed 29241762

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing eltrombopag in people with Thrombocytopaenia, run by Novartis Pharmaceuticals at 120 sites in 22 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 15 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 5 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 120 sites in United States, Brazil, Israel, Spain, Belgium and 17 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. 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?

Yes. Results are posted in the registry (first posted 14 Jun 2016). 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?

Number of Participants With Platelet Response up to Week 8 During Part 1 (time frame: From Baseline up to Week 8 during Part 1). 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 NCT01440374, 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 5 Jun 2017.

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