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Myelodysplastic SyndromePhase 1NMDSG10ANCT01481220

Safety Study of Eltrombopag Combined With Azacitidine to Treat Myelodysplastic Syndrome (MDS)

A Phase 1 study testing Eltrombopag in people with Myelodysplastic Syndrome and Thrombocytopenia, run by Nordic MDS Group at 1 site in Sweden.

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

Patients with Myelodysplastic Syndromes (MDS) often suffer from low platelet levels which may lead to bleeding complications. Treatment with cytotoxic agents can decrease the platelet levels further. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndrome, Thrombocytopenia
What is being tested?
Eltrombopag
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteSweden
Study dates
Oct 2011to May 2013
Phase
Phase 1
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 advanced MDS or sAML/MDS requiring treatment with Azacitidine as approved by EMEA:
  • MDS classified as Intermediate 2-risk or high risk according to the international prognostic scoring system (IPSS) or
  • Chronic myelomonocytic leukaemia (CMML) with 10-29% bone marrow blasts without myeloproliferative disease or
  • Acute myeloblastic leukaemia (AML) with 20-30% bone marrow blasts with multilineage dysplasia according to the WHO classification.
  • Platelet counts < 75 x 109 /L at start of Azacitidine treatment.
  • Subjects must have platelet count and platelet transfusion data available over a period of 4 weeks prior to inclusion.
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  • During the 8 weeks prior to inclusion in study, subjects must have a baseline bone marrow examination including all of the following:
  • cytomorphology to confirm bone marrow blasts
  • cytogenetics
  • ECOG Status 0-2.
  • 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) </= 1.5xULN
  • ALT and AST </= 3xULN
  • creatinine </= 2.5 xULN
  • Subject is practicing an acceptable method of contraception (documented in CRF).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 1 of the following highly effective methods of contraception (i.e., Pearl Index < 1.0%) from 2 weeks prior to administration of study medication, throughout the study, and 28 days after completion or premature discontinuation from the study:
  • Complete abstinence from intercourse;
  • Intrauterine device (IUD);
  • Two forms of barrier contraception (diaphragm plus spermicide, and for males condom plus spermicide);
  • Male partner is sterile prior to entry into the study and is the only partner of the female;
  • Systemic contraceptives (combined or progesterone only).

Who cannot join

  • Subjects with a diagnosis of acute promyelocytic leukemia.
  • Patients with short life expectancy (less than 3 months)
  • Patients with bone marrow fibrosis that does not allow bone marrow aspiration (so-called "dry tap") or fibrosis grade II or III (grading according to European consensus on grading bone marrow fibrosis.
  • History of treatment for cancer other than MDS or sAML/MDS with systemic chemotherapy and/or radiotherapy within the last 2 years.
  • Patients with clinically significant splenomegaly, or > 16 cm spleen in length measured by ultrasound
  • Patients with known liver cirrhosis
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  • Patients with East Asian ancestry such as Chinese, Japanese, Taiwanese or Korean.
  • History of treatment with romiplostim or other TPO-R agonists.
  • subjects with a QTc > 450 msec (QTc > 480 msec for subjects with Bundle Branch Block).
  • 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.
  • 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 drug 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

1 site in 1 country.

Sweden1 site

  • 4 Locations

    Uppsala, Stockholm, Göteborg, Umeå

Other trials in Sweden

About this study

Official title: A Pilot Phase I Dose Finding Safety Study of a Thrombopoietin-receptor Agonist, Eltrombopag, in Patients With Myelodysplastic Syndrome Treated With Azacitidine

Patients with Myelodysplastic Syndromes (MDS) often suffer from low platelet levels which may lead to bleeding complications. Treatment with cytotoxic agents can decrease the platelet levels further. Eltrombopag is a relatively new drug that increases the platelet level in the blood by working directly on the bone marrow. It is available for treatment of the disease Immunological Thrombocytopenic Purpura (ITP). In this study patients with MDS and low platelet levels that are treated with the cytotoxic agent Azacitidine will also receive Eltrombopag. The administration of Eltrombopag to MDS patients treated with Azacitidine may result in less dose reductions and less treatment delays for Azacitidine and may reduce the need for thrombocyte transfusions and lower the risk of bleeding complications.

This is a phase I study, meaning that our major goal is to investigate the safety and tolerability for Eltrombopag in this patient group. It will also generate a basis for a phase II-III-study.

Being tested
Eltrombopag
Keywords
Myelodysplastic syndrome, azacitidine, Eltrombopag, Thrombopoietin-receptor agonist, Thrombocytopenia, Transfusion dependency, Safety study
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.

  • EltrombopagDrug

    In this study 4 different doses of oral eltrombopag (50mg, 100mg, 200mg and 300mg) will be tested. A modified 3+3 patient cohorts design will be used so no new patients are accepted to start on a higher dose without prior tolerance at the previous dose. Patients will be given eltrombopag once daily starting one week before the start of azacitidine treatment and then continue throughout the study, which duration will be approximately 3 months (three Azacytidine cycles). Patients will be evaluated continuously by clinical and laboratory assessments as well as bone marrow examinations during the treatment period until 4 weeks after discontinuation of Eltrombopag. Response, AEs/SAEs and DLTs will be monitored throughout the study.

Study groups in NCT01481220
Study groupRoleWhat they receive
Azacitidine + EltrombopagExperimentalNot described
Receives: Drug: Eltrombopag

What is being measured

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

  1. Safety and tolerability parameters

    Including:

    • Non-hematological clinical, laboratory Grade 3/Grade 4 toxicities
    • Change in bone marrow or peripheral blood blast counts from baseline
    • Adverse events and interactions at increasing doses of eltrombopag

    Measured: week 26

Secondary outcomes (4)

  1. Azacitidine treatment delays and dose reductions

    Measured: week 26

  2. Need for thrombocyte transfusions

    Measured: week 26

  3. Bleeding complications

    Measured: week 26

  4. Possible signs of antineoplastic effects (blood values and bone marrow picture)

    Measured: week 26

Timeline

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

  1. First posted29 Nov 2011 (estimated)
  2. Study startOct 2011
  3. Primary completionMay 2013
  4. Study completionMay 2013

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01481220
Sponsor's protocol ID
2010-023663-18
Acronym
NMDSG10A
Status last verified
May 2013
First submitted
17 Nov 2011
Last updated
3 May 2013
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Eltrombopag in people with Myelodysplastic Syndrome and Thrombocytopenia, run by Nordic MDS Group at 1 site in Sweden.

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 22 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 3 May 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Sweden. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Safety and tolerability parameters (time frame: week 26). 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 NCT01481220.

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 3 May 2013.

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