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Essential ThrombocythemiaPhase 2ET/PVNCT01243073

Open Label Study to Evaluate the Activity of Imetelstat in Patients With Essential Thrombocythemia or Polycythemia Vera

A Phase 2 study testing Imetelstat in people with Essential Thrombocythemia and Polycythemia Vera, run by Geron Corporation at 12 sites in 3 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

This is a phase II open-label study of single agent imetelstat in patients with essential thrombocytopenia or with polycythemia vera who have failed or are intolerant to at least one prior therapy, or who refuse standard therapy. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Essential Thrombocythemia, Polycythemia Vera
What is being tested?
Imetelstat
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
12 sitesin 3 countries
Study dates
Dec 2010to Apr 2015
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

  • ET-Specific Criteria
  • Confirmed diagnosis of ET by WHO criteria
  • Patients with ET requiring cytoreduction who have failed or are intolerant to at least one prior therapy, or who refuse standard therapy
  • Laboratory criteria (within 14 days of first study drug administration):
  • Platelets > 600,000/μL
  • ANC ≥ 1500/μL
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  • Hemoglobin ≥ 10 g/dL
  • PV-Specific Criteria
  • Confirmed diagnosis of PV by WHO criteria
  • Patients with PV requiring cytoreduction with phlebotomy and/or myelosuppressive agents
  • Patients may have failed or are intolerant to at least one prior therapy, or refuse standard therapy
  • For those patients receiving phlebotomy only, the frequency over the past year must be at least one phlebotomy every 3 months.
  • Undergone phlebotomy and attained a Hct < 47% (men) or < 45% (women) (or pre-specified Hct count that is tolerable) within 14 days prior to the start of study treatment
  • Cessation of myelosuppressive agents prior to initiation of study treatment (unless approved by Geron Medical Monitor for unusual circumstances)
  • Hydroxyurea or anagrelide: Cessation 1 day prior to initiation of study treatment. Consideration to the timing of cessation of this therapy prior to the start of study treatment should take into account the requirement for phlebotomy.
  • INF-α or pegylated- INF-α: Cessation 4 weeks prior to initiation of study treatment
  • Laboratory criteria (within 14 days of first study drug administration):
  • Platelets > the lower limit of normal (LLN)
  • ANC ≥ 1500/μL
  • General Criteria (All Patients)
  • Willing and able to sign an informed consent
  • Male or female, aged 18 years or older
  • ECOG performance status 0-2
  • Laboratory criteria (within 14 days of first study drug administration):
  • INR (or PT) and aPTT < 1.5 x the upper limit of normal (ULN)
  • Serum creatinine ≤ 2 mg/dL
  • Serum bilirubin < 2.0 mg/dL (patients with Gilbert's syndrome: serum bilirubin < 3 x ULN)
  • AST (SGOT) and ALT (SGPT) ≤ 2.5 x ULN
  • Alkaline phosphatase < 2.5 x ULN
  • Any clinically significant toxicity from previous cancer treatments and/or major surgery must have recovered to Grade 0-1 prior to initiation of study treatment
  • Women of childbearing potential must have a negative pregnancy test and agree to use effective birth control during and for at least 12 weeks after the last study treatment with imetelstat
  • Male patients must agree to use effective birth control for themselves or their partner during and for 12 weeks after the last study treatment with imetelstat.

Who cannot join

  • Patients who meet any of the following criteria will be excluded from screening and study entry:
  • Women who are pregnant or breast feeding
  • Prior stem cell transplantation
  • Investigational therapy within 4 weeks prior to first study drug administration
  • Clinically significant cardiovascular disease or condition including:
  • Uncontrolled congestive heart failure (CHF)
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  • Need for anti-arrhythmic therapy for a ventricular arrhythmia
  • Clinically significant severe conduction disturbance per the Investigator's discretion
  • Ongoing angina pectoris requiring therapy
  • New York Heart Association (NYHA) Class II, III, or IV cardiovascular disease (see Appendix E)
  • Known positive serology for human immunodeficiency virus (HIV)
  • Serious co-morbid medical conditions, including active or chronically recurrent bleeding, clinically relevant active infection, cirrhosis, and chronic obstructive or chronic restrictive pulmonary disease per the Investigator's discretion
  • Any other severe, acute, or chronic medical or psychiatric condition, laboratory abnormality, or difficulty complying with protocol requirements that may increase the risk associated with study participation or study drug administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the patient inappropriate for this study.

Trial locations and contacts

12 sites in 3 countries.

12 sites in 3 countries

United States6 sites

  • Johns Hopkins University - Bunting Blaustein Cancer Research Building

    Baltimore, Maryland, 21205

  • University of Chicago

    Chicago, Illinois, 60637

  • City of Hope

    Duarte, California, 91010

  • Saint Francis Hospital

    Greenville, South Carolina, 29601

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • MDACC - Orlando

    Orlando, Florida, 32806

Other trials in United States

Germany5 sites

  • University Hospital of Essen - West German Cancer Center

    Essen

  • Medizinische Klinik II, Abt. Hämatologie und Onkologie - Johann Wolfgang Goethe Universität

    Frankfurt, D-60590

  • SLK-Kliniken GmbH

    Heilbronn

  • Hematology Oncology Center - Ludwig-Maximilians, University Munich Medical School

    Munich, 80331

  • University Hospital Regensburg - Uniklinik Regensburg

    Regensburg

Other trials in Germany

Switzerland1 site

  • INSELSPITAL, University Hospital Bern

    Bern, CH - 3010

Other trials in Switzerland

About this study

Official title: A Phase II Trial to Evaluate the Activity of Imetelstat (GRN163L) in Patients With Essential Thrombocythemia or Polycythemia Vera Who Require Cytoreduction and Have Failed or Are Intolerant to Previous Therapy, or Who Refuse Standard Therapy

This is a phase II open-label study of single agent imetelstat in patients with essential thrombocytopenia or with polycythemia vera who have failed or are intolerant to at least one prior therapy, or who refuse standard therapy.

Read the detailed description

For patients with ET: To obtain a preliminary estimate of efficacy of imetelstat, as measured by best hematologic response within the first year of therapy in patients with ET who have failed or are intolerant to at least one prior therapy, or who have refused standard therapy.

For patients with PV: To obtain a preliminary estimate of efficacy of imetelstat, as measured by maintenance of Hct < 45% in men and < 42% in women (or pre-specified Hct count that is tolerable) without phlebotomy or myelosuppressive therapy within the first year of therapy in patients with PV who have failed or are intolerant to at least one prior therapy, or who have refused standard therapy.

Being tested
Imetelstat
Keywords
imetelstat, imetelstat sodium, GRN163L, telomerase inhibitor, telomerase, essential thrombocytosis, essential thrombocythemia, ET, polycythemia vera, PV
Medical subject headings
Thrombocythemia, Essential, Polycythemia Vera

What participants receive

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

  • ImetelstatDrug

Study groups in NCT01243073
Study groupRoleWhat they receive
imetelstatExperimentalInduction dosing of 9.4 mg/kg weekly, followed by intermittent maintenance dosing.
Receives: Drug: Imetelstat

What is being measured

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

  1. Hematologic Response

    Primary objectives are as follows: ET patients - best hematologic response within the first year of therapy and PV patients - maintenance of Hct < 45% in men and < 42% in women (or pre-specified Hct count that is tolerable) without phlebotomy or myelosuppressive therapy within the first year of therapy. Secondary objectives, to determine the durability of hematologic response and to determine the rate of phlebotomy required within the first year of therapy.

    Measured: From time of first dose (cycle 1 day 1) through end of study (12 mos after last participant is dosed)

Secondary outcomes (1)

  1. Safety and tolerability: Number of Patients with Hematological Toxicities, Non-Heme Grade 3 and 4 AEs, and Hemorrhagic Events

    The safety and tolerability of imtelstat will be assessed by the incidence, nature, relatedness and severity of adverse events, laboratory abnormalities and vital signs.

    Measured: From time of first dose (cycle 1 day 1) through end of study (12 mos after last paricipant is dosed)

Timeline

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

  1. First posted18 Nov 2010 (estimated)
  2. Study startDec 2010
  3. Primary completionOct 2013
  4. Study completionApr 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
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01243073
Sponsor's protocol ID
CP14B015
Acronym
ET/PV
Status last verified
Dec 2015
First submitted
11 Nov 2010
Last updated
26 Jan 2016
Results posted
No
Data monitoring committee
No

Publications

  • Oppliger Leibundgut E, Haubitz M, Burington B, Ottmann OG, Spitzer G, Odenike O, McDevitt MA, Roth A, Snyder DS, Baerlocher GM. Dynamics of mutations in patients with essential thrombocythemia treated with imetelstat. Haematologica. 2021 Sep 1;106(9):2397-2404. doi: 10.3324/haematol.2020.252817.

    PubMed 32732354

  • Baerlocher GM, Oppliger Leibundgut E, Ottmann OG, Spitzer G, Odenike O, McDevitt MA, Roth A, Daskalakis M, Burington B, Stuart M, Snyder DS. Telomerase Inhibitor Imetelstat in Patients with Essential Thrombocythemia. N Engl J Med. 2015 Sep 3;373(10):920-8. doi: 10.1056/NEJMoa1503479.

    PubMed 26332546

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Imetelstat in people with Essential Thrombocythemia and Polycythemia Vera, run by Geron Corporation at 12 sites in 3 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 32 inclusion and 13 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 26 Jan 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United States, Germany, Switzerland. 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?

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?

Hematologic Response (time frame: From time of first dose (cycle 1 day 1) through end of study (12 mos after last participant is dosed)). 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 NCT01243073, 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 26 Jan 2016.

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