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Open Label Study to Evaluate the Activity of Imetelstat in Patients With Essential Thrombocythemia or Polycythemia Vera (ET/PV)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Geron Corporation
ClinicalTrials.gov Identifier:
NCT01243073
First received: November 11, 2010
Last updated: November 11, 2014
Last verified: November 2014

November 11, 2010
November 11, 2014
December 2010
January 2014   (final data collection date for 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) ] [ Designated as safety issue: No ]
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.
Hematologic Response [ Time Frame: From time of first dose (cycle 1 day 1) through end of study (12 mos after last participant is dosed) ] [ Designated as safety issue: No ]
The primary objective of this study is to obtain a preliminary estimate of efficacy of imetelstat, as measured by hematologic response - defined by the Modified European Leukemianet Consensus Conference Response Criteria, in patients with essential thrombocythemia (ET) who have failed or are intolerant to at least one prior therapy, or who have refused standard therapy. Secondly, the study will evaluate the durability of the hematologic response.
Complete list of historical versions of study NCT01243073 on ClinicalTrials.gov Archive Site
Safety and tolerability: Number of Patients with Hematological Toxicities, Non-Heme Grade 3 and 4 AEs, and Hemorrhagic Events [ Time Frame: From time of first dose (cycle 1 day 1) through end of study (12 mos after last paricipant is dosed) ] [ Designated as safety issue: Yes ]
The safety and tolerability of imtelstat will be assessed by the incidence, nature, relatedness and severity of adverse events, laboratory abnormalities and vital signs.
Same as current
Not Provided
Not Provided
 
Open Label Study to Evaluate the Activity of Imetelstat in Patients With Essential Thrombocythemia or Polycythemia Vera
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.

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.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Essential Thrombocythemia
  • Polycythemia Vera
Drug: Standard of Care
Standard of care
Experimental: imetelstat
Induction dosing of 9.4 mg/kg weekly, followed by intermittent maintenance dosing.
Intervention: Drug: Standard of Care
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
40
June 2015
January 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

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
    • 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.

Exclusion Criteria

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)
    • 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.
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Germany,   Switzerland
 
NCT01243073
CP14B015
No
Geron Corporation
Geron Corporation
Not Provided
Not Provided
Geron Corporation
November 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP