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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


Phase 2
18 Years
N/A
Open (Enrolling)
Both
Essential Thrombocythemia, Polycythemia Vera

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Trial Information

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


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.


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.

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Hematologic Response

Outcome Description:

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.

Outcome Time Frame:

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

Safety Issue:

No

Principal Investigator

Stephen Kelsey, MD FRCP FRCPath

Investigator Role:

Study Director

Investigator Affiliation:

Geron Corporation

Authority:

United States: Food and Drug Administration

Study ID:

CP14B015

NCT ID:

NCT01243073

Start Date:

December 2010

Completion Date:

January 2016

Related Keywords:

  • Essential Thrombocythemia
  • Polycythemia Vera
  • imetelstat
  • imetelstat sodium
  • GRN163L
  • telomerase inhibitor
  • telomerase
  • essential thrombocytosis
  • essential thrombocythemia
  • ET
  • polycythemia vera
  • PV
  • Polycythemia
  • Polycythemia Vera
  • Thrombocythemia, Essential
  • Thrombocytosis

Name

Location

MD Anderson Cancer CenterHouston, Texas  77030-4096
City of HopeDuarte, California  91010
University of ChicagoChicago, Illinois  60637
Saint Francis HospitalGreenville, South Carolina  29601
MDACC - OrlandoOrlando, Florida  32806
Johns Hopkins University - Bunting Blaustein Cancer Research BuildingBaltimore, Maryland  21205
Mount Sinai School of Medicine - Tisch Cancer InstituteNew York, New York  10029