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History of Changes for Study: NCT01731951
Imetelstat Sodium in Treating Patients With Primary or Secondary Myelofibrosis
Latest version (submitted August 24, 2021) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 November 18, 2012 None (earliest Version on record)
2 January 28, 2014 Recruitment Status, Study Status, Arms and Interventions, Contacts/Locations and Study Design
3 July 28, 2014 Sponsor/Collaborators and Study Status
4 September 23, 2014 Study Identification, Arms and Interventions, Outcome Measures, Study Status, Study Design, Sponsor/Collaborators, Study Description and Conditions
5 June 18, 2015 Arms and Interventions, Contacts/Locations, Study Status, Study Design, Study Identification, Outcome Measures, Sponsor/Collaborators, Eligibility, Conditions and Study Description
6 September 23, 2015 Recruitment Status, Study Status, Contacts/Locations and Study Design
7 November 10, 2015 Recruitment Status, Study Status, Contacts/Locations, Arms and Interventions, Study Design and Study Description
8 February 8, 2016 Study Status
9 May 2, 2016 Study Status
10 June 22, 2016 Study Status
11 October 5, 2016 Study Status
12 November 4, 2016 Study Status
13 December 2, 2016 Study Status
14 June 16, 2017 Study Status and Oversight
15 June 29, 2018 Study Status
16 July 19, 2018 Recruitment Status and Study Status
17 May 17, 2019 Study Status, Contacts/Locations, Sponsor/Collaborators, Study Identification
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Results Submission Events
18 August 24, 2021 Study Status, Arms and Interventions, More Information, Outcome Measures, Study Description, Document Section, Adverse Events, Baseline Characteristics, Participant Flow, Contacts/Locations, Eligibility, Study Design, Oversight and Study Identification
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Study NCT01731951
Submitted Date:  November 18, 2012 (v1)

Open or close this module Study Identification
Unique Protocol ID: MC1285
Brief Title: Imetelstat Sodium in Treating Patients With Primary or Secondary Myelofibrosis
Official Title: A Pilot Open-Label Study of the Efficacy and Safety of Imetelstat (GRN163L) in Patients With DIPSS_plus Intermediate-2 or High Risk Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), or Post-Essential Thrombocythemia Myelofibrosis (Post-ET-MF).
Secondary IDs: NCI-2012-02036 [Registry Identifier: CTRP (Clinical Trial Reporting Program)]
Open or close this module Study Status
Record Verification: November 2012
Overall Status: Recruiting
Study Start: October 2012
Primary Completion: March 2014 [Anticipated]
Study Completion:
First Submitted: November 18, 2012
First Submitted that
Met QC Criteria:
November 18, 2012
First Posted: November 22, 2012 [Estimate]
Last Update Submitted that
Met QC Criteria:
November 18, 2012
Last Update Posted: November 22, 2012 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Mayo Clinic
Responsible Party: Principal Investigator
Investigator: Ayalew Tefferi, M.D.
Official Title: Principal Investigator
Affiliation: Mayo Clinic
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: Yes
Open or close this module Study Description
Brief Summary: This pilot clinical trial studies how well imetelstat sodium works in treating patients with primary or secondary myelofibrosis. Imetelstat sodium may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth
Detailed Description:

PRIMARY OBJECTIVES:

I. To evaluate overall response rate.

SECONDARY OBJECTIVES:

I. To evaluate the safety and tolerability of imetelstat (imetelstat sodium) in myelofibrosis (MF) (per common terminology criteria for adverse events, version 4.03).

II. To evaluate the efficacy of imetelstat in the reduction of spleen size, as measured by physical examination (palpable distance from the left costal margin).

III. To evaluate the efficacy of imetelstat in inducing red blood cell transfusion-independence in previously transfusion-dependent patients (per International Working Group for Myelofibrosis Research and Treatment [IWG-MRT] criteria).

TERTIARY OBJECTIVES:

I. To evaluate the effect of imetelstat on bone marrow histology and karyotype. II. To evaluate the effect of imetelstat on leukocytosis and thrombocytosis.

OUTLINE: Patients receive imetelstat sodium intravenously (IV) over 2 hours on day 1. Treatment repeats every 21 days for up to 52 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 6 months for 5 years.

Open or close this module Conditions
Conditions: Primary Myelofibrosis
Secondary Myelofibrosis
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Not Applicable
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 29 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: Treatment (imetelstat sodium)
Patients receive imetelstat sodium IV over 2 hours on day 1. Treatment repeats every 21 days for up to 52 courses in the absence of disease progression or unacceptable toxicity.
Drug: imetelstat sodium
Given IV
Other Names:
  • GRN163L
  • telomerase inhibitor GRN163L
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Overall response rate defined as a clinical improvement (CI), partial remission (PR), or complete remission (CR) according to the IWG-MRT consensus criteria
[ Time Frame: Up to 27 weeks ]

Will be estimated by the number of successes divided by the total number of evaluable patients. Ninety-five percent Duffy Santner confidence intervals for the true success proportion will be calculated.
Secondary Outcome Measures:
1. Maximum grade for each type of adverse event for each patient
[ Time Frame: Up to 5 years ]

Frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.
2. Spleen response defined as either a minimum 50% reduction in palpable splenomegaly of a spleen that is at least 10 cm at baseline or a spleen that is palpable at more than 5 cm at baseline becomes not palpable
[ Time Frame: Up to 27 weeks ]

Will be calculated by the number of patients who achieve spleen response divided by the total number of evaluable patients with spleen involvement at baseline (estimated to be approximately 80% of patients). Ninety-five percent exact binomial confidence intervals for the true proportion of patients with spleen response will be calculated.
3. Proportion of patients achieving transfusion independence
[ Time Frame: Up to 27 weeks ]

Will be estimated by the number of patients who achieve transfusion independence divided by the total number of evaluable patients who were transfusion dependent at baseline Ninety-five percent exact binomial confidence intervals for the true proportion of patients who become transfusion independent will be calculated.
Open or close this module Eligibility
Minimum Age: 18 Years
Maximum Age:
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  • Diagnosis of one of the following:
    • PMF per the revised World Health Organization (WHO) criteria
    • Post-ET/PV MF per the IWG-MRT criteria
  • High-risk or Intermediate-2 risk MF (as defined by the Dynamic International Prognostic Scoring System [DIPSS-plus])
  • Life expectancy of >= 12 weeks
  • Able to provide informed consent and be willing to sign an informed consent form
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
  • Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) =< 2.5 x upper limit of normal (ULN) (or =< 5 x ULN if in the investigator's opinion the elevation is due to extramedullary hematopoiesis)
  • Serum glutamic pyruvate transaminase (SGPT) alanine aminotransferase (ALT) =< 2.5 x upper limit of normal (ULN) (or =<5 x ULN if in the investigator's opinion the elevation is due to extramedullary hematopoiesis)
  • Total bilirubin =< 3.0 mg/dL (or direct bilirubin < 1 mg/dL)
  • Creatinine =< 3.0 mg/dL
  • Absolute neutrophil count >= 1000/uL
  • Platelet count >= 50,000/uL
  • Absence of active treatment with systemic anticoagulation and a baseline prothrombin time (PT) and activated partial thromboplastin time (aPTT) that does not exceed 1.5 x ULN
  • Females of childbearing potential must have a negative pregnancy test =< 7 days prior to registration, unless they are surgically sterile for at least 3 months (i.e., hysterectomy), OR postmenopausal for at least 12 months (follicle-stimulating hormone [FSH] >30 U/mL)
  • Females of childbearing potential must agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through end of study; permitted methods for preventing pregnancy must be communicated to study subjects and their understanding confirmed
  • Males must agree to take appropriate precautions to avoid fathering a child (with at least 99% certainty) from screening through follow-up; permitted methods for preventing pregnancy should be communicated to the subjects and their understanding confirmed

Exclusion Criteria:

  • Females who are pregnant or are currently breastfeeding
  • Any chemotherapy (e.g., hydroxyurea), immunomodulatory drug therapy (e.g., thalidomide), immunosuppressive therapy, corticosteroids > 10 mg/day prednisone or equivalent, growth factor treatment (e.g., erythropoietin) or Janus kinase (JAK) inhibitor therapy =< 14 days prior to registration
  • Subjects with another active malignancy
    • Note: patients with early stage squamous cell carcinoma of the skin, basal cell carcinoma of the skin or cervical intraepithelial neoplasia are eligible for enrollment
  • Known positive status for human immunodeficiency virus (HIV)
  • Any unresolved toxicity greater or equal to grade 2 from previous anticancer therapy, except for stable chronic toxicities not expected to resolve
  • Incomplete recovery from any prior surgical procedures or had surgery =< 4 weeks prior to registration, excluding the placement of vascular access
  • Presence of acute active infection requiring antibiotics
  • Uncontrolled intercurrent illness or any concurrent condition that, in the Investigator's opinion, would jeopardize the safety of the patient or compliance with the protocol
Open or close this module Contacts/Locations
Study Officials: Ayalew Tefferi, M.D.
Study Chair
Mayo Clinic
Locations: United States, Minnesota
Mayo Clinic
[Recruiting]
Rochester, Minnesota, United States, 55905
Contact:Contact: Mayo Clinic Clinical Trials Referral Office 507-538-7623
Contact:Principal Investigator: Ayalew Tefferi, M.D.
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links:
Available IPD/Information:

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