Clinical trial · Interventional
Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis
A Pilot Open-Label Study of the Efficacy and Safety of Imetelstat (GRN163L) in Myelofibrosis and Other Myeloid Malignancies
NCT01731951CI-TRIAL-00053984completedPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This pilot clinical trial studies how well imetelstat sodium works in treating participants with primary or secondary myelofibrosis and other myeloid malignancies. Imetelstat sodium may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Myeloid Malignancies | — | UNRESOLVED | — |
| Primary Myelofibrosis | Primary Myelofibrosis | ONTOLOGY_EXACT | 0.98 |
| Secondary Myelofibrosis | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Imetelstat | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (6)
- type
- EXPERIMENTAL
- label
- Arm A: Imetelstat 9.4 mg/kg (Myelofibrosis [MF])
- description
- Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 milligram per kilogram (mg/kg), intravenously (IV) as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
- interventionNames
- Drug: Imetelstat
- type
- EXPERIMENTAL
- label
- Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)
- description
- Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Diagnosis of one of the following: * Primary myelofibrosis (PMF) per the revised World Health Organization (WHO) criteria. * Post-polycythemia vera/essential thrombocythemia myelofibrosis (Post-ET/PV MF) per the International Working Group for Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria. * High-risk or Intermediate-2 risk MF (as defined by the Dynamic International Prognostic Scoring System \[DIPSS-plus\]). * Life expectancy of greater than or equal to (\>=) 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 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/microliter (mcL). * Platelet count \>=50,000/mcL. * 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 participants 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 participants 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. * Participants with another active malignancy. * Note: participants 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 participant or compliance with the protocol.
References
Publications (1)
- DERIVEDTefferi A, Lasho TL, Begna KH, Patnaik MM, Zblewski DL, Finke CM, Laborde RR, Wassie E, Schimek L, Hanson CA, Gangat N, Wang X, Pardanani A. A Pilot Study of the Telomerase Inhibitor Imetelstat for Myelofibrosis. N Engl J Med. 2015 Sep 3;373(10):908-19. doi: 10.1056/NEJMoa1310523. PMID 26332545