Phase 2
N=80
Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis
Primary Myelofibrosis · Secondary Myelofibrosis · Myeloid Malignancies
Bottom Line
View on ClinicalTrials.gov: NCT01731951 ↗Enrolled (actual)
80
Serious AEs
85.0%
Results posted
Sep 2021
Primary outcome: Primary: MF Participants: Percentage of Participants With Overall Response (OR) - (Clinical Improvement[CI] or Partial Remission[PR] or Complete Remission[CR]) Per International Working Group - Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Criteria — 36.84; 35.7; 0; 33.3 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Imetelstat (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Geron Corporation
- Primary completion
- May 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY MF Participants: Percentage of Participants With Overall Response (OR) - (Clinical Improvement[CI] or Partial Remission[PR] or Complete Remission[CR]) Per International Working Group - Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Criteria |
36.84; 35.7; 0; 33.3 | — |
| PRIMARY Blastic MF/AML Participants: Percentage of Participants With Overall Response |
— | — |
| PRIMARY MDS Participants: Percentage of Participants With Overall Response (Hematologic Improvement [HI] or PR or CR) Per IWG Criteria |
33.3 | — |
| SECONDARY Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Grade >= 3 TEAEs and Treatment Related Adverse Events |
100; 100; 100; 100; 100; 100 | — |
| SECONDARY Number of Participants With Spleen Response Per IWG-MRT Criteria |
3; 1; 1; 0; 2 | — |
| SECONDARY MF and Blastic MF/AML Participants: Number of Participants With Transfusion Independence (CI by Anemia Response) Per IWG-MRT |
1; 0; 0; 0; 3 | — |
| SECONDARY MDS Participants: Number of Participants With Transfusion Independence (HI by Erythroid Response) Per IWG Criteria |
3 | — |
| SECONDARY MF Participants: Time to Response |
2.1; 1.4; 2.9 | — |
| SECONDARY Blastic MF/AML Participants: Time to Response |
— | — |
| SECONDARY MDS Participants: Time to Response |
3.7 | — |
| SECONDARY MF Participants: Duration of Response (DOR) Per IWG-MRT Criteria |
24.36; NA; 35.52 | — |
| SECONDARY Blastic MF/AML Participants: Duration of Response |
— | — |
| SECONDARY MDS Participants: Duration of Response Per IWG Criteria |
NA | — |
| SECONDARY Overall Survival (OS) |
42.61; 26.73; 4.93; 12.09; NA; 28.42 | — |
Summary
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.
Eligibility Criteria
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]) = =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 = 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.
Data sourced from ClinicalTrials.gov (NCT01731951). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.