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Trial · NCT01731951

Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis

NCT01731951, Phase 2, Completed, Geron Corporation; results first posted 2021-09-21

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis. Trials, record NCT01731951. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01731951

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title
Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis
NCT number
NCT01731951
Status
Completed
Phases
Phase 2
Enrollment
80 participants, actual
Lead sponsor
Geron Corporation
Conditions
Primary Myelofibrosis, Secondary Myelofibrosis, Myeloid Malignancies
Interventions
Imetelstat
First posted
2012-11-22
Last updated
2021-09-21
Results first posted
2021-09-21

Source ClinicalTrials.gov

Registered outcomes14

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

ListOutcomeTime frameDescription
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
Up to first 9 cycles of treatment (each cycle was of 21 days for Arms A and B and 28 days for Arms E and F)
OR:CI/PR/CR per IWG-MRT. CR:Bone marrow (BM):\<5% blasts;≤Grade 1 MF, AND Peripheral blood:Hemoglobin (Hb) ≥100 g/liter (g/L) and \<upper normal limit (UNL);Neutrophil count(NC) ≥1x10\^9/L and \<UNL; Platelet(PLT) count ≥100x10\^9/L and \<UNL;\<2% immature myeloid cells(IMCs), AND Clinical: Resolution of disease symptoms; Spleen and liver not palpable; No evidence of extramedullary hematopoeisis(EMH). PR:All CR criteria plus peripheral blood: Hb≥85 but \<100g/L and \<UNL; NC ≥1x10\^9/L and \<UNL; PLT count ≥50 but \<100x10\^9/L and \<UNL;\<2%IMCs. CI:achievement of anemia (≥20 g/L increase in Hb level), spleen(baseline splenomegaly palpable at 5-10 cm, below left costal margin(LCM), becomes not palpable), symptoms response (50% reduction in total symptom score) without progressive disease (PD) (appearance of new splenomegaly- palpable at least \[≥\]5 cm below LCM)/increase in severity of anemia, thrombocytopenia/neutropenia. Data is reported separately for arms whose response was assessed per IWG-MRT.
PrimaryBlastic MF/AML Participants: Percentage of Participants With Overall ResponseUp to first 9 cycles of treatment (each cycle was of 28 days for Arm D)
For this pilot study, overall response was defined as achievement of a leukemic response (i.e. a clinically meaningful reduction in Blast cells). This was quantified as \<5% peripheral blood and bone marrow blasts % that lasts for at least two months. Data is reported separately for this arm where response was assessed by this specific criterion.
PrimaryMDS Participants: Percentage of Participants With Overall Response (Hematologic Improvement [HI] or PR or CR) Per IWG CriteriaUp to first 9 cycles of treatment (each cycle was of 28 days for Arm G)
OR: HI/PR/CR per IWG. CR: BM:≤5% myeloblasts (all cell lines normal maturation), Peripheral blood:Hgb ≥11g/dL, PLTs ≥100x10\^9/L, Neutrophils ≥1.0x10\^9/L, Blasts 0%. PR: All CR criteria if abnormal before treatment except- BM blasts decreased ≥50% over pretreatment but still \>5%, Cellularity, morphology not relevant. HI responses:1) Erythroid: Hgb increase ≥1.5 g/dL, Relevant reduction of red blood cell(RBC) units transfusions by absolute ≥4 RBC transfusions/8 week (wk) compared with pretreatment transfusion number in previous 8 wk. Only RBC transfusions given for Hgb of ≤9.0 g/dL.2)PLTs:Absolute increase ≥30x10\^9/L starting \>20x10\^9/L PLTs; Increase from \<20x10\^9/L to \>20x10\^9/L and by ≥100%; 3)Neutrophil: ≥100% increase, absolute increase \>0.5x10\^9/L; 4)Progression/relapse after HI:≥1 of following:≥50% decrement from maximum response levels in granulocytes/PLTs, Reduction in Hgb ≥1.5 g/dL,Transfusion dependence. Data is reported separately for arm whose response was assessed per IWG.
SecondaryPercentage of Participants With Treatment Emergent Adverse Events (TEAEs), Grade >= 3 TEAEs and Treatment Related Adverse EventsFrom first dose of study drug up to 30 days from the last dose of study drug or until subsequent anti-cancer therapy if earlier (Up to approximately 5.7 years)
TEAEs defined as those events that 1) occur on or after the first dose of study drug, through the treatment phase, and for 30 days following the last dose of study drug or until subsequent anti-cancer therapy if earlier; 2) any event that is considered study drug-related regardless of the start date of the event; or 3) any event that is present at baseline but worsens in severity or is subsequently considered drug-related by the investigator. Grade \>=3 TEAE defined as events that are severe, life-threatening or disabling, or fatal and considered related to imetelstat as per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.03. An AE was considered related to the study drug if the event was assessed by the investigator as probably or possibly related.
SecondaryNumber of Participants With Spleen Response Per IWG-MRT CriteriaUp to approximately 5.7 years
Spleen response per IWG-MRT criteria defined as baseline splenomegaly that is palpable at 5-10 cm, below the LCM, becomes not palpable, OR A baseline splenomegaly that is palpable at \>10 cm, below the LCM, decreases by ≥50%. Baseline splenomegaly that is palpable at \<5 cm, below the LCM, is not eligible for spleen response. Confirmation by Magnetic resonance imaging (MRI) or computerized tomography (CT) showing ≥35% spleen volume reduction is recommended (but not required). Spleen response was assessed only in participants with MF.
SecondaryMF and Blastic MF/AML Participants: Number of Participants With Transfusion Independence (CI by Anemia Response) Per IWG-MRTUp to approximately 5.7 years
Transfusion dependency was defined by a history of at least 2 units of red blood cell transfusions in the last month for a hemoglobin level of less than 85 g/L that was not associated with clinically overt bleeding. A participant who was transfusion dependent at baseline was considered transfusion independent if the participant met either of the following conditions: Received no more than 1 unit of red blood cell transfusions in a rolling time interval of 30 days or more, and had a hemoglobin level increase over baseline more than 2 g/dL if participant baseline hemoglobin level was less than 85 g/L and received no transfusion. Anemia response per IWG-MRT Criteria- Transfusion-independent participants: a ≥20 g/L increase in hemoglobin level. Transfusion-dependent participants: becoming transfusion-independent. Data is reported separately for arms assessed as per the IWG-MRT criteria.
SecondaryMDS Participants: Number of Participants With Transfusion Independence (HI by Erythroid Response) Per IWG CriteriaUp to approximately 5.7 years
Transfusion dependency was defined by a history of at least 2 units of red blood cell transfusions in the last month for a hemoglobin level of less than 85 g/L that was not associated with clinically overt bleeding. A participant who was transfusion dependent at baseline was considered transfusion independent if the participant met either of the following conditions: Received no more than 1 unit of red blood cell transfusions in a rolling time interval of 30 days or more, and had a hemoglobin level increase over baseline more than 2 g/dL if participant baseline hemoglobin level was less than 85 g/L and received no transfusion. HI responses included: 1) Erythroid: Hgb increase ≥1.5 g/dL, Relevant reduction of RBC units transfusions by absolute number of \>=4 RBC transfusions/8 week compared with pretreatment transfusion number in previous 8 week. Only RBC transfusions given for Hgb of ≤9.0 g/dL. Data is reported separately for arm assessed as per IWG criteria.
SecondaryMF Participants: Time to ResponseFrom study Day 1 to the earliest date that a response was first documented (Up to approximately 5.7 years)Time to response was defined as the duration from study Day 1 to the earliest date that a response is first documented. The response CI/CR/PR was assessed by IWG-MRT criteria.
SecondaryBlastic MF/AML Participants: Time to ResponseFrom study Day 1 to the earliest date that a response was first documented (Up to approximately 5.7 years)
Time to response was defined as the duration from study Day 1 to the earliest date that a response is first documented. Response was defined as achievement of a leukemic response (i.e. a clinically meaningful reduction in Blast cells). This was quantified as \<5% peripheral blood and bone marrow blasts % that lasts for at least two months. Data for time to response is reported as per criteria of response assessment.
SecondaryMDS Participants: Time to ResponseFrom study Day 1 to the earliest date that a response was first documented (Up to approximately 5.7 years)
Time to response was defined as the duration from study Day 1 to the earliest date that a response is first documented. Response was defined as HI, PR or CR per IWG criteria. Data for time to response is reported as per criteria of response assessment.

Outcomes14

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

TypeOutcomeTime framePopulationUnitsMeasured as
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
Up to first 9 cycles of treatment (each cycle was of 21 days for Arms A and B and 28 days for Arms E and F)
Eligible Analysis Set included subset of all participants who received at least one dose of study drug that excludes ineligible participants who did not satisfy each and every eligibility criteria for study entry. Data is reported for arm groups (Arms A, B, E and F) whose overall response was assessed by IWG-MRT criteria.
percentage of participantsNUMBER
PRIMARYBlastic MF/AML Participants: Percentage of Participants With Overall ResponseUp to first 9 cycles of treatment (each cycle was of 28 days for Arm D)
All Treated Analysis Set included all participants who received at least one dose of study drug. Data is reported for Arm D: Imetelstat 9.4 mg/kg (Blastic MF/AML) participants whose overall response was assessed by a specific criterion.
percentage of participantsNUMBER
PRIMARYMDS Participants: Percentage of Participants With Overall Response (Hematologic Improvement [HI] or PR or CR) Per IWG CriteriaUp to first 9 cycles of treatment (each cycle was of 28 days for Arm G)
All Treated Analysis Set included all participants who received at least one dose of the study drug. Data is reported for Arm G: Imetelstat 9.4 mg/kg (MDS/MPN or MDS and spliceosome mutations or ring sideroblasts) participants whose overall response was assessed by IWG criteria.
percentage of participantsNUMBER
SECONDARYPercentage of Participants With Treatment Emergent Adverse Events (TEAEs), Grade >= 3 TEAEs and Treatment Related Adverse EventsFrom first dose of study drug up to 30 days from the last dose of study drug or until subsequent anti-cancer therapy if earlier (Up to approximately 5.7 years)All Treated Analysis Set included all participants who received at least one dose of imetelstat.percentage of participantsNUMBER
SECONDARYNumber of Participants With Spleen Response Per IWG-MRT CriteriaUp to approximately 5.7 years
Arms A, B, E and F: Eligible Analysis Set included subset of all participants who received at least one dose of study drug that excludes ineligible participants who did not satisfy each and every eligibility criteria for study entry. Arm D: All Treated Analysis Set included all participants who received at least one dose of imetelstat.
ParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYMF and Blastic MF/AML Participants: Number of Participants With Transfusion Independence (CI by Anemia Response) Per IWG-MRTUp to approximately 5.7 years
Arms A, B, E, F: Eligible Analysis Set included subset of all participants who received at least one dose of study drug that excludes ineligible participants who did not satisfy each and every eligibility criteria for study entry. Arm D: All Treated Analysis Set included all participants who received at least one dose of imetelstat, assessed per IWG-MRT.
ParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYMDS Participants: Number of Participants With Transfusion Independence (HI by Erythroid Response) Per IWG CriteriaUp to approximately 5.7 years
All Treated Analysis Set included all participants who received at least one dose of study drug. Data is reported for Arm G: Imetelstat 9.4 mg/kg (MDS/MPN or MDS and spliceosome mutations or ring sideroblasts) participants whose transfusion independence (HI by erythroid response) was assessed by IWG criteria.
ParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYMF Participants: Time to ResponseFrom study Day 1 to the earliest date that a response was first documented (Up to approximately 5.7 years)
Eligible Analysis Set included subset of all participants who received at least one dose of study drug that excludes ineligible participants who did not satisfy each and every eligibility criteria for study entry. Data is reported for arm groups (Arms A, B, E and F) whose overall response was assessed by IWG-MRT criteria. Only responders were analyzed for this outcome measure.
monthsMEDIAN
SECONDARYBlastic MF/AML Participants: Time to ResponseFrom study Day 1 to the earliest date that a response was first documented (Up to approximately 5.7 years)
All Treated Analysis Set included all participants who received at least one dose of the study drug. Only responders were analyzed for this outcome measure. Data is reported for Arm D: Imetelstat 9.4 mg/kg (Blastic MF/AML) participants whose overall response was assessed by a specific criterion.
SECONDARYMDS Participants: Time to ResponseFrom study Day 1 to the earliest date that a response was first documented (Up to approximately 5.7 years)
All Treated Analysis Set included all participants who received at least one dose of the study drug. Only responders were analyzed for this outcome measure. Data is reported for Arm G: Imetelstat 9.4 mg/kg (MDS/MPN or MDS and spliceosome mutations or ring sideroblasts) participants whose overall response was assessed by IWG criteria.
monthsMEDIAN

Results by group49

Each measurement the trial posted, group by group, in the words and numbers it posted.

OutcomeGroupCategoryValueSpreadUnits
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
Arm A: Imetelstat 9.4 mg/kg (MF)36.8495% Confidence Interval 15.2 to 58.5percentage of participants
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
Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)35.795% Confidence Interval 10.6 to 60.8percentage of participants
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
Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts])095% Confidence Interval NA to NApercentage of participants
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
Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])33.395% Confidence Interval 11.6 to 55.1percentage of participants
Blastic MF/AML Participants: Percentage of Participants With Overall ResponseArm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia)095% Confidence Interval NA to NApercentage of participants
MDS Participants: Percentage of Participants With Overall Response (Hematologic Improvement [HI] or PR or CR) Per IWG CriteriaArm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)33.395% Confidence Interval 2.5 to 64.1percentage of participants
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Grade >= 3 TEAEs and Treatment Related Adverse EventsArm A: Imetelstat 9.4 mg/kg (MF)TEAEs100percentage of participants
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Grade >= 3 TEAEs and Treatment Related Adverse EventsArm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)TEAEs100percentage of participants
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Grade >= 3 TEAEs and Treatment Related Adverse EventsArm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia)TEAEs100percentage of participants
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Grade >= 3 TEAEs and Treatment Related Adverse EventsArm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts])TEAEs100percentage of participants

Participant flow18

How many started, completed or left each group, period by period.

PeriodMilestoneGroupParticipantsUnits counted
Overall StudySTARTEDArm A: Imetelstat 9.4 mg/kg (MF)19
Overall StudySTARTEDArm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)16
Overall StudySTARTEDArm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia)9
Overall StudySTARTEDArm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts])9
Overall StudySTARTEDArm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])18
Overall StudySTARTEDArm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)9
Overall StudyCOMPLETEDArm A: Imetelstat 9.4 mg/kg (MF)2
Overall StudyCOMPLETEDArm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)1
Overall StudyCOMPLETEDArm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia)0
Overall StudyCOMPLETEDArm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts])0

Adverse events510

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

TypeEventOrgan systemGroupAffectedAt riskEvents
otherAnaemiaBlood and lymphatic system disordersArm A: Imetelstat 9.4 mg/kg (MF)1619
otherAnaemiaBlood and lymphatic system disordersArm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)916
otherAnaemiaBlood and lymphatic system disordersArm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia49
otherAnaemiaBlood and lymphatic system disordersArm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts])69
otherAnaemiaBlood and lymphatic system disordersArm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])1118
otherAnaemiaBlood and lymphatic system disordersArm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)79
otherAtrial fibrillationCardiac disordersArm A: Imetelstat 9.4 mg/kg (MF)219
otherAtrial fibrillationCardiac disordersArm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)016
otherAtrial fibrillationCardiac disordersArm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia19
otherAtrial fibrillationCardiac disordersArm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts])09

Adverse event totals18

The totals the trial posted for each group.

TotalGroupAffectedAt risk
Total, all-cause mortalityArm A: Imetelstat 9.4 mg/kg (MF)1319
Total, all-cause mortalityArm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)1516
Total, all-cause mortalityArm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia99
Total, all-cause mortalityArm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts])89
Total, all-cause mortalityArm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])918
Total, all-cause mortalityArm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)69
Total, other adverse eventsArm A: Imetelstat 9.4 mg/kg (MF)1919
Total, other adverse eventsArm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)1616
Total, other adverse eventsArm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia99
Total, other adverse eventsArm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts])99

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMIDListed as
26332545DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov
2026-11-12
ClinicalTrials.gov
Results, from AACT
2026-03-23
AACT
NIH grant links
2026-11-13
NIH RePORTER

Sources 3

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ClinicalTrials.govNo license stated
  • NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ClinicalTrials.govnone stated
  • NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01731951

GET https://conductscience.org/api/trials/v1/NCT01731951/results?page=2