NCT02598661 · OUTCOME

Phase 2 and Phase 3: Overall Survival (OS)

Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) · Source last updated 2026-07-17

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure: MEDIAN
Unit: months
Interval / dispersion: 95% Confidence Interval
Time frame: Up to 6.6 years in Phase 2 and up to 4 years in Phase 3

What was measured

OS was defined as the interval from Study Day 1 to death from any cause. Survival time of living participants was censored on the last date a participant is known to be alive or lost to follow-up. The Kaplan-Meier method was used to estimate overall survival.

Analysis population: Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug. Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.

Groups in this outcome

Phase 2: Imetelstat Sodium

Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.

Phase 3: Imetelstat Sodium

Phase 3: Placebo

Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

Group	Source count
Phase 2: Imetelstat Sodium	57
Phase 3: Imetelstat Sodium	118
Phase 3: Placebo	60

Reported measurements

Source class 1

Values in months · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Phase 2: Imetelstat Sodium	55.3	Not reported	39.16	NA	Upper limit of 95% CI was not estimable due to low number of participants with events.
Phase 3: Imetelstat Sodium	40.4	Not reported	37.06	NA	Upper limit of 95% CI was not estimable due to low number of participants with events.
Phase 3: Placebo	NA	Not reported	32.16	NA	Median and upper limit of 95% CI were not estimable due to low number of participants with events.

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

ci Lower Limit
0.526
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
1.823
estimate Comment
Hazard ratio and 95% CI were calculated from the Cox proportional hazard model, stratified by prior RBC transfusion burden (≤6 vs. \>6 units RBC) and IPSS risk group (low vs. intermediate-1), with treatment as the only covariate.
group Ids
OG001
OG002
non Inferiority Type
SUPERIORITY
p Value
0.949
p Value Comment
P value (two-sided) for superiority of imetelstat sodium versus placebo in hazard ratio was calculated using stratified log-rank test.
param Type
Hazard Ratio (HR)
param Value
0.98
statistical Method
Log Rank

Complete source fields

analyses

ci Lower Limit
0.526
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
1.823
estimate Comment
Hazard ratio and 95% CI were calculated from the Cox proportional hazard model, stratified by prior RBC transfusion burden (≤6 vs. \>6 units RBC) and IPSS risk group (low vs. intermediate-1), with treatment as the only covariate.
group Ids
OG001
OG002
non Inferiority Type
SUPERIORITY
p Value
0.949
p Value Comment
P value (two-sided) for superiority of imetelstat sodium versus placebo in hazard ratio was calculated using stratified log-rank test.
param Type
Hazard Ratio (HR)
param Value
0.98
statistical Method
Log Rank

classes

categories
measurements
comment
Upper limit of 95% CI was not estimable due to low number of participants with events.
group Id
OG000
lower Limit
39.16
upper Limit
NA
value
55.3
comment
Upper limit of 95% CI was not estimable due to low number of participants with events.
group Id
OG001
lower Limit
37.06
upper Limit
NA
value
40.4
comment
Median and upper limit of 95% CI were not estimable due to low number of participants with events.
group Id
OG002
lower Limit
32.16
upper Limit
NA
value
NA

denoms

counts
group Id
OG000
value
57
group Id
OG001
value
118
group Id
OG002
value
60
units
Participants

description

dispersion Type

95% Confidence Interval

groups

description
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
id
OG000
title
Phase 2: Imetelstat Sodium
description
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
id
OG001
title
Phase 3: Imetelstat Sodium
description
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
id
OG002
title
Phase 3: Placebo

param Type

MEDIAN

population Description

Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug. Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.

reporting Status

POSTED

time Frame

Up to 6.6 years in Phase 2 and up to 4 years in Phase 3

title

Phase 2 and Phase 3: Overall Survival (OS)

type

SECONDARY

unit Of Measure

months

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Preserved source evidence · Not medical advice