NCT02598661 · OUTCOME

Phase 2 and Phase 3: Percentage of Participants With Hematologic Improvement Including Erythroid Response (HI-E) as Per International Working Group (IWG) Response Criteria 2006

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: NUMBER
Unit: percentage of participants
Interval / dispersion: 95% Confidence Interval
Time frame: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3

What was measured

As per IWG Response Criteria 2006: HI-E was defined as a hemoglobin (Hb) increase by greater than or equal to (≥)1.5 grams per deciliter (g/dL) relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusion units/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hb of less than or equal to (≤)9 g/dL pretreatment were counted in the RBC transfusion response evaluation. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.

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 percentage of participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Phase 2: Imetelstat Sodium	61.4	Not reported	47.57	74.00	Not reported
Phase 3: Imetelstat Sodium	63.6	Not reported	54.20	72.22	Not reported
Phase 3: Placebo	51.7	Not reported	38.39	64.77	Not reported

Source statistical analyses

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

ci Lower Limit
-4.10
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
27.56
estimate Comment
95% CI was calculated based on the Wilson Score Method.
group Ids
OG001
OG002
non Inferiority Type
SUPERIORITY
p Value
0.112
p Value Comment
The p-value was calculated based on CMH controlling for prior RBC transfusion burden (≤ 6 vs. \> 6 units RBC) and IPSS risk group (low vs. intermediate-1) applied to randomization.
param Type
Percentage Difference
param Value
11.9
statistical Method
Cochran-Mantel-Haenszel

Complete source fields

analyses

ci Lower Limit
-4.10
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
27.56
estimate Comment
95% CI was calculated based on the Wilson Score Method.
group Ids
OG001
OG002
non Inferiority Type
SUPERIORITY
p Value
0.112
p Value Comment
The p-value was calculated based on CMH controlling for prior RBC transfusion burden (≤ 6 vs. \> 6 units RBC) and IPSS risk group (low vs. intermediate-1) applied to randomization.
param Type
Percentage Difference
param Value
11.9
statistical Method
Cochran-Mantel-Haenszel

classes

categories
measurements
group Id
OG000
lower Limit
47.57
upper Limit
74.00
value
61.4
group Id
OG001
lower Limit
54.20
upper Limit
72.22
value
63.6
group Id
OG002
lower Limit
38.39
upper Limit
64.77
value
51.7

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

NUMBER

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 5 years in Phase 2 and up to 3.7 years in Phase 3

title

Phase 2 and Phase 3: Percentage of Participants With Hematologic Improvement Including Erythroid Response (HI-E) as Per International Working Group (IWG) Response Criteria 2006

type

SECONDARY

unit Of Measure

percentage of participants

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