NCT02604433 · OUTCOME

Percentage Of Participants Who Were Transfusion Independent For ≥ 8 Weeks During Treatment

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia · Source last updated 2023-04-18

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: Not reported
Time frame: From first dose through 3 weeks post last dose (up to approximately 218 weeks)

What was measured

Transfusion independence was defined as the absence of any transfusion during any consecutive "rolling" 8-week time interval within the treatment period, i.e, Days 1 to 56, Days 2 to 57 and so on.

Analysis population: All randomized participants

Groups in this outcome

Luspatercept + BSC

Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care

Placebo + BSC

Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care

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
Luspatercept + BSC	224
Placebo + BSC	112

Reported measurements

Source class 1

Values in Percentage of participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
Luspatercept + BSC	12.1	Not reported	Not reported	Not reported	Not reported
Placebo + BSC	1.8	Not reported	Not reported	Not reported	Not reported

Source statistical analyses

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

ci Lower Limit
1.8
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
32.9
group Description
Odds ratio 95% confidence intervals (CIs), and p-value were estimated from the Cochran Mantel-Haenszel (CMH) test stratified by the geographical regions defined at randomization.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.0015
p Value Comment
Significance level of 0.050 for 2-sided tests.
param Type
Odds Ratio (OR)
param Value
7.6
statistical Method
Cochran-Mantel-Haenszel

Complete source fields

analyses

ci Lower Limit
1.8
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
32.9
group Description
Odds ratio 95% confidence intervals (CIs), and p-value were estimated from the Cochran Mantel-Haenszel (CMH) test stratified by the geographical regions defined at randomization.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.0015
p Value Comment
Significance level of 0.050 for 2-sided tests.
param Type
Odds Ratio (OR)
param Value
7.6
statistical Method
Cochran-Mantel-Haenszel

classes

categories
measurements
group Id
OG000
value
12.1
group Id
OG001
value
1.8

denoms

counts
group Id
OG000
value
224
group Id
OG001
value
112
units
Participants

description

Transfusion independence was defined as the absence of any transfusion during any consecutive "rolling" 8-week time interval within the treatment period, i.e, Days 1 to 56, Days 2 to 57 and so on.

groups

description
Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care
id
OG000
title
Luspatercept + BSC
description
Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care
id
OG001
title
Placebo + BSC

param Type

NUMBER

population Description

All randomized participants

reporting Status

POSTED

time Frame

From first dose through 3 weeks post last dose (up to approximately 218 weeks)

title

Percentage Of Participants Who Were Transfusion Independent For ≥ 8 Weeks During Treatment

type

SECONDARY

unit Of Measure

Percentage of participants

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice