NCT01520909 · OUTCOME

Number of Participants Achieving a Platelet Count >=50 Giga Cells Per Liter (Gi/L) for at Least 6 Out of 8 Weeks, Between Weeks 5 and 12 of Part 1

Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body. · Source last updated 2015-03-10

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

Measure: NUMBER
Unit: Participants
Interval / dispersion: Not reported
Time frame: From Week 5 up to Week 12 of Part 1

What was measured

Participants who achieved a platelet count \>=50 Gi/L for at least 6 out of 8 weeks, between Weeks 5 and 12 of Part 1, were reported.

Analysis population: Intent-to-Treat (ITT) Population: all randomized participants. The ITT Population was the primary population used for assessing efficacy.

Groups in this outcome

Part 1 (Randomized Period)- Placebo

In Part 1, participants aged between 6 and 17 years with a body weight \<27 kilograms (kg) received placebo 37.5 milligrams (mg) once daily (QD), and those with a body weight \>=27 kg received placebo 50 mg QD. Participants of East Asian ancestry received a starting dose of placebo 25 mg QD. Participants aged between 1 and 5 years received placebo 1.2 milligrams per kilogram (mg/kg) QD; participants of East Asian ancestry received a starting dose of placebo 0.8 milligrams per kilograms per day (mg/kg/day).

Part 1 (Randomized Period)-Eltrombopag

In Part 1, participants aged between 6 and 17 years with a body weight \<27 kg received eltrombopag 37.5 mg QD, and those with a body weight \>=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day.

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
Part 1 (Randomized Period)- Placebo	29
Part 1 (Randomized Period)-Eltrombopag	63

Reported measurements

Source class 1

Values in Participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
Part 1 (Randomized Period)- Placebo	1	Not reported	Not reported	Not reported	Not reported
Part 1 (Randomized Period)-Eltrombopag	25	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
2.29
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
140.93
group Description
Indicated significance at the 5% (two-sided) level of significance
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY_OR_OTHER
p Value
<0.001
param Type
Odds Ratio (OR)
param Value
17.96
statistical Comment
The proportion of participants achieving platelet counts \>=50 Gi/L for those participants receiving eltrombopag versus placebo was compared.
statistical Method
Cochran-Mantel-Haenszel
tested Non Inferiority
false

Complete source fields

analyses

ci Lower Limit
2.29
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
140.93
group Description
Indicated significance at the 5% (two-sided) level of significance
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY_OR_OTHER
p Value
<0.001
param Type
Odds Ratio (OR)
param Value
17.96
statistical Comment
The proportion of participants achieving platelet counts \>=50 Gi/L for those participants receiving eltrombopag versus placebo was compared.
statistical Method
Cochran-Mantel-Haenszel
tested Non Inferiority
false

classes

categories
measurements
group Id
OG000
value
1
group Id
OG001
value
25

denoms

counts
group Id
OG000
value
29
group Id
OG001
value
63
units
Participants

description

Participants who achieved a platelet count \>=50 Gi/L for at least 6 out of 8 weeks, between Weeks 5 and 12 of Part 1, were reported.

groups

description
In Part 1, participants aged between 6 and 17 years with a body weight \<27 kilograms (kg) received placebo 37.5 milligrams (mg) once daily (QD), and those with a body weight \>=27 kg received placebo 50 mg QD. Participants of East Asian ancestry received a starting dose of placebo 25 mg QD. Participants aged between 1 and 5 years received placebo 1.2 milligrams per kilogram (mg/kg) QD; participants of East Asian ancestry received a starting dose of placebo 0.8 milligrams per kilograms per day (mg/kg/day).
id
OG000
title
Part 1 (Randomized Period)- Placebo
description
In Part 1, participants aged between 6 and 17 years with a body weight \<27 kg received eltrombopag 37.5 mg QD, and those with a body weight \>=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day.
id
OG001
title
Part 1 (Randomized Period)-Eltrombopag

param Type

NUMBER

population Description

Intent-to-Treat (ITT) Population: all randomized participants. The ITT Population was the primary population used for assessing efficacy.

reporting Status

POSTED

time Frame

From Week 5 up to Week 12 of Part 1

title

Number of Participants Achieving a Platelet Count >=50 Giga Cells Per Liter (Gi/L) for at Least 6 Out of 8 Weeks, Between Weeks 5 and 12 of Part 1

type

PRIMARY

unit Of Measure

Participants

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

Preserved source evidence · Not medical advice