Trial Outcomes & Findings for Clinical Study to Evaluate the Efficacy and Safety of Octagam 10% in Idiopathic Thrombocytopenic Purpura in Adults (NCT NCT00426270)

Results Overview

A clinical response is defined as an increase in platelet count to ≥ 50\*10\^9/L on any day from Day 2 to Day 7.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

116 participants

Primary outcome timeframe

Day 2 to Day 7

Results posted on

2014-08-01

Participant Flow

One participant was incorrectly enrolled in the study and was not included in the efficacy analyses but was included in the safety population.

Participant milestones

Participant milestones
Measure	Octagam 10% 1 g/kg/Day Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Overall Study STARTED	116
Overall Study COMPLETED	110
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Octagam 10% 1 g/kg/Day Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Overall Study Incorrectly Enrolled in the Study	1
Overall Study Adverse Event	5

Baseline Characteristics

Clinical Study to Evaluate the Efficacy and Safety of Octagam 10% in Idiopathic Thrombocytopenic Purpura in Adults

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Octagam 10% 1 g/kg/Day n=116 Participants Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Age, Continuous	47.7 Years STANDARD_DEVIATION 19.1 • n=5 Participants
Sex: Female, Male Female	74 Participants n=5 Participants
Sex: Female, Male Male	42 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 2 to Day 7

Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count.

A clinical response is defined as an increase in platelet count to ≥ 50\*10\^9/L on any day from Day 2 to Day 7.

Outcome measures

Outcome measures
Measure	Octagam 10% 1 g/kg/Day n=115 Participants Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Percentage of Participants With a Clinical Response	80.0 Percentage of participants

SECONDARY outcome

Timeframe: Day 2 to Day 7

Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count. Only participants with a clinical response were included in the analysis.

A clinical response is defined as an increase in platelet count to ≥ 50\*10\^9/L on any day from Day 2 to Day 7.

Outcome measures

Outcome measures
Measure	Octagam 10% 1 g/kg/Day n=92 Participants Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Time to Achieve a Clinical Response	2.1 Days Standard Deviation 1.08

SECONDARY outcome

Timeframe: Day 2 to the end of the study (Day 63)

Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count.

Platelet count was assessed on Days 2 through 7 and on Days 14, 21, and 63. The maximum measured platelet count is reported.

Outcome measures

Outcome measures
Measure	Octagam 10% 1 g/kg/Day n=115 Participants Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Maximum Platelet Count	221.6 *10^9/L Standard Deviation 142.66

SECONDARY outcome

Timeframe: Day 2 to the end of the study (Day 63)

Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count. Only participants with a clinical response were included in the analysis.

The duration of the clinical response was the number of days that the platelet count remained ≥ 50\*10\^9/L. Platelet count was assessed on Days 2 through 7 and on Days 14, 21, and 63. A conservative method was used to calculate the duration of the clinical response. For example, if the platelet count was ≥ 50\*10\^9/L on Day 7 and dropped below 50\*10\^9/L at Day 14, Day 7 was used as the last day to calculate the duration of the clinical response. The same procedure was used if the platelet count dropped below 50\*10\^9/L at Day 21 from Day 14 or Day 63 from Day 21.

Outcome measures

Outcome measures
Measure	Octagam 10% 1 g/kg/Day n=92 Participants Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Duration of the Clinical Response	24.1 Days Standard Deviation 23.88

SECONDARY outcome

Timeframe: Day 7

Population: Full analysis set: All participants who received at least 1 dose of study medication, satisfied all major entry criteria, and had at least 1 post-baseline measurement of platelet count.

The investigator evaluated the severity of bleeding using the following rating scale: None (definitely no haemorrhage of any kind), Minor (few petechiae \[≤ 100 total\] and/or ≤ 5 small bruises \[≤ 3 cm diameter\], no mucosal bleeding), Mild (many petechiae \[\> 100 total\] and/or \> 5 large bruises \[\> 3 cm diameter\], no mucosal bleeding), Moderate (overt mucosal bleeding \[epistaxis, gum bleeding, oropharyngeal blood blisters, menorrhagia, gastrointestinal bleeding, etc\] that does not require immediate medical attention or intervention).

Outcome measures

Outcome measures
Measure	Octagam 10% 1 g/kg/Day n=115 Participants Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 None	80.9 Percentage of participants
Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 Minor	13.0 Percentage of participants
Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 Mild	2.6 Percentage of participants
Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 Moderate	0.9 Percentage of participants
Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 Missing	2.6 Percentage of participants

Adverse Events

Octagam 10% 1 g/kg/Day

Serious events: 14 serious events

Other events: 79 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Octagam 10% 1 g/kg/Day n=116 participants at risk Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Blood and lymphatic system disorders Idiopathic thrombocytopenic purpura	4.3% 5/116 Safety population: All participants who received at least 1 dose of study medication.
Blood and lymphatic system disorders Thrombocytopenia	1.7% 2/116 Safety population: All participants who received at least 1 dose of study medication.
Injury, poisoning and procedural complications Extradural haematoma	0.86% 1/116 Safety population: All participants who received at least 1 dose of study medication.
Injury, poisoning and procedural complications Injury	0.86% 1/116 Safety population: All participants who received at least 1 dose of study medication.
Injury, poisoning and procedural complications Overdose	0.86% 1/116 Safety population: All participants who received at least 1 dose of study medication.
Nervous system disorders Headache	1.7% 2/116 Safety population: All participants who received at least 1 dose of study medication.
Nervous system disorders Transient ischaemic attack	0.86% 1/116 Safety population: All participants who received at least 1 dose of study medication.
Infections and infestations Pneumonia	0.86% 1/116 Safety population: All participants who received at least 1 dose of study medication.
Investigations Platelet count decreased	0.86% 1/116 Safety population: All participants who received at least 1 dose of study medication.

Other adverse events

Other adverse events
Measure	Octagam 10% 1 g/kg/Day n=116 participants at risk Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days.
Blood and lymphatic system disorders Idiopathic thrombocytopenic purpura	5.2% 6/116 Safety population: All participants who received at least 1 dose of study medication.
Gastrointestinal disorders Nausea	6.0% 7/116 Safety population: All participants who received at least 1 dose of study medication.
General disorders Pyrexia	18.1% 21/116 Safety population: All participants who received at least 1 dose of study medication.
Investigations Heart rate decreased	15.5% 18/116 Safety population: All participants who received at least 1 dose of study medication.
Investigations Heart rate increased	21.6% 25/116 Safety population: All participants who received at least 1 dose of study medication.
Nervous system disorders Headache	30.2% 35/116 Safety population: All participants who received at least 1 dose of study medication.
Vascular disorders Hypertension	6.0% 7/116 Safety population: All participants who received at least 1 dose of study medication.

Additional Information

Michael Eppolito

Octapharma USA

Phone: 201 604-1155

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place