Trial Outcomes & Findings for A Study to Find How Safe and Effective GAMMAPLEX® is in Subjects With Chronic Idiopathic Thrombocytopenic Purpura (ITP) (NCT NCT00504075)

Last Updated: 2013-03-01

Results Overview

The number of subjects with chronic ITP treated following treatment with Gammaplex who attained a platelet count of ≥ 50 x 10\^9/L by Day 9.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

35 participants

Primary outcome timeframe

9 days

Results posted on

2013-03-01

Participant Flow

First enrollment: 04 September 2007. Last Subject completed: 05 August 2011. Thirty-five sites in the United States, India and Argentina, all hospital clinics.

This was an open label study. All enrolled subjects received study medication.

Participant milestones

Participant milestones
Measure	GAMMAPLEX The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.
Overall Study STARTED	35
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	8

Reasons for withdrawal

Reasons for withdrawal
Measure	GAMMAPLEX The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.
Overall Study Adverse Event	1
Overall Study Lost to Follow-up	5
Overall Study Withdrawal by Subject	1
Overall Study Required other therapeutic intervention	1

Baseline Characteristics

A Study to Find How Safe and Effective GAMMAPLEX® is in Subjects With Chronic Idiopathic Thrombocytopenic Purpura (ITP)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	GAMMAPLEX n=35 Participants The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.
Age, Categorical <=18 years	3 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	29 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Age Continuous	36 years STANDARD_DEVIATION 18 • n=5 Participants
Sex: Female, Male Female	26 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Region of Enrollment United States	9 participants n=5 Participants
Region of Enrollment Argentina	5 participants n=5 Participants
Region of Enrollment India	21 participants n=5 Participants

PRIMARY outcome

Timeframe: 9 days

Population: Any subject who started treatment with GAMMAPLEX and whose central laboratory results taken prior to infusion on Day 1 were within specified protocol parameters, was included in the intent-to-treat (ITT) population for analysis of efficacy and safety.

The number of subjects with chronic ITP treated following treatment with Gammaplex who attained a platelet count of ≥ 50 x 10\^9/L by Day 9.

Outcome measures

Outcome measures
Measure	GAMMAPLEX n=35 Participants The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.
The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L.	29 participants Interval 60.0 to 100.0

SECONDARY outcome

Timeframe: AEs were documented from the date the informed consent form was signed until the End of Study visit on Day 90.

The safety variables used to assess safety were the following: * Adverse events * The number and percent of infusions with at least 1 adverse event(AE) that occurs during an infusion or within 72 hours after the infusion stops * Nature, severity, and frequency of AEs * Suspected unexpected serious adverse reactions (SUSARs) * Vital signs * Clinical laboratory tests and Direct Coombs' Test * Transmission of viruses * Physical examination

Outcome measures

Outcome measures
Measure	GAMMAPLEX n=35 Participants The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.
The Safety of GAMMAPLEX at the Dosage Used in This Study.	8.6 % of subjects with product related SAEs Interval 1.8 to 23.1

SECONDARY outcome

Timeframe: Days 1, 2, 3, 5, 9, 14, 21, 32.

Blood samples were collected to measure platelet counts and the duration of time for which the platelet count remained ≥50 x 10\^9/L was measured.

Outcome measures

Outcome measures
Measure	GAMMAPLEX n=35 Participants The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.
Duration of Time That the Platelet Count of Subjects With Chronic ITP Treated With Gammaplex Remained ≥ 50 x 10^9/L.	10 days Interval 7.0 to 14.0

Adverse Events

GAMMAPLEX

Serious events: 4 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	GAMMAPLEX n=35 participants at risk The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.
Metabolism and nutrition disorders Dehydration	2.9% 1/35 • Number of events 1 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Gastrointestinal disorders Diarrhoea	2.9% 1/35 • Number of events 1 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Nervous system disorders Headache	8.6% 3/35 • Number of events 3 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Infections and infestations Pneumonia	2.9% 1/35 • Number of events 1 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Infections and infestations Sepsis	2.9% 1/35 • Number of events 1 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Gastrointestinal disorders Vomiting	2.9% 1/35 • Number of events 1 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.

Other adverse events

Other adverse events
Measure	GAMMAPLEX n=35 participants at risk The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.
Gastrointestinal disorders Abdominal pain	5.7% 2/35 • Number of events 2 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Gastrointestinal disorders Abdominal pain upper	5.7% 2/35 • Number of events 2 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Blood and lymphatic system disorders Anaemia	8.6% 3/35 • Number of events 5 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Musculoskeletal and connective tissue disorders Arthralgia	5.7% 2/35 • Number of events 4 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Injury, poisoning and procedural complications Contusion	8.6% 3/35 • Number of events 6 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Respiratory, thoracic and mediastinal disorders Cough	5.7% 2/35 • Number of events 2 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Skin and subcutaneous tissue disorders Ecchymosis	8.6% 3/35 • Number of events 5 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Respiratory, thoracic and mediastinal disorders Epistaxis	5.7% 2/35 • Number of events 2 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
General disorders Fatigue	5.7% 2/35 • Number of events 3 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Gastrointestinal disorders Gastritis	5.7% 2/35 • Number of events 2 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Gastrointestinal disorders Gingival bleeding	11.4% 4/35 • Number of events 7 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Vascular disorders Hypertension	8.6% 3/35 • Number of events 3 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Gastrointestinal disorders Nausea	20.0% 7/35 • Number of events 8 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Musculoskeletal and connective tissue disorders Neck pain	5.7% 2/35 • Number of events 2 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
General disorders Pain	8.6% 3/35 • Number of events 4 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Musculoskeletal and connective tissue disorders Pain in extremity	5.7% 2/35 • Number of events 2 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Skin and subcutaneous tissue disorders Petechiae	20.0% 7/35 • Number of events 7 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
General disorders Pyrexia	17.1% 6/35 • Number of events 10 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Reproductive system and breast disorders Vaginal haemorrhage	5.7% 2/35 • Number of events 2 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Gastrointestinal disorders Vomiting	22.9% 8/35 • Number of events 11 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
Nervous system disorders Headache	31.4% 11/35 • Number of events 21 • An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.

Additional Information

Tim Aldwinckle, MD

Bio Products Laboratory Limited

Phone: +44 2089572565

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Publication of the Study results will be allowed only with prior written approval from Sponsor, said consent not to be unreasonably withheld. At the request of Sponsor, the Institution and/or Investigator shall delete any Confidential Information pertaining to Sponsor's Inventions from any proposed publications prior to submitting or presenting the materials. Any and all publications will give appropriate recognition of any support received from Sponsor.
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Restriction type: OTHER