Trial Outcomes & Findings for Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases. (NCT NCT00278954)
NCT ID: NCT00278954
Last Updated: 2013-01-24
Results Overview
By assessing the number of serious, acute, bacterial infections per subject per year in subjects with Primary Immunodeficiency disease.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
50 participants
Primary outcome timeframe
12 months
Results posted on
2013-01-24
Participant Flow
First enrollment: 06 February 2006 Last subject completed: 06 November 2007 Seven investigative sites, all hospital clinics
This was an open study. All enrolled subjects received study medication.
Participant milestones
| Measure |
Gammaplex
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
Overall Study
STARTED
|
50
|
|
Overall Study
COMPLETED
|
45
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Gammaplex
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.
Baseline characteristics by cohort
| Measure |
Gammaplex
n=50 Participants
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
Age, Categorical
<=18 years
|
6 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
37 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
7 Participants
n=5 Participants
|
|
Age Continuous
|
44.0 years
STANDARD_DEVIATION 19.10 • n=5 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
50 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: Intent to Treat (ITT).
By assessing the number of serious, acute, bacterial infections per subject per year in subjects with Primary Immunodeficiency disease.
Outcome measures
| Measure |
Gammaplex
n=50 Participants
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
Number of Serious, Acute, Bacterial Infections (SABIs) Per Subject Per Year in Subjects With Primary Immunodeficiency Disease.
|
0 SABIs/subject/year
|
SECONDARY outcome
Timeframe: -5, 0, 60 minutes (min), 24, 48 hours (hrs), 4, 7, 14, 21 and 28 daysPopulation: Only 24 of the 45 evaluable subjects participated in the Pharmacokinetic part of the protocol. No data imputation.
Blood samples for PK analysis were obtained and analysed at 10 different time points, i.e. -5, 0, 60 minutes (min), 24, 48 hours (hrs), 4, 7, 14, 21 and 28 days, at an infusion visit following 6 months of treatment.
Outcome measures
| Measure |
Gammaplex
n=24 Participants
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
The Pharmacokinetic (PK) Half-Life of Immunoglobulin G (IgG)
|
41.19 Days
Standard Deviation 19.19
|
SECONDARY outcome
Timeframe: -5, 0, 60 min, 24, 48 hrs, 4, 7, 14, 21 and 28 daysPopulation: Only 24 of the 45 evaluable subjects participated in the Pharmacokinetic part of the protocol. No data imputation.
Blood samples for PK analysis were obtained and analysed at 10 different time points, i.e. -5, 0, 60 minutes (min), 24, 48 hours (hrs), 4, 7, 14, 21 and 28 days, at an infusion visit following 6 months of treatment.
Outcome measures
| Measure |
Gammaplex
n=24 Participants
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
The Pharmacokinetic (PK) Clearance of Immunoglobulin G (IgG)
|
0.585 mL/day/kg
Standard Deviation 0.2508
|
SECONDARY outcome
Timeframe: -5, 0, 60 min, 24, 48 hrs, 4, 7, 14, 21 and 28 daysPopulation: Only 24 of the 45 evaluable subjects participated in the Pharmacokinetic part of the protocol. No data imputation.
Blood samples for PK analysis were obtained and analysed at 10 different time points, i.e. -5, 0, 60 minutes (min), 24, 48 hours (hrs), 4, 7, 14, 21 and 28 days, at an infusion visit following 6 months of treatment.
Outcome measures
| Measure |
Gammaplex
n=24 Participants
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
The Pharmacokinetic (PK) Volume of Distribution (Vz)of Immunoglobulin G (IgG)
|
0.297 dL/kg
Standard Deviation 0.0539
|
SECONDARY outcome
Timeframe: -5, 0, 60 min, 24, 48 hrs, 4, 7, 14, 21 and 28 daysBlood samples for PK analysis were obtained and analysed at 10 different time points, i.e. -5, 0, 60 minutes (min), 24, 48 hours (hrs), 4, 7, 14, 21 and 28 days, at an infusion visit following 6 months of treatment.
Outcome measures
| Measure |
Gammaplex
n=50 Participants
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
The Pharmacokinetic (PK) Mean Residence Time (MRT) for Inmuunoglobulin G (IgG)
|
56.1 Days
Standard Deviation 23.06
|
Adverse Events
Gammaplex
Serious events: 6 serious events
Other events: 47 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Gammaplex
n=50 participants at risk
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma
|
2.0%
1/50 • 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.
|
|
General disorders
Chest Pain
|
2.0%
1/50 • 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
Gastroenteritis viral
|
2.0%
1/50 • 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
Pneumonia bacterial
|
2.0%
1/50 • 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.
|
|
Pregnancy, puerperium and perinatal conditions
Pregnancy
|
2.0%
1/50 • 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.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma
|
2.0%
1/50 • 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
Syncope vasovagal
|
2.0%
1/50 • 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.
|
|
Vascular disorders
Thrombosis
|
2.0%
1/50 • 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
| Measure |
Gammaplex
n=50 participants at risk
All subjects received between 300 to 800 mg/kg/infusion of Gammaplex intravenously, every 21 day or 28 days.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
8.0%
4/50 • Number of events 19 • 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
Athralgia
|
6.0%
3/50 • 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
Back pain
|
10.0%
5/50 • 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.
|
|
Infections and infestations
Bronchitis
|
12.0%
6/50 • 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.
|
|
Infections and infestations
Bronchitis acute
|
8.0%
4/50 • 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.
|
|
General disorders
Chest pain
|
6.0%
3/50 • 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.
|
|
General disorders
Chills
|
8.0%
4/50 • 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.
|
|
Eye disorders
Conjunctivitis
|
8.0%
4/50 • 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.
|
|
Injury, poisoning and procedural complications
Contusion
|
6.0%
3/50 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.0%
3/50 • 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.
|
|
Infections and infestations
Cystitis
|
6.0%
3/50 • 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
Diarrhea
|
20.0%
10/50 • Number of events 13 • 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
|
14.0%
7/50 • Number of events 22 • 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
Gastroenteritis viral
|
12.0%
6/50 • 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.
|
|
Nervous system disorders
Headache
|
36.0%
18/50 • Number of events 97 • 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
|
6.0%
3/50 • 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.
|
|
Infections and infestations
Influenza
|
6.0%
3/50 • 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.
|
|
Psychiatric disorders
Insomnia
|
6.0%
3/50 • 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
Muscle spasms
|
6.0%
3/50 • 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
Myalgia
|
10.0%
5/50 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
12.0%
6/50 • 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.
|
|
Infections and infestations
Nasopharyngitis
|
14.0%
7/50 • Number of events 15 • 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
|
18.0%
9/50 • Number of events 12 • 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
|
10.0%
5/50 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
14.0%
7/50 • 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.
|
|
General disorders
Pyrexia
|
30.0%
15/50 • Number of events 26 • 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
Rash
|
10.0%
5/50 • 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
Sinus congestion
|
8.0%
4/50 • 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.
|
|
Nervous system disorders
Sinus headache
|
6.0%
3/50 • 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.
|
|
Infections and infestations
Sinusitis
|
34.0%
17/50 • Number of events 26 • 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
Upper abdominal pain
|
10.0%
5/50 • 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.
|
|
Infections and infestations
Upper respiratory tract infection
|
26.0%
13/50 • Number of events 19 • 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
Urinary tract infection
|
8.0%
4/50 • 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.
|
|
Gastrointestinal disorders
Vomiting
|
6.0%
3/50 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
6.0%
3/50 • 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.
|
Additional Information
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.
- Publication restrictions are in place
Restriction type: OTHER