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A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency

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ClinicalTrials.gov Identifier: NCT01289847
Recruitment Status : Completed
First Posted : February 4, 2011
Results First Posted : December 23, 2014
Last Update Posted : December 23, 2014
Sponsor:
Information provided by (Responsible Party):
Bio Products Laboratory

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Prevention
Conditions Primary Immune Deficiency Disorders
Common Variable Immunodeficiency
X-linked Agammaglobulinemia
Hyper-IgM Syndrome
Wiskott-Aldrich Syndrome
Intervention Biological: Gammaplex
Enrollment 25
Recruitment Details First enrollment: 06 April 2011; Last Subject completed 23 April 2014; Nine recruiting sites globally: United States (US) (seven sites), Chile (one site) and Israel (one site)
Pre-assignment Details This was a Phase IV, multicentre, open-label, non-randomised study. All enrolled subjects received study medication.
Arm/Group Title Gammaplex
Hide Arm/Group Description Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300–800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Period Title: Overall Study
Started 25
Completed 24
Not Completed 1
Reason Not Completed
Patient could not comply with visits             1
Arm/Group Title Gammaplex
Hide Arm/Group Description Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300–800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Overall Number of Baseline Participants 25
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 25 participants
2 to 5 year age group 3
6 to 11 year age group 12
12 to 16 year age group 10
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants
Female
6
  24.0%
Male
19
  76.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 25 participants
United States 22
Israel 2
Chile 1
1.Primary Outcome
Title Adverse Events
Hide Description Number of subjects with serious, acute, bacterial infections as a measure of efficacy
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat (ITT)
Arm/Group Title Gammaplex
Hide Arm/Group Description:
Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300–800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Overall Number of Participants Analyzed 25
Measure Type: Number
Unit of Measure: participants
2
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gammaplex
Comments For the primary efficacy analysis, the SABI rate for GAMMAPLEX and the upper bound of its one-sided 99% confidence interval (CI) were estimated by using the exact method for a one-sample Poisson rate.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.01
Comments [Not Specified]
Method one-sample Poisson rate
Comments [Not Specified]
2.Secondary Outcome
Title Therapeutic Efficacy
Hide Description Number and proportion of subjects who maintain trough IgG levels at least as high as the average of the 2 previous trough levels before the first Gammaplex infusion
Time Frame From week 15 onwards
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Seven subjects (28.0%) maintained trough IgG levels at all visits that were at least as high as the average of the two previous levels before the first infusion
Arm/Group Title Gammaplex
Hide Arm/Group Description:
Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300–800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Overall Number of Participants Analyzed 25
Measure Type: Number
Unit of Measure: participants
7
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gammaplex
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.01
Comments [Not Specified]
Method one-sample Poisson method
Comments [Not Specified]
3.Secondary Outcome
Title Therapeutic Efficacy
Hide Description Number of days off school
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Gammaplex
Hide Arm/Group Description:
Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300–800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Overall Number of Participants Analyzed 25
Mean (Standard Deviation)
Unit of Measure: days
7.8  (12.06)
4.Secondary Outcome
Title Therapeutic Efficacy
Hide Description Number of days in hospital
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Gammaplex
Hide Arm/Group Description:
Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300–800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Overall Number of Participants Analyzed 25
Mean (Standard Deviation)
Unit of Measure: days
0.3  (0.87)
5.Secondary Outcome
Title Therapeutic Efficacy
Hide Description Visits to physicians and/or emergency room
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Gammaplex
Hide Arm/Group Description:
Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300–800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Overall Number of Participants Analyzed 25
Mean (Standard Deviation)
Unit of Measure: visits
4.0  (4.67)
6.Secondary Outcome
Title Therapeutic Efficacy
Hide Description Number of days on therapeutic antibiotics
Time Frame 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Gammaplex
Hide Arm/Group Description:
Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300–800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
Overall Number of Participants Analyzed 25
Mean (Standard Deviation)
Unit of Measure: days
32.0  (28.28)
Time Frame AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Gammaplex
Hide Arm/Group Description Gammaplex: GAMMAPLEX 5g/100 mL, dose is 300–800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.
All-Cause Mortality
Gammaplex
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Gammaplex
Affected / at Risk (%) # Events
Total   3/25 (12.00%)    
Gastrointestinal disorders   
Acute Gastroenterirtis (AGE)  1 [1]  1/25 (4.00%)  1
Respiratory, thoracic and mediastinal disorders   
left lower lobe pneumonia  1 [2]  1/25 (4.00%)  1
left lower lobe pneumonia  1 [3]  1/25 (4.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 8.1
[1]
Three days prior to hospital admission the subject experienced watery diarrhea and vomiting. It was reported that the subject would not eat or drink and had a one-time fever of 38.1 degrees celsius. Resolved.
[2]
A chest X-ray revealed a 4 cm retrocardiac left lung base infiltrate. Laboratory results included a WBC count of 26.4 × 109/L (ref range 4.8-10.8 × 109/L), band neutrophils 23% (ref range 50-80%) and lymphocytes 6% (ref range 20-50%). Resolved.
[3]
The subject was hospitalised with a fever (101.8°F), tachycardia, dyspnoea, hypoxia and vomiting. No bacterial aetiology was identified by blood cultures. Resolved.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Gammaplex
Affected / at Risk (%) # Events
Total   25/25 (100.00%)    
Blood and lymphatic system disorders   
Lymphadenopathy  2  2/25 (8.00%)  3
Cardiac disorders   
Tachycardia  2  3/25 (12.00%)  6
Gastrointestinal disorders   
Nausea  2  6/25 (24.00%)  8
Vomiting  2  5/25 (20.00%)  8
Diarrhoea  2  3/25 (12.00%)  4
Abdominal pain upper  2  2/25 (8.00%)  2
General disorders   
Pyrexia  2  9/25 (36.00%)  15
Fatigue  2  5/25 (20.00%)  11
Malaise  2  5/25 (20.00%)  7
Chest discomfort  2  3/25 (12.00%)  6
Infusion site erythema  2  2/25 (8.00%)  2
Pain  2  2/25 (8.00%)  2
Infections and infestations   
Nasopharyngitis  1  8/25 (32.00%)  11
Acute sinusitis  2  7/25 (28.00%)  9
Upper respiratory tract infection  2  6/25 (24.00%)  8
Viral upper respiratory tract infection  1  5/25 (20.00%)  7
Pharyngitis streptococcal  2  4/25 (16.00%)  4
Sinusitis  2  3/25 (12.00%)  7
Gastroenteritis viral  2  3/25 (12.00%)  4
Pharyngitis  2  3/25 (12.00%)  4
Bronchitis  2  2/25 (8.00%)  3
Lobar pneumonia  2  2/25 (8.00%)  3
Bronchitis acute  2  2/25 (8.00%)  2
Influenza  2  2/25 (8.00%)  2
Otitis media  2  2/25 (8.00%)  2
Otitis media acute  2  2/25 (8.00%)  2
Pneumonia  2  2/25 (8.00%)  2
Rhinitis  2  2/25 (8.00%)  2
Injury, poisoning and procedural complications   
Joint sprain  2  2/25 (8.00%)  2
Skin laceration  2  2/25 (8.00%)  2
Musculoskeletal and connective tissue disorders   
Arthralgia  2  4/25 (16.00%)  4
Myalgia  2  2/25 (8.00%)  13
Nervous system disorders   
Headache  2  13/25 (52.00%)  39
Respiratory, thoracic and mediastinal disorders   
Cough  2  8/25 (32.00%)  19
Nasal congestion  2  8/25 (32.00%)  17
Rhinorrhoea  2  4/25 (16.00%)  4
Dyspnoea  2  3/25 (12.00%)  3
Pharyngolaryngeal pain  2  3/25 (12.00%)  3
Epistaxis  2  2/25 (8.00%)  3
Nasal oedema  2  2/25 (8.00%)  3
Wheezing  2  2/25 (8.00%)  3
Skin and subcutaneous tissue disorders   
Eczema  2  2/25 (8.00%)  3
Vascular disorders   
Hypotension  2  4/25 (16.00%)  12
Diastolic hypertension  1  3/25 (12.00%)  5
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (8.1)
2
Term from vocabulary, MedDRA 8.1
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

There is an agreement between the PI and Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results.

Publication of Study results will be allowed only with prior written approval from Sponsor. 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.

Results Point of Contact
Name/Title: Head of Medical Affairs
Organization: Bio Products Laboratory
Phone: +44 20 8957 2200
Responsible Party: Bio Products Laboratory
ClinicalTrials.gov Identifier: NCT01289847     History of Changes
Other Study ID Numbers: GMX04
IND 12569 ( Registry Identifier: CBER )
First Submitted: January 27, 2011
First Posted: February 4, 2011
Results First Submitted: December 8, 2014
Results First Posted: December 23, 2014
Last Update Posted: December 23, 2014