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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Bio Products Laboratory
ClinicalTrials.gov Identifier:
NCT01289847
First received: January 27, 2011
Last updated: December 15, 2014
Last verified: December 2014
Results First Received: December 8, 2014  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: 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

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
This was a Phase IV, multicentre, open-label, non-randomised study. All enrolled subjects received study medication.

Reporting Groups
  Description
Gammaplex 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.

Participant Flow:   Overall Study
    Gammaplex  
STARTED     25  
COMPLETED     24  
NOT COMPLETED     1  
Patient could not comply with visits                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Gammaplex 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.

Baseline Measures
    Gammaplex  
Number of Participants  
[units: participants]
  25  
Age, Customized  
[units: participants]
 
2 to 5 year age group     3  
6 to 11 year age group     12  
12 to 16 year age group     10  
Gender  
[units: participants]
 
Female     6  
Male     19  
Region of Enrollment  
[units: participants]
 
United States     22  
Israel     2  
Chile     1  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Adverse Events   [ Time Frame: 12 months ]

2.  Secondary:   Therapeutic Efficacy   [ Time Frame: From week 15 onwards ]

3.  Secondary:   Therapeutic Efficacy   [ Time Frame: 12 months ]

4.  Secondary:   Therapeutic Efficacy   [ Time Frame: 12 months ]

5.  Secondary:   Therapeutic Efficacy   [ Time Frame: 12 months ]

6.  Secondary:   Therapeutic Efficacy   [ Time Frame: 12 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Head of Medical Affairs
Organization: Bio Products Laboratory
phone: +44 20 8957 2200
e-mail: medinfo@bpl.co.uk


No publications provided


Responsible Party: Bio Products Laboratory
ClinicalTrials.gov Identifier: NCT01289847     History of Changes
Other Study ID Numbers: GMX04, IND 12569
Study First Received: January 27, 2011
Results First Received: December 8, 2014
Last Updated: December 15, 2014
Health Authority: United States: Food and Drug Administration