Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency (KIDS)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT01465958
First received: October 24, 2011
Last updated: February 23, 2015
Last verified: February 2015
Results First Received: January 19, 2015  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Pharmacokinetics Study;   Intervention Model: Crossover Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Primary Immunodeficiency
Intervention: Biological: GAMUNEX-C

  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
A total of 13 subjects were screened for participation in this study. After screening, one subject discontinued the study and did not receive study drug. Eleven subjects entered the Run-in phase. One subject entered intravenous Gamunex-C phase directly. A total of 11 subjects entered the IV phase and subsequently entered the SC phase.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Intravenous GAMUNEX-C GAMUNEX-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified: In the Run-in Phase, subjects received intravenous infusions of Gamunex-C at a dose between 200 to 600 mg/kg every three or four weeks for 3 months. In the subsequent IV phase, subjects received two IV infusions of Gamunex-C at a dose between 200 to 600 mg/kg for four to five weeks.
Subcutaneous GAMUNEX-C GAMUNEX-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified: weekly subcutaneous infusion at a mg/kg dose based on intravenous dose of subject and dosing interval x 1.37 conversion factor for 12 weeks.

Participant Flow for 3 periods

Period 1:   Run-in Phase
    Intravenous GAMUNEX-C     Subcutaneous GAMUNEX-C  
STARTED     11     0  
COMPLETED     10     0  
NOT COMPLETED     1     0  
Protocol Violation                 1                 0  

Period 2:   Intravenous (IV) Phase
    Intravenous GAMUNEX-C     Subcutaneous GAMUNEX-C  
STARTED     11 [1]   0  
COMPLETED     11     0  
NOT COMPLETED     0     0  
[1] One subject entered the IV phase directly.

Period 3:   Subcutaneous (SC) Phase
    Intravenous GAMUNEX-C     Subcutaneous GAMUNEX-C  
STARTED     0 [1]   11 [2]
COMPLETED     0     10  
NOT COMPLETED     0     1  
Adverse Event                 0                 1  
[1] After completing the intravenous (IV) phase, subjects entered the subcutaneous (SC) phase.
[2] Eleven subjects completed the IV phase and entered the SC phase.



  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.
All 12 enrolled subjects who were infused with any amount of study drug (GAMUNEX-C) were included in the safety population. The demographic data of the safety population are provided.

Reporting Groups
  Description
Safety Population The safety population consisted of all subjects who received any amount of GAMUNEX-C. In the Run-in Phase, subjects received intravenous infusions of Gamunex-C at a dose between 200 to 600 mg/kg every three or four weeks for 3 months. In the subsequent IV phase, subjects received two IV infusions of Gamunex-C at a dose between 200 to 600 mg/kg for four to five weeks. In the SC Phase, subjects received weekly subcutaneous infusion of Gamunex-C at a mg/kg dose based on intravenous dose of the subject and dosing interval x 1.37 conversion factor for 12 weeks.

Baseline Measures
    Safety Population  
Number of Participants  
[units: participants]
  12  
Age  
[units: participants]
 
<=18 years     12  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean (Standard Deviation)
  10.8  (3.7)  
Gender  
[units: participants]
 
Female     5  
Male     7  
Region of Enrollment  
[units: participants]
 
United States     12  



  Outcome Measures
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1.  Primary:   Steady-state Area Under the Curve (AUC) for Serum Total Immunoglobulin (IgG)   [ Time Frame: 4 to 5 weeks for IV administration; 12 weeks for SC administration ]

2.  Primary:   Mean Trough of Serum Total IgG   [ Time Frame: 4 - 5 weeks of IV administration and 12 weeks for SC administration ]


  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 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.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Henry Li, PhD
Organization: Grifols Therapeutics Inc
phone: 800-20-2807
e-mail: henry.li@grifols.com


No publications provided


Responsible Party: Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT01465958     History of Changes
Other Study ID Numbers: T5004-401
Study First Received: October 24, 2011
Results First Received: January 19, 2015
Last Updated: February 23, 2015
Health Authority: United States: Food and Drug Administration