Tolerability and Safety of Immune Globulin Subcutaneous Solution (IGSC) and rHuPH20 in PID

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Baxalta US Inc.
ClinicalTrials.gov Identifier:
NCT01175213
First received: August 3, 2010
Last updated: May 24, 2016
Last verified: May 2016
Results First Received: February 26, 2016  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Primary Immunodeficiency Diseases (PID)
Interventions: Biological: IGSC - rHuPH20 then IGSC or IGIV
Biological: IGIV, 10% only

  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
Recruitment was conducted 11 clinical sites in the United States.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
66 participants who enrolled from study 160603 were screened and all were enrolled on the study. Of these, 3 participants were treated with intravenous administration of Immune Globulin Intravenous (Human) (IGIV), 10% only i.e. did not receive rHuPH20 during this study.

Reporting Groups
  Description
IGSC - rHuPH20 Then IGSC, 10% or IGIV, 10% Only

Efficacy and safety of subcutaneous (SC) administration of Immune Globulin Subcutaneous Solution (IGSC), 10% after SC administration of recombinant human hyaluronidase (rHuPH20). Participants then went into a safety follow-up with either SC administration of IGSC, 10% or intravenous (IV) administration of Immune Globulin Intravenous (Human) (IGIV), 10%, only. The IV or SC administration route was at the discretion of the participant and the investigator.

Note: IGIV, 10% is the same product as IGSC, 10%.

IGIV, 10% Only

Participants were treated with Immune Globulin Intravenous (Human) (IGIV), 10% only, via the intravenous (IV) route throughout the study.

Note: IGIV, 10% is the same product as IGSC, 10%.


Participant Flow for 2 periods

Period 1:   IGSC, 10%/rHuPH20
    IGSC - rHuPH20 Then IGSC, 10% or IGIV, 10% Only     IGIV, 10% Only  
STARTED     63     3 [1]
COMPLETED     48     3  
NOT COMPLETED     15     0  
Withdrawal by Subject                 4                 0  
Death                 1                 0  
bone marrow transplant                 1                 0  
clinical site closed by sponsor                 6                 0  
site elected to exit study                 3                 0  
[1] 3 participants treated IV with IGIV,10% without rHuPH20 throughout the study.

Period 2:   Safety Follow-up
    IGSC - rHuPH20 Then IGSC, 10% or IGIV, 10% Only     IGIV, 10% Only  
STARTED     48     3  
COMPLETED     47     3  
NOT COMPLETED     1     0  
Adverse Event                 1                 0  



  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.
Safety Analysis Set

Reporting Groups
  Description
Participants Aged 2 to <12 Years No text entered.
Participants Aged 12 to <16 Years No text entered.
Participants Aged 16 to <65 Years No text entered.
Participants Aged 65 Years and Older No text entered.
Total Total of all reporting groups

Baseline Measures
    Participants Aged 2 to <12 Years     Participants Aged 12 to <16 Years     Participants Aged 16 to <65 Years     Participants Aged 65 Years and Older     Total  
Number of Participants  
[units: participants]
  4     7     47     8     66  
Age  
[units: Years]
Median (Full Range)
  10.5  
  (9 to 11)  
  15.0  
  (13 to 15)  
  47.0  
  (16 to 64)  
  71.0  
  (65 to 80)  
  43.0  
  (9 to 80)  
Gender  
[units: Participants]
         
Female     1     1     23     7     32  
Male     3     6     24     1     34  
Region of Enrollment  
[units: Participants]
         
United States     4     7     47     8     66  



  Outcome Measures
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1.  Primary:   Annual Rate of Serious Bacterial Infections   [ Time Frame: Throughout the efficacy period only (from 60 to 729 days) ]

2.  Primary:   Annual Rate of All Infections   [ Time Frame: Throughout the efficacy period only (from 60 to 729 days) ]

3.  Primary:   Trough Levels of IgG Maintained During the Study Period in Relation to Dose Frequency   [ Time Frame: Throughout the efficacy period only (from 60 to 729 days) ]

4.  Secondary:   The Annual Rate of Serious Adverse Events (SAEs), Related and Not Related to Study Drugs   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

5.  Secondary:   Percentage of Infusions for Which the Infusion Rate Was Reduced and/or the Infusion Interrupted or Stopped for Tolerability Concerns or for AEs   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

6.  Secondary:   Percentage of Infusions Associated With One or More Moderate or Severe AEs (Including and Excluding Infections) That Begin During or Within 72 Hours of Completion of an Infusion   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

7.  Secondary:   Number of Participants Who Develop Antibodies and Neutralizing Antibodies to rHuPH20   [ Time Frame: Throughout entire study period for 160603 (1 year 11 months) and 160902 (2 years 11 months) ]

8.  Secondary:   Percentage of Participants Who Develop Antibodies and Neutralizing Antibodies to rHuPH20   [ Time Frame: Throughout entire study period for 160603 (1 year 11 months) and 160902 (2 years 11 months) ]

9.  Secondary:   Number of Participants With AEs Related to Anti-rHuPH20 Titers   [ Time Frame: Throughout entire study period for 160603 (1 year 11 months) and 160902 (2 years 11 months) ]

10.  Secondary:   Percentage of Participants With AEs Related to Anti-rHuPH20 Titers   [ Time Frame: Throughout entire study period for 160603 (1 year 11 months) and 160902 (2 years 11 months) ]

11.  Secondary:   Number of All AEs Categorized by MedDRA Preferred Terms, Seriousness, Relatedness to the Study Drug, and Severity (A-F).   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

12.  Secondary:   Number of All AEs Categorized by MedDRA Preferred Terms, Seriousness, Relatedness to the Study Drug, and Severity (G-M).   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

13.  Secondary:   Number of All AEs Categorized by MedDRA Preferred Terms, Seriousness, Relatedness to the Study Drug, and Severity (N-Z).   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

14.  Secondary:   Rate of All AEs Per Participant Categorized by MedDRA Preferred Terms, Seriousness and Severity (A-F).   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

15.  Secondary:   Rate of All AEs Per Participant Categorized by MedDRA Preferred Terms, Seriousness and Severity (G-M).   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

16.  Secondary:   Rate of All AEs Per Participant Categorized by MedDRA Preferred Terms, Seriousness and Severity (N-Z).   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

17.  Secondary:   Rate of All AEs Per Infusion Categorized by MedDRA Preferred Terms, Seriousness and Severity (A-F).   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

18.  Secondary:   Rate of All AEs Per Infusion Categorized by MedDRA Preferred Terms, Seriousness and Severity (G-M).   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

19.  Secondary:   Rate of All AEs Per Infusion Categorized by MedDRA Preferred Terms, Seriousness and Severity (N-Z).   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

20.  Secondary:   Rate of AEs Per Participant (Including and Excluding Infections) Determined by the Investigator to be Related to the Study Drug That Occur at Any Time During the Study (“Related”)   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

21.  Secondary:   Rate of AEs Per Infusion (Including and Excluding Infections) Determined by the Investigator to be Related to the Study Drug That Occur at Any Time During the Study (“Related”)   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

22.  Secondary:   Rate of AEs Per Participant (Including and Excluding Infections) Temporarily Associated With the Infusion   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

23.  Secondary:   Rate of AEs Per Infusion (Including and Excluding Infections) Temporarily Associated With the Infusion   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]

24.  Secondary:   Percentage of Infusions Associated With One or More Local AEs (Including and Excluding Infections), at Any Time During the Study   [ Time Frame: Throughout entire study period (up to 3 years). Duration of participation for each participant is variable depending on date of enrollment and their anti-rHuPH20 binding antibody titer. ]


  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
No text entered.


  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: Clinical Trial Registries and Results Disclosure
Organization: Baxalta US Inc.
e-mail: ClinicalTrialsDisclosure@baxalta.com



Responsible Party: Baxalta US Inc.
ClinicalTrials.gov Identifier: NCT01175213     History of Changes
Other Study ID Numbers: 160902
Study First Received: August 3, 2010
Results First Received: February 26, 2016
Last Updated: May 24, 2016
Health Authority: United States: Food and Drug Administration