Phase 3
N=66
Tolerability and Safety of Immune Globulin Subcutaneous Solution (IGSC) and rHuPH20 in PID
Primary Immunodeficiency Diseases (PID)
Bottom Line
View on ClinicalTrials.gov: NCT01175213 ↗Enrolled (actual)
66
Serious AEs
10.5%
Results posted
May 2016
Primary outcome: Primary: Annual Rate of Serious Bacterial Infections — 0.020 Estimated infections/year — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- SC treatment with IGSC, 10% with rHuPH20 followed by SC/IGSC, 10% only (safety) (Biological); SC treatment with IGSC, 10% with rHuPH20 followed by IV/IGSC, 10% only (safety) (Biological); IV treatment with IGSC, 10% (Biological)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Baxalta now part of Shire
- Primary completion
- Aug 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annual Rate of Serious Bacterial Infections |
0.020 | <0.0001 sig |
| PRIMARY Annual Rate of All Infections |
2.86 | — |
| PRIMARY Trough Levels of IgG Maintained During the Study Period in Relation to Dose Frequency |
10.900; 12.300; 9.760 | — |
| SECONDARY The Annual Rate of Serious Adverse Events (SAEs), Related and Not Related to Study Drugs |
0.0000; 0.0000; 0.0000; 0.0000; 0.0768; 0.0000 | — |
| SECONDARY Percentage of Infusions for Which the Infusion Rate Was Reduced and/or the Infusion Interrupted or Stopped for Tolerability Concerns or for AEs |
1.3; 0.3; 0.8; 0.2; 0.1; 0.2 | — |
| 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 |
3.4; 4.7; 2.9; 4.0 | — |
| SECONDARY Number of Participants Who Develop Antibodies and Neutralizing Antibodies to rHuPH20 |
13; 0 | — |
| SECONDARY Percentage of Participants Who Develop Antibodies and Neutralizing Antibodies to rHuPH20 |
19.7; 0 | — |
| SECONDARY Number of Participants With AEs Related to Anti-rHuPH20 Titers |
12 | — |
| SECONDARY Percentage of Participants With AEs Related to Anti-rHuPH20 Titers |
92.3 | — |
| SECONDARY Number of All AEs Categorized by MedDRA Preferred Terms, Seriousness, Relatedness to the Study Drug, and Severity (A-F). |
6; 0; 1; 0; 0; 1 | — |
| SECONDARY Number of All AEs Categorized by MedDRA Preferred Terms, Seriousness, Relatedness to the Study Drug, and Severity (G-M). |
1; 0; 1; 0; 3; 0 | — |
| SECONDARY Number of All AEs Categorized by MedDRA Preferred Terms, Seriousness, Relatedness to the Study Drug, and Severity (N-Z). |
11; 5; 2; 0; 1; 0 | — |
| SECONDARY Rate of All AEs Per Participant Categorized by MedDRA Preferred Terms, Seriousness and Severity (A-F). |
0.095; 0; 0.016; 0; 0; 0.020 | — |
| SECONDARY Rate of All AEs Per Participant Categorized by MedDRA Preferred Terms, Seriousness and Severity (G-M). |
0.016; 0; 0.016; 0; 0.048; 0 | — |
| SECONDARY Rate of All AEs Per Participant Categorized by MedDRA Preferred Terms, Seriousness and Severity (N-Z). |
0.175; 0.098; 0.032; 0; 0.016; 0 | — |
| SECONDARY Rate of All AEs Per Infusion Categorized by MedDRA Preferred Terms, Seriousness and Severity (A-F). |
0.004; 0; 0.001; 0; 0.002; 0 | — |
| SECONDARY Rate of All AEs Per Infusion Categorized by MedDRA Preferred Terms, Seriousness and Severity (G-M). |
0.001; 0; 0.001; 0; 0.002; 0 | — |
| SECONDARY Rate of All AEs Per Infusion Categorized by MedDRA Preferred Terms, Seriousness and Severity (N-Z). |
0.007; 0.008; 0.001; 0; 0.001; 0 | — |
| 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") |
4.635; 2.137; 0.476; 0.392; 0.016; 0.059 | — |
| 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") |
0.183; 0.182; 0.019; 0.033; 0.001; 0.005 | — |
| SECONDARY Rate of AEs Per Participant (Including and Excluding Infections) Temporarily Associated With the Infusion |
5.921; 2.275; 1.111; 0.647; 0.032; 0.059 | — |
| SECONDARY Rate of AEs Per Infusion (Including and Excluding Infections) Temporarily Associated With the Infusion |
0.233; 0.194; 0.044; 0.055; 0.001; 0.005 | — |
| SECONDARY Percentage of Infusions Associated With One or More Local AEs (Including and Excluding Infections), at Any Time During the Study |
8.8; 3.7; 8.7; 3.7 | — |
Summary
The original purpose of the study is to assess the long-term safety, tolerability, and practicability of the subcutaneous (SC) treatment with Immune Globulin Subcutaneous Solution (IGSC), 10% facilitated with recombinant human hyaluronidase (rHuPH20) in participants with Primary Immunodeficiency Diseases (PID) who have completed Baxter Clinical Study Protocol No. 160603.
Following a discussion with the FDA, all participants still active in the study stopped treatment with rHuPH20 to assure safety of the participants participating in the study and went into a safety follow-up.
During this safety follow-up period, participants underwent either intravenous (IV) or SC treatment with IGSC, 10%. The IV or SC administration route was at the discretion of the participant and the investigator.
Eligibility Criteria
Inclusion Criteria
- Participant has completed or is about to complete Baxter Clinical Study Protocol No. 160603. Participants who have discontinued rHuPH20 and reverted to intravenous or subcutaneous treatment due to an anti-rHuPH20 antibody also may enroll for long-term safety monitoring.
- Participant/caretaker has reviewed, signed and dated informed consent
- Participant is willing and able to comply with the requirements of the protocol
Exclusion Criteria
- Participant has a serious medical condition such that the participant's safety or medical care would be impacted by participation in Study 160902
- Participant is scheduled to participate in another non-Baxter clinical study involving an investigational product or investigational device during the course of this study
- If female of childbearing potential, participant is pregnant or has a negative pregnancy test and does not agree to employ adequate birth control measures for the duration of the study
Data sourced from ClinicalTrials.gov (NCT01175213). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.