Post-Authorization Study of HyQvia in Pediatric PIDD subjects
Research type
Research Study
Full title
Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases
IRAS ID
219189
Contact name
Andrew Cant
Contact email
Sponsor organisation
Baxalta US Inc.
Eudract number
2016-003438-26
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
Research Summary
The purpose of this study is to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study. Participation for one patient will last at maximum 4 years.
Approximately 40 participants will be enrolled across 20 sites in the European Union. 5 sites will be in the United Kingdom.
The treatment schedule consists of three study parts (Epochs), as below:
1) During Epoch 1, the dose and infusion intervals of HyQvia will be determined (maximum 6 weeks).
2) During Epoch 2, the participant will receive an infusion of HyQvia once every 3 or 4 weeks, for a maximum of 3 years. Depending on how the participant responds to treatment, they may either switch to Cuvitru or KIOVIG (to start Epoch 3) or stop the study.
3) During Epoch 3 (safety follow-up), the participant will receive Cuvitru or KIOVIG for up to 1 year.Safety events will be documented throughout the study, and blood and urine samples will be collected at specified study visits and sent to a lab for analysis.
Summary of Results
In conclusion, HyQvia was found to be safe and tolerable among pediatric subjects (2 to <18 years old) with PIDD. During the course of this study, the rate of TEAEs, both local and systemic, was low; a total of
2 treatment-related severe TEAEs at a rate of <0.1 (rate: 0.003) TEAE per infusion were reported and none of them were deemed serious. The rate per infusion of serious TEAEs (excluding infections) was <0.1 (rate: 0.005) with only 4 serious TEAEs reported in 3 HyQvia pre-treated subjects. None of these were related to the treatment.
Most of the ARs reported during the study were mild in severity. The rates of local ARs and systemic ARs were lower among the HyQvia pre-treated group compared to the HyQvia new starter group.
No substantial difference in the total IgG trough levels across timepoints from baseline was observed, and the protective IgG levels were maintained throughout Epoch 2 among all the study subjects with similar levels observed across age groups. The mean trough levels of specific antibodies against Clostridium tetani toxoid, Haemophilus influenzae B, and HBV were maintained at 10 times the levels considered to be the minimal protective titers. A similar trend was observed for IgG subclass levels with no substantial changes during the treatment period and no notable age group differences. In this study, no subject developed binding anti-rHuPH20 antibodies with a titer ≥160 at any timepoint. Because no subject was positive (titer ≥160) for binding antirHuPH20 antibodies, neutralizing anti-rHuPH20 antibodies were not assessed for any subject. The rate of ASBI occurrences among the pediatric subjects with PIDD was as low as 0.019 per subject-year. The efficacy of HyQvia was further demonstrated by the annualized rate of all infections per subject, consistent with results obtained in Clinical Studies 160603 and 160902. The rate of all infections per subject-year was 1.486; the rate was similar across both the HyQvia new starter and HyQvia pre-treated group.
The majority of the subjects achieved a treatment interval of 4 weeks and maintained a treatment interval of 3 or
4 weeks for 12 months during the Epoch 2. Overall, HyQvia was able to be administered subcutaneously at the same dosing interval as IV immunoglobulin therapies for PIDD, with a mean of 1.28 infusions per month and duration of 89.0 mins, with a good tolerability profile for both local and systemic ARs. For all the analyzed subjects, HyQvia was the preferred mode of immunoglobulin therapy for continuation. No new safety concerns were identified following HyQvia treatment in pediatric subjects with PIDD who had received immunoglobulin therapy prior to study enrollment.REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
17/NW/0022
Date of REC Opinion
14 Feb 2017
REC opinion
Further Information Favourable Opinion