HYPOPHOSPHATASIA
Conditions
Brief summary
Number and percentage of participants who achieve IST complete response at Week 100. The definition of IST complete response at Week 100 is defined as follows: ADA or NAb titer is decreased from Baseline by at least 2 titer steps or becomes negative, and Radiographic evidence of improvement on RSS by a score of at least 1 or more points from Baseline
Detailed description
ADA incidence, ADA response categories, ADA titer, NAb incidence and NAb titer in participants treated with IST undergoing asfotase alfa treatment through the duration of the study, Plasma concentrations of TNSALP as measured by asfotase alfa enzyme activity (PK), PLP, and PPi, at prespecified time points, Incidence of TEAEs and TESAEs, clinical safety laboratory test results, vital signs, 12-lead ECGs, physical examination findings, and for bortezomib treated participants echocardiography, and pediatric-modified Total Neuropathy Scale, Enumeration of CD19 B cells in participants treated with IST undergoing asfotase alfa treatment for the duration of the study
Interventions
DRUGIMMUNOGLOBULINS
DRUGNORMAL HUMAN
DRUGBortezomib Hikma 3
DRUGMTX HEXAL 2
DRUG5 mg Tabletten
DRUG-
DRUGMethotrexat-GRY® 50 mg/2 ml Injektionslösung
DRUGASFOTASE ALFA
Sponsors
Alexion Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number and percentage of participants who achieve IST complete response at Week 100. The definition of IST complete response at Week 100 is defined as follows: ADA or NAb titer is decreased from Baseline by at least 2 titer steps or becomes negative, and Radiographic evidence of improvement on RSS by a score of at least 1 or more points from Baseline | — |
Secondary
| Measure | Time frame |
|---|---|
| ADA incidence, ADA response categories, ADA titer, NAb incidence and NAb titer in participants treated with IST undergoing asfotase alfa treatment through the duration of the study, Plasma concentrations of TNSALP as measured by asfotase alfa enzyme activity (PK), PLP, and PPi, at prespecified time points, Incidence of TEAEs and TESAEs, clinical safety laboratory test results, vital signs, 12-lead ECGs, physical examination findings, and for bortezomib treated participants echocardiography, and pediatric-modified Total Neuropathy Scale, Enumeration of CD19 B cells in participants treated with IST undergoing asfotase alfa treatment for the duration of the study | — |
Countries
France
Outcome results
None listed