Clinical Trial Search

Primary: Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP) — 2.00 scores on a scale

Primary: Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate (PLP) — -254.96; -564.27; 3.13; -397.72 ng/mL — p=0.0285

Primary: Survival — 270.5 days

Primary: Change in Rickets Severity From Baseline to Week 24, Based on Assessment of Skeletal Radiographs Using Radiologic Global Impression of Change (RGI-C) — 2.00 Units on a…

Primary: Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi) — -36.46; -77.31; -56.88 percent change — p=0.0005

Primary: Change in Rickets Severity on Skeletal Radiographs From Baseline to Week 24 as Measured by the Radiographic Global Impression of Change (RGI-C) Scale — 0; 2.00 units on…

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) — 3; 5; 4; 0 Participants

Primary: Change In Plasma PPi From Baseline To Pre-3rd Dose At Week 9 — -2.604; -3.797; -4.484 μM — p=<0.0001

Primary: Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09…

Primary: Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa — 10 Participants

Primary: Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs) — 9; 2; 0 Participants

Primary: Radiographic Global Impression of Change - RGI-C — 0.33 units on a scale — p=0.0755

Primary: Requirements for Calcium Supplementation — 3.03; 1.66 grams per day of calcium supplementation

Primary: Modified Performance-Oriented Mobility Assessment-Gait Subtest (MPOMA-G) - Change From Baseline to Last Overall — 1.50 units on a scale — p=0.0625

Primary: Change in Urine cAMP — 3048 fm/uL

Primary: Number of Participants With Treatment Emergent Adverse Event (TEAE) and Treatment Emergent Serious Adverse Event (TESAE) — 13; 48 Participants

Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale

Primary: Serum Calcium Level — 2; 1.92 mmol/liter

Primary: Area Under the Curve for FGF23 — 3172.34; 3215.34 pg/ml*hr

Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm

Primary: The Percentage of Subjects Who Met the Triple Efficacy Endpoint Criteria at Week 24. — 2.5; 54.8 percentage of participants — p=<0.001

Primary: Area Under the Curve for Parathyroid Hormone (PTHauc) Measurement — 1223; 1449; 987; 1616 nlEq*26hr/ml — p=0.007

Primary: Change in Bone Mineral Density in Response to Pamidronate — -3.54; -4.98 Z-score

Primary: Amount of Bone Density Lost (%) From pre-to Post-operative DXA Scan — 0.043; -0.025 percent change

Primary: Change in Urine Total Protein — 1681; 59 mg/day

Primary: Change From Baseline (402) in Concentration of Albumin-corrected Serum Calcium (ACSC) at Month 6 — -0.005 Millimole per liter (mmol/L)

Primary: Annualized New Heterotopic Ossification (HO) — 9427.1; 23720.2 cubic millimeters (mm^3)

Primary: Lumbar Spine Bone Mineral Content — 41.4; 38.7 Average grams

Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615

Primary: Change in Dose of Calcium Supplementation — 37 percent change in dose