Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01203826 | Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP) | PHASE2 | COMPLETED | 12 | — | — | Apr 1, 2010 | Jun 1, 2016 | Mar 13, 2019 | 2 | United States, Canada |
| NCT00952484 | Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP) | PHASE2 | COMPLETED | 13 | — | — | Sep 1, 2009 | Jul 1, 2010 | Apr 1, 2019 | 2 | United States, Canada |
| NCT00744042 | Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP) | PHASE1 | COMPLETED | 11 | — | — | Sep 1, 2008 | May 1, 2010 | Apr 1, 2019 | 10 | United States, Canada +2 |
| NCT00739505 | Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP) | PHASE1 | COMPLETED | 6 | — | — | Aug 1, 2008 | Feb 1, 2009 | Mar 29, 2019 | 3 | United States, Canada |
Evaluation of radiographic change in rickets severity (as assessed by skeletal radiographs of the hands/wrists and knees) from the Baseline of Study ENB-006-09 (NCT00952484) to the End of Study (EOS) visit in Study ENB-008-10 using an ordinal RGI-C scale score. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP associated rickets) to +3 (indicative of complete or near complete healing of HPP associated rickets). The time points will be pre-treatment (Baseline from Study ENB-006-09) to the last radiographic assessment in Study ENB-008-10, which represents at least 72 months of treatment.
A 7-point RGI-C (radiographic global impression of change) score was used to rate change in rickets severity. Only those patients with a minimum score of +2 indicating substantial healing of rickets) were considered responders. Three pediatric radiologists not affiliated with the conduct of the study performed the ratings.
A 7-point RGI-C (Radiographic Global Impression of Change) score was used to rate change in rickets severity. Scores ranged from -3 (severe worsening of rickets) to +3 (complete healing of rickets). Only those patients with a minimum score of +2 indicating substantial healing of rickets) were considered "responders". Three pediatric radiologists not affiliated with the conduct of the study performed the ratings. Average scores were derived for each patient at each assessment.
| Arm | Type | Description |
|---|---|---|
| asfotase alfa | EXPERIMENTAL | asfotase alfa starting dose 3 mg/kg/week SC injection, increased to 6 mg/kg/week SC injection |
| 2 mg/kg | ACTIVE_COMPARATOR | 2 mg/kg subcutaneous injection three times per week. |
| 3 mg/kg | ACTIVE_COMPARATOR | 3 mg/kg subcutaneous injection three times per week. |
| Cohort 1 | EXPERIMENTAL | 3 HPP patients are to be enrolled in Cohort 1 and receive a single IV dose and three weekly SC doses of Asfotase Alfa . End of Study for patients in Cohort 1 is at 8 weeks. |
| Cohort 2 | EXPERIMENTAL | Cohort 2 will begin when the safety and PK data for Cohort 1 weeks 1-4 has been reviewed by the DSMB. Cohort 2 will enroll 3 HPP patients and will receive a higher dose level than Cohort 1. Cohort 2 patients will have a single IV dose and three weekly SC doses of Asfotase Alfa . End of Study for patients in Cohort 2 is at 8 weeks. |
| Name | Type | Description |
|---|---|---|
| Asfotase Alfa | BIOLOGICAL | - |
Inclusion Criteria: * Compliant and satisfactory completion of Enobia-sponsored clinical trial ENB-006-09 * Written informed consent by parent or other legal guardian prior to any study procedures being performed * Parent or other legal guardian willing to comply with study requirements Exclusion ...