A Phase 2 interventional study of Asfotase Alfa in Hypophosphatasia (HPP), sponsored by Alexion Pharmaceuticals, Inc.. Completed at 2 sites in 2 countries. Open to participants aged 5 Years to 12 Years. Per ClinicalTrials.gov, last updated 2019-03-13.
Sponsored by Alexion Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
Asfotase alfa was formerly referred to as ENB-0040
Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.
45 studies on the registry are indexed under Hypophosphatasia; 10 are open to participants now.
This study's enrollment of 12 is below the median of 14 across 18 interventional studies indexed under Hypophosphatasia.
Browse Hypophosphatasia studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 249 studies on the registry; 25 are open to participants now.
Of its 98 completed or terminated interventional studies of FDA-regulated products, 70 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
asfotase alfa starting dose 3 mg/kg/week SC injection, increased to 6 mg/kg/week SC injection
Biological: Asfotase Alfa
Also known as: human recombinant tissue nonspecific alkaline phosphatase fusion protein
Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09.
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.
Time frame: At least 72 months of treatment with asfotase alfa
The main criteria for inclusion in Study ENB-006-09 were patients ages 5 to 12 years inclusive, with open growth plates at time of study entry and a documented diagnosis of HPP. To enter the extension, Study ENB-008-10, patients had to successfully complete Study ENB-006-09 and provide consent.
| Milestone | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa |
|---|---|---|
| Started | 6 | 6 |
| Completed | 6 | 6 |
| Not completed | 0 | 0 |
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.
| units on a scale | Asfotase Alfa Combined |
|---|---|
| Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09. | 2.83 (2.0 to 3.0) |
Collected over Treatment emergent AEs are events starting on or after the day of first dose of asfotase alfa and recorded in Study ENB-008-10 (at least 66 months of treatment in Study ENB-008-10).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 2 mg/kg Asfotase Alfa | 0/6 (0%) | 0/6 (0%) | 6/6 (100%) |
| 3 mg/kg Asfotase Alfa | 0/6 (0%) | 0/6 (0%) | 6/6 (100%) |
| Combined Asfotase Alfa Group | 0/12 (0%) | 0/12 (0%) | 12/12 (100%) |
| Event | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa | Combined Asfotase Alfa Group |
|---|---|---|---|
| Injection site erythemaGeneral disorders | 4/6 | 6/6 | 10/12 |
| Injection site maculeGeneral disorders | 4/6 | 5/6 | 9/12 |
| Upper respiratory tract infectionInfections and infestations | 5/6 | 4/6 | 9/12 |
| Injection site hypertrophyGeneral disorders | 4/6 | 4/6 | 8/12 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 4/6 | 1/6 | 5/12 |
| Injection site atrophyGeneral disorders | 3/6 | 3/6 | 6/12 |
| Gait disturbanceGeneral disorders | 0/6 | 3/6 | 3/12 |
| GastroenteritisInfections and infestations | 2/6 | 3/6 | 5/12 |
| PneumoniaInfections and infestations | 0/6 | 3/6 | 3/12 |
| ContusionInjury, poisoning and procedural complications | 3/6 | 0/6 | 3/12 |
| Age, Continuous(years) | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa | Total |
|---|---|---|---|
| Mean | 8.4 ± 2.21 | 9.0 ± 2.51 | 8.7 ± 2.27 |
| Age, Customized(Participants) | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa | Total |
|---|---|---|---|
| Age Group at Enrollment: 2 to 11 Years; % | 5 | 5 | 10 |
| Age Group at Enrollment: 12 to 17 Years; % | 1 | 1 | 2 |
| Sex: Female, Male(Participants) | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa | Total |
|---|---|---|---|
| Female | 1 | 1 | 2 |
| Male | 5 | 5 | 10 |
| Ethnicity (NIH/OMB)(Participants) | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 0 | 1 |
| Not Hispanic or Latino | 5 | 6 | 11 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 6 | 6 | 12 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(Participants) | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa | Total |
|---|---|---|---|
| Canada | 2 | 1 | 3 |
| United States | 4 | 5 | 9 |
| Hypophosphatasia Phenotype(Participants) | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa | Total |
|---|---|---|---|
| Infantile (< 6 months) | 3 | 1 | 4 |
| Juvenile (≥ 6 months to < 18 yrs) | 3 | 5 | 8 |
| Age at Onset of Hypophosphatasia Symptoms(Months) | 2 mg/kg Asfotase Alfa | 3 mg/kg Asfotase Alfa | Total |
|---|---|---|---|
| Mean | 10.8 ± 8.66 | 11.5 ± 5.54 | 11.2 ± 6.94 |
1 further baseline measures are reported on the registry.
This study is completed, as verified in Mar 2019. You cannot join it, but the record below documents what was studied.
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Alexion Pharmaceuticals, Inc.