An observational study in Hypophosphatasia (HPP), sponsored by Alexion Pharmaceuticals, Inc.. Completed at 12 sites in 7 countries. Per ClinicalTrials.gov, last updated 2019-04-01.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Observational
This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.
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 no approved disease-modifying treatments for patients with this disease. There is also limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.
46 studies on the registry are indexed under Hypophosphatasia; 10 are open to participants now.
This study's enrollment of 48 is below the median of 55 across 23 observational studies indexed under Hypophosphatasia.
Browse Hypophosphatasia studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 24 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 73 (72%) have results posted.
First submitted before Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Patients with perinatal and/or infantile onset HPP. Thirty-six patients were invasively ventilated or died, and 12 patients were censored.
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Exclusion Criteria:
Patients will be excluded from study participation if they have 1 or more of the following exclusion criteria:
Both living and deceased patients will be considered for study participation
Patients with a confirmed diagnosis of perinatal or infantile onset hypophosphatasia (HPP)
Survival
Overall survival is defined as the time from birth to time of death.
Time frame: Retrospective data collected on or before the data of abstraction.
Invasive Ventilator-free Survival Time
Invasive ventilator-free survival is defined as the time during which the patient is alive and not invasively ventilated. For the purpose of this study, invasive ventilation is defined as mechanical ventilation via intubation of trachaeostomy.
Time frame: Retrospective data collected on or before the date of abstraction.
Patients were enrolled between Sept 2012 and Apr 2013. 65 patients were screened and 48 were enrolled at 12 sites in 7 countries. Patients were recruited from investigational sites established at academic research and medical centers known to diagnose and/or treat patients with HPP. Both living and deceased patients were considered to partricipate.
| Milestone | Retrospective Observational |
|---|---|
| Started | 48 |
| Completed | 48 |
| Not completed | 0 |
Overall survival is defined as the time from birth to time of death.
| days | Retrospective Observational |
|---|---|
| Survival | 270.5 (155.0 to 428.0) |
Invasive ventilator-free survival is defined as the time during which the patient is alive and not invasively ventilated. For the purpose of this study, invasive ventilation is defined as mechanical ventilation via intubation of trachaeostomy.
| days | Retrospective Observational |
|---|---|
| Invasive Ventilator-free Survival Time | 236 (78 to 300) |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Retrospective Observational | — | — | — |
Thirteen participants were alive at the time of data abstraction and were censored in the analysis
| Age, Continuous(weeks) | Retrospective Observational |
|---|---|
| Mean | 506.9 ± 319.24 |
| Age, Customized(weeks) | Retrospective Observational |
|---|---|
| <=18 years | 10 |
| Between 18 and 65 years | 3 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Retrospective Observational |
|---|---|
| Female | 22 |
| Male | 26 |
| Race (NIH/OMB)(Participants) | Retrospective Observational |
|---|---|
| American Indian or Alaska Native | 1 |
| Asian | 2 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 3 |
| White | 40 |
| More than one race | 0 |
| Unknown or Not Reported | 2 |
| Ethnicity (NIH/OMB)(Participants) | Retrospective Observational |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 41 |
| Unknown or Not Reported | 6 |
| Region of Enrollment(participants) | Retrospective Observational |
|---|---|
| Australia | 2 |
| Canada | 11 |
| Germany | 6 |
| Spain | 1 |
| Switzerland | 1 |
| Taiwan | 1 |
| United States | 26 |
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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