Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
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| ClinicalTrials.gov Identifier: NCT02235493 |
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Recruitment Status :
Completed
First Posted : September 10, 2014
Results First Posted : September 28, 2015
Last Update Posted : March 13, 2019
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Sponsor:
Alexion Pharmaceuticals
Information provided by (Responsible Party):
Alexion Pharmaceuticals
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Brief Summary:
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
| Condition or disease | Intervention/treatment |
|---|---|
| Hypophosphatasia Bone Disease | Biological: Retrospective Case Only |
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.
| Study Type : | Observational |
| Actual Enrollment : | 6 participants |
| Observational Model: | Other |
| Time Perspective: | Retrospective |
| Official Title: | A Single-Center, Non-interventional Substudy of ALX-HPP-502 to Assess Functional Natural History Data of Patients With Juvenile Historical Controls in ENB-006-09 |
| Study Start Date : | August 2014 |
| Actual Primary Completion Date : | September 2014 |
| Actual Study Completion Date : | September 2014 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Hypophosphatasia
| Group/Cohort | Intervention/treatment |
|---|---|
| Retrospective Case Only |
Biological: Retrospective Case Only |
Primary Outcome Measures :
- Modified Performance-Oriented Mobility Assessment-Gait Subtest (MPOMA-G) - Change From Baseline to Last Overall [ Time Frame: The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive. ]The MPOMA-G is a 5-component assessment that is used to evaluate gait performance. The first component has 4 sub-components. For 2 components and 2 sub-components, scores of 0 or 1 are assigned while scores of 0, 1 or 2 are assigned to the rest of the 2 components and 2 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.
Secondary Outcome Measures :
- Performance-Oriented Mobility Assessment-Gait Subtest (POMA-G) - Change From Baseline to Last Overall [ Time Frame: The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive. ]The POMA-G is a 7-component assessment that is used to evaluate gait performance. The second component has 4 sub-components. Scores of 0, 1 or 2 are assigned to 2 components while scores of 0 or 1 are assigned to the rest of the 4 components and 4 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.
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| Ages Eligible for Study: | 5 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Study Population
Patient has juvenile-onset HPP and was selected as a historical-control for Study ENB-006-09.
Criteria
Inclusion Criteria:
- Documented informed consent/assent prior to video collection and data abstraction.
- Documented informed consent/assent in Study ALX-HPP-502.
- Patients with juvenile-onset HPP and was selected as a historical-control for Study ENB-006-09.
- Patients with at least 2 videos of basic mobility that were recorded at 2 different clinic visits when the patient was ≥5 to ≤15 ye
Exclusion Criteria:
- There are no exclusion criteria for this study
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02235493
Locations
| United States, Missouri | |
| Saint Louis, Missouri, United States | |
Sponsors and Collaborators
Alexion Pharmaceuticals
Additional Information:
| Responsible Party: | Alexion Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT02235493 |
| Other Study ID Numbers: |
ALX-HPP-502s |
| First Posted: | September 10, 2014 Key Record Dates |
| Results First Posted: | September 28, 2015 |
| Last Update Posted: | March 13, 2019 |
| Last Verified: | March 2019 |
Keywords provided by Alexion Pharmaceuticals:
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HPP Soft Bones low Alkaline Phosphatase genetic metabolic disorder |
alkaline phosphatase tissue non-specific alkaline phosphatase rickets osteomalacia |
Additional relevant MeSH terms:
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Bone Diseases Hypophosphatasia Musculoskeletal Diseases Metal Metabolism, Inborn Errors |
Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases |