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

Hypophosphatasia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02235493
• Other study ID #: ALX-HPP-502s
• Status: Completed
• Start date: August 2014
• Est. completion date: September 2014

Study information

• Verified date: March 2019
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: September 2014
• Est. primary completion date: September 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Bone Disease
• Bone Diseases
• Hypophosphatasia

Intervention

Biological:
• Retrospective Case Only

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Modified Performance-Oriented Mobility Assessment-Gait Subtest (MPOMA-G) - Change From Baseline to Last Overall	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.	The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.
Secondary	Performance-Oriented Mobility Assessment-Gait Subtest (POMA-G) - Change From Baseline to Last Overall	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.	The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.

External Links

•   HPP support group
•   Hypophosphatasia Website
•   Hypophosphatasia Website for Health Care Professionals
•   US Hypophosphatasia Group (Soft Bones)
•   Yahoo health group for HPP with an international list serve where patients can correspond and post questions to the group