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

NCT02235493 | Completed Results

• 1 other identifier: ALX-HPP-502s
• Study Type: observational
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Hypophosphatasia; Bone Disease

Keywords

HPP; Soft Bones; low Alkaline Phosphatase; genetic metabolic disorder; alkaline phosphatase; tissue non-specific alkaline phosphatase; rickets; osteomalacia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Performance-Oriented Mobility Assessment-Gait Subtest (POMA-G) - Change From Baseline to Last Overall

  The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.

Study Arms (1)

Retrospective Case Only

Biological: Retrospective Case Only

Interventions

Retrospective Case Only BIOLOGICAL

Retrospective Case Only

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patient has juvenile-onset HPP and was selected as a historical-control for Study ENB-006-09.

You may qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (1)

Unknown Facility

St Louis, Missouri, United States

Location

Related Links

• Hypophosphatasia Website
• Hypophosphatasia Website for Health Care Professionals
• HPP support group
• US Hypophosphatasia Group (Soft Bones)

MeSH Terms

Conditions

Hypophosphatasia; Bone Diseases; Rickets; Osteomalacia

Condition Hierarchy (Ancestors)

Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Musculoskeletal Diseases; Bone Diseases, Metabolic; Calcium Metabolism Disorders; Vitamin D Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders

Results Point of Contact

• Title: Senior Medical Director, Clinical Development
• Organization: Alexion Pharmaceuticals

clinicaltrials@alxn.com

203-272-2596

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 5, 2014
• First Posted: September 10, 2014
• Study Start: August 1, 2014
• Primary Completion: September 1, 2014
• Study Completion: September 1, 2014
• Last Updated: March 13, 2019
• Results First Posted: September 28, 2015

Record last verified: 2019-03

Locations

US (1)