Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

NCT02104219 | Completed Results

• 1 other identifier: ALX-HPP-502
• Study Type: observational
• Target: 32
• Locations: 8 countries
• Sites: 19

Brief Summary

The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.

Conditions

Hypophosphatasia (HPP)

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Radiographic Global Impression of Change - RGI-C

  The RGI-C scale is a 7-point ordinal scale that is used to evaluate musculoskeletal characteristics of HPP (eg, metaphyseal fraying, demineralization of distal metaphyses). The scores range from -3 (severe worsening) to +3 (complete or near-complete healing).

  Between Baseline (earliest available, complete, and readable x-ray set) and all available, readable post-Baseline x-ray sets during the period of patients' aged 5 to 15 years, inclusive.

Secondary Outcomes (2)

• Change in Height Z-score From Baseline to Last Assessment

  Any available growth data during the period of patients' aged 5 to 15 years, inclusive. Baseline is the earliest available assessment while post baselines are time points after Baseline during the defined age period.

• Change in Weight Z-score From Baseline to Last Assessment

  Any available growth data during the period of patients' aged 5 to 15 years, inclusive. Baseline is the earliest available assessment while Post baselines are time points after Baseline during the defined age period.

Other Outcomes (1)

• Rickets Severity Sale - RSS

  Any available data during the period of patients' aged 5 to 15 years, inclusive. Baseline is the earliest available assessment value during the period.

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

Juvenile-onset HPP (≥ 6 months and ˂18 years)

You may qualify if:

• Documented informed consent unless patient is deceased
• Patients with Juvenile-onset HPP, defined as documented onset of first signs/symptoms at ≥ 6 months to ˂18 years
• Documented diagnosis of HPP as indicated by skeletal manifestations and low alkaline phosphatase or genotyping

You may not qualify if:

• Received treatment with asfotase alfa in the ENB-006-09 study and/or currently enrolled in the ENB-008-10 study
• Received other treatment and/or intervention to treat HPP up to 15 years old
• Other clinically significant disease

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (19)

Unknown Facility

Atlanta, Georgia, United States

Location

Unknown Facility

Indianapolis, Indiana, United States

Location

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Boston, Massachusetts, United States

Location

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Springfield, Missouri, United States

Location

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St Louis, Missouri, United States

Location

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Durham, North Carolina, United States

Location

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Cincinnati, Ohio, United States

Location

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Houston, Texas, United States

Location

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Seattle, Washington, United States

Location

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Sydney, Australia

Location

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Winnipeg, Manitoba, Canada

Location

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Le Kremlin-Bicêtre, France

Location

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Paris, France

Location

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Toulouse, France

Location

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Rotterdam, Netherlands

Location

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Moscow, Russia

Location

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Istanbul, Turkey (Türkiye)

Location

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Manchester, United Kingdom

Location

Unknown Facility

Sheffield, United Kingdom

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: March 25, 2014
• First Posted: April 4, 2014
• Study Start: March 1, 2014
• Primary Completion: September 1, 2014
• Study Completion: September 1, 2014
• Last Updated: April 1, 2019
• Results First Posted: December 18, 2015

Record last verified: 2019-03

Locations

CA (1); NL (1); FR (3); RU (1); GB (2); AU (1); US (9); TU (1)