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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02104219
Other study ID # ALX-HPP-502
Secondary ID
Status Completed
Phase
First received
Last updated
Start date March 2014
Est. completion date September 2014

Study information

Verified date March 2019
Source Alexion Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
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.


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.


Other known NCT identifiers
  • NCT02104206

Recruitment information / eligibility

Status Completed
Enrollment 32
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 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

Exclusion Criteria:

- 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

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Netherlands,  Russian Federation,  Turkey,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Other Rickets Severity Sale - RSS The RSS is a 10-point scale, developed for nutritional rickets, that evaluates the degree of metaphyseal cupping and fraying and the proportion of growth plate affected (10 points = severe cupping/fraying, 0 points = absence of cupping/fraying). Any available data during the period of patients' aged 5 to 15 years, inclusive. Baseline is the earliest available assessment value during the period.
Primary 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 Change in Height Z-score From Baseline to Last Assessment Height measurements were assigned to Z-scores which were calculated using the Centers for Disease Control and Prevention (CDC) 2000 growth charts and methodology. The Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. 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.
Secondary Change in Weight Z-score From Baseline to Last Assessment Weight measurements were assigned a Z-score which was calculated using the Centers for Disease Control and Prevention (CDC) 2000 growth charts and methodology. The Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. 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.
See also
  Status Clinical Trial Phase
Completed NCT00952484 - Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP) Phase 2
Completed NCT00744042 - Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP) Phase 1/Phase 2
Active, not recruiting NCT04181164 - Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)
Completed NCT01419028 - A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
Completed NCT00739505 - Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP) Phase 1
Completed NCT01203826 - Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP) Phase 2
Enrolling by invitation NCT02306720 - Registry of Patients With Hypophosphatasia