This clinical trial studied the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed study ENB-002-08 (NCT00744042). Partial funding for this study was provided by the Office of Orphan Product Development (OOPD).
Asfotase Alfa was formerly referred to as ENB-0040 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 limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Alfred I. DuPont Hospital for Children
Wilmington, Delaware, United States
St. John's Medical Research Institute
Springfield, Missouri, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Vanderbilt University Medical Center
Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa
Outcome measure is the number of patients with 1 or more treatment-emergent adverse event. The time period is from Baseline in the ENB-003-08 study to the end of the ENB-003-08 study.
Time frame: 84 months
Long-term Efficacy of Asfotase Alfa in Treating Rickets in Infants and Young Children With Hypophosphatasia (HPP).
Outcome measure is the evaluation of radiographic change in rickets severity using a qualitative Radiographic Global Impression of Change (RGI-C) Scale. Skeletal radiographs obtained at the patient's last assessment were compared with skeletal radiographs obtained before initiation of treatment (Baseline in Study ENB-002-08 \[NCT00744042\]). The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP-associated rickets) to +3 (indicative of complete or near complete healing of HPP-associated rickets). The time period is pre-dose (Baseline from ENB-002-08 study) to the last assessment for each patient in the ENB-003-08 study, which represents up to 90 months of exposure for the combined studies.
Time frame: Up to 90 Months
Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Plasma Inorganic Pyrophosphate (PPi) Levels
Outcome measure is the change from Baseline in plasma inorganic pyrophosphate (PPi) levels. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment for each patient in the ENB-003-08 study, which represents up to 90 months of exposure for the combined studies.
Time frame: Up to 90 Months
Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Pyridoxal-5-phosphate (PLP) Levels
Outcome measure is the change from Baseline in pyridoxal-5-phosphate (PLP) levels. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment for each patient in the ENB-003-08 study, which represents up to 90 months of exposure for the combined studies.
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Nashville, Tennessee, United States
St. Vincent Hospital
Green Bay, Wisconsin, United States
Tawam Hospital
Al Ain City, Abu Dhabi Emirate, United Arab Emirates
Royal Maternity Hospital, Royal Belfast Hospital for Sick Children
Belfast, Northern Ireland, United Kingdom
Sheffield Children's Hospital
Sheffield, United Kingdom
Time frame: Up to 90 Months
Effect of SC Asfotase Alfa on Growth: Weight Z-scores
Outcome measure is the change from Baseline in Z-scores for weight. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment in the ENB-003-08 study, which represents up to 90 months of exposure in the combined studies.
Time frame: Up to 90 Months
Effect of SC Asfotase Alfa on Growth: Height/Length Z-scores
Outcome measure is the change from Baseline in Z-scores for height/length. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment in the ENB-003-08 study, which represents up to 90 months of exposure in the combined studies.
Time frame: Up to 90 Months
Effect of SC Asfotase Alfa on Respiratory Function
Outcome measure is the shift in the proportion of patients requiring respiratory support at their last assessment in Study ENB-003-08 compared with Baseline. The time period is pre-dose (Baseline from the ENB-002-08 study \[NCT00744042\]) to the last assessment in the ENB-003-08 study, which represents up to 90 months of exposure in the combined studies.
Time frame: Up to 90 Months