Long-Term Safety and Efficacy of Asfotase Alfa in Children with Hypophosphatasia (HPP)

• Register: EUCTR
• Last refreshed on: 21 August 2017
• Main ID: EUCTR2017-003153-42-Outside-EU/EEA
• Date of registration: 03/08/2017
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharma GmbH
• Public title: Long-Term Safety and Efficacy of Asfotase Alfa in Children with Hypophosphatasia (HPP)
• Scientific title: Extension Study of Protocol ENB-006-09 Evaluating the Long-Term Safety and Efficacy of Asfotase Alfa (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Children with Hypophosphatasia (HPP)
• Target sample size: 12
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-003153-42
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Canada, United States

Contacts

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

Key inclusion & exclusion criteria

Inclusion criteria:
Patients must meet all of the following criteria for enrollment in this study:
? Compliant and satisfactory completion of Alexion-sponsored clinical trial ENB-006-09
? Written informed consent by parent or other legal guardian) prior to any study procedures being performed
? Parent or other legal guardian willing to comply with study requirements
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Patients will be excluded from enrollment in this study if they meet any of the following exclusion criteria:
? Clinically significant disease that precludes study participation, in the Investigator’s opinion
? Treatment with an investigational drug other than asfotase alfa
? Prior treatment with bisphosphonates
? Enrollment in any study (other than ENB-006-09) involving an investigational drug, device, or treatment
for HPP


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Hypophosphatasia
MedDRA version: 20.0 Level: PT Classification code 10049933 Term: Hypophosphatasia System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Intervention(s)

Trade Name: Strensiq
Product Name: Asfotase alfa
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ASFOTASE ALFA
Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-
INN or Proposed INN: ASFOTASE ALFA
Other descriptive name: Human recombinant tissue non-specific alkaline phosphatase fusion protein
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-

Primary Outcome(s)

Main Objective: To assess the long-term tolerability of subcutaneous (SC) asfotase alfa
To assess the proportion of asfotase alfa-treated patients showing radiographic change in the skeletal manifestations of HPP

Secondary Objective: To evaluate the long-term pharmacokinetics (PK) of SC asfotase alfa
To evaluate the effect of SC asfotase alfa on reduction in plasma inorganic pyrophosphate (PPi) and plasma pyridoxal-5’-phosphate (PLP)

Primary end point(s): - Long-term tolerability of subcutaneous (SC) asfotase alfa
- Proportion of asfotase alfa-treated patients showing radiographic change in the skeletal manifestations of HPP

Timepoint(s) of evaluation of this end point: Throughout the study

Secondary Outcome(s)

Secondary end point(s): To evaluate the long-term pharmacokinetics (PK) of SC asfotase alfa
To evaluate the effect of SC asfotase alfa on reduction in plasma inorganic pyrophosphate (PPi) and plasma pyridoxal-5’-phosphate (PLP)

Timepoint(s) of evaluation of this end point: Throughout the study

Secondary ID(s)

ENB-008-10

Source(s) of Monetary Support

Alexion Pharma GmbH