Extension open-label use of ENB-0040 for infantile hypophosphatasia

• Register: EUCTR
• Last refreshed on: 30 April 2019
• Main ID: EUCTR2009-009369-32-GB
• Date of registration: 14/04/2009
• Prospective Registration: No
• Primary sponsor: Alexion Pharma GmbH
• Public title: Extension open-label use of ENB-0040 for infantile hypophosphatasia
• Scientific title: Extension Study of ENB-0040 (Human Recombinant Tissue-Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children with Hypophosphatasia (HPP)
• Date of first enrolment: 30/03/2009
• Target sample size: 10
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-009369-32
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: Open: yes; Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other: 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

United Arab Emirates, United Kingdom, 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:
· Compliant and satisfactory completion, in the opinion of the Sponsor and Investigator, of study
ENB-002-08
· 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: 6
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:
- The patient has a history of sensitivity to any ENB 0040 constituents
- The patient has a clinically significant disease that precludes study participation, in the Investigator’s opinion
- The patient has been enrolled in any study involving an investigational drug, device, or treatment for HPP (e.g., one marrow transplantation)



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

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Infantile hypoposphatasia
MedDRA version: 19.1 Level: PT Classification code 10049933 Term: Hypophosphatasia System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Trade Name: Strensiq
Product Name: Asfotase alfa
Product Code: Asfotase alfa
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Asfotase alfa
Current Sponsor code: 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-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: 84 months

Main Objective: - To determine the long-term tolerability of subcutaneous (SC) Asfotase alfa
- To assess the long-term efficacy of Asfotase alfa in treating rickets in infants and young children with HPP

Primary end point(s): Skeletal radiograph using a qualitative Radiographic Global Impression of Change (RGI-C) scale.

Secondary Objective: - To assess the long-term pharmacodynamics (PD) of SC Asfotase alfa
- To assess the effect of SC Asfotase alfa on growth and development
- To assess the effect of SC Asfotase alfa on mortality and other clinical signs and symptoms of HPP in infants and young children

Secondary Outcome(s)

Secondary end point(s): Long-term pharmacodynamics of SC Asfotase alfa.
Effect of SC Asfotase alfa on growth and development, mortality, and other clinical signs and symptoms of HPP in infants and young children.

Timepoint(s) of evaluation of this end point: 84 months

Secondary ID(s)

ENB-003-08

Source(s) of Monetary Support

Alexion Pharma GmbH

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 17/09/2017
• Date Completed: 07/02/2017
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2009-009369-32/results