Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharma International Sarl
ClinicalTrials.gov Identifier:
NCT01205152
First received: September 17, 2010
Last updated: May 6, 2014
Last verified: May 2014
  Purpose

This clinical trial studies the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed the ENB-002-08 study.

Partial funding for this study was provided by the Office of Orphan Product Development (OOPD).


Condition Intervention Phase
Hypophosphatasia
Biological: asfotase alfa
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Extension Study of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children With Hypophosphatasia (HPP)

Resource links provided by NLM:


Further study details as provided by Alexion Pharma International Sarl:

Primary Outcome Measures:
  • To determine the long-term tolerability of subcutaneous (SC) asfotase alfa [ Time Frame: 84 months ] [ Designated as safety issue: No ]
    The time points will be pre-dose (Baseline from the ENB-002-08 study) to Month 84 of the ENB-003-08 study which represents 90 months.

  • To assess the long-term efficacy of asfotase alfa in treating rickets in infants and young children with HPP [ Time Frame: 84 Months ] [ Designated as safety issue: No ]
    The time points will be pre-dose (Baseline from ENB-002-08 study) to Month 84 of the ENB-003-08 study which represents 90 months.


Secondary Outcome Measures:
  • To assess the long-term pharmacodynamics (PD) of SC asfotase alfa [ Time Frame: 84 Months ] [ Designated as safety issue: No ]
    The time points will be pre-dose (baseline from the ENB-002-08 study) to Month 84 of the ENB-003-08 study whih represents 90 months.

  • To assess the effect of SC asfotase alfa on growth and development [ Time Frame: 84 Months ] [ Designated as safety issue: No ]
    The time points will be pre-dose (Baseline from the ENB-002-08 study) to Month 84 in the ENB-003-08 study which represents 90 months.

  • To assess the effect of SC asfotase alfa on survival, respiratory function, and other clinical signs and symptoms of HPP in infants and young children [ Time Frame: 84 Months ] [ Designated as safety issue: No ]
    The time points will be pre-dose (Baseline from the ENB-002-08 study) to Month 84 of the ENB-003-08 study which represents 90 months.


Enrollment: 9
Study Start Date: April 2009
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: asfotase alfa Biological: asfotase alfa
Other Name: ENB-0040

Detailed Description:

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 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.

  Eligibility

Ages Eligible for Study:   6 Months to 42 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  • Patient completed participation in 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

Exclusion Criteria

  • History of sensitivity to any of the constituents of the study drug
  • Clinically significant disease that precludes study participation
  • Enrollment in any study involving an investigational drug, device, or treatment for HPP(e.g., bone marrow transplantation)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01205152

Locations
United States, Missouri
St. John's Medical Research Institute
Springfield, Missouri, United States, 65807
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198
United States, Tennessee
Vanderbilt Univesity Medical Center
Nashville, Tennessee, United States, 37232
United States, Wisconsin
St. Vincent Hospital
Green Bay, Wisconsin, United States, 54301
United Arab Emirates
Tawam Hospital
Al Ain, Abu-Dhabi, United Arab Emirates
United Kingdom
Royal Belfast Hospital for Sick Children
Belfast, Northern Ireland, United Kingdom
Sheffield Children's Hospital
Sheffield, United Kingdom, S10 2TH
Sponsors and Collaborators
Alexion Pharma International Sarl
  More Information

Additional Information:
No publications provided

Responsible Party: Alexion Pharma International Sarl
ClinicalTrials.gov Identifier: NCT01205152     History of Changes
Other Study ID Numbers: ENB-003-08, FD-R-003745-03
Study First Received: September 17, 2010
Last Updated: May 6, 2014
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Arab Emirates: General Authority for Health Services for Abu Dhabi

Keywords provided by Alexion Pharma International Sarl:
Hypophosphatasia
HPP
Bone Disease
Soft Bones
Low Alkaline Phosphatase
genetic metabolic disorder
alkaline phosphatase
tissue non-specific alkaline phosphatase
rickets
osteomalacia

Additional relevant MeSH terms:
Hypophosphatasia
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases

ClinicalTrials.gov processed this record on August 27, 2014