Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in 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:
NCT01203826
First received: September 15, 2010
Last updated: May 27, 2014
Last verified: May 2014
  Purpose

This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed the ENB-006-09 study.


Condition Intervention Phase
Hypophosphatasia (HPP)
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 Protocol ENB-006-09 Evaluating the Long-term Safety and Efficacy of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP)

Resource links provided by NLM:


Further study details as provided by Alexion Pharma International Sarl:

Primary Outcome Measures:
  • Skeletal radiograph using a qualitative Radiographic Global Impression of Change (RGI-C)scale compared to baseline of treatment in ENB-006-09. [ Time Frame: 60 months ] [ Designated as safety issue: No ]
    The time points will be pre-treatment (Baseline from the 006 study) to Month 60 of the 008 study which represents 66 months of treatment.


Enrollment: 12
Study Start Date: April 2010
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
asfotase alfa 6 mg/kg/week SC injection
Biological: Asfotase Alfa
Other Name: human recombinant tissue non-specific alkaline phosphatase fusion protein

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:   5 Years to 13 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Compliant and satisfactory completion of Enobia-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

Exclusion Criteria:

  • Clinically significant disease that precludes study participation, in the Investigator's opinion
  • Treatment with an investigational drug other than Asfotase Alfa
  • Enrollment in any study involving an investigational drug, device, or treatment for HPP
  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: NCT01203826

Locations
United States, Missouri
Shriner's Hospital for Children
St. Louis, Missouri, United States, 63131
Canada, Manitoba
Children's Hospital Health Sciences Centre
Winnipeg, Manitoba, Canada, R3B 0A6
Sponsors and Collaborators
Alexion Pharma International Sarl
  More Information

Additional Information:
No publications provided

Responsible Party: Alexion Pharma International Sarl
ClinicalTrials.gov Identifier: NCT01203826     History of Changes
Other Study ID Numbers: ENB-008-10
Study First Received: September 15, 2010
Last Updated: May 27, 2014
Health Authority: United States: Food and Drug Administration
Canada: Health Canada

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 21, 2014