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

September 15, 2010
May 27, 2014
April 2010
December 2015   (final data collection date for primary outcome measure)
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.
Skeletal radiograph using a qualitative Radiographic Global Inpression of Change (RGI-C)scale compared to historical controls [ Time Frame: 12 months ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01203826 on ClinicalTrials.gov Archive Site
Not Provided
PK using serum peak and trough levels and PD of Plasma PPi, PLP and serum PTH as biomarkers for HPP [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
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)

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

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.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Hypophosphatasia (HPP)
Biological: Asfotase Alfa
Other Name: human recombinant tissue non-specific alkaline phosphatase fusion protein
Experimental: asfotase alfa
asfotase alfa 6 mg/kg/week SC injection
Intervention: Biological: Asfotase Alfa
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
12
December 2015
December 2015   (final data collection date for primary outcome measure)

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
Both
5 Years to 13 Years
No
Contact information is only displayed when the study is recruiting subjects
Canada,   United States
 
NCT01203826
ENB-008-10
Yes
Alexion Pharma International Sarl
Alexion Pharma International Sarl
Not Provided
Not Provided
Alexion Pharma International Sarl
May 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP