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: April 9, 2014
Last verified: April 2014

September 17, 2010
April 9, 2014
April 2009
December 2015   (final data collection date for primary outcome measure)
  • 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.
Skeletal radiograph using a qualitative Radiographic Global Impression of Change (RGI-C) scale [ Time Frame: 24 months ] [ Designated as safety issue: No ]
The time points will be pre-treatment (Baseline from the 002 study) to Month 24 of the 003 study which represents 30 months of treatment.
Complete list of historical versions of study NCT01205152 on ClinicalTrials.gov Archive Site
  • 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.
Pharmacokinetics (PK) using serum PK and trough levels and pharmacodynamics (PD) of plasma inorganic pyrophosphate (PPi), pyridoxal 5'-phosphate (PLP) and serum parathyroid hormone (PTH) as biomarkers for hypophosphatasia (HPP) [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
Extension Study of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Severely Affected Infants and Young Children With Hypophosphatasia (HPP)

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.

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
Biological: asfotase alfa
Other Name: ENB-0040
Experimental: asfotase alfa
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
9
December 2015
December 2015   (final data collection date for primary outcome measure)

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)
Both
6 Months to 42 Months
No
Contact information is only displayed when the study is recruiting subjects
United States,   United Arab Emirates,   United Kingdom
 
NCT01205152
ENB-003-08
Yes
Alexion Pharma International Sarl
Alexion Pharma International Sarl
Not Provided
Principal Investigator: Jill Simmons, MD Vanderbilt Children's Hospital
Principal Investigator: Nick Bishop, MD Sheffield Children's Hospital
Principal Investigator: John Taylor, DO St.Vincent Hospital
Principal Investigator: Nada Salman, MD Tawam Hospital
Principal Investigator: Mairead McGinn, MD Royal Belfast Hospital for Sick Children
Principal Investigator: Jean Moore, MD St. John's Research Institute
Principal Investigator: Richard Lutz, MD University of Nebraska
Alexion Pharma International Sarl
April 2014

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