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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharma GmbH
ClinicalTrials.gov Identifier:
NCT01205152
First received: September 17, 2010
Last updated: July 5, 2017
Last verified: July 2017
September 17, 2010
July 5, 2017
April 2009
August 2016   (Final data collection date for primary outcome measure)
  • To determine the long-term tolerability of subcutaneous (SC) asfotase alfa [ Time Frame: 84 months ]
    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 ]
    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 ]
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 ]
    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 ]
    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 ]
    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 ]
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.

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

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
Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Hypophosphatasia
Biological: asfotase alfa
Other Name: ENB-0040
Experimental: asfotase alfa
Intervention: Biological: asfotase alfa
Whyte MP, Rockman-Greenberg C, Ozono K, Riese R, Moseley S, Melian A, Thompson DD, Bishop N, Hofmann C. Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia. J Clin Endocrinol Metab. 2016 Jan;101(1):334-42. doi: 10.1210/jc.2015-3462. Epub 2015 Nov 3.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
10
August 2016
August 2016   (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)
Sexes Eligible for Study: All
6 Months to 42 Months   (Child)
No
Contact information is only displayed when the study is recruiting subjects
United Arab Emirates,   United Kingdom,   United States
 
 
NCT01205152
ENB-003-08
FD-R-003745-03 ( Other Grant/Funding Number: Office of Orphan Product Development )
Yes
Not Provided
Not Provided
Alexion Pharma GmbH
Alexion Pharma GmbH
Not Provided
Not Provided
Alexion Pharma GmbH
July 2017

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