Extension Study of Protocol ENB-006-09 -Asfotase Alfa Treatment in Children With Hypophosphatasia

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: January 24, 2013
Last verified: July 2012

September 15, 2010
January 24, 2013
April 2010
December 2014   (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 -Asfotase Alfa Treatment in Children With Hypophosphatasia
Extension Study of Protocol ENB-006-09 Evaluating the Long-term Safety and Efficacy of Asfotase Alfa (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Children With Hypophosphatasia (HPP)

This clinical trial is being conducted to study long term safety and efficacy outcomes in children with hypophosphatasia (HPP) being treated with an investigational study drug called ENB-0040 (Asfotase Alfa).

Asfotase Alfa was formerly referred to as ENB-0040

Hypophosphatasia (HPP) is a rare inherited form of rickets and osteomalacia caused by inactivating mutations in the gene encoding the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP). The prevalence of the disease is thought to be about 1:100,000 although it is markedly higher in a small Canadian Mennonite population (Fraser 1957, Chodirker 1990). Inheritance can be autosomal recessive or dominant, and penetrance is variable resulting in a wide range of clinical expressivity. HPP differs from other forms of rickets and osteomalacia in that serum levels of calcium and phosphorus are generally normal or even elevated (Whyte 2002). Low circulating levels of alkaline phosphatase with elevated serum or urine levels of the TNSALP substrates inorganic pyrophosphate (PPi), pyridoxal 5'-phosphate (PLP) and phosphoethanolamine (PEA) are the biochemical hallmarks of this inborn error of metabolism.

Disease severity in HPP is inversely related to the age at symptom presentation. The most severe cases occur in utero and almost invariably result in death, generally due to pulmonary compromise. Infants who present in the first six months of life have about 50% mortality. Children and adults have less severe disease but can have frequent fractures, bone pain, bowing of the long bones and muscle weakness, and morbidity is generally cumulative. Some patients cannot ambulate independently and end up wheelchair-bound.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Hypophosphatasia
Biological: Asfotase Alfa
2 mg/kg SC injection 3 times per week for 60 months or 1mg/kg sc injection 6 times per week for 60 months.
Other Name: human recombinant tissue non-specific alkaline phosphatase fusion protein
Experimental: Asfotase Alfa
Asfotase Alfa 6 mg/kg/week SC injection for 60 months
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 2014
December 2014   (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
United States,   Canada
 
NCT01203826
ENB-008-10
Yes
Alexion Pharma International Sarl
Alexion Pharma International Sarl
Not Provided
Principal Investigator: Michael Whyte, MD Shriners Hospitals for Children
Principal Investigator: Cheryl Greenberg, MD Children's Hospital Health Sciences Centre
Alexion Pharma International Sarl
July 2012

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