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Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharma GmbH
ClinicalTrials.gov Identifier:
NCT01203826
First received: September 15, 2010
Last updated: June 27, 2017
Last verified: June 2017
September 15, 2010
June 27, 2017
April 2010
June 2016   (Final data collection date for primary outcome measure)
Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09. [ Time Frame: At least 72 months of treatment with asfotase alfa ]

Evaluation of radiographic change in rickets severity (as assessed by skeletal radiographs of the hands/wrists and knees) from the Baseline of Study ENB-006-09 relative to the End of Study (EOS) visit in Study ENB-008-10 using an ordinal RGI-C scale score. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP associated rickets) to +3 (indicative of complete or near complete healing of HPP associated rickets).

The time points will be pre-treatment (Baseline from Study ENB-006-09) to the last radiographic assessment in Study ENB-008-10, which represents at least 72 months of treatment.

Skeletal radiograph using a qualitative Radiographic Global Inpression of Change (RGI-C)scale compared to historical controls [ Time Frame: 12 months ]
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 ]
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 Asfotase Alfa (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 Study ENB-006-09.

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: None (Open Label)
Primary Purpose: Treatment
Hypophosphatasia (HPP)
Biological: Asfotase Alfa
Other Name: human recombinant tissue nonspecific alkaline phosphatase fusion protein
Experimental: asfotase alfa
asfotase alfa starting dose 3 mg/kg/week SC injection, increased to 6 mg/kg/week SC injection
Intervention: Biological: Asfotase Alfa
Whyte MP, Madson KL, Phillips D, Reeves AL, McAlister WH, Yakimoski A, Mack KE, Hamilton K, Kagan K, Fujita KP, Thompson DD, Moseley S, Odrljin T, Rockman-Greenberg C. Asfotase alfa therapy for children with hypophosphatasia. JCI Insight. 2016 Jun 16;1(9):e85971.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
12
June 2016
June 2016   (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 (other than ENB-006-09) involving an investigational drug, device, or treatment for HPP
  • Prior treatment with bisphosphonates
Sexes Eligible for Study: All
5 Years to 12 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Canada,   United States
 
 
NCT01203826
ENB-008-10
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Alexion Pharma GmbH
Alexion Pharma GmbH
Not Provided
Not Provided
Alexion Pharma GmbH
June 2017

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