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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:
ClinicalTrials.gov Identifier:
NCT01203826
First Posted: September 16, 2010
Last Update Posted: July 26, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Alexion Pharma GmbH
  Purpose
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09.

Condition Intervention Phase
Hypophosphatasia (HPP) Biological: Asfotase Alfa Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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)

Resource links provided by NLM:


Further study details as provided by Alexion Pharma GmbH:

Primary Outcome Measures:
  • 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.



Enrollment: 12
Study Start Date: April 2010
Study Completion Date: June 2016
Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: asfotase alfa
asfotase alfa starting dose 3 mg/kg/week SC injection, increased to 6 mg/kg/week SC injection
Biological: Asfotase Alfa
Other Name: human recombinant tissue nonspecific alkaline phosphatase fusion protein

Detailed Description:

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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01203826


Locations
United States, Missouri
Shriners Hospital for Children
Saint Louis, Missouri, United States, 63110
Canada, Manitoba
Children's Hospital Health Sciences Centre
Winnipeg, Manitoba, Canada, R3A 1S1
Sponsors and Collaborators
Alexion Pharma GmbH
  More Information

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alexion Pharma GmbH
ClinicalTrials.gov Identifier: NCT01203826     History of Changes
Other Study ID Numbers: ENB-008-10
First Submitted: September 15, 2010
First Posted: September 16, 2010
Results First Submitted: June 27, 2017
Results First Posted: July 26, 2017
Last Update Posted: July 26, 2017
Last Verified: June 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Alexion Pharma GmbH:
Hypophosphatasia
HPP
Bone Disease
Soft Bones
Low Alkaline Phosphatase
genetic metabolic disorder
alkaline phosphatase
tissue non-specific alkaline phosphatase
rickets
osteomalacia

Additional relevant MeSH terms:
Hypophosphatasia
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Immunoglobulin G
Immunologic Factors
Physiological Effects of Drugs