Skip to main content
OpenTrials
Completed

NCT Number: NCT02456038

Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)

The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Hypophosphatasia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Kurume University Hospital, Kurume, Fukuoka, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."

  • Patient who has been already treated with Asfotae Alfa (ALXN1215) out of this clinical trial
  • Patient who has been diagnosed as HPP
  • Documented diagnosis of HPP as indicated by:

<!-- -->

  • Total serum alkaline phosphatase below the lower limit of normal for age
  • Ultrasonographic features of prenatal, characterized by:
  • severe short extremities (femur length <-4SD in second and third trimesters) 2) extending into the metaphysis (femur metaphysis length or femur length >0.33) 3) craniotabes 4) Hypoplastic thorax (Thoracic or abdominal circumference <0.6) (3) Computed tomographic findings of prenatal, characterized by:
  • Generalized decreased ossification
  • Extreme shortening of tubular bones
  • Hypoplastic thorax (4) Radiographic evidence of HPP, characterized by:
  • Flared and frayed metaphyses 2) Severe, generalized osteopenia 3) Widened growth plates 4) Areas of radiolucency or sclerosis (5) Two or more of the following HPP-related findings:
  • History or presence of:
  • Nontraumatic post-natal fracture
  • Delayed fracture healing
  • Nephrocalcinosis or history of elevated serum calcium
  • Functional craniosynostosis
  • Respiratory compromise or rachitic chest deformity
  • Vitamin B6 dependent seizures
  • Failure to thrive
  • Premature tooth loss (6) Patient who have the mutation of tissue non-specific ALP gene 4. Parent or legal guardian(s) must provide written informed consent prior to any study procedures being performed and must be willing to comply with all study-required procedures

Exclusion criteria

  • Current evidence of treatable form of rickets
  • Serum calcium or phosphate levels below the normal range
  • Pregnant women and nursing mothers
  • Patient who cannot enforce suitable contraceptive measures during the clinical trial
  • Prior treatment with bisphosphonates
  • Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
  • Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
  • Clinically significant disease that precludes study participation, in the opinion of the Investigator

Treatment and study plan

Asfotase Alfa (ALXN1215)

Drug

Other names: ALXN1215

Primary outcomes

  1. Number of subjects with Adverse Events as an assessment of the Safety of repeated subcutaneous (SC) injections of asfotase alfa

    Time frame: Up to 50 months or until regulatory approval

    Safety of repeated subcutaneous (SC) injections of asfotase alfa for all treated patients

Secondary outcomes

  1. Overall survival

    Time frame: Up to 50 months or until regulatory approval

    Overall survival is defined as the time from birth to time of death.

  2. Effect of asfotase alfa treatment on skeletal manifestations of HPP as measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale

    Time frame: Up to 50 months or until regulatory approval

    Effect of asfotase alfa treatment on skeletal manifestations of HPP as measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale for all treated patients

  3. Effect of asfotase alfa treatment on ventilator-free survival: (percentage of patients who are alive and ventilator-free after receiving asfotase alfa)

    Time frame: Up to 50 months or until regulatory approval

    For patients who are not mechanically ventilated at the time of enrollment, the percentage who are alive and ventilator-free after receiving asfotase alfa

  4. Profile of asfotase alfa treatment on respiratory function

    Time frame: Up to 50 months or until regulatory approval

    Effect of asfotase alfa treatment on respiratory function as measured by ventilator status, time on respiratory support (including time on ventilator or supplemental oxygen), ventilator rate or oxygen volume, ventilator pressures, and fraction of inspired oxygen (FiO2) for all treated patients

  5. Profile of asfotase alfa treatment on physical growth

    Time frame: Up to 50 months or until regulatory approval

    Effect of asfotase alfa treatment on physical growth as measured by body weight, length, arm span, head circumference, and chest circumference for all treated patients

  6. Effect of asfotase alfa treatment on development

    Time frame: Up to 50 months or until regulatory approval

    Assessment of changes in gross motor development as measured by the developmental motor milestones for all treated patients

Sponsors and collaborators

Lead sponsor

Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan

Other

Collaborators

  • Osaka University Graduate School of Medicine

Registry information

Official study title

A Multicenter Study of the Safety and Efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Patients With Hypophosphatasia (HPP)

Important dates

Study start
2014
Primary completion
2015
Study completion
2015
First posted
May 28, 2015
Registry last updated
Mar 31, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.