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Clinical Trials/EUCTR2010-019850-42-DE
EUCTR2010-019850-42-DEActive, not recruitingPhase 1

An Open-Label, Multicenter, Multinational Study of the Safety, Efficacy and Pharmacokinetics of Asfotase alfa (human recombinant tissue nonspecific alkaline phosphatase fusion protein) in Infants and Children =5 Years of Age with Hypophosphatasia (HPP)

Alexion Pharma GmbH0 sites100 target enrollmentStarted: October 15, 2010Last updated:
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Conditions
Drugs

Trial Snapshot

Phase
Phase 1
Status
Active, not recruiting
Enrollment
100
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional clinical trial of medicinal product

Eligibility Criteria

Sex
All

Inclusion Criteria

  • •Patients must meet all of the following criteria for enrollment in this study:
  • •- 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-related procedures. If the minor is in a position to comprehend the nature, significance, and implications of the clinical trial and to form a rational intention in the light of these facts, then he/she must provide his/her assent prior to any study procedures being performed.
  • •- Documented diagnosis of HPP as indicated by:
  • •o Total serum alkaline phosphatase (ALP) below the lower limit of normal for age
  • •o Plasma PLP above the upper limit of normal (unless patient is receiving pyridoxine for seizures)
  • •o Radiographic evidence of HPP at screening, characterized by:
  • •* Flared and frayed metaphyses and
  • •* Severe, generalized osteopenia and
  • •* Widened growth plates and
  • •* Areas of radiolucency or sclerosis
  • •o 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
  • •- Onset of symptoms prior to 6 months of age
  • •- Chronological age of = 5 years or adjusted age for premature infants born = 37 weeks gestation
  • •- Otherwise medically stable in the opinion of the Investigator and/or Sponsor
  • •Are the trial subjects under 18? yes
  • •Number of subjects for this age range: 100
  • •F.1.2 Adults (18-64 years) no
  • •F.1.2.1 Number of subjects for this age range
  • •F.1.3 Elderly (>=65 years) no
  • •F.1.3.1 Number of subjects for this age range

Exclusion Criteria

  • •Patients will be excluded from enrollment in this study if they meet any of the following exclusion criteria:
  • •- Clinically significant disease, such as, but not limited to, hepatitis C virus (HCV) / human immunodeficiency virus (HIV) / hepatitis B virus (HBV), that precludes study participation, in the opinion of the Investigator and/or Sponsor
  • •- Serum calcium or phosphate levels below the normal range
  • •- Serum 25-hydroxy (25[OH]) vitamin D below 20 ng/mL
  • •- Current evidence of treatable form of rickets
  • •- 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)
  • •- Intolerance to the study drug or any of its excipients
  • •- Previous participation in the same study
  • •- Close relation to the Investigator

Investigators

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