Safety and efficacy of asfotase alfa in children with hypophosphatasia

Conditions: Hypophosphatasia (HPP)
MedDRA version: 18.0Level: PTClassification code 10049933Term: HypophosphatasiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Registration Number: EUCTR2015-001128-52-Outside-EU/EEA

Lead Sponsor: Alexion Pharma International Sàrl

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 13

Inclusion Criteria

1) Patients > 5 and < 12 years of age with open growth plates at time of enrollment
2) Tanner stage of 2 or less indicating pre-pubescence
3) Documented history of HPP, as evidenced by:
-Presence of HPP-related rickets on skeletal radiographs of the wrist and knee
-Serum alkaline phosphatase (ALP) below age-adjusted normal range
-Plasma PLP at least twice the upper limit of normal
4) 25(OH) vitamin D level > 20 ng/mL
Are the trial subjects under 18? yes
Number of subjects for this age range: 13
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

1) Serum calcium or phosphorus below age-adjusted normal range
2) History of sensitivity to any study drug constituent
3) Medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities
4) Treatment with an investigational drug within 1 month before start of study drug
5) Current enrollment in any other study involving an investigational new drug, device, or treatment for HPP (e.g., bone marrow transplantation)
6) Current evidence of a treatable form of rickets
7) Prior treatment with bisphosphonates
8) Bone fracture or orthopedic surgery within the past 12 months that, in the opinion of the Investigator would interfere with the ability of study patient to comply with study protocol
9) Major congenital abnormality other than those associated with HPP