CompletedPhase 2

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

United States, Canada13 participantsStarted 2009-09

Plain-language summary

This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.

Age range5 Years – 12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Written informed consent from parent or legal guardian prior to participation
✓. Patients \> 5 and \< 12 years of age with open growth plates at time of enrollment
✓. Tanner stage of 2 or less indicating pre-pubescence
✓. Documented history of HPP, as evidenced by:
✓. 25(OH) vitamin D level \> 20 ng/mL
✓. Ability of patient and parent/guardian to comply with study requirements

✕. Serum calcium or phosphorus below age-adjusted normal range
✕. History of sensitivity to any study drug constituent
✕. 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
✕. Treatment with an investigational drug within 1 month before start of study drug
✕. Current enrollment in any other study involving an investigational new drug, device, or treatment for HPP (e.g., bone marrow transplantation)
✕. Current evidence of a treatable form of rickets
✕. Prior treatment with bisphosphonates
✕. 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

Change in Rickets Severity on Skeletal Radiographs From Baseline to Week 24 as Measured by the Radiographic Global Impression of Change (RGI-C) Scale

Timeframe: Baseline and Week 24

NCT IDNCT00952484

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2010-07

Results submitted2011-05-14

Who can participate

Age range5 Years – 12 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Written informed consent from parent or legal guardian prior to participation
✓. Patients \> 5 and \< 12 years of age with open growth plates at time of enrollment
✓. Tanner stage of 2 or less indicating pre-pubescence
✓. Documented history of HPP, as evidenced by:
✓. 25(OH) vitamin D level \> 20 ng/mL
✓. Ability of patient and parent/guardian to comply with study requirements

✕. Serum calcium or phosphorus below age-adjusted normal range
✕. History of sensitivity to any study drug constituent
✕. 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
✕. Treatment with an investigational drug within 1 month before start of study drug
✕. Current enrollment in any other study involving an investigational new drug, device, or treatment for HPP (e.g., bone marrow transplantation)
✕. Current evidence of a treatable form of rickets
✕. Prior treatment with bisphosphonates
✕. 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