Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease (NCT04189315) | Clinical Trial Compass
WithdrawnPhase 4
Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease
Stopped: R\&D portfolio review and in light of the global impact of the COVID-19 pandemic
0Started 2020-04
Plain-language summary
The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).
Who can participate
Age range18 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Male or female aged ≥ 18 years at the time of signing the informed consent form
* Clinical diagnosis of pediatric-onset HPP based on signs and symptoms consistent with HPP
* Past medical history that includes at least one nonvertebral fracture (or pseudofracture) incurred without evidence of significant trauma.
* The presence of a current fracture is not necessary, but for participants with current unhealed fracture(s) or pseudofracture(s) of the lower extremity(ies) (that is, femoral, tibial, fibular, metatarsal) documentation must be provided of the presence of these fractures for at least 3 months prior to screening (with or without surgical intervention)
Exclusion Criteria:
* Medical condition, serious concurrent or recurrent illness and/or injury, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance or the assessment of study endpoints, including all protocol required evaluations and follow up activities, or may put the patient at risk
* Primary or secondary hyperparathyroidism or hypoparathyroidism
* History of hypersensitivity to any ingredient contained in asfotase alfa
* Oral bisphosphonate use within 6 to 12 months (depending on the half-life of the drug as assessed by the investigator) and intravenous (IV) bisphosphonate use within 12 months prior to screening
* Denosumab use within 18 months prior to screening
* Asfotase alfa use within 6 months prior to screening
* T…
What they're measuring
1
Change From Baseline To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1