Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease (REBUILD)

Alexion Pharmaceuticals

Status and phase

Withdrawn

Phase 4

Conditions

Hypophosphatasia

Treatments

Drug: asfotase alfa

Study type

Interventional

Funder types

Industry

Identifiers

NCT04189315

AA-HPP-406

Details and patient eligibility

About

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).

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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
Teriparatide/parathyroid hormone analog use within 2 months prior to screening
Treatment with strontium or sclerostin inhibitors within 6 months prior to the first dose of study drug
Vitamin B6 use for at least 2 weeks prior to screening
Serum 25-hydroxy (25-OH) vitamin D below 20 nanogram (ng)/milliliter (mL), with repletion and recheck allowed at screening (results from local laboratory may be used if within 4 weeks of screening)
Female patients who are pregnant, planning to become pregnant, or breastfeeding