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

Hypophosphatasia Clinical Trial

— REBUILD  

Official title:

A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04189315
• Other study ID #: AA-HPP-406
• Status: Withdrawn
• Phase: Phase 4
• Start date: April 2020
• Est. completion date: August 2022

Study information

• Verified date: June 2020
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: August 2022
• Est. primary completion date: August 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hypophosphatasia

Intervention

Drug:
• asfotase alfa
Asfotase alfa is administered subcutaneously.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1		Baseline, Week 36
Secondary	Change From Baseline To Week 36 In Plasma Concentrations Of PPi In Group 2		Baseline, Week 36
Secondary	Change From Baseline To Week 36 In The 36-item Short-Form Survey (SF-36) Physical Component Summary (PCS) Score In Groups 1 And 2		Baseline, Week 36
Secondary	Change From Baseline To Week 36 In The Repeated Chair Stand Test (A Component Of The Short Physical Performance Battery [SPPB]) In Groups 1 And 2		Baseline, Week 36