Study of ALXN1850 in Participants With Hypophosphatasia (HPP)

Hypophosphatasia Clinical Trial

Official title:

A Phase 1, Open-label, Dose-escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ALXN1850 in Adults With Hypophosphatasia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04980248
• Other study ID #: ALXN1850-HPP-101
• Status: Completed
• Phase: Phase 1
• Start date: August 16, 2021
• Est. completion date: August 24, 2022

Study information

• Verified date: December 2023
• Source: Alexion Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is an open-label, dose-escalating study to assess safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and immunogenicity of ALXN1850 when given intravenous (IV) and subcutaneous (SC) to adults with HPP.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: August 24, 2022
• Est. primary completion date: August 24, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Confirmed clinical diagnosis of HPP
• Not anticipated to require further treatment with enzyme replacement therapy to treat participant's HPP after study completion
• Willing and able to follow protocol-specified contraception requirements
• Willing and able to give informed consent

Exclusion Criteria:

• Primary or secondary hyperparathyroidism or hypoparathyroidism
• Fracture within 12 weeks of screening
• Current or relevant history of unstable physical or psychiatric illness
• Significant allergies
• Asfotase alfa use within 6 months and/or positive for asfotase alfa antidrug antibody/neutralizing antibodies

Related Conditions & MeSH terms

• Hypophosphatasia

Intervention

Biological:
• ALXN1850
ALXN1850 will be administered as an IV infusion and via the SC route.

Locations

Country	Name	City	State
United States	Clinical Trial Site	Columbus	Ohio
United States	Clinical Trial Site	Nashville	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence Of Treatment-emergent Adverse Events And Treatment-emergent Serious Adverse Events		Up to Day 85
Secondary	Area Under The Plasma Concentration Versus Time Curve (AUC) Of Single IV And Multiple SC Doses Of ALXN1850		Predose through up to 168 hours following each dose administration
Secondary	Maximum Observed Plasma Concentration (Cmax) Of Single IV And Multiple SC Doses Of ALXN1850		Predose through up to 168 hours following each dose administration
Secondary	Area Under The Plasma Concentration Versus Time Curve Within The Dosing Interval (AUCtau) Values Of The First SC Versus IV Administration		Predose through up to 168 hours postdose
Secondary	Change From Baseline In Plasma Concentrations Of Inorganic Pyrophosphate (PPi)		Up to Day 85
Secondary	Change From Baseline In Plasma Concentrations Of Pyridoxal-5'-Phosphate (PLP)		Up to Day 85
Secondary	Change From Baseline In Plasma Concentrations Of PLP/Pyridoxal (PL) Ratio		Up to Day 85
Secondary	Assess incidence of Antidrug Antibody (ADA) and Neutralizing Antibody (NAbs)		Up to Day 85