Study of ALXN1850 in Participants With Hypophosphatasia (HPP)

December 13, 2023 updated by: Alexion Pharmaceuticals, Inc.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Clinical Trial Site
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ALXN1850
Three experimental cohorts will be administered 3 dosages (low, medium, high) of ALXN1850, respectively, via IV infusion and/or SC over multiple administration intervals.
Biological: ALXN1850
ALXN1850 will be administered as an IV infusion and via the SC route.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence Of Treatment-emergent Adverse Events And Treatment-emergent Serious Adverse Events
Time Frame: Up to Day 85
Up to Day 85

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 16, 2021

Primary Completion (Actual)

August 24, 2022

Study Completion (Actual)

August 24, 2022

Study Registration Dates

First Submitted

July 23, 2021

First Submitted That Met QC Criteria

July 23, 2021

First Posted (Actual)

July 28, 2021

Study Record Updates

Last Update Posted (Estimated)

December 14, 2023

Last Update Submitted That Met QC Criteria

December 13, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALXN1850-HPP-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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