Study of ALXN1850 in Participants With Hypophosphatasia (HPP)

NCT ID: NCT04980248

Last Updated: 2024-12-27

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-28

Study Completion Date

2022-08-24

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hypophosphatasia

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

HPP enzyme replacement therapy low alkaline phosphatase tissue-nonspecific alkaline phosphatase TNSALP

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SEQUENTIAL

Study participants will be enrolled into 3 cohorts in a sequential fashion.
Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type EXPERIMENTAL

ALXN1850

Intervention Type BIOLOGICAL

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

ALXN1850

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

Intervention Type BIOLOGICAL

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

64 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Research Site

Las Vegas, Nevada, United States

Site Status

Research Site

Columbus, Ohio, United States

Site Status

Research Site

Nashville, Tennessee, United States

Site Status

Research Site

Austin, Texas, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

Dahir KM, Shannon A, Dunn D, Voegtli W, Dong Q, Hasan J, Pradhan R, Pelto R, Pan WJ. Safety, pharmacokinetics, and pharmacodynamics of efzimfotase alfa, a second-generation enzyme replacement therapy: phase 1, dose-escalation study in adults with hypophosphatasia. J Bone Miner Res. 2024 Sep 26;39(10):1412-1423. doi: 10.1093/jbmr/zjae128.

Reference Type DERIVED
PMID: 39135540 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

ALXN1850-HPP-101

Identifier Type: -

Identifier Source: org_study_id