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Completed

NCT Number: NCT04980248

Study of ALXN1850 in Participants With Hypophosphatasia (HPP)

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.

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Key information

Conditions

Hypophosphatasia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year–64 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Research Site, Las Vegas, Nevada, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ALXN1850

Biological

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

Primary outcomes

  1. Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)

    Time frame: Day 1 up to Day 85

    TEAEs were defined as any adverse events (AEs) that began or worsened on or after the first dose of treatment until the final follow-up visit. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. TESAEs were defined as any serious AEs that began or worsened on or after the first dose of treatment until the final follow-up visit. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Secondary outcomes

  1. Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3

    Time frame: Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29

  2. Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity Following IV Dose of ALXN1850

    Time frame: Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29

  3. Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Following SC Dose 1, SC Dose 2 and SC Dose 3

    Time frame: Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29

  4. Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Values of the First SC Versus IV Administration of ALXN1850

    Time frame: Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29

  5. Absolute Change From Baseline in Plasma Concentration of Pyridoxal-5' Phosphate (PLP) at Week 1

    Time frame: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3

  6. Absolute Change From Baseline in Plasma Concentration of Inorganic Pyrophosphate (PPi) at Week 1

    Time frame: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3

  7. Absolute Change From Baseline in Plasma Concentration of Pyridoxal Phosphate/ Pyridoxal (PLP/PL) Ratio at Week 1

    Time frame: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3

  8. Percent Change From Baseline in Plasma Concentration of PLP at Week 1

    Time frame: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3

  9. Percent Change From Baseline in Plasma Concentration of PPi at Week 1

    Time frame: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3

  10. Percent Change From Baseline in Plasma Concentration of PLP/PL Ratio Over Time at Week 1

    Time frame: Baseline, 1 Week post Day 1 IV dose and 1 Week post Day 29 SC dose 3

  11. Number of Participants With Anti-drug Antibody (ADA) Positive and Neutralizing Antibody (NAb) Positive Status

    Time frame: Baseline up to Day 85

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Jul 28, 2021
Registry last updated
Dec 27, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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