Study of ALXN1850 in Participants With Hypophosphatasia (HPP)

Alexion Pharmaceuticals

Status and phase: Completed; Phase 1

Conditions: Hypophosphatasia

Treatments: Biological: ALXN1850

Study type: Interventional

Funder types: Industry

Identifiers

NCT04980248

ALXN1850-HPP-101

Details and patient eligibility

About

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.

Enrollment: 15 patients

Sex: All

Ages: 18 to 64 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Basic Science

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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15 participants in 1 patient group

ALXN1850

Experimental group

Description:

Three experimental cohorts will be administered 3 dosages (low, medium, high) of ALXN1850, respectively, via IV infusion and/or SC over multiple administration intervals.

Treatment:

Biological: ALXN1850