Study of ALXN1850 in Participants With Hypophosphatasia (HPP)
Purpose
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.
Condition
- Hypophosphatasia
Eligibility
- Eligible Ages
- Between 18 Years and 64 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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 Design
- Phase
- Phase 1
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Sequential Assignment
- Intervention Model Description
- Study participants will be enrolled into 3 cohorts in a sequential fashion.
- Primary Purpose
- Basic Science
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
|
More Details
- Status
- Completed
- Sponsor
- Alexion Pharmaceuticals, Inc.