Phase 3 Study of ALXN1850 versus Placebo in Adolescent and Adult Participants with HPP who have not previously been treated with Asfotase Alfa - HICKORY

Study identifier:D8590C00002

ClinicalTrials.gov identifier:NCT06079281

EudraCT identifier:N/A

CTIS identifier:N/A

Recruiting

Official Title

A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 (Recombinant Alkaline Phosphatase) Administered Subcutaneously in Adolescent (12 to < 18 years of age) and Adult Participants with Hypophosphatasia Who Have Not Previously Been Treated with Asfotase Alfa

Medical condition

Hypophosphatasia

Phase

Phase 3

Healthy volunteers

Study drug

ALXN1850, Placebo

Sex

All

Estimated Enrollment

114

Study type

Interventional

Age

12 Years - 130 Years

Date

Study Start Date: 03 Jan 2024

Estimated Primary Completion Date: 23 Dec 2025

Estimated Study Completion Date: 24 Jul 2028

Study design

Allocation: Randomized
Endpoint Classification: -
Intervention Model: Parallel Assignment
Masking: -
Primary Purpose: Treatment

Verification:

Verified 01 Aug 2024 by Alexion Pharmaceuticals, Inc.

Collaborators

Inclusion and exclusion criteria

Inclusion Criteria

- Diagnosis of HPP documented in the medical records
- Must meet 1 of the following criteria:
a. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of
unknown significance) from a Clinical Laboratory Improvement Amendments
(CLIA) or ISO 15189 certified laboratory (Section 8.7 )
b. Plasma PLP above the upper limit of normal (ULN) during the Screening Period
(central or local laboratory results allowed per local regulations)
- Must meet 1 of the following criteria without a probably cause other than HPP:
a. Serum ALP activity below the age- and sex-adjusted normal range during the screening period as measured by the Central Laboratory
b. Two documented serum ALP activity results, at least 15 days apart, below the age- and sex-adjusted local laboratory normal range during the 24 months before the Day 1 Visit. Note: Local laboratories need to be CLIA or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion’s approval.
- Two separate 6MWTs at below 85% of the predicted distance (for age, sex, weight, and height) during the Screening Period without a probable cause other than HPP

Exclusion Criteria

- History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal,
endocrinological, hematological, neurological disorders, or any other disorders that are
capable of significantly altering the absorption, metabolism, or elimination of drugs;
constituting a risk when taking the study intervention; or interfering with the
interpretation of data as determined by the Investigator
- Diagnosis of primary or secondary hyperparathyroidism
- Hypoparathyroidism, unless secondary to HPP
- Any new fracture within 12 weeks before Day 1 (excluding pseudofractures)
- Planned surgical intervention which may impact the results of study assessments (in the opinion of the Investigator) during the Randomized Evaluation Period
- History of allergy or hypersensitivity to any ingredient contained in ALXN1850 or the
placebo comparator (Table 9)

Sort by status

No locations available

Arms	Assigned Interventions
Placebo Comparator: Placebo Group During the Randomized Evaluation Period, the placebo group will receive placebo on Day 1, followed by once every 2 weeks (q2w) via SC injection for 24 weeks. Participants will enter the OLE Period and receive bodyweight dependent doses of either 20mg, 35mg, or 50mg of ALXN1850 and continue q2w dosing with ALXN1850 for up to 132 weeks.	Drug: Placebo Placebo will be administered via SC injection.
Experimental: ALXN1850 Group Starting at Day 1 of the Randomized Evaluation Period, the ALXN1850 group will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once q2w via SC injection, for 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks.	Drug: ALXN1850 ALXN1850 will be administered via subcutaneous (SC) injection. Drug: Placebo Placebo will be administered via SC injection.

Documents available

Trial Results Summaries
Available here

Contacts

Contact

Alexion Pharmaceuticals, Inc. (Sponsor)

1-855-752-2356

[email protected]