Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants with HPP - MULBERRY

Study identifier:D8590C00003

ClinicalTrials.gov identifier:NCT06079359

EudraCT identifier:N/A

CTIS identifier:2023-505675-73-00

Recruiting

Official Title

A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 Versus Placebo Administered Subcutaneously in Pediatric (2 to < 12 years of age) Participants with Hypophosphatasia Who Have Not Received Previous Treatment with Asfotase Alfa

Medical condition

Hypophosphatasia

Phase

Phase 3

Healthy volunteers

Study drug

ALXN1850, Placebo

Sex

All

Estimated Enrollment

Study type

Interventional

Age

2 Years - 11 Years

Date

Study Start Date: 14 May 2024

Estimated Primary Completion Date: 12 Sept 2025

Estimated Study Completion Date: 21 Apr 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, and the following criteria fulfilled without other probable cause than HPP:
a. Presence of HPP-related rickets on skeletal X-rays during the Screening Period, with a minimum Rickets Severity
Score (RSS) of 1.0 AND
b. Serum ALP activity below the age- and sex-adjusted normal range during the Screening Period as measured by the Central Laboratory OR 2 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 Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion’s approval.
- Must meet 1 of the following criteria:
a. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of
unknown significance) from a CLIA 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)
- Tanner stage 2 or less during the Screening Period

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

Sort by status

No locations available

Arms	Assigned Interventions
Experimental: ALXN1850 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 every 2 weeks (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 Participants will receive a ALXN1850 via subcutaneous (SC) injection. Drug: Placebo Participants will receive placebo via SC injection.
Placebo Comparator: Placebo Starting at Day 1 during the Randomized Evaluation Period, participants will receive placebo q2w for a total of 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks.	Drug: Placebo Participants will receive placebo via SC injection.

Contacts

Contact

Alexion Pharmaceuticals, Inc. (Sponsor)

1-855-752-2356

[email protected]