Ignite Creation Date:
2025-12-24 @ 3:04 PM
Ignite Modification Date:
2026-01-03 @ 12:08 PM
Study NCT ID:
NCT06079359
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-09-03
First Post:
2023-10-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
Sponsor:
Organization:
Raw JSON
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Presence of HPP-related rickets on skeletal X-rays during the Screening Period, with a minimum Rickets Severity Score (RSS) of 1.0 AND\n 2. 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.\n* Must meet 1 of the following criteria:\n\n 1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a CLIA certified laboratory (Section 8.7)\n 2. Plasma PLP above the upper limit of normal (ULN) during the Screening Period (central or local laboratory results allowed per local regulations)\n* Tanner stage 2 or less during the Screening Period\n\nExclusion Criteria:\n\n* 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\n* Diagnosis of primary or secondary hyperparathyroidism\n* Hypoparathyroidism, unless secondary to HPP\n* Any new fracture within 12 weeks before Day 1 (excluding pseudofractures)\n* Planned surgical intervention which may impact the results of study assessments (in the opinion of the Investigator) during the Randomized Evaluation Period\n* History of allergy or hypersensitivity to any ingredient contained in ALXN1850 or the placebo comparator"}, 'identificationModule': {'nctId': 'NCT06079359', 'acronym': 'MULBERRY', 'briefTitle': 'Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'D8590C00003'}, 'secondaryIdInfos': [{'id': 'ALXN1850-HPP-305', 'type': 'OTHER', 'domain': 'Alexion Pharmaceuticals, Inc.'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ALXN1850', 'description': '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.', 'interventionNames': ['Drug: ALXN1850', 'Drug: Placebo']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': '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.', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'ALXN1850', 'type': 'DRUG', 'description': 'Participants will receive a ALXN1850 via subcutaneous (SC) injection.', 'armGroupLabels': ['ALXN1850']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Participants will receive placebo via SC injection.', 'armGroupLabels': ['ALXN1850', 'Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '64108', 'city': 'Kansas City', 'state': 'Missouri', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 35.99403, 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