Hypophosphatasia Clinical Trial
— MULBERRYOfficial 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
The primary purpose of this study is to evaluate the efficacy of ALXN1850 versus placebo on radiographic outcomes in pediatric participants with HPP who have not previously been treated with asfotase alfa.
Status | Recruiting |
Enrollment | 30 |
Est. completion date | August 20, 2027 |
Est. primary completion date | July 14, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 11 Years |
Eligibility | Inclusion Criteria: - Diagnosis of HPP documented in the medical records, and the following criteria fulfilled during the Screening Period without other probable cause than HPP: 1. Presence of HPP-related rickets on skeletal X-rays, with a minimum Rickets Severity Score (RSS) of 1.0 AND 2. Serum ALP activity below the age- and sex-adjusted normal range - Must meet 1 of the following criteria: 1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory (Section 8.7) 2. 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 |
Country | Name | City | State |
---|---|---|---|
Argentina | Research Site | Buenos Aires | |
Argentina | Research Site | Ciudad Autónoma de Buenos Aires | |
Argentina | Research Site | Mar del Plata | |
Argentina | Research Site | Mar del Plata | |
Australia | Research Site | Nedlands | |
Australia | Research Site | Parkville | |
Australia | Research Site | Westmead | |
Belgium | Research Site | Brussels | |
Brazil | Research Site | Brasilia | |
Brazil | Research Site | Porto Alegre | |
Brazil | Research Site | Recife | |
Brazil | Research Site | São Paulo | |
Canada | Research Site | Calgary | Alberta |
Canada | Research Site | Edmonton | Alberta |
Canada | Research Site | Ottawa | Ontario |
Canada | Research Site | Winnepeg | Manitoba |
China | Research Site | Beijing | |
China | Research Site | Guangzhou | |
China | Research Site | Shenzhen | |
France | Research Site | Le Kremlin Bicêtre | |
France | Research Site | Paris | |
Germany | Research Site | Homburg | |
Israel | Research Site | Ashkelon | |
Israel | Research Site | Beer Sheva | |
Israel | Research Site | Petach-Tikva | |
Israel | Research Site | Ramat Gan | |
Italy | Research Site | Bologna | |
Italy | Research Site | Genova | |
Italy | Research Site | Milano | |
Italy | Research Site | Roma | |
Italy | Research Site | Rome | |
Korea, Republic of | Research Site | Seoul | |
Mexico | Research Site | Campeche | |
Mexico | Research Site | Chihuahua | |
Mexico | Research Site | Ciudad de Mexico | |
Mexico | Research Site | Zapopan | |
Poland | Research Site | Kraków | |
Poland | Research Site | Lódz | |
Poland | Research Site | Warszawa | |
Spain | Research Site | Vitoria | |
Sweden | Research Site | Stockholm | |
Taiwan | Research Site | Taipei | |
Taiwan | Research Site | Taoyuan | |
Thailand | Research Site | Bangkok | |
Thailand | Research Site | Chaingmai | |
Thailand | Research Site | Hat Yai | |
Thailand | Research Site | Muang | |
Thailand | Research Site | Ratchathewi | |
Thailand | Research Site | Songkla | |
Turkey | Research Site | Ankara | |
Turkey | Research Site | Ankara | |
Turkey | Research Site | Bursa | |
Turkey | Research Site | Edirne | |
Turkey | Research Site | Erzurum | |
Turkey | Research Site | Istambul | |
United Kingdom | Research Site | Birmingham | |
United Kingdom | Research Site | Manchester | |
United States | Research Site | Baltimore | Maryland |
United States | Research Site | Durham | North Carolina |
United States | Research Site | Kansas City | Missouri |
Vietnam | Research Site | Hanoi |
Lead Sponsor | Collaborator |
---|---|
Alexion Pharmaceuticals, Inc. |
United States, Vietnam, Argentina, Australia, Belgium, Brazil, Canada, China, France, Germany, Israel, Italy, Korea, Republic of, Mexico, Poland, Spain, Sweden, Taiwan, Thailand, Turkey, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169) | Day 169 | ||
Secondary | Change from Baseline in Rickets Severity Score (RSS) at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 | ||
Secondary | Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 | ||
Secondary | Change from Baseline in Percent Predicted 6MWT at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 | ||
Secondary | Change from Baseline in Bruininks Oseretsky Test of Motor Proficiency, Second Edition (BOT2) Score at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 | ||
Secondary | Change from Baseline in Peabody Developmental Motor Scales, Third Edition (PDMS-3) Score at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 | ||
Secondary | RGI-C Responder at the end of the Randomized Evaluation Period (Day 169) | Day 169 |
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