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Активно (без набиране) Фаза 3 NCT06079359

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

Фаза 3 – широко изпитване преди одобрение
Заболявания: Hypophosphatasia

Спонсор: Alexion Pharmaceuticals, Inc.

Налично на: БГ
Обобщение
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.
Кой може да участва
Inclusion Criteria: * Diagnosis of HPP documented in the medical records, and the following criteria fulfilled without other probable cause than HPP: 1. Presence of HPP-related rickets on skeletal X-rays during the Screening Period, with a minimum Rickets Severity Score (RSS) of 1.0 AND 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. * Must meet 1 of the following criteria: 1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a 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
Места на провеждане 16
Австралия (1)
Research Site
Nedlands
Белгия (1)
Research Site
Brussels
Бразилия (1)
Research Site
Brasília
Канада (1)
Research Site
Calgary , Alberta
Китай (1)
Research Site
Beijing
Финландия (1)
Research Site
Helsinki
Израел (1)
Research Site
Ashkelon
Mexico (1)
Research Site
Chihuahua City
Полша (1)
Research Site
Lodz
Румъния (1)
Research Site
Bucharest
Испания (1)
Research Site
Madrid
Швеция (1)
Research Site
Stockholm
Taiwan (1)
Research Site
Taipei
Турция (1)
Research Site
Ankara
Великобритания (1)
Research Site
Birmingham
САЩ (1)
Research Site
Baltimore , Maryland
Технически детайли
Статус
Активно (без набиране)
Фаза
Фаза 3
Вид изследване
INTERVENTIONAL
Пол
Мъже и жени
Минимална възраст
2 Years
Максимална възраст
11 Years
Здрави доброволци
Не
Начална дата
14.05.2024
Крайна дата
24.08.2028
Регистрационен номер
NCT06079359
Източник
anzctr
Запитване за медицински туризъм

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