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Активно (без набиране) Фаза 3 NCT06079281

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa

Фаза 3 – широко изпитване преди одобрение
Заболявания: Hypophosphatasia

Спонсор: Alexion Pharmaceuticals, Inc.

Налично на: БГ
Обобщение
The primary objective of this study is to assess the efficacy of ALXN1850 versus placebo on functional outcomes in adolescent and adult 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 * 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) or ISO 15189 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) * Must meet 1 of the following criteria without a probably cause other than HPP: 1. Serum ALP activity below the age- and sex-adjusted normal range during the screening period as measured by the Central Laboratory 2. 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)
Места на провеждане 17
Argentina (1)
Research Site
Ciudad de Buenos Aires
Австралия (1)
Research Site
Clayton
Бразилия (1)
Research Site
Belo Horizonte
Канада (1)
Research Site
Calgary , Alberta
Китай (1)
Research Site
Beijing
Франция (1)
Research Site
Paris
Германия (1)
Research Site
Bad Reichenhall
Израел (1)
Research Site
Ashkelon
Италия (1)
Research Site
Florence
Япония (1)
Research Site
Hiroshima
Полша (1)
Research Site
Lodz
Южна Корея (1)
Research Site
Seoul
Испания (1)
Research Site
Barcelona
Taiwan (1)
Research Site
Taipei
Турция (1)
Research Site
Ankara
Великобритания (1)
Research Site
London
САЩ (1)
Research Site
Indianapolis , Indiana
Технически детайли
Статус
Активно (без набиране)
Фаза
Фаза 3
Вид изследване
INTERVENTIONAL
Пол
Мъже и жени
Минимална възраст
12 Years
Максимална възраст
130 Years
Здрави доброволци
Не
Начална дата
03.01.2024
Крайна дата
29.03.2028
Регистрационен номер
NCT06079281
Източник
anzctr
Запитване за медицински туризъм

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