Clinical trial NCT06079359: Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Hypophosphatasia
Keywords: Hypophosphatasia
HPP
ALXN1850
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2023-10-12
Enrollment: 30
Sex: ALL
Minimum age (years): 2
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)
timeFrame: Day 169
Secondary outcomes:
measure: Change from Baseline in Rickets Severity Score (RSS) at the end of the Randomized Evaluation Period (Day 169)
timeFrame: Baseline, Day 169
measure: Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169)
timeFrame: Baseline, Day 169
measure: Change from Baseline in Percent Predicted 6MWT at the end of the Randomized Evaluation Period (Day 169)
timeFrame: Baseline, Day 169
measure: Change from Baseline in Bruininks Oseretsky Test of Motor Proficiency, Second Edition (BOT2) Score at the end of the Randomized Evaluation Period (Day 169)
timeFrame: Baseline, Day 169
measure: Change from Baseline in Peabody Developmental Motor Scales, Third Edition (PDMS-3) Score at the end of the Randomized Evaluation Period (Day 169)
timeFrame: Baseline, Day 169
measure: RGI-C Responder at the end of the Randomized Evaluation Period (Day 169)
timeFrame: Day 169
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Research Site
state: Maryland
city: Kansas City
country: United States
facility: Research Site
state: Missouri
city: Durham
country: United States
facility: Research Site
state: North Carolina
city: Buenos Aires
country: Argentina
facility: Research Site
city: Buenos Aires
country: Argentina
facility: Research Site
city: Mar del Plata
country: Argentina
facility: Research Site
city: Mar del Plata
country: Argentina
facility: Research Site
city: Nedlands
country: Australia
facility: Research Site
city: Parkville
country: Australia
facility: Research Site
city: Westmead
country: Australia
facility: Research Site
city: Brussels
country: Belgium
facility: Research Site
city: Brasilia
country: Brazil
facility: Research Site
city: Porto Alegre
country: Brazil
facility: Research Site
city: Recife
country: Brazil
facility: Research Site
city: Salvador
country: Brazil
facility: Research Site
city: Sao Paulo
country: Brazil
facility: Research Site
city: São Paulo
country: Brazil
facility: Research Site
city: Calgary
country: Canada
facility: Research Site
state: Alberta
city: Edmonton
country: Canada
facility: Research Site
state: Alberta
city: Winnepeg
country: Canada
facility: Research Site
state: Manitoba
city: Ottawa
country: Canada
facility: Research Site
state: Ontario
city: Beijing
country: China
facility: Research Site
city: Guangzhou
country: China
facility: Research Site
city: Shanghai
country: China
facility: Research Site
city: Shenzhen
country: China
facility: Research Site
city: Helsinki
country: Finland
facility: Research Site
city: Le Kremlin Bicêtre
country: France
facility: Research Site
city: Paris
country: France
facility: Research Site
city: Homburg
country: Germany
facility: Research Site
city: Ashkelon
country: Israel
facility: Research Site
city: Beer Sheva
country: Israel
facility: Research Site
city: Petach-Tikva
country: Israel
facility: Research Site
city: Ramat Gan
country: Israel
facility: Research Site
city: Bologna
country: Italy
facility: Research Site
city: Genova
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Rome
country: Italy
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Campeche
country: Mexico
facility: Research Site
city: Chihuahua
country: Mexico
facility: Research Site
city: Chihuahua
country: Mexico
facility: Research Site
city: Ciudad de Mexico
country: Mexico
facility: Research Site
city: Ciudad de Mexico
country: Mexico
facility: Research Site
city: Zapopan
country: Mexico
facility: Research Site
city: Zapopan
country: Mexico
facility: Research Site
city: Kraków
country: Poland
facility: Research Site
city: Warszawa
country: Poland
facility: Research Site
city: Łódź
country: Poland
facility: Research Site
city: Bucharest
country: Romania
facility: Research Site
city: Madrid
country: Spain
facility: Research Site
city: Vitoria
country: Spain
facility: Research Site
city: Stockholm
country: Sweden
facility: Research Site
city: Taipei
country: Taiwan
facility: Research Site
city: Taoyuan
country: Taiwan
facility: Research Site
city: Bangkok
country: Thailand
facility: Research Site
city: Chaingmai
country: Thailand
facility: Research Site
city: Hat Yai
country: Thailand
facility: Research Site
city: Muang
country: Thailand
facility: Research Site
city: Ratchathewi
country: Thailand
facility: Research Site
city: Songkla
country: Thailand
facility: Research Site
city: Ankara
country: Turkey
facility: Research Site
city: Ankara
country: Turkey
facility: Research Site
city: Bursa
country: Turkey
facility: Research Site
city: Edirne
country: Turkey
facility: Research Site
city: Erzurum
country: Turkey
facility: Research Site
city: Istambul
country: Turkey
facility: Research Site
city: Birmingham
country: United Kingdom
facility: Research Site
city: Manchester
country: United Kingdom
facility: Research Site
city: Hanoi
country: Vietnam
facility: Research Site
Study officials:
References: