Clinical trial NCT06079372: Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa
Official title: A Phase 3, Randomized, Open-label, Parallel-arm, Active-controlled, Multicenter Study to Evaluate Safety and Efficacy of ALXN1850 Versus Asfotase Alfa Administered Subcutaneously in Pediatric Participants (2 to < 12 Years of Age) With Hypophosphatasia (HPP) Previously Treated With Asfotase Alfa
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Hypophosphatasia
Keywords: Hypophosphatasia
HPP
Asfotase Alfa
ALXN1850
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2023-10-12
Enrollment: 42
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 assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.


Eligibility criteria: Inclusion Criteria:

* Diagnosis of HPP documented in the medical records
* Presence of open growth plates by X-ray during Screening Period
* Tanner stage 2 or less during the Screening Period
* Must have been treated with 6 mg/kg/ week of asfotase alfa via SC injection administered as either 2mg/kg 3 times per week or 1 mg/kg 6 times per week for ≥ 6 months before Day 1. Note: participants currently treated with 9 mg/kg (eg, 3 mg/kg 3 times per week) will not be allowed in the study.

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 asfotase alfa or ALXN1850
* Body weight \< 10 kg during the Screening Period

Primary outcomes:
measure: Number of Participants with Treatment-emergent Adverse Events (TEAEs)
timeFrame: Baseline Through Day 169
Secondary outcomes:
measure: Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)
timeFrame: Baseline, Day 169
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
Other outcomes:
Locations:
city: Hartford
country: United States
facility: Research Site
state: Connecticut
city: Baltimore
country: United States
facility: Research Site
state: Maryland
city: Minneapolis
country: United States
facility: Research Site
state: Minnesota
city: Kansas City
country: United States
facility: Research Site
state: Missouri
city: Durham
country: United States
facility: Research Site
state: North Carolina
city: Nashville
country: United States
facility: Research Site
state: Tennessee
city: Mar del Plata
country: Argentina
facility: Research Site
city: South Brisbane
country: Australia
facility: Research Site
city: Ottawa
country: Canada
facility: Research Site
state: Ontario
city: Bunkyo-ku
country: Japan
facility: Research Site
city: Minato-ku
country: Japan
facility: Research Site
city: Suita-shi
country: Japan
facility: Research Site
city: Yonago-shi
country: Japan
facility: Research Site
city: Altındağ
country: Turkey
facility: Research Site
city: Ankara
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: Sheffield
country: United Kingdom
facility: Research Site
Study officials:
References: