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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06079359
Other study ID # D8590C00003
Secondary ID ALXN1850-HPP-305
Status Recruiting
Phase Phase 3
First received
Last updated
Start date May 14, 2024
Est. completion date August 20, 2027

Study information

Verified date June 2024
Source Alexion Pharmaceuticals, Inc.
Contact Alexion Pharmaceuticals, Inc. (Sponsor)
Phone 1-855-752-2356
Email clinicaltrials@alexion.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
ALXN1850
Participants will receive a ALXN1850 via subcutaneous (SC) injection.
Placebo
Participants will receive placebo via SC injection.

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

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, 

Outcome

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
See also
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Recruiting NCT02237625 - Natural History Study of Patients With Hypophosphatasia (HPP)
Completed NCT02291497 - Burden of Disease in Hypophosphatasia (HPP) N/A
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Active, not recruiting NCT04195763 - Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
Not yet recruiting NCT05596539 - Prospective, Longitudinal, Observational Registry of Adult Patients With Hypophosphatasia (REG-HYPO)
Completed NCT02796885 - Characterisation of Adult-Onset Hypophosphatasia
Completed NCT02751801 - Health Burden of Hypophosphatasia
Completed NCT05890794 - Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia Phase 1/Phase 2
Recruiting NCT05234567 - A Prospective Sub-Study of the Global Hypophosphatasia Registry
Completed NCT02797821 - Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP) Phase 2
Completed NCT01163149 - Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP) Phase 2
Completed NCT04925804 - Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)
Completed NCT02531867 - Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan Phase 4
Completed NCT01406977 - Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP) Phase 2
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Completed NCT01176266 - Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP) Phase 2/Phase 3
Withdrawn NCT00894075 - Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP) Phase 2
Active, not recruiting NCT04222452 - The PORTRAIT Study
Recruiting NCT06079281 - Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa Phase 3