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

Hypophosphatasia Clinical Trial

— HICKORY  

Official title:

A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 (Recombinant Alkaline Phosphatase) Administered Subcutaneously in Adolescent (12 to < 18 Years of Age) and Adult Participants With Hypophosphatasia Who Have Not Previously Been Treated With Asfotase Alfa

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06079281
• Other study ID #: D8590C00002
• Secondary ID: ALXN1850-HPP-301
• Status: Recruiting
• Phase: Phase 3
• Start date: January 3, 2024
• Est. completion date: August 31, 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
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 114
• Est. completion date: August 31, 2027
• Est. primary completion date: September 22, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 130 Years

Eligibility

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) 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)

• Serum ALP activity below the age- and sex-adjusted normal range during the screening period, without a probable cause other than HPP

• 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)

Related Conditions & MeSH terms

• Hypophosphatasia

Intervention

Drug:
• ALXN1850
ALXN1850 will be administered via subcutaneous (SC) injection.
• Placebo
Placebo will be administered via SC injection.

Locations

Country	Name	City	State
Argentina	Research Site	Buenos Aires
Argentina	Research Site	Caba
Argentina	Research Site	Ciudad Autónoma de Buenos Aires
Argentina	Research Site	Córdoba
Argentina	Research Site	Mar del Plata
Australia	Research Site	Clayton
Australia	Research Site	Nedlands
Australia	Research Site	Parkville
Australia	Research Site	st Leonards
Australia	Research Site	Westmead
Austria	Research Site	Wien
Belgium	Research Site	Brussels
Belgium	Research Site	Edegem
Belgium	Research Site	Leuven
Brazil	Research Site	Belo Horizonte
Brazil	Research Site	Brasilia
Brazil	Research Site	Porto Alegre
Brazil	Research Site	Recife
Brazil	Research Site	Salvador
Brazil	Research Site	Sao Paulo
Brazil	Research Site	São Paulo
Canada	Research Site	Calgary	Alberta
Canada	Research Site	Edmonton	Alberta
Canada	Research Site	Oakville	Ontario
Canada	Research Site	Trois-Rivières	Quebec
Canada	Research Site	Vancouver	British Columbia
Canada	Research Site	Winnepeg	Manitoba
China	Research Site	Beijing
China	Research Site	Beijing
China	Research Site	Changsha
China	Research Site	Chengdu
China	Research Site	Nanchang
China	Research Site	Nanjing
China	Research Site	Qingdao
China	Research Site	Shanghai
China	Research Site	Shenzhen
China	Research Site	Wuhan
Czechia	Research Site	Praha
Czechia	Research Site	Praha
France	Research Site	Lille
France	Research Site	Paris
France	Research Site	Poitiers
France	Research Site	Toulouse
Germany	Research Site	Bad Reichenhall
Germany	Research Site	Berlin
Germany	Research Site	Bonn
Germany	Research Site	Hamburg
Germany	Research Site	Würzburg
India	Research Site	Bangalore
India	Research Site	Chandigarh
Israel	Research Site	Ashkelon
Israel	Research Site	Beer Sheva
Israel	Research Site	Petach-Tikva
Israel	Research Site	Ramat Gan
Italy	Research Site	Firenze
Italy	Research Site	Genova
Italy	Research Site	Milano
Italy	Research Site	Milano
Italy	Research Site	Padova
Italy	Research Site	Pisa
Italy	Research Site	Roma
Italy	Research Site	Rome
Italy	Research Site	San Giovanni Rotondo
Italy	Research Site	Verona
Japan	Research Site	Minato-ku
Japan	Research Site	Sapporo-shi
Japan	Research Site	Yaizu-shi
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Suwon-si
Poland	Research Site	Kraków
Poland	Research Site	Lódz
Poland	Research Site	Warszawa
Slovakia	Research Site	Bratislava
Spain	Research Site	Barcelona
Spain	Research Site	Granada
Spain	Research Site	La Laguna
Spain	Research Site	Madrid
Spain	Research Site	Santander
Spain	Research Site	Vitoria
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	Songkla
Turkey	Research Site	Ankara
Turkey	Research Site	Ankara
Turkey	Research Site	Erzurum
Turkey	Research Site	Istambul
United Kingdom	Research Site	London
United Kingdom	Research Site	Oxford
United Kingdom	Research Site	Sheffield
United Kingdom	Research Site	Stanmore
United States	Research Site	Baltimore	Maryland
United States	Research Site	Boston	Massachusetts
United States	Research Site	Columbus	Ohio
United States	Research Site	Durham	North Carolina
United States	Research Site	Indianapolis	Indiana
United States	Research Site	Madison	Wisconsin
United States	Research Site	Mineola	New York
United States	Research Site	Nashville	Tennessee
United States	Research Site	Pittsburgh	Pennsylvania
United States	Research Site	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Austria,  Belgium,  Brazil,  Canada,  China,  Czechia,  France,  Germany,  India,  Israel,  Italy,  Japan,  Korea, Republic of,  Poland,  Slovakia,  Spain,  Taiwan,  Thailand,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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 30-second Sit to Stand (STS) Test Score at the end of the Randomized Evaluation Period (Day 169)		Baseline, Day 169
Secondary	Change from Baseline in Lower Extremity Functional Scale (LEFS) Score at the end of the Randomized Evaluation Period (Day 169)		Baseline, Day 169
Secondary	Change from Baseline in Timed Up-and-Go (TUG) 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	Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)		Day 169
Secondary	RGI-C Responder 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