Hypophosphatasia Clinical Trial
— HICKORYOfficial 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
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.
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) |
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 |
Lead Sponsor | Collaborator |
---|---|
Alexion Pharmaceuticals, Inc. |
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,
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 |
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