Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

Last updated: February 16, 2026
Sponsor: Alexion Pharmaceuticals, Inc.
Overall Status: Active - Not Recruiting

Phase

3

Condition

N/A

Treatment

ALXN1850

Placebo

Clinical Study ID

NCT06079359
D8590C00003
ALXN1850-HPP-305
  • Ages 2-11
  • All Genders

Study 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

Inclusion Criteria:

  • Diagnosis of HPP documented in the medical records, and the following criteriafulfilled 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 theScreening Period as measured by the Central Laboratory OR 2 documented serumALP activity results, at least 15 days apart, below the age- and sex-adjustedlocal 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 laboratorycertification with Alexion's approval.

  • Must meet 1 of the following criteria:
  1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant ofunknown 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

Exclusion Criteria:

  • History or presence of cardiovascular, respiratory, hepatic, renal,gastrointestinal, endocrinological, hematological, neurological disorders, or anyother disorders that are capable of significantly altering the absorption,metabolism, or elimination of drugs; constituting a risk when taking the studyintervention; or interfering with the interpretation of data as determined by theInvestigator

  • 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 (inthe opinion of the Investigator) during the Randomized Evaluation Period

  • History of allergy or hypersensitivity to any ingredient contained in ALXN1850 orthe placebo comparator

Study Design

Total Participants: 30
Treatment Group(s): 2
Primary Treatment: ALXN1850
Phase: 3
Study Start date:
May 14, 2024
Estimated Completion Date:
August 24, 2028

Connect with a study center

  • Research Site

    Buenos Aires, C1199
    Argentina

    Site Not Available

  • Research Site

    Ciudad Autónoma de Buenos Aires, C1199
    Argentina

    Site Not Available

  • Research Site

    Ciudad de Buenos Aires, C1199
    Argentina

    Site Not Available

  • Research Site

    Mar del Plata, B7600FYK
    Argentina

    Site Not Available

  • Research Site

    Nedlands, 6009
    Australia

    Site Not Available

  • Research Site

    Nedlands 2064874, 6009
    Australia

    Site Not Available

  • Research Site

    Parkville, 3052
    Australia

    Site Not Available

  • Research Site

    Parkville 2153770, 3052
    Australia

    Site Not Available

  • Research Site

    Westmead, 2145
    Australia

    Site Not Available

  • Research Site

    Brussel, 1020
    Belgium

    Site Not Available

  • Research Site

    Brussels, 1020
    Belgium

    Site Not Available

  • Research Site

    Brussels 2800866, 1020
    Belgium

    Site Not Available

  • Research Site

    Brasilia, 71625-009
    Brazil

    Site Not Available

  • Research Site

    Brasília, 71625-009
    Brazil

    Site Not Available

  • Research Site

    Brasília 3469058, 71625-009
    Brazil

    Site Not Available

  • Research Site

    Porto Alegre, 90610-261
    Brazil

    Site Not Available

  • Research Site

    Porto Alegre 3452925, 90610-261
    Brazil

    Site Not Available

  • Research Site

    Recife, 50740-465
    Brazil

    Site Not Available

  • Research Site

    Recife 3390760, 50740-465
    Brazil

    Site Not Available

  • Research Site

    Salvador, 40050-410
    Brazil

    Site Not Available

  • Research Site

    Salvador 3450554, 40050-410
    Brazil

    Site Not Available

  • Research Site

    Sao Paulo, 05403-900
    Brazil

    Site Not Available

  • Research Site

    São Paulo, 05403-900
    Brazil

    Site Not Available

  • Research Site

    São Paulo 3448439, 01409-902
    Brazil

    Site Not Available

  • Research Site

    Calgary, Alberta T2E 7Z4
    Canada

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  • Research Site

    Edmonton, Alberta T6G 2H7
    Canada

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  • Research Site

    Calgary 5913490, Alberta 5883102 T2E 7Z4
    Canada

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    Winnepeg, Manitoba 6065171 R3E 3P4
    Canada

    Site Not Available

  • Research Site

    Ottawa, Ontario K1H 8L1
    Canada

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  • Research Site

    Beijing, 100045
    China

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  • Research Site

    Beijing 1816670, 100045
    China

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  • Research Site

    Guangzhou, 510623
    China

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  • Research Site

    Guangzhou 1809858, 510623
    China

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  • Research Site

    Shanghai, 2000127
    China

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  • Research Site

    Shanghai 1796236, 2000127
    China

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  • Research Site

    Shenzhen, 518053
    China

    Site Not Available

  • Research Site

    Shenzhen 1795565, 518053
    China

    Site Not Available

  • Research Site

    Helsinki, 00290
    Finland

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  • Research Site

    Helsinki 658225, 00290
    Finland

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    Le Kremlin Bicêtre, 94270
    France

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  • Research Site

    Le Kremlin-Bicêtre, 94270
    France

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    Paris, 75015
    France

    Site Not Available

  • Research Site

    Homburg, 66421
    Germany

    Site Not Available

  • Research Site

    Ashkelon, 7830604
    Israel

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  • Research Site

    Ashkelon 295620, 7830604
    Israel

    Site Not Available

  • Research Site

    Beer Sheva, 8410101
    Israel

    Site Not Available

  • Research Site

    Petach-Tikva, 4920235
    Israel

    Site Not Available

  • Research Site

    Petah Tikva 293918, 4920235
    Israel

    Site Not Available

  • Research Site

    Petah-Tikva, 4920235
    Israel

    Site Not Available

  • Research Site

    Ramat Gan, 52621
    Israel

    Site Not Available

  • Research Site

    Ramat Gan 293788, 52621
    Israel

    Site Not Available

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    Bologna, 40138
    Italy

    Site Not Available

  • Research Site

    Genova, 16147
    Italy

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    Milano, 20133
    Italy

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    Roma, 00190
    Italy

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  • Research Site

    Rome, 00050
    Italy

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    Seoul, 05505
    Korea, Republic of

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  • Research Site

    Campeche, 24050
    Mexico

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    Chihuahua, 31238
    Mexico

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    Chihuahua City 4014338, 31238
    Mexico

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  • Research Site

    Ciudad de Mexico, 14389
    Mexico

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  • Research Site

    Mexico City, '14080
    Mexico

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  • Research Site

    Zapopan, 45040
    Mexico

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  • Research Site

    Kraków, 31-503
    Poland

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  • Research Site

    Lodz 3093133, 93-338
    Poland

    Site Not Available

  • Research Site

    Warszawa, 01-211
    Poland

    Site Not Available

  • Research Site

    Łódź, 93-338
    Poland

    Site Not Available

  • Research Site

    Bucharest, 011863
    Romania

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  • Research Site

    Bucharest 683506, 011863
    Romania

    Site Not Available

  • Research Site

    Bucureşti, 011863
    Romania

    Site Not Available

  • Research Site

    Madrid, 28046
    Spain

    Site Not Available

  • Research Site

    Madrid 3117735, 28046
    Spain

    Site Not Available

  • Research Site

    Vitoria, 01009
    Spain

    Site Not Available

  • Research Site

    Vitoria-Gasteiz 3104499, 01009
    Spain

    Site Not Available

  • Research Site

    Stockholm, 17176
    Sweden

    Site Not Available

  • Research Site

    Stockholm 2673730, 17176
    Sweden

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  • Research Site

    Taipei, 100
    Taiwan

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  • Research Site

    Taipei 1668341, 100
    Taiwan

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  • Research Site

    Taoyuan, 333
    Taiwan

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  • Research Site

    Taoyuan District 1667905, 333
    Taiwan

    Site Not Available

  • Research Site

    Bangkok, 10400
    Thailand

    Site Not Available

  • Research Site

    Chaingmai, 50200
    Thailand

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  • Research Site

    Hat Yai, 90110
    Thailand

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  • Research Site

    Muang, 50200
    Thailand

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  • Research Site

    Ratchathewi, 10400
    Thailand

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  • Research Site

    Songkla, 90110
    Thailand

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  • Research Site

    Altındağ-Ankara, 06230
    Turkey

    Site Not Available

  • Research Site

    Ankara, 06100
    Turkey

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  • Research Site

    Bursa, 16059
    Turkey

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  • Research Site

    Edirne, 22030
    Turkey

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  • Research Site

    Erzurum, 25240
    Turkey

    Site Not Available

  • Research Site

    Istambul, 34899
    Turkey

    Site Not Available

  • Research Site

    Ankara 323786, 06560
    Turkey (Türkiye)

    Site Not Available

  • Research Site

    Bursa 750269, 16059
    Turkey (Türkiye)

    Site Not Available

  • Research Site

    Erzurum 315368, 25240
    Turkey (Türkiye)

    Site Not Available

  • Research Site

    Istanbul 745044, 34899
    Turkey (Türkiye)

    Site Not Available

  • Research Site

    Birmingham, B4 6NH
    United Kingdom

    Site Not Available

  • Research Site

    Birmingham 2655603, B4 6NH
    United Kingdom

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  • Research Site

    Manchester,
    United Kingdom

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  • Research Site

    Baltimore, Maryland 21287
    United States

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  • Research Site

    Baltimore 4347778, Maryland 4361885 21287
    United States

    Site Not Available

  • Research Site

    Kansas City, Missouri 64108
    United States

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  • Research Site

    Kansas City 4393217, Missouri 4398678 64108
    United States

    Site Not Available

  • Research Site

    Durham, North Carolina 27705
    United States

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  • Research Site

    Durham 4464368, North Carolina 4482348 27705
    United States

    Site Not Available

  • Research Site

    Hanoi, 100000
    Vietnam

    Site Not Available

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