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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06079281




Registration number
NCT06079281
Ethics application status
Date submitted
6/10/2023
Date registered
12/10/2023

Titles & IDs
Public title
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa
Scientific 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
Secondary ID [1] 0 0
ALXN1850-HPP-301
Secondary ID [2] 0 0
D8590C00002
Universal Trial Number (UTN)
Trial acronym
HICKORY
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypophosphatasia 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ALXN1850
Treatment: Drugs - Placebo

Placebo comparator: Placebo Group - During the Randomized Evaluation Period, the placebo group will receive placebo on Day 1, followed by once every 2 weeks (q2w) via SC injection for 24 weeks. Participants will enter the OLE Period and receive bodyweight dependent doses of either 20mg, 35mg, or 50mg of ALXN1850 and continue q2w dosing with ALXN1850 for up to 132 weeks.

Experimental: ALXN1850 Group - Starting at Day 1 of the Randomized Evaluation Period, the ALXN1850 group will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once q2w via SC injection, for 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks.


Treatment: Drugs: ALXN1850
ALXN1850 will be administered via subcutaneous (SC) injection.

Treatment: Drugs: Placebo
Placebo will be administered via SC injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169)
Timepoint [1] 0 0
Baseline, Day 169
Secondary outcome [1] 0 0
Change from Baseline in 30-second Sit to Stand (STS) Test Score at the end of the Randomized Evaluation Period (Day 169)
Timepoint [1] 0 0
Baseline, Day 169
Secondary outcome [2] 0 0
Change from Baseline in Lower Extremity Functional Scale (LEFS) Score at the end of the Randomized Evaluation Period (Day 169)
Timepoint [2] 0 0
Baseline, Day 169
Secondary outcome [3] 0 0
Change from Baseline in Timed Up-and-Go (TUG) at the end of the Randomized Evaluation Period (Day 169)
Timepoint [3] 0 0
Baseline, Day 169
Secondary outcome [4] 0 0
Change from Baseline in Percent Predicted 6MWT at the end of the Randomized Evaluation Period (Day 169)
Timepoint [4] 0 0
Baseline, Day 169
Secondary outcome [5] 0 0
Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)
Timepoint [5] 0 0
Day 169
Secondary outcome [6] 0 0
RGI-C Responder at the end of the Randomized Evaluation Period (Day 169)
Timepoint [6] 0 0
Day 169
Secondary outcome [7] 0 0
Change from Baseline in Rickets Severity Score (RSS) at the end of the Randomized Evaluation Period (Day 169)
Timepoint [7] 0 0
Baseline, Day 169

Eligibility
Key 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) or ISO 15189 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)
* Must meet 1 of the following criteria without a probably cause other than HPP:

1. Serum ALP activity below the age- and sex-adjusted normal range during the screening period as measured by the Central Laboratory
2. Two documented serum ALP activity results, at least 15 days apart, below the age- and sex-adjusted local laboratory normal range during the 24 months before the Day 1 Visit. Note: Local laboratories need to be CLIA or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval.
* 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
Minimum age
12 Years
Maximum age
130 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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)

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/01/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
29/03/2028
Actual
Sample size
Target
114
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Clayton
Recruitment hospital [2] 0 0
Research Site - Herston
Recruitment hospital [3] 0 0
Research Site - Nedlands
Recruitment hospital [4] 0 0
Research Site - Parkville
Recruitment hospital [5] 0 0
Research Site - st Leonards
Recruitment hospital [6] 0 0
Research Site - Westmead
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
4029 - Herston
Recruitment postcode(s) [3] 0 0
6009 - Nedlands
Recruitment postcode(s) [4] 0 0
3052 - Parkville
Recruitment postcode(s) [5] 0 0
2065 - st Leonards
Recruitment postcode(s) [6] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
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California
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Indiana
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Maryland
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Massachusetts
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Missouri
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New York
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North Carolina
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Ohio
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Pennsylvania
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Tennessee
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Wisconsin
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Argentina
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Buenos Aires
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Argentina
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Caba
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Argentina
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Córdoba
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Mar del Plata
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Austria
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Wien
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Belgium
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Brussels
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Belgium
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Edegem
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Belgium
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Leuven
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Brazil
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Belo Horizonte
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Brazil
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Brasilia
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Brazil
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Porto Alegre
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Recife
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Salvador
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British Columbia
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Manitoba
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Ontario
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Quebec
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Beijing
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Changsha
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Chengdu
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Bad Reichenhall
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Bonn
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Ashkelon
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Israel
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Beer Sheva
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Pisa
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Italy
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Roma
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Italy
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Rome
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Italy
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San Giovanni Rotondo
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Verona
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Muang
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Oxford
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Sheffield
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United Kingdom
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Stanmore

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alexion Pharmaceuticals, Inc. (Sponsor)
Address 0 0
Country 0 0
Phone 0 0
1-855-752-2356
Fax 0 0
Email 0 0
clinicaltrials@alexion.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06079281