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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06079359




Registration number
NCT06079359
Ethics application status
Date submitted
6/10/2023
Date registered
12/10/2023

Titles & IDs
Public title
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
Scientific title
A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 Versus Placebo Administered Subcutaneously in Pediatric (2 to < 12 Years of Age) Participants With Hypophosphatasia Who Have Not Received Previous Treatment With Asfotase Alfa
Secondary ID [1] 0 0
ALXN1850-HPP-305
Secondary ID [2] 0 0
D8590C00003
Universal Trial Number (UTN)
Trial acronym
MULBERRY
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

Experimental: ALXN1850 - 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 every 2 weeks (q2w) via SC injection, for 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks.

Placebo comparator: Placebo - Starting at Day 1 during the Randomized Evaluation Period, participants will receive placebo q2w for a total of 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks.


Treatment: Drugs: ALXN1850
Participants will receive a ALXN1850 via subcutaneous (SC) injection.

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

Outcomes
Primary outcome [1] 0 0
Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)
Timepoint [1] 0 0
Day 169
Secondary outcome [1] 0 0
Change from Baseline in Rickets Severity Score (RSS) 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 6-Minute Walk Test (6MWT) 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 Percent Predicted 6MWT 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 Bruininks Oseretsky Test of Motor Proficiency, Second Edition (BOT2) Score at the end of the Randomized Evaluation Period (Day 169)
Timepoint [4] 0 0
Baseline, Day 169
Secondary outcome [5] 0 0
Change from Baseline in Peabody Developmental Motor Scales, Third Edition (PDMS-3) Score at the end of the Randomized Evaluation Period (Day 169)
Timepoint [5] 0 0
Baseline, 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

Eligibility
Key inclusion criteria
* Diagnosis of HPP documented in the medical records, and the following criteria fulfilled 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 the Screening Period as measured by the Central Laboratory OR 2 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 Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval.
* Must meet 1 of the following criteria:

1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown 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
Minimum age
2 Years
Maximum age
11 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

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
14/05/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
21/04/2028
Actual
Sample size
Target
30
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Nedlands
Recruitment hospital [2] 0 0
Research Site - Parkville
Recruitment hospital [3] 0 0
Research Site - Westmead
Recruitment postcode(s) [1] 0 0
6009 - Nedlands
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Maryland
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United States of America
State/province [2] 0 0
Missouri
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United States of America
State/province [3] 0 0
North Carolina
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Argentina
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Buenos Aires
Country [5] 0 0
Argentina
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Ciudad Autónoma de Buenos Aires
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Argentina
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Mar del Plata
Country [7] 0 0
Belgium
State/province [7] 0 0
Brussels
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Brazil
State/province [8] 0 0
Brasilia
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Brazil
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Porto Alegre
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Brazil
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Recife
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Brazil
State/province [11] 0 0
Salvador
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Brazil
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São Paulo
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Canada
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Alberta
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Canada
State/province [14] 0 0
Manitoba
Country [15] 0 0
Canada
State/province [15] 0 0
Ontario
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China
State/province [16] 0 0
Beijing
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China
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Guangzhou
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China
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Shenzhen
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France
State/province [19] 0 0
Le Kremlin Bicêtre
Country [20] 0 0
France
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Paris
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Germany
State/province [21] 0 0
Homburg
Country [22] 0 0
Israel
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Ashkelon
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Israel
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Beer Sheva
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Israel
State/province [24] 0 0
Petach-Tikva
Country [25] 0 0
Israel
State/province [25] 0 0
Ramat Gan
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Italy
State/province [26] 0 0
Bologna
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Italy
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Genova
Country [28] 0 0
Italy
State/province [28] 0 0
Milano
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Italy
State/province [29] 0 0
Roma
Country [30] 0 0
Italy
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Rome
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Korea, Republic of
State/province [31] 0 0
Seoul
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Mexico
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Campeche
Country [33] 0 0
Mexico
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Chihuahua
Country [34] 0 0
Mexico
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Ciudad de Mexico
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Mexico
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Mexico City
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Mexico
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Zapopan
Country [37] 0 0
Poland
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Kraków
Country [38] 0 0
Poland
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Warszawa
Country [39] 0 0
Poland
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Lódz
Country [40] 0 0
Spain
State/province [40] 0 0
Vitoria
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Sweden
State/province [41] 0 0
Stockholm
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Taiwan
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Taipei
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Taiwan
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Taoyuan
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Thailand
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Bangkok
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Thailand
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Chaingmai
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Thailand
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Hat Yai
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Thailand
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Muang
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Thailand
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Ratchathewi
Country [49] 0 0
Thailand
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Songkla
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Turkey
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Ankara
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Turkey
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Bursa
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Turkey
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Edirne
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Turkey
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Erzurum
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Turkey
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Istambul
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United Kingdom
State/province [55] 0 0
Birmingham
Country [56] 0 0
United Kingdom
State/province [56] 0 0
Manchester
Country [57] 0 0
Vietnam
State/province [57] 0 0
Hanoi

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
[email protected]
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/NCT06079359