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

February 26, 2024 updated by: Alexion Pharmaceuticals, Inc.

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

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

30

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Argentina
      • Buenos Aires, Argentina, C1425BPE
        • Research Site
      • Ciudad Autónoma de Buenos Aires, Argentina, C1199
        • Research Site
      • Mar del Plata, Argentina, B7600FYK
        • Research Site
      • Mar del Plata, Argentina, B7600
        • Research Site
  • Australia
      • Nedlands, Australia, 6009
        • Research Site
      • Parkville, Australia, 3052
        • Research Site
      • Westmead, Australia, 2145
        • Research Site
  • Belgium
      • Brussels, Belgium, 1020
        • Research Site
  • Brazil
      • Brasilia, Brazil, 71625-009
        • Research Site
      • Porto Alegre, Brazil, 90610-261
        • Research Site
      • Recife, Brazil, 50740-465
        • Research Site
      • São Paulo, Brazil, 01409-902
        • Research Site
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2E 7Z4
        • Research Site
      • Edmonton, Alberta, Canada, T6G 2H7
        • Research Site
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L1
        • Research Site
  • China
      • Beijing, China, 100045
        • Research Site
      • Guangzhou, China, 510623
        • Research Site
      • Shenzhen, China, 518053
        • Research Site
  • France
      • Le Kremlin Bicêtre, France, 94270
        • Research Site
      • Paris, France, 75015
        • Research Site
  • Germany
      • Homburg, Germany, 66421
        • Research Site
  • Israel
      • Ashkelon, Israel, 7830604
        • Research Site
      • Beer Sheva, Israel, 8410101
        • Research Site
      • Petach-Tikva, Israel, 4920235
        • Research Site
      • Ramat Gan, Israel, 52621
        • Research Site
  • Italy
      • Bologna, Italy, 40138
        • Research Site
      • Genova, Italy, 16147
        • Research Site
      • Milano, Italy, 20133
        • Research Site
      • Roma, Italy, 00190
        • Research Site
      • Rome, Italy, 00050
        • Research Site
  • Korea, Republic of
      • Seoul, Korea, Republic of, 05505
        • Research Site
  • Mexico
      • Campeche, Mexico, 24050
        • Research Site
      • Chihuahua, Mexico, 31238
        • Research Site
      • Ciudad de Mexico, Mexico, 14389
        • Research Site
      • Zapopan, Mexico, 45040
        • Research Site
  • Poland
      • Kraków, Poland, 31-503
        • Research Site
      • Warszawa, Poland, 01-211
        • Research Site
      • Łódź, Poland, 93-338
        • Research Site
  • Spain
      • Vitoria, Spain, 01009
        • Research Site
  • Taiwan
      • Taipei, Taiwan, 100
        • Research Site
      • Taoyuan, Taiwan, 333
        • Research Site
  • Thailand
      • Bangkok, Thailand, 10400
        • Research Site
      • Muang, Thailand, 50200
        • Research Site
      • Songkla, Thailand, 90110
        • Research Site
  • Turkey
      • Ankara, Turkey, 06100
        • Research Site
      • Ankara, Turkey, 06560
        • Research Site
      • Bursa, Turkey, 16059
        • Research Site
      • Edirne, Turkey, 22030
        • Research Site
      • Erzurum, Turkey, 25240
        • Research Site
      • Istambul, Turkey, 34899
        • Research Site
  • United Kingdom
      • Birmingham, United Kingdom, B4 6NH
        • Research Site
      • Manchester, United Kingdom
        • Research Site
  • United States
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Research Site
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Research Site
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Research Site
  • Vietnam
      • Hanoi, Vietnam, 100000
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosis of HPP documented in the medical records, and the following criteria fulfilled during the Screening Period without other probable cause than HPP:

    1. Presence of HPP-related rickets on skeletal X-rays, with a minimum Rickets Severity Score (RSS) of 1.0 AND
    2. Serum ALP activity below the age- and sex-adjusted normal range

  • 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)
  • Tanner stage 2 or less during the Screening Period

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
Drug: ALXN1850
Participants will receive a ALXN1850 via subcutaneous (SC) injection.
Drug: Placebo
Participants will receive placebo via SC injection.
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.
Drug: Placebo
Participants will receive placebo via SC injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)
Time Frame: Day 169
Day 169

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from Baseline in Rickets Severity Score (RSS) at the end of the Randomized Evaluation Period (Day 169)
Time Frame: Baseline, Day 169
Baseline, Day 169
Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169)
Time Frame: Baseline, Day 169
Baseline, Day 169
Change from Baseline in Percent Predicted 6MWT at the end of the Randomized Evaluation Period (Day 169)
Time Frame: Baseline, Day 169
Baseline, Day 169
Change from Baseline in Bruininks Oseretsky Test of Motor Proficiency, Second Edition (BOT2) Score at the end of the Randomized Evaluation Period (Day 169)
Time Frame: Baseline, Day 169
Baseline, Day 169
Change from Baseline in Peabody Developmental Motor Scales, Third Edition (PDMS-3) Score at the end of the Randomized Evaluation Period (Day 169)
Time Frame: Baseline, Day 169
Baseline, Day 169
RGI-C Responder at the end of the Randomized Evaluation Period (Day 169)
Time Frame: Day 169
Day 169

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 1, 2024

Primary Completion (Estimated)

May 30, 2025

Study Completion (Estimated)

July 30, 2027

Study Registration Dates

First Submitted

October 6, 2023

First Submitted That Met QC Criteria

October 6, 2023

First Posted (Actual)

October 12, 2023

Study Record Updates

Last Update Posted (Actual)

February 28, 2024

Last Update Submitted That Met QC Criteria

February 26, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • D8590C00003
  • ALXN1850-HPP-305 (Other Identifier: Alexion Pharmaceuticals, Inc.)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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