A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804

NCT ID: NCT03118570

Last Updated: 2023-07-05

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

112 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-09-11

Study Completion Date

2020-11-12

Brief Summary

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The purpose of this study is to select a suitable dose of BPS804 by measuring the strength/quality of bone using a special type of CT scanner. Participants will be treated for 12 months and followed up for a further 12 months.

Detailed Description

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This study was previously posted by Mereo Biopharma and was transferred to Ultragenyx in February 2021.

Conditions

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Osteogenesis Imperfecta, Type I Osteogenesis Imperfecta Type III Osteogenesis Imperfecta Type IV

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Double-blind, Dose-finding Study, incorporating an open-label substudy
Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators
Sponsor will be masked until the primary analysis of the study except for open-label substudy treatment arm. The study site pharmacist will be unmasked to treatment allocation throughout. Study treatment will be monitored by a separate unmasked monitoring team.

Study Groups

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Setrusumab 20 mg/kg (Blinded)

Setrusumab 20 mg/kg intravenous (IV) infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Group Type EXPERIMENTAL

setrusumab

Intervention Type DRUG

Intravenous infusion

Calcium

Intervention Type DIETARY_SUPPLEMENT

tablets

Vitamin D

Intervention Type DIETARY_SUPPLEMENT

capsules

zoledronic acid (optional)

Intervention Type DRUG

Following completion of the study treatment (Month 12) participants can receive an optional single dose of zoledronic acid. Participants can receive an optional further dose of zoledronic acid at Month 18 at the discretion of their treating physician.

Setrusumab 8 mg/kg (Blinded)

Setrusumab 8 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Group Type EXPERIMENTAL

setrusumab

Intervention Type DRUG

Intravenous infusion

Calcium

Intervention Type DIETARY_SUPPLEMENT

tablets

Vitamin D

Intervention Type DIETARY_SUPPLEMENT

capsules

zoledronic acid (optional)

Intervention Type DRUG

Following completion of the study treatment (Month 12) participants can receive an optional single dose of zoledronic acid. Participants can receive an optional further dose of zoledronic acid at Month 18 at the discretion of their treating physician.

Setrusumab 2 mg/kg (Blinded)

Setrusumab 2 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Group Type EXPERIMENTAL

setrusumab

Intervention Type DRUG

Intravenous infusion

Calcium

Intervention Type DIETARY_SUPPLEMENT

tablets

Vitamin D

Intervention Type DIETARY_SUPPLEMENT

capsules

zoledronic acid (optional)

Intervention Type DRUG

Following completion of the study treatment (Month 12) participants can receive an optional single dose of zoledronic acid. Participants can receive an optional further dose of zoledronic acid at Month 18 at the discretion of their treating physician.

Setrusumab 20 mg/kg (Open-Label)

Setrusumab 20 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Group Type EXPERIMENTAL

setrusumab

Intervention Type DRUG

Intravenous infusion

Calcium

Intervention Type DIETARY_SUPPLEMENT

tablets

Vitamin D

Intervention Type DIETARY_SUPPLEMENT

capsules

zoledronic acid (optional)

Intervention Type DRUG

Following completion of the study treatment (Month 12) participants can receive an optional single dose of zoledronic acid. Participants can receive an optional further dose of zoledronic acid at Month 18 at the discretion of their treating physician.

Placebo

Placebo IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Group Type PLACEBO_COMPARATOR

Calcium

Intervention Type DIETARY_SUPPLEMENT

tablets

Vitamin D

Intervention Type DIETARY_SUPPLEMENT

capsules

Interventions

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setrusumab

Intravenous infusion

Intervention Type DRUG

Calcium

tablets

Intervention Type DIETARY_SUPPLEMENT

Vitamin D

capsules

Intervention Type DIETARY_SUPPLEMENT

zoledronic acid (optional)

Following completion of the study treatment (Month 12) participants can receive an optional single dose of zoledronic acid. Participants can receive an optional further dose of zoledronic acid at Month 18 at the discretion of their treating physician.

Intervention Type DRUG

Other Intervention Names

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BPS804

Eligibility Criteria

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Inclusion Criteria

* Patients with a clinical diagnosis of OI Type I, III or IV with a confirmed defect in the COL1A1/COL1A2 genes, as confirmed by genetic testing
* One or more fractures in the past 5 years
* Capable of giving signed consent

Exclusion Criteria

* History of skeletal malignancies or other bone diseases (other than OI)
* History of neural foraminal stenosis (except if due to scoliosis)
* History of myocardial infarction, angina pectoris, ischaemic stroke or transient ischaemic attack
* History of endocrine or thyroid/parathyroid conditions that could affect bone metabolism
* Treatment with bisphosphonates within 3 months of randomisation
* Treatment with teraparatide, denosumab or other anabolic/anti-reabsorptive medications within 6 months of randomisation
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Mereo BioPharma

INDUSTRY

Sponsor Role collaborator

Ultragenyx Pharmaceutical Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Mereo BioPharma

Locations

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Mereo Investigator Site

Birmingham, Alabama, United States

Site Status

Mereo Investigator Site

Jacksonville, Florida, United States

Site Status

Mereo Investigator Site

Baltimore, Maryland, United States

Site Status

Mereo Investigator Site

Boston, Massachusetts, United States

Site Status

Mereo Investigator Site

Saint Paul, Minnesota, United States

Site Status

Mereo Investigator Site

St Louis, Missouri, United States

Site Status

Mereo Investigator Site

Albuquerque, New Mexico, United States

Site Status

Mereo Investigator Site

Cincinnati, Ohio, United States

Site Status

Mereo Investigator Site

Portland, Oregon, United States

Site Status

Mereo Investigator Site

Pittsburgh, Pennsylvania, United States

Site Status

Mereo Investigator Site

Nashville, Tennessee, United States

Site Status

Mereo Investigator Site

Houston, Texas, United States

Site Status

Mereo Investigator Site

Toronto, Ontario, Canada

Site Status

Mereo Investigator Site

Montreal, Quebec, Canada

Site Status

Mereo Investigator Site

Québec, Quebec, Canada

Site Status

Mereo Investigator Site

Aarhus, , Denmark

Site Status

Mereo Investigator Site

Odense, , Denmark

Site Status

Mereo Investigator Site

Paris, Paris Cedex 14, France

Site Status

Mereo Investigator Site

Lyon, , France

Site Status

Mereo Investigator Site

Paris, , France

Site Status

Mereo Investigator Site

Cambridge, Cambridgeshire, United Kingdom

Site Status

Mereo Investigator Site

Newcastle upon Tyne, Newcastle, United Kingdom

Site Status

Mereo Investigator Site

Oxford, Oxfordshire, United Kingdom

Site Status

Mereo Investigator Site

Bristol, , United Kingdom

Site Status

Mereo Investigator Site

London, , United Kingdom

Site Status

Countries

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United States Canada Denmark France United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2016-005096-27

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MBPS205

Identifier Type: -

Identifier Source: org_study_id

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