Vanderbilt Clinical Trials

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

Purpose

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.

Conditions

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

Eligibility

Eligible Ages: Between 18 Years and 75 Years
Eligible Genders: All
Accepts Healthy Volunteers: No

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

Study Design

Phase: Phase 2
Study Type: Interventional
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Double-blind, Dose-finding Study, incorporating an open-label substudy
Primary Purpose: Treatment
Masking: Double (Participant, Investigator)
Masking Description: 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.

Arm Groups

Arm	Description	Assigned Intervention
Experimental 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.	Drug: setrusumab Intravenous infusion Other names: BPS804 Dietary Supplement: Calcium tablets Dietary Supplement: Vitamin D capsules Drug: 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.
Experimental 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.	Drug: setrusumab Intravenous infusion Other names: BPS804 Dietary Supplement: Calcium tablets Dietary Supplement: Vitamin D capsules Drug: 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.
Experimental 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.	Drug: setrusumab Intravenous infusion Other names: BPS804 Dietary Supplement: Calcium tablets Dietary Supplement: Vitamin D capsules Drug: 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.
Experimental 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.	Drug: setrusumab Intravenous infusion Other names: BPS804 Dietary Supplement: Calcium tablets Dietary Supplement: Vitamin D capsules Drug: 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.
Placebo Comparator Placebo	Placebo IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.	Dietary Supplement: Calcium tablets Dietary Supplement: Vitamin D capsules

More Details

Status: Completed
Sponsor: Ultragenyx Pharmaceutical Inc

Study Contact

Detailed Description

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.