CompletedPhase 2

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

United States, Canada, Denmark112 participantsStarted 2017-09-11

Plain-language summary

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.

Who can participate

Age range

18 Years – 75 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12

Timeframe: Baseline, Month 12 (end of treatment [EOT])

Change From Baseline in Radial Bone Strength (Failure Load) at Month 12

Timeframe: Baseline, Month 12 (EOT)

Change From Baseline in Radial Bone Strength (Stiffness) at Month 12

Timeframe: Baseline, Month 12 (EOT)

Trial details

NCT IDNCT03118570

SponsorUltragenyx Pharmaceutical Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2019-10-01

Results submitted2021-12-13

View on ClinicalTrials.gov More Osteogenesis Imperfecta, Type I trials

Who can participate

Age range

18 Years – 75 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12

Timeframe: Baseline, Month 12 (end of treatment [EOT])

Change From Baseline in Radial Bone Strength (Failure Load) at Month 12

Timeframe: Baseline, Month 12 (EOT)

Change From Baseline in Radial Bone Strength (Stiffness) at Month 12

Timeframe: Baseline, Month 12 (EOT)