Osteogenesis Imperfecta
Conditions
Brief summary
The primary objective of the study is to evaluate bone mineral density (BMD) after 12 months of retreatment with monthly setrusumab in adults with osteogenesis imperfecta (OI).
Detailed description
UX143-CL203 is a long-term extension study in adults with OI who participated in the ASTEROID study (also referred to as MBPS205) \[NCT03118570\]. The UX143-CL203 study comprises Observation, Retreatment, and Extension Periods. There is no intervention during the Observation Period. During the single-arm Retreatment Period, participants receive open-label setrusumab once a month (QM) for 12 months. Following the Retreatment Period, participants enter an Extension Period in which they will continue treatment with open-label setrusumab at different dosing frequencies.
Interventions
BIOLOGICALSetrusumab
A fully human anti-sclerostin monoclonal antibody (mAb)
Sponsors
Mereo BioPharma
Ultragenyx Pharmaceutical Inc
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
19 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Males or females who participated in the Phase 2b ASTEROID study * Females of childbearing potential must consent to use highly effective contraception during the Observation, Retreatment, and Extension Periods through 2 months after the last dose of setrusumab and agree not to become pregnant
Exclusion criteria
* Known hypersensitivity to setrusumab or its excipients that, in the judgment of the Investigator, places the participant at increased risk for adverse effects * Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results * Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study * Not willing or able to discontinue bisphosphonates 6 months prior to the Retreatment Screening Visit Once enrolled in UX143-CL203, individuals who meet any of the following
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percentage Change From Retreatment Baseline in Lumbar Spine Bone Mineral Density (BMD) Measured by Dual-Energy X-Ray Absorptiometry (DXA) After 12 Months of Setrusumab | Retreatment Baseline, Month 12 |
Secondary
| Measure | Time frame |
|---|---|
| Percentage Change From Retreatment Baseline in Total Hip BMD Measured by DXA at Month 12 of the Retreatment Period | Retreatment Baseline, Month 12 |
| Annualized New Fracture Rate as Confirmed by Radiograph During the Retreatment Period | Up to Month 12 |
Countries
United States
Participant flow
Recruitment details
This study recruited adults with osteogenesis imperfecta (OI) who previously participated in the phase 2b, double-blind, dose-finding MBPS205 (ASTEROID) study (NCT03118570).
Pre-assignment details
A total of 2 participants were screened and enrolled. Both participants completed the Baseline visit as part of the Observation Period, and then were discontinued from the study prior to entering the retreatment period as the trial was terminated by the Sponsor.
Participants by arm
| Arm | Count |
|---|---|
| Observation Period --> Setrusumab QM --> Setrusumab Q2M/Setrusumab Q2M Following an Observation Period, participants enter a Retreatment Period and receive setrusumab administered via intravenous (IV) infusion once a month (QM) for 12 months. During the planned Extension Period, participants are randomized 1:1 to either continue treatment with open-label setrusumab QM or transition to open-label setrusumab every 2 months (Q2M) for at least 12 months or until setrusumab becomes commercially available. | 2 |
| Total | 2 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Sponsor Terminated Trial | 2 |
Baseline characteristics
| Characteristic | Observation Period --> Setrusumab QM --> Setrusumab Q2M/Setrusumab Q2M |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 2 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race/Ethnicity, Customized White | 2 Participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 2 |
| other Total, other adverse events | 0 / 2 |
| serious Total, serious adverse events | 0 / 2 |
Outcome results
Primary
Percentage Change From Retreatment Baseline in Lumbar Spine Bone Mineral Density (BMD) Measured by Dual-Energy X-Ray Absorptiometry (DXA) After 12 Months of Setrusumab
Time frame: Retreatment Baseline, Month 12
Population: No statistical analyses were performed. No primary analysis occurred, as it was planned to occur at the end of the Retreatment Period, reflecting 12 months of retreatment with setrusumab.
Secondary
Annualized New Fracture Rate as Confirmed by Radiograph During the Retreatment Period
Time frame: Up to Month 12
Population: No statistical analyses were performed. No primary analysis occurred, as it was planned to occur at the end of the Retreatment Period, reflecting 12 months of retreatment with setrusumab.
Secondary
Percentage Change From Retreatment Baseline in Total Hip BMD Measured by DXA at Month 12 of the Retreatment Period
Time frame: Retreatment Baseline, Month 12
Population: No statistical analyses were performed. No primary analysis occurred, as it was planned to occur at the end of the Retreatment Period, reflecting 12 months of retreatment with setrusumab.