Trial Outcomes & Findings for Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta (NCT NCT05312697)

NCT ID: NCT05312697

Last Updated: 2025-07-23

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

Retreatment Baseline, Month 12

Results posted on

2025-07-23

Participant Flow

This study recruited adults with osteogenesis imperfecta (OI) who previously participated in the phase 2b, double-blind, dose-finding MBPS205 (ASTEROID) study (NCT03118570).

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.

Participant milestones

Participant milestones
Measure
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.
Overall Study
STARTED
2
Overall Study
COMPLETED
0
Overall Study
NOT COMPLETED
2

Reasons for withdrawal

Reasons for withdrawal
Measure
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.
Overall Study
Sponsor Terminated Trial
2

Baseline Characteristics

Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Observation Period --> Setrusumab QM --> Setrusumab Q2M/Setrusumab Q2M
n=2 Participants
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.
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
2 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Sex: Female, Male
Female
2 Participants
n=5 Participants
Sex: Female, Male
Male
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
Race/Ethnicity, Customized
White
2 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

Adverse Events

Observation Period --> Setrusumab QM --> Setrusumab Q2M/Setrusumab Q2M

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Medical Information

Ultragenyx Pharmaceutical Inc

Phone: 1-888-756-8567

Results disclosure agreements

  • Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
  • Publication restrictions are in place

Restriction type: OTHER