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

• Register: ClinicalTrials.gov
• Last refreshed on: 10 July 2023
• Main ID: NCT03118570
• Date of registration: 03/04/2017
• Prospective Registration: Yes
• Primary sponsor: Ultragenyx Pharmaceutical Inc
• Public title: A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 Asteroid
• Scientific title: A Phase 2b, Multicentre, Multinational, Double-blind, Dose-finding Study, Incorporating an Open Label Substudy, in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With Setrusumab (BPS804)
• Date of first enrolment: September 11, 2017
• Target sample size: 112
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03118570
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2

Countries of recruitment

Canada, Denmark, France, United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Mereo BioPharma

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Osteogenesis Imperfecta Type III

Osteogenesis Imperfecta Type IV

Osteogenesis Imperfecta, Type I

Intervention(s)

Dietary Supplement: Calcium

Drug: zoledronic acid (optional)

Dietary Supplement: Vitamin D

Drug: setrusumab

Primary Outcome(s)

Change From Baseline in Radial Bone Strength (Failure Load) at Month 12 [Time Frame: Baseline, Month 12 (EOT)]

Change From Baseline in Radial Bone Strength (Stiffness) at Month 12 [Time Frame: Baseline, Month 12 (EOT)]

Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 [Time Frame: Baseline, Month 12 (end of treatment [EOT])]

Secondary Outcome(s)

Change From Baseline in Total vBMD (Radial and Tibial) Over Time [Time Frame: Baseline, Months 6, 12 (EOT), 18, and 24]

Change From Baseline in Index (Utility) Score on EuroQol 5-Dimension 5-Level Descriptive System (EQ-5D-5L) Score at Months 6 and 12 [Time Frame: Baseline, Months 6 and 12 (EOT)]

Change From Baseline in Lumbar, Total Body, and Femoral Neck Bone Mineral Density (BMD) T-score at Month 6 [Time Frame: Baseline, Month 6]

Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) Over Time: Full Analysis Set [Time Frame: Baseline, Months 6, 12 (EOT), 18, 24]

Change From Baseline in Amino-Terminal Propeptide of Type 1 Procollagen (P1NP) up to Month 12 [Time Frame: Baseline, Months 1, 3, 6, 9, 12 (EOT)]

Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) at Months 6 and 12: Open-Label Arm [Time Frame: Baseline, Months 6, 12 (EOT)]

Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 12 [Time Frame: Baseline, Month 12 (EOT)]

Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 6 [Time Frame: Baseline, Month 6]

Changes From Baseline in Radial and Tibial Tr VBMD at Months 6 and 12: Open-Label Arm [Time Frame: Baseline, Months 6, 12 (EOT)]

Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD T-score at Month 12 [Time Frame: Baseline, Month 12 (EOT)]

Percentage of Participants Who Were Positive for Anti-Setrusumab Antibodies at Any Time During the Study up to Month 14 [Time Frame: up to Month 14]

Percentage of Participants With Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation or Death [Time Frame: Non-serious AEs: up to Month 14; Serious AEs: up to Month 24. (Average duration of exposure to placebo was 5 months and for setrusumab was 11 month plus follow-up to 24 months.)]

Percentage of Participants With at Least 1 New Fracture (Peripheral, Vertebral, Long-Bone, Any) at Month 12 [Time Frame: Month 12 (EOT)]

Change From Baseline in Carboxy-Terminal Telo-Peptide [CTX-1] up to Month 12 [Time Frame: Baseline, Months 1, 3, 6, 9, 12 (EOT)]

Change From Baseline in OIQoL-A Pain Subscale Score at Months 6 and 12 [Time Frame: Baseline, Months 6, 12 (EOT)]

Change From Baseline in Osteogenesis Imperfecta Specific Quality of Life Questionnaire for Adults (OIQoL-A) Total Score at Months 6 and 12 [Time Frame: Baseline, Months 6, 12 (EOT)]

Change From Baseline in Cortical vBMD (Radial and Tibial) Over Time [Time Frame: Baseline, Months 6, 12 (EOT), 18, and 24]

Change From Baseline in Radial and Tibial Tr VBMD Over Time: Full Analysis Set [Time Frame: Baseline, Months 6, 12 (EOT), 18, 24]

Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) Over Time: Full Analysis Set [Time Frame: Baseline, Months 6, 12 (EOT), 18, 24]

Number of Participants With Clinically Significant Changes From Baseline in Body Height, Weight and Body Mass Index (BMI) at 6 and 12 Months: Full Analysis Set [Time Frame: Baseline, Month 6, Month 12 (EOT)]

Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) at Months 6 and 12: Open-Label Arm [Time Frame: Baseline, Months 6, 12 (EOT)]

Change From Baseline in Lean and Fat Body Mass From Whole Body at Months 6 and 12 [Time Frame: Baseline, Months 6, 12 (EOT)]

Change From Baseline in OIQoL-A Activity Subscale Score at Months 6 and 12 [Time Frame: Baseline, Months 6, 12 (EOT)]

Change From Baseline in SF-12 Mental Component Summary Score at Months 6 and 12 [Time Frame: Baseline, Months 6, 12 (EOT)]

Change From Baseline in Short Form 12 Health Survey (SF-12) Physical Component Summary Score at Months 6 and 12 [Time Frame: Baseline, Months 6, 12 (EOT)]

Secondary ID(s)

MBPS205

2016-005096-27

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Mereo BioPharma

Results

• Results available: Yes
• Date Posted: 02/03/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03118570