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

Ultragenyx

Status and phase: Completed; Phase 2

Conditions: Osteogenesis Imperfecta, Type I; Osteogenesis Imperfecta Type IV; Osteogenesis Imperfecta Type III

Treatments: Drug: setrusumab; Dietary Supplement: Vitamin D; Dietary Supplement: Calcium; Drug: zoledronic acid (optional)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03118570

MBPS205

2016-005096-27 (EudraCT Number)

Details and patient eligibility

About

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.

Full description

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

Enrollment: 112 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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112 participants in 5 patient groups, including a placebo group

Setrusumab 20 mg/kg (Blinded)

Experimental group

Description:

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.

Treatment:

Dietary Supplement: Calcium

Drug: zoledronic acid (optional)

Drug: setrusumab

Dietary Supplement: Vitamin D

Setrusumab 8 mg/kg (Blinded)

Experimental group

Description:

Setrusumab 8 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Treatment:

Dietary Supplement: Calcium

Drug: zoledronic acid (optional)

Drug: setrusumab

Dietary Supplement: Vitamin D

Setrusumab 2 mg/kg (Blinded)

Experimental group

Description:

Setrusumab 2 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Treatment:

Dietary Supplement: Calcium

Drug: zoledronic acid (optional)

Drug: setrusumab

Dietary Supplement: Vitamin D

Setrusumab 20 mg/kg (Open-Label)

Experimental group

Description:

Setrusumab 20 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Treatment:

Dietary Supplement: Calcium

Drug: zoledronic acid (optional)

Drug: setrusumab

Dietary Supplement: Vitamin D

Placebo

Placebo Comparator group

Description:

Placebo IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Treatment:

Dietary Supplement: Calcium

Dietary Supplement: Vitamin D