Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta

Ultragenyx

Status and phase: Completed; Phase 2

Conditions: Osteogenesis Imperfecta

Treatments: Drug: BPS804

Study type: Interventional

Funder types: Industry

Identifiers

NCT01417091

CBPS804A2201

2011-001465-41 (EudraCT Number)

Details and patient eligibility

About

This is a randomized, open label intra-patient dose escalation study to evaluate safety and tolerability, pharmacokinetics, and pharmacodynamics of BPS804 in adults with osteogenesis imperfecta (OI).

Pharmacodynamic effect will be determined by serological biomarkers and radiologic assessments. In addition, tolerability and pharmacokinetics (PK) will be evaluated.

Full description

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

Enrollment: 10 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Osteogenesis imperfecta
• Two or more previous fractures
• Bone mineral density Z-score of ≤ -1.0 and > -4.0

Exclusion criteria

• Open epiphyses
• Fracture within last 2 weeks
• Treatment with bisphosphonates/teriparatide (last 6 months)
• Surgery within last year

Other protocol-defined inclusion/exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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10 participants in 2 patient groups

Treatment group

Experimental group

Treatment:

Drug: BPS804

Untreated reference group

No Intervention group