Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta

• Status: Completed
• Trial end date: 2012-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals; Ultragenyx Pharmaceutical Inc

Criteria

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