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Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01417091
First received: August 4, 2011
Last updated: March 18, 2013
Last verified: March 2013
History of Changes
  Purpose

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.

Twelve to 15 patients will be enrolled.


Condition Intervention Phase
Osteogenesis Imperfecta
 Drug: BPS804
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Randomized, Open Label Intra-patient Dose Escalation Study With an Untreated Reference Group to Evaluate Safety and Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Infusions of BPS804 in Adults With Moderate Osteogenesis Imperfecta

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Safety and tolerability (composite outcome: physical examination, vital signs, standard laboratory, (serious) adverse events, ECG) [ Time Frame: Days 1, 2, 8, 15, 16, 29, 30, 36, 43, 57, 85, 113 and 141 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Determination of pharmacodynamic effect by means of biomarkers and radiologic measurement [ Time Frame: 21 weeks ] [ Designated as safety issue: No ]
  • Determination of pharmacokinetics (Composite of Pharmacokinetics). [ Time Frame: Day 1 (prior to administration and 3x after administration) and Days 2, 8, 15 (2x), 16, 29 (4x), 30, 36, 43, 57, 85, 113,141 ] [ Designated as safety issue: No ]

Enrollment: 11
Study Start Date: June 2011
Study Completion Date: December 2012
Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment group Drug: BPS804
No Intervention: Untreated reference group

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01417091

Locations
United States, California
Novartis Investigative Site
Anaheim, California, United States, 92801
United States, Florida
Novartis Investigative Site
Miramar, Florida, United States, 33025
Belgium
Novartis Investigative Site
Bruxelles, Belgium, 1200
Novartis Investigative Site
Gent, Belgium, 9000
Canada, Quebec
Novartis Investigative Site
Montreal, Quebec, Canada, H3GIA6
Germany
Novartis Investigative Site
Wuerzburg, Germany, 97074
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Publications:

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01417091     History of Changes
Other Study ID Numbers: CBPS804A2201, 2011-001465-41
Study First Received: August 4, 2011
Last Updated: March 18, 2013
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Belgium HA: Federal Agency for Medicines and Health Products
Germany: Paul-Ehrlich-Institut

Keywords provided by Novartis:
Brittle bone disease
inherited
connective tissue disorder
fracture

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
 Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases

ClinicalTrials.gov processed this record on May 21, 2013