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Translational Therapy in Patients With Osteogenesis Imperfecta - A Pilot Trial on Treatment With the Rankl-Antibody Denosumab (OI-AK)

This study has been completed.
Information provided by (Responsible Party):
Dr. med. Joerg Oliver Semler, University of Cologne Identifier:
First received: February 14, 2013
Last updated: January 26, 2015
Last verified: January 2015

Pilot study to assess the efficacy of a therapy with the RANKL-antibody denosumab in children 5-10 years of age with mutation in COL1A1 or COL1A2 leading to Osteogenesis imperfecta. Efficacy will be assessed by DXA measurements at the lumbar spine of the areal bone mineral density (BMD) which is the most frequently used parameter in trials investigating osteoporosis.

The hypothesis of the study is:

Osteoclastic activity which is increased in OI could be reduced by inhibition of osteoclast maturation. Denosumab inhibits maturation of the osteoclasts by inhibiting RANKL. BMD could be increased during a 36 week treatment course with denosumab measured after 48 weeks.

Condition Intervention Phase
Osteogenesis Imperfecta Drug: Denosumab Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Dr. med. Joerg Oliver Semler, University of Cologne:

Primary Outcome Measures:
  • Changes of bone mineral density (BMD [g/cm2]) in lumbar spine after 36 weeks of treatment with denosumab. Changes will be calculated between baseline and study week 48. [ Time Frame: 48 weeks ]

Secondary Outcome Measures:
  • Decrease of osteoclastic activity measured by urinary deoxypyridinoline (DPD). [ Time Frame: 14 days (DPD) ]
  • Parathormone in study week 12, 24, 36 and 48 compared to baseline. [ Time Frame: 12 weeks ]
    Descriptive statistical analysis

  • N-Telopeptides in study week 12, 24, 36 and 48 compared to baseline. [ Time Frame: 12 weeks ]
    descriptive statistical analysis

  • Osteocalcin in study week 12, 24, 36 and 48. [ Time Frame: 12 weeks ]
    descriptive statistical analysis

Enrollment: 10
Study Start Date: February 2013
Study Completion Date: January 2015
Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Denosumab subcutaneously Drug: Denosumab
Denosumab will be given subcutaneously in a dosage of 1mg/kg body weight every 12 weeks. 4 interventions are planned until trial week 36. There is no control group planned.


Ages Eligible for Study:   5 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria

  • Male or female subjects between 5 years and 10 years of age with molecular proven Osteogenesis imperfecta (COL1A1/A2 mutation)
  • Subjects must have been treated for a minimum of 2 years with bisphosphonates prior to study entry

Exclusion Criteria:

  • Hypocalcemia (<1.03 mmol/l ionized Calcium)
  • Subjects with reduced renal function (estimated GFR (Schwartz formula) <30ml/min/1.73m2)
  • Any other abnormal finding such as physical examination or laboratory evaluation, in the opinion of the investigator that is indicative of a disease that would compromise the safety of the patient when getting denosumab s.c.
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Please refer to this study by its identifier: NCT01799798

University Cologne, Childrens Hospital, Cologne, Germany
Cologne, NRW, Germany, 50924
Sponsors and Collaborators
University of Cologne
Principal Investigator: Joerg Oliver Semler, MD University Cologne, Childrens Hospital, Cologne, Germany
  More Information

Responsible Party: Dr. med. Joerg Oliver Semler, Head of the outpatient center for sceletal dysplasias, University of Cologne Identifier: NCT01799798     History of Changes
Other Study ID Numbers: Uni-Koeln-1574
Study First Received: February 14, 2013
Last Updated: January 26, 2015

Keywords provided by Dr. med. Joerg Oliver Semler, University of Cologne:
Osteogenesis imperfecta
Areal bone mineral density

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
Immunologic Factors
Physiological Effects of Drugs
Bone Density Conservation Agents processed this record on September 21, 2017