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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01799798
Recruitment Status : Completed
First Posted : February 27, 2013
Last Update Posted : January 27, 2015
Information provided by (Responsible Party):
Dr. med. Joerg Oliver Semler, University of Cologne

Brief Summary:

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 or disease Intervention/treatment Phase
Osteogenesis Imperfecta Drug: Denosumab Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : February 2013
Actual Primary Completion Date : January 2015
Actual Study Completion Date : January 2015

Arm Intervention/treatment
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.

Primary Outcome Measures :
  1. 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 :
  1. Decrease of osteoclastic activity measured by urinary deoxypyridinoline (DPD). [ Time Frame: 14 days (DPD) ]
  2. Parathormone in study week 12, 24, 36 and 48 compared to baseline. [ Time Frame: 12 weeks ]
    Descriptive statistical analysis

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

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

Information from the National Library of Medicine

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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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01799798

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University Cologne, Childrens Hospital, Cologne, Germany
Cologne, NRW, Germany, 50924
Sponsors and Collaborators
University of Cologne
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Principal Investigator: Joerg Oliver Semler, MD University Cologne, Childrens Hospital, Cologne, Germany
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Responsible Party: Dr. med. Joerg Oliver Semler, Head of the outpatient center for sceletal dysplasias, University of Cologne Identifier: NCT01799798    
Other Study ID Numbers: Uni-Koeln-1574
First Posted: February 27, 2013    Key Record Dates
Last Update Posted: January 27, 2015
Last Verified: January 2015
Keywords provided by Dr. med. Joerg Oliver Semler, University of Cologne:
Osteogenesis imperfecta
Areal bone mineral density
Additional relevant MeSH terms:
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Osteogenesis Imperfecta
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
Bone Density Conservation Agents
Physiological Effects of Drugs