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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.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01799798
First Posted: February 27, 2013
Last Update Posted: January 27, 2015
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.
Information provided by (Responsible Party):
Dr. med. Joerg Oliver Semler, University of Cologne
  Purpose

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
Official Title: TRANSLATIONAL THERAPY IN PATIENTS WITH OSTEOGENESIS IMPERFECTA - A PILOT TRIAL ON TREATMENT WITH THE RANKL-ANTIBODY DENOSUMAB

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.

  Eligibility

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

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.
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT01799798


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

Publications:
Responsible Party: Dr. med. Joerg Oliver Semler, Head of the outpatient center for sceletal dysplasias, University of Cologne
ClinicalTrials.gov Identifier: NCT01799798     History of Changes
Other Study ID Numbers: Uni-Koeln-1574
First Submitted: February 14, 2013
First Posted: February 27, 2013
Last Update Posted: January 27, 2015
Last Verified: January 2015

Keywords provided by Dr. med. Joerg Oliver Semler, University of Cologne:
Osteogenesis imperfecta
COL1A1/A2
Denosumab
Bisphosphonates
Children
Areal bone mineral density

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
Antibodies
Denosumab
Immunologic Factors
Physiological Effects of Drugs
Bone Density Conservation Agents