Trial record 13 of 20 for:
"Osteogenesis imperfecta"
Translational Therapy in Patients With Osteogenesis Imperfecta - A Pilot Trial on Treatment With the Rankl-Antibody Denosumab (OI-AK)
This study is currently recruiting participants.
Verified February 2013 by University of Cologne
Sponsor:
University of Cologne
Information provided by (Responsible Party):
Dr. med. Joerg Oliver Semler, University of Cologne
ClinicalTrials.gov Identifier:
NCT01799798
First received: February 14, 2013
Last updated: February 24, 2013
Last verified: February 2013
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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: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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 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 ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Decrease of osteoclastic activity measured by urinary deoxypyridinoline (DPD). [ Time Frame: 14 days (DPD) ] [ Designated as safety issue: Yes ]
- Parathormone in study week 12, 24, 36 and 48 compared to baseline. [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]Descriptive statistical analysis
- N-Telopeptides in study week 12, 24, 36 and 48 compared to baseline. [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]descriptive statistical analysis
- Osteocalcin in study week 12, 24, 36 and 48. [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]descriptive statistical analysis
| Estimated Enrollment: | 10 |
| Study Start Date: | February 2013 |
| Estimated Study Completion Date: | June 2015 |
| Estimated Primary Completion Date: | March 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| Ages Eligible for Study: | 5 Years to 11 Years |
| Genders Eligible for Study: | Both |
| 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01799798
Contacts
| Contact: Heike Katharina Hoyer-Kuhn, MD | +49-221-478-4360 | heike-katharina.hoyer-kuhn@uk-koeln.de |
| Contact: Joerg Oliver Semler, MD | +49-221-478-4360 | joerg.semler@uk-koeln.de |
Locations
| Germany | |
| University Cologne, Childrens Hospital, Cologne, Germany | Recruiting |
| Cologne, NRW, Germany, 50924 | |
| Contact: Heike Katharina Hoyer-Kuhn, MD +49-221-478-4360 heike-katharina.hoyer-kuhn@uk-koeln.de | |
| Contact: Barbara Hero, MD +49-221-478-6831 barbara.hero@uk-koeln.de | |
| Sub-Investigator: Heike Katharina Hoyer-Kuhn, MD | |
| Sub-Investigator: Eckhard Schönau, MD | |
Sponsors and Collaborators
University of Cologne
Investigators
| Principal Investigator: | Joerg Oliver Semler, MD | University Cologne, Childrens Hospital, Cologne, Germany |
More Information
Additional 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 |
| Study First Received: | February 14, 2013 |
| Last Updated: | February 24, 2013 |
| Health Authority: | Germany: Paul-Ehrlich-Institut |
Keywords provided by 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 |
ClinicalTrials.gov processed this record on May 23, 2013