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CompletedNCT01799798OI-AKUpdated Jan 27, 2015

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

A Phase 2 interventional study of Denosumab in Osteogenesis Imperfecta, sponsored by University of Cologne. Completed at 1 site in Germany. Open to participants aged 5 Years to 11 Years. Per ClinicalTrials.gov, last updated 2015-01-27.

Sponsored by University of Cologne · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Ages
5 Years to 11 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Osteogenesis Imperfecta

Keywords

  • Osteogenesis imperfecta
  • COL1A1/A2
  • Denosumab
  • Bisphosphonates
  • Children
  • Areal bone mineral density
03

In context

Osteogenesis Imperfecta

85 studies on the registry are indexed under Osteogenesis Imperfecta; 20 are open to participants now.

This study's enrollment of 10 is below the median of 26 across 59 interventional studies indexed under Osteogenesis Imperfecta.

Browse Osteogenesis Imperfecta studies →

Lead sponsor

University of Cologne is the lead sponsor of 199 studies on the registry; 26 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
5 Years to 11 Years
Sexes eligible
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

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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
10 participants (actual)

Study arms

  • Experimental
    Denosumab subcutaneously

    Drug: Denosumab

Interventions

  • DrugDenosumab

    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.

06

What researchers measure

Primary outcomes

  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 outcomes

  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.

    Descriptive statistical analysis

    Time frame: 12 weeks

  3. N-Telopeptides in study week 12, 24, 36 and 48 compared to baseline.

    descriptive statistical analysis

    Time frame: 12 weeks

  4. Osteocalcin in study week 12, 24, 36 and 48.

    descriptive statistical analysis

    Time frame: 12 weeks

07

Study locations

1 site
  • University Cologne, Childrens Hospital, Cologne, Germany
    Cologne, NRW 50924, Germany
08

References and documents

Publications

  • Semler O, Netzer C, Hoyer-Kuhn H, Becker J, Eysel P, Schoenau E. First use of the RANKL antibody denosumab in osteogenesis imperfecta type VI. J Musculoskelet Neuronal Interact. 2012 Sep;12(3):183-8. PubMed 22947550 ↗
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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 27, 2015, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT01799798
Lead sponsor
University of Cologne
Responsible party
Dr. med. Joerg Oliver Semler (Head of the outpatient center for sceletal dysplasias, University of Cologne) — Principal investigator
First posted
Feb 27, 2013
Start date
Feb 2013
Primary completion
Jan 2015
Completion
Jan 2015
Last update
Jan 27, 2015

Study contacts

Joerg Oliver Semler, MD
principal investigator · University Cologne, Childrens Hospital, Cologne, Germany

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jan 2015. You cannot join it, but the record below documents what was studied.

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