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

January 26, 2015 updated by: Dr. med. Joerg Oliver Semler, University of Cologne

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
    • NRW
      • Cologne, NRW, Germany, 50924
        • University Cologne, Childrens Hospital, Cologne, Germany

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 11 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
48 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Decrease of osteoclastic activity measured by urinary deoxypyridinoline (DPD).
Time Frame: 14 days (DPD)
14 days (DPD)
Parathormone in study week 12, 24, 36 and 48 compared to baseline.
Time Frame: 12 weeks
Descriptive statistical analysis
12 weeks
N-Telopeptides in study week 12, 24, 36 and 48 compared to baseline.
Time Frame: 12 weeks
descriptive statistical analysis
12 weeks
Osteocalcin in study week 12, 24, 36 and 48.
Time Frame: 12 weeks
descriptive statistical analysis
12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Cologne

Investigators

  • Principal Investigator: Joerg Oliver Semler, MD, University Cologne, Childrens Hospital, Cologne, Germany

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2013

Primary Completion (Actual)

January 1, 2015

Study Completion (Actual)

January 1, 2015

Study Registration Dates

First Submitted

February 14, 2013

First Submitted That Met QC Criteria

February 24, 2013

First Posted (Estimate)

February 27, 2013

Study Record Updates

Last Update Posted (Estimate)

January 27, 2015

Last Update Submitted That Met QC Criteria

January 26, 2015

Last Verified

January 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Uni-Koeln-1574

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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