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

Phase 2

Completed

• Conditions: Osteogenesis Imperfecta
• Interventions: Drug: Denosumab

• Registration Number: NCT01799798
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-02
• Study First Submitted: 2013-02-14
• Study First Submitted QC: 2013-02-24
• Study First Posted: 2013-02-27
• Status Verified: 2015-01
• Primary Completion: 2015-01
• Study Completion: 2015-01
• Last Update Submitted: 2015-01-26
• Last Update Posted: 2015-01-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Not provided

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Denosumab subcutaneously	Denosumab	-

Primary Outcome Measures

Name	Time	Method
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.	48 weeks

Secondary Outcome Measures

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

Trial Locations

Locations (1)

University Cologne, Childrens Hospital, Cologne, Germany; 🇩🇪 Cologne, NRW, Germany