Completed

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

What is being tested

Denosumab

Drug

Who is being recruted

Bone Diseases+7

+ Bone Diseases, Developmental

+ Collagen Diseases

From 5 to 11 Years

+5 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 2

Interventional

Study Start: February 2013

See protocol details

Summary

Principal SponsorUniversity of Cologne

Last updated: January 27, 2015

Sourced from a government-validated database.Claim as a partner

Study start date: February 1, 2013

Actual date on which the first participant was enrolled.

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.

NCT01799798

Principal SponsorUniversity of Cologne

Last updated: January 27, 2015

Sourced from a government-validated database.Claim as a partner

Protocol

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

Design Details

10 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 5 to 11 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Bone DiseasesBone Diseases, DevelopmentalCollagen DiseasesConnective Tissue DiseasesMusculoskeletal DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesOsteochondrodysplasiasSkin and Connective Tissue DiseasesGenetic Diseases, InbornOsteogenesis Imperfecta

Criteria

2 inclusion criteria required to participate

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

3 exclusion criteria prevent from participating

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

Hypocalcemia ( < 1.03 mmol/l ionized Calcium)

Subjects with reduced renal function (estimated GFR (Schwartz formula) < 30ml/min/1.73m2)

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

University Cologne, Childrens Hospital, Cologne, Germany

Cologne, GermanyOpen University Cologne, Childrens Hospital, Cologne, Germany in Google Maps

CompletedOne Study Center