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NCT Number: NCT01799798

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.

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Key information

Conditions

Osteogenesis Imperfecta Bone Diseases Bone Diseases, Developmental Collagen Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias Skin and Connective Tissue Diseases

Age range

5 year–11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University Cologne, Childrens Hospital, Cologne, Germany

Cologne, North Rhine-Westphalia, 50924, Germany

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Denosumab

Drug

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.

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.

    Time frame: 12 weeks

    Descriptive statistical analysis

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

    Time frame: 12 weeks

    descriptive statistical analysis

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

    Time frame: 12 weeks

    descriptive statistical analysis

Sponsors and collaborators

Lead sponsor

University of Cologne

Other

Registry information

Acronym: OI-AK

Important dates

Study start
2013
Primary completion
2015
Study completion
2015
First posted
Feb 27, 2013
Registry last updated
Jan 27, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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