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Completed

NCT Number: NCT00106028

Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children

Children with Osteogenesis Imperfecta (OI) have bone pain, low bone mass and fractures. There are no approved drugs for the treatment of OI in children, even though some intravenous (IV) bisphosphonates are used off-label in some countries. In a single dose, pharmacokinetic study, data showed that risedronate was well tolerated in 28 children with OI. This three year study will test the safety and efficacy of risedronate in the treatment of children with OI. For the first year, patients will be randomized to the risedronate and placebo groups in a 2:1 ratio. For the second and third years of the study, all patients will receive risedronate.

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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

4 year–15 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

The Children's Hospital at Westmead, Westmead, New South Wales, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • OI diagnosis
  • increased risk of fracture: either has a history of at least 1 radiographically confirmed, non-traumatic or low impact fracture plus low bone mineral density (BMD) or has very low BMD with or without a history of fractures.

Exclusion criteria

  • Any bisphosphonate use within one year of enrollment

Treatment and study plan

risedronate sodium (Actonel)

Drug

risedronate tablet once a day for one year followed by risedronate once a day for two years

Placebo

Drug

placebo tablet once a day for one year followed by risedronate once a day for two years

Primary outcomes

  1. Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 12, ITT Population

    Time frame: Baseline and Month 12

    Lumbar Spine Bone Mineral Density (BMD) measured by dual-energy x-ray absorptiometry (DXA)and read by central reader. Duplicate scans obtained at screening and Month 12.

Secondary outcomes

  1. Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 24, ITT Population

    Time frame: Baseline and Month 24

    Lumbar Spine Bone Mineral Density (BMD) measured by dual-energy x-ray absorptiometry (DXA)and read by central reader.

  2. Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 36, ITT Population

    Time frame: Baseline and Month 36

    Lumbar Spine Bone Mineral Density (BMD) measured by dual-energy x-ray absorptiometry (DXA)and read by central reader.

  3. Percent Change From Baseline in Total Body BMD at Month 12, ITT Population

    Time frame: Baseline and Month 12

    Percent Change from baseline in Total Body Bone Mineral Density (BMD) measured by DXA.

  4. Percent Change From Baseline in Total Body BMD at Month 24, ITT Population

    Time frame: Baseline and Month 24

    Percent Change from baseline in Total Body Bone Mineral Density (BMD) measured by DXA.

  5. Percent Change From Baseline in Total Body BMD at Month 36, ITT Population

    Time frame: Baseline and Month 36

    Percent Change from baseline in Total Body Bone Mineral Density (BMD) measured by DXA.

  6. Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 12, ITT Population

    Time frame: Baseline and Month 12

  7. Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 24, ITT Population

    Time frame: Baseline and Month 24

  8. Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 36, ITT Population

    Time frame: Baseline and Month 36

  9. Percent Change From Baseline in Total Body BMC at Month 12, ITT Population

    Time frame: Baseline and Month 12

  10. Percent Change From Baseline in Total Body BMC at Month 24, ITT Population

    Time frame: Baseline and Month 24

  11. Percent Change From Baseline in Total Body BMC at Month 36, ITT Population

    Time frame: Baseline and Month 36

  12. Lumbar Spine Z-score - Percent Change From Baseline to Month 12, ITT Population

    Time frame: Baseline and Month 12

    Lumbar Spine Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".

  13. Lumbar Spine Z-score - Percent Change From Baseline to Month 24, ITT Population

    Time frame: Baseline and Month 24

    Lumbar Spine Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".

  14. Lumbar Spine Z-score - Percent Change From Baseline to Month 36, ITT Population

    Time frame: Baseline and Month 36

    Lumbar Spine Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".

  15. Total Body Z-score- Percent Change From Baseline to Month 12, ITT Population

    Time frame: Baseline and Month 12

    Total Body Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".

  16. Total Body Z-score- Percent Change From Baseline to Month 24, ITT Population

    Time frame: Baseline and Month 24

    Total Body Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".

  17. Total Body Z-score- Percent Change From Baseline to Month 36, ITT Population

    Time frame: Baseline and Month 36

    Total Body Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".

  18. Percent Change From Baseline in Lumbar Spine Bone Area at Month 12, ITT Population

    Time frame: Baseline and Month 12

    Measured by DXA.

  19. Percent Change From Baseline in Lumbar Spine Bone Area at Month 24, ITT Population

    Time frame: Baseline and Month 24

    Measured by DXA.

  20. Percent Change From Baseline in Lumbar Spine Bone Area at Month 36, ITT Population

    Time frame: Baseline and Month 36

    Measured by DXA.

  21. Percent Change From Baseline in Total Body Bone Area Month 12, ITT Population

    Time frame: Baseline and Month 12

  22. Percent Change From Baseline in Total Body Bone Area Month 24, ITT Population

    Time frame: Baseline and Month 24

  23. Percent Change From Baseline in Total Body Bone Area Month 36, ITT Population

    Time frame: Baseline and Month 36

  24. New Morphometric Vertebral Fracture at Month 12, ITT Population

    Time frame: Baseline and Month 12

    Morphometric Vertebral Fracture measured by semi-quantitative (SQ) analysis of x-rays using the Genant scoring system at endpoint. (Ref: Genant 1993). SQ-Scores range from 0 (no fracture) to 3 (severe fracture). New fracture = SQ score is 0 at baseline and >0 at the specified end visit.

  25. New Morphometric Vertebral Fracture at Month 36, ITT Population

    Time frame: Baseline and Month 36

    Morphometric Vertebral Fracture measured by SQ analysis of x-rays using the Genant scoring system. (Ref: Genant 1993). SQ-Scores range from 0 (no fracture) to 3 (severe fracture). New fracture = SQ score is 0 at baseline and >0 at the specified end visit.

  26. Categorization by Number of New Morphometric Vertebral Fracture at Month 12, ITT

    Time frame: Baseline and Month 12

    Patients with 1 or more New Morphometric Vertebral Fracture as measured by SQ analysis of x-rays using the Genant scoring system (Ref: Genant 1993). SQ-Scores range from 0 (no fracture) to 3 (severe fracture). Incidence = SQ score is 0 at baseline and >0 at post-baseline.

  27. Categorization by Number of New Morphometric Vertebral Fracture at Month 36, ITT

    Time frame: Baseline and Month 36

    Patients with 1 or more New Morphometric Vertebral Fracture as measured by SQ analysis of x-rays using the Genant scoring system (Ref: Genant 1993). SQ-Scores range from 0 (no fracture) to 3 (severe fracture). Incidence = SQ score is 0 at baseline and >0 at post-baseline.

  28. Incidence New Vertebral Fractures by SQ (Semi-Quantitative) Score, Patients Aged 4-9 Years, Month 12, ITT Population

    Time frame: Month 12

    Patients aged 4-9 years with new morphometric vertebral fractures as measured by SQ analysis of x-rays using the Genant scoring system at Month 12 +/- 14 days. (Ref: Genant 1993). SQ Score mild - 0/no fracture to Grade 1, Moderate to Severe - change from 0/no fracture to Grade 2-3.

  29. Incidence New Vertebral Fractures by SQ Score, Patients Aged 10-15 Years, Month 12, ITT Population

    Time frame: Month 12

    Patients aged 10-15 years with new morphometric vertebral fractures as measured by SQ analysis of x-rays using the Genant scoring system at Month 12 +/- 14 days. (Ref: Genant 1993). SQ Score mild - 0/no fracture to Grade 1, Moderate to Severe - change from 0/no fracture to Grade 2-3.

  30. Probability of Fracture in 12 Months (Kaplan-Meier Cumulative Incidence), ITT Population

    Time frame: Time to First Event (days) up to 12 Months

    Long bones include radius, ulna, humerus, tibia, fibula, femur, upper limb and lower limb fracture.

  31. Number of Clinical Fractures, Month 12, ITT Population

    Time frame: 12 Months

    Long bones include radius, ulna, humerus, tibia, fibula, femur, upper limb and lower limb fracture.

  32. Serum BAP - Percent Change From Baseline to Month 12, ITT Population

    Time frame: Baseline and 12 Months

    Serum Bone Alkaline Phosphatase (BAP - bone formation marker). Negative percent changes indicate response to treatment.

  33. Serum BAP - Percent Change From Baseline to Month 24, ITT Population

    Time frame: Baseline and 24 Months

    Serum Bone Alkaline Phosphatase (BAP - bone formation marker). Negative percent changes indicate response to treatment.

  34. Serum BAP - Percent Change From Baseline to Month 36, ITT Population

    Time frame: Baseline and 36 Months

    Serum Bone Alkaline Phosphatase (BAP - bone formation marker). Negative percent changes indicate response to treatment.

  35. Urine NTX/Cr - Percent Change From Baseline at Month 12, ITT Population

    Time frame: Baseline and Endpoint / Month 12

    Urine type-I collagen N-telopeptide/creatinine (NTX/Cr; bone resorption marker). Negative percent changes indicate response to treatment.

  36. Urine NTX/Cr - Percent Change From Baseline at Month 24, ITT Population

    Time frame: Baseline and Month 24

    Urine type-I collagen N-telopeptide/creatinine (NTX/Cr; bone resorption marker). Negative percent changes indicate response to treatment.

  37. Urine NTX/Cr - Percent Change From Baseline at Month 36, ITT Population

    Time frame: Baseline and Month 36

    Urine type-I collagen N-telopeptide/creatinine (NTX/Cr; bone resorption marker). Negative percent changes indicate response to treatment.

  38. Wong-Baker FACES Pain Rating Scale - Change From Baseline to Month 12, ITT Population

    Time frame: Baseline and Month 12

    Wong-Baker FACES Pain Rating Scale (pain assessment scale using facial expressions, translated into a range from 0= no pain [smiling face] to 10= worst pain possible [distorted face with tears]; negative values indicate decrease in pain). Reference: Wong DL et al.

  39. Bone Age (Years), Change From Baseline to Month 12, ITT Population

    Time frame: Baseline and Month 12

    Bone Age determined by visual assessment of hand / wrist radiographs.

  40. Bone Age (Years), Change From Baseline to Month 24, ITT Population

    Time frame: Baseline and Month 24

    Bone Age determined by visual assessment of hand / wrist radiographs.

  41. Bone Age (Years), Change From Baseline to Month 36, ITT Population

    Time frame: Baseline and Month 36

    Bone Age determined by visual assessment of hand / wrist radiographs.

  42. Annualized Growth Velocity - Change From Baseline to Month 12, ITT Population

    Time frame: Baseline and Month 12

    Annualized Growth Velocity [= bone age change from baseline x (365.25/time in days between baseline and the bone age measurement)]

  43. Annualized Growth Velocity - Change From Baseline to Month 36, ITT Population

    Time frame: Baseline and Month 36

    Annualized Growth Velocity [= bone age change from baseline x (365.25/time in days between baseline and the bone age measurement)]

Sponsors and collaborators

Lead sponsor

Warner Chilcott

Industry

Registry information

Important dates

Study start
2004
Primary completion
2008
Study completion
2010
First posted
Mar 21, 2005
Registry last updated
Apr 22, 2013

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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