Clinical trial NCT00106028: Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children
Official title: Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Osteogenesis Imperfecta
Keywords: Primary disease: Osteogenesis Imperfecta
Lead sponsor: Warner Chilcott
Sponsor class: INDUSTRY
First posted: 2005-03-21
Enrollment: 143
Sex: ALL
Minimum age (years): 4
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 12, ITT Population
timeFrame: Baseline and Month 12
Secondary outcomes:
measure: Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Percent Change From Baseline in Total Body BMD at Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Percent Change From Baseline in Total Body BMD at Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Percent Change From Baseline in Total Body BMD at Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Percent Change From Baseline in Total Body BMC at Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Percent Change From Baseline in Total Body BMC at Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Percent Change From Baseline in Total Body BMC at Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Lumbar Spine Z-score - Percent Change From Baseline to Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Lumbar Spine Z-score - Percent Change From Baseline to Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Lumbar Spine Z-score - Percent Change From Baseline to Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Total Body Z-score- Percent Change From Baseline to Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Total Body Z-score- Percent Change From Baseline to Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Total Body Z-score- Percent Change From Baseline to Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Percent Change From Baseline in Lumbar Spine Bone Area at Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Percent Change From Baseline in Lumbar Spine Bone Area at Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Percent Change From Baseline in Lumbar Spine Bone Area at Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Percent Change From Baseline in Total Body Bone Area Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Percent Change From Baseline in Total Body Bone Area Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Percent Change From Baseline in Total Body Bone Area Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: New Morphometric Vertebral Fracture at Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: New Morphometric Vertebral Fracture at Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Categorization by Number of New Morphometric Vertebral Fracture at Month 12, ITT
timeFrame: Baseline and Month 12
measure: Categorization by Number of New Morphometric Vertebral Fracture at Month 36, ITT
timeFrame: Baseline and Month 36
measure: Incidence New Vertebral Fractures by SQ (Semi-Quantitative) Score, Patients Aged 4-9 Years, Month 12, ITT Population
timeFrame: Month 12
measure: Incidence New Vertebral Fractures by SQ Score, Patients Aged 10-15 Years, Month 12, ITT Population
timeFrame: Month 12
measure: Probability of Fracture in 12 Months (Kaplan-Meier Cumulative Incidence), ITT Population
timeFrame: Time to First Event (days) up to 12 Months
measure: Number of Clinical Fractures, Month 12, ITT Population
timeFrame: 12 Months
measure: Serum BAP - Percent Change From Baseline to Month 12, ITT Population
timeFrame: Baseline and 12 Months
measure: Serum BAP - Percent Change From Baseline to Month 24, ITT Population
timeFrame: Baseline and 24 Months
measure: Serum BAP - Percent Change From Baseline to Month 36, ITT Population
timeFrame: Baseline and 36 Months
measure: Urine NTX/Cr - Percent Change From Baseline at Month 12, ITT Population
timeFrame: Baseline and Endpoint / Month 12
measure: Urine NTX/Cr - Percent Change From Baseline at Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Urine NTX/Cr - Percent Change From Baseline at Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Wong-Baker FACES Pain Rating Scale - Change From Baseline to Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Bone Age (Years), Change From Baseline to Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Bone Age (Years), Change From Baseline to Month 24, ITT Population
timeFrame: Baseline and Month 24
measure: Bone Age (Years), Change From Baseline to Month 36, ITT Population
timeFrame: Baseline and Month 36
measure: Annualized Growth Velocity - Change From Baseline to Month 12, ITT Population
timeFrame: Baseline and Month 12
measure: Annualized Growth Velocity - Change From Baseline to Month 36, ITT Population
timeFrame: Baseline and Month 36
Other outcomes:
Locations:
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Omaha
country: United States
facility: University of Nebraska Medical Center, Children's Hospital
state: Nebraska
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: Dayton
country: United States
facility: Wright State University BioMedical Imaging Laboratory and Miami Valley Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Westmead
country: Australia
facility: The Children's Hospital at Westmead
state: New South Wales
city: Perth
country: Australia
facility: Princess Margaret Hospital for Children
city: Bruxelles
country: Belgium
facility: Cliniques Universitaires Saint Luc
city: Santiago
country: Chile
facility: Pontificia Universidad Catolica de Chile
city: Plzen
country: Czech Republic
facility: Osteocentrum, II. Interní klinika, Fakultní nemocnice Plzeň-Bory
city: Helsinki
country: Finland
facility: Hospital for Children and Adolescents
city: Koln
country: Germany
facility: Klinikum und Poliklinik für Kinderheilkunde der Universität zu Köln
city: Budapest
country: Hungary
facility: 2nd Department of Pediatrics, Semmelwies University, Faculty of Medicine
city: Valeggio sul Mincio
country: Italy
facility: Rheumatologic Rehabilitation Unit of the University of Verona
city: Warzawa-Międzylesie
country: Poland
facility: Zaklad Biochemii i Medycyny Doswiadczalnej (Biochemisty Dept, Institute "Monument-Children Health Centre"
city: Pretoria
country: South Africa
facility: Little Company of Mary Hospital
state: Gauteng
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deu
city: Bristol
country: United Kingdom
facility: Bristol Royal Hospital for Children,
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Sick Children
city: Sheffield
country: United Kingdom
facility: Sheffield Children's Hospital
Study officials:
affiliation: Procter and Gamble
name: Dietrich H Wenderoth, MD
role: STUDY_DIRECTOR
References:
citation: Bishop N, Adami S, Ahmed SF, Anton J, Arundel P, Burren CP, Devogelaer JP, Hangartner T, Hosszu E, Lane JM, Lorenc R, Makitie O, Munns CF, Paredes A, Pavlov H, Plotkin H, Raggio CL, Reyes ML, Schoenau E, Semler O, Sillence DO, Steiner RD. Risedronate in children with osteogenesis imperfecta: a randomised, double-blind, placebo-controlled trial. Lancet. 2013 Oct 26;382(9902):1424-32. doi: 10.1016/S0140-6736(13)61091-0. Epub 2013 Aug 6.