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

NCT ID: NCT00106028

Last Updated: 2013-04-22

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

143 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-11-30

Study Completion Date

2010-03-31

Brief Summary

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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.

Detailed Description

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Conditions

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Osteogenesis Imperfecta

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Placebo Daily

placebo tablet, once a day for one year then for two years open label risedronate

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Risedronate Daily

risedronate tablet, once a day for one year then for two years open label risedronate once a day

Group Type EXPERIMENTAL

risedronate sodium (Actonel)

Intervention Type DRUG

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

Interventions

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risedronate sodium (Actonel)

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

Intervention Type DRUG

Placebo

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

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

4 Years

Maximum Eligible Age

15 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Warner Chilcott

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Dietrich H Wenderoth, MD

Role: STUDY_DIRECTOR

Procter and Gamble

Locations

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Miami Children's Hospital

Miami, Florida, United States

Site Status

University of Nebraska Medical Center, Children's Hospital

Omaha, Nebraska, United States

Site Status

Hospital for Special Surgery

New York, New York, United States

Site Status

Wright State University BioMedical Imaging Laboratory and Miami Valley Hospital

Dayton, Ohio, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

The Children's Hospital at Westmead

Westmead, New South Wales, Australia

Site Status

Princess Margaret Hospital for Children

Perth, , Australia

Site Status

Cliniques Universitaires Saint Luc

Brussels, , Belgium

Site Status

Pontificia Universidad Catolica de Chile

Santiago, , Chile

Site Status

Osteocentrum, II. Interní klinika, Fakultní nemocnice Plzeň-Bory

Pilsen, , Czechia

Site Status

Hospital for Children and Adolescents

Helsinki, , Finland

Site Status

Klinikum und Poliklinik für Kinderheilkunde der Universität zu Köln

Cologne, , Germany

Site Status

2nd Department of Pediatrics, Semmelwies University, Faculty of Medicine

Budapest, , Hungary

Site Status

Rheumatologic Rehabilitation Unit of the University of Verona

Valeggio sul Mincio, , Italy

Site Status

Zaklad Biochemii i Medycyny Doswiadczalnej (Biochemisty Dept, Institute "Monument-Children Health Centre"

Warzawa-Międzylesie, , Poland

Site Status

Little Company of Mary Hospital

Pretoria, Gauteng, South Africa

Site Status

Hospital Sant Joan de Deu

Barcelona, , Spain

Site Status

Royal Hospital for Sick Children

Glasgow, Glasgow, United Kingdom

Site Status

Sheffield Children's Hospital

Sheffield, Sheffield, United Kingdom

Site Status

Bristol Royal Hospital for Children,

Bristol, , United Kingdom

Site Status

Countries

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United States Australia Belgium Chile Czechia Finland Germany Hungary Italy Poland South Africa Spain United Kingdom

References

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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.

Reference Type DERIVED
PMID: 23927913 (View on PubMed)

Other Identifiers

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HMR4003I/3001

Identifier Type: -

Identifier Source: secondary_id

2003100

Identifier Type: -

Identifier Source: org_study_id

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