NCT00106028

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.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
143

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Nov 2004

Longer than P75 for phase_3

Geographic Reach
13 countries

20 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2004

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

March 18, 2005

Completed
3 days until next milestone

First Posted

Study publicly available on registry

March 21, 2005

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2008

Completed
1.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2010

Completed
4 months until next milestone

Results Posted

Study results publicly available

June 28, 2010

Completed
Last Updated

April 22, 2013

Status Verified

April 1, 2013

Enrollment Period

3.4 years

First QC Date

March 18, 2005

Results QC Date

April 15, 2009

Last Update Submit

April 15, 2013

Conditions

Osteogenesis Imperfecta

Keywords

Primary disease: Osteogenesis Imperfecta

Outcome Measures

Primary Outcomes (1)

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

    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.

    Baseline and Month 12

Secondary Outcomes (43)

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

    Baseline and Month 24

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

    Baseline and Month 36

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

    Baseline and Month 12

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

    Baseline and Month 24

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

    Baseline and Month 36

  • +38 more secondary outcomes

Study Arms (2)

Placebo Daily

PLACEBO COMPARATOR

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

Drug: Placebo

Risedronate Daily

EXPERIMENTAL

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

Drug: risedronate sodium (Actonel)

Interventions

risedronate sodium (Actonel)DRUG

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

Risedronate Daily
PlaceboDRUG

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

Placebo Daily

Eligibility Criteria

Age4 Years - 15 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • Any bisphosphonate use within one year of enrollment

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

University of Nebraska Medical Center, Children's Hospital

Omaha, Nebraska, 68114, United States

Location

Hospital for Special Surgery

New York, New York, 10021, United States

Location

Wright State University BioMedical Imaging Laboratory and Miami Valley Hospital

Dayton, Ohio, 45409, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

The Children's Hospital at Westmead

Westmead, New South Wales, Australia

Location

Princess Margaret Hospital for Children

Perth, Australia

Location

Cliniques Universitaires Saint Luc

Brussels, Belgium

Location

Pontificia Universidad Catolica de Chile

Santiago, Chile

Location

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

Pilsen, Czechia

Location

Hospital for Children and Adolescents

Helsinki, Finland

Location

Klinikum und Poliklinik für Kinderheilkunde der Universität zu Köln

Cologne, Germany

Location

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

Budapest, Hungary

Location

Rheumatologic Rehabilitation Unit of the University of Verona

Valeggio sul Mincio, Italy

Location

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

Warzawa-Międzylesie, Poland

Location

Little Company of Mary Hospital

Pretoria, Gauteng, South Africa

Location

Hospital Sant Joan de Deu

Barcelona, Spain

Location

Royal Hospital for Sick Children

Glasgow, Glasgow, G3 8SJ, United Kingdom

Location

Sheffield Children's Hospital

Sheffield, Sheffield, S210 2TH, United Kingdom

Location

Bristol Royal Hospital for Children,

Bristol, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 23927913DERIVED

MeSH Terms

Conditions

Osteogenesis Imperfecta

Interventions

Risedronic Acid

Condition Hierarchy (Ancestors)

OsteochondrodysplasiasBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCollagen DiseasesConnective Tissue DiseasesSkin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

DiphosphonatesOrganophosphonatesOrganophosphorus CompoundsOrganic ChemicalsPyridinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Limitations and Caveats

The clinical meaningfulness of new fractures in the thoracic spine scored as Genant Grade I is unclear, thus the morphometric vertebral fracture data are evaluated further.

Results Point of Contact

Title
Grexan Wulff, Manager Regulatory Affairs
Organization
Warner Chilcott
gwulff@wcrx.com
973-442-3376

Study Officials

  • Dietrich H Wenderoth, MD

    Procter and Gamble

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 18, 2005

First Posted

March 21, 2005

Study Start

November 1, 2004

Primary Completion

April 1, 2008

Study Completion

March 1, 2010

Last Updated

April 22, 2013

Results First Posted

June 28, 2010

Record last verified: 2013-04

Locations

AU(2)HU(1)GB(3)IT(1)US(5)CL(1)DE(1)FI(1)PL(1)BE(1)ES(1)ZA(1)CZ(1)