NCT00159419

Brief Summary

The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." We, the researchers at Indiana University School of Medicine, are characterizing the changes effected by oral bisphosphonate therapy and comparing them to a regimen of intravenous bisphosphonate therapy in a group of children with OI and also in children with other disorders that result in low bone mass and fractures.

Trial Health

100
On Track

Trial Health Score

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

Enrollment
18

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Aug 1999

Longer than P75 for phase_4

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

August 1, 1999

Completed
6.1 years until next milestone

First Submitted

Initial submission to the registry

September 7, 2005

Completed
5 days until next milestone

First Posted

Study publicly available on registry

September 12, 2005

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2008

Completed
7.7 years until next milestone

Results Posted

Study results publicly available

March 31, 2016

Completed
Last Updated

June 14, 2017

Status Verified

May 1, 2017

Enrollment Period

9 years

First QC Date

September 7, 2005

Results QC Date

November 12, 2013

Last Update Submit

May 17, 2017

Conditions

Keywords

Osteogenesis ImperfectaFracturesPediatricOsteoporosisJuvenile Pagets

Outcome Measures

Primary Outcomes (1)

  • Bone Mineral Density

    By Dual-energy x-ray absorptiometry. Results were reported as z-scores as well as as absolute values. The Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z scores indicate a better outcome", or similar, as accurate and appropriate.

    2 years

Study Arms (2)

Alendronate

ACTIVE COMPARATOR

1 mg/kg po qd rounded to nearest 10 or 20 mg dose

Drug: Alendronate

Pamidronate

ACTIVE COMPARATOR

3 mg/kg IV q4 months

Drug: Pamidronate

Interventions

Also known as: fosamax
Alendronate
Pamidronate

Eligibility Criteria

Age3 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of OI, as defined by genetic analysis revealing a defect of type I collagen, OR by bone mineral density (BMD) \<2.5 standard deviations (SD) for age plus two of the following:
  • Family history of OI
  • Frequent fractures
  • Blue sclerae
  • Multiple wormian bones on skull x-ray
  • Hearing disturbance
  • Dentinogenesis imperfecta
  • Age between 3 and 21 years at the start of the study period.
  • Children must be able to swallow whole tablets
  • Parents of children must be able to understand protocol and give informed consent.

You may not qualify if:

  • Therapy with bisphosphonates during the past 12 months.
  • Other "non-traditional" therapy for OI in the last 6 months, such as growth hormone or anabolic steroids.
  • Other chronic diseases besides OI that interfere with bone morphology or gastrointestinal absorption

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Osteogenesis ImperfectaOsteoporosisOsteitis DeformansFractures, Bone

Interventions

AlendronatePamidronate

Condition Hierarchy (Ancestors)

OsteochondrodysplasiasBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCollagen DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesBone Diseases, MetabolicMetabolic DiseasesNutritional and Metabolic DiseasesWounds and Injuries

Intervention Hierarchy (Ancestors)

DiphosphonatesOrganophosphonatesOrganophosphorus CompoundsOrganic Chemicals

Results Point of Contact

Title
Dr. Linda DiMeglio
Organization
Indiana University School of Medicine

Study Officials

  • Linda A DiMeglio, MD, MPH

    Indiana University School of Medicine

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

September 7, 2005

First Posted

September 12, 2005

Study Start

August 1, 1999

Primary Completion

August 1, 2008

Study Completion

August 1, 2008

Last Updated

June 14, 2017

Results First Posted

March 31, 2016

Record last verified: 2017-05