Do Bisphosphonates Alter the Skeletal Response to Mechanical Stimulation in Children With Osteogenesis Imperfecta?

NCT03208582 | Completed Phase 2 DMC

• 1 other identifier: SCH-2013
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

Osteogenesis Imperfecta(OI) is an inherited disorder characterised by extreme fragility of the bones. Bones often break from little or no apparent cause. Current available medicine can increase bone strength by making bones wider and "filling in" the holes in the bone walls that weaken it. These medicines are bisphosphonates, given either by a drip intravenously (eg pamidronate), or taken by mouth (eg risedronate). Their major action is to prevent bone breakdown by stopping the normal process of removing and then replacing old bone tissue, so in some parts of the bone, new bone formation is actually reduced. Most studies of bisphosphonates in children with OI have shown increased bone mineral density and improved exercise tolerance that could positively affect new bone formation; some have shown reduced fracture rate. Bone is highly responsive to mechanical stimulation. Whole body vibration (WBV) is a form of mechanical stimulation that has been shown to improve bone mineral density in some individuals with narrow bones. Little is known whether bisphosphonates affect the response of the skeleton to mechanical stimulation. We will determine the response to mechanical stimulation in children with OI by looking at bone turnover markers following WBV in those who are and are not treated with bisphosphonates. The results from this study will help us to understand whether skeleton in children with OI is normally responsive to mechanical stimulation, and whether bisphosphonates alter that responsiveness in a way that is either beneficial or not for increasing bone strength.

Conditions

Osteogenesis Imperfecta

Outcome Measures

Primary Outcomes (1)

• Change in P1NP response to 1 week of vibration without risedronate treatment, followed by a washout period. Change in P1NP response to vibration will be reassessed following Risedonate treatment. Serial bone markers will be done over a 99 day period.

  To assess if risedronate alters the response to mechanical stimulation

  99 days

Study Arms (1)

Single arm trial

OTHER

Intervention : Risedronate Sodium (oral) Dosage: 1mg/kg/week Frequency: once/week Duration: 6 weeks

Drug: Risedronate Sodium; Dietary Supplement: Calcichew tablets

Interventions

Risedronate Sodium DRUG

Participants will be initially tested on the response to mechanical stimulation as a baseline and then tested again after 6 weeks treatment with Risedronate

Also known as: Bisphosphonate

Single arm trial

Calcichew tablets DIETARY_SUPPLEMENT

Participants will take calcichew tablets during the 6 week period of risedronate treatment

Single arm trial

Eligibility Criteria

• Age: 4 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age 4-16 years
• Able to speak fluent English
• Diagnosed with osteogenesis imperfecta
• Able to stand
• Not treated with bisphosphonates

You may not qualify if:

• Presence of other chronic illnesses
• Balance problems
• Recent fracture (in the last 6 months)
• Recent (last 12 months) or current treatment likely to affect bone - this does not include inhaled or intermittent oral therapy with steroids for asthma
• Involvement in another interventional research project

Sponsors & Collaborators

• Sheffield Children's NHS Foundation Trust: lead

Study Sites (1)

Clinical Research Facility

Sheffield, South Yorkshire, S10 2TH, United Kingdom

Location

MeSH Terms

Conditions

Osteogenesis Imperfecta

Interventions

Risedronic Acid; Diphosphonates; Calcium Carbonate

Condition Hierarchy (Ancestors)

Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Organophosphonates; Organophosphorus Compounds; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Calcium Compounds; Inorganic Chemicals; Carbonates; Carbonic Acid; Carbon Compounds, Inorganic; Minerals

Study Officials

• Nick Bishop, MD, FRCPCH

  Sheffield Children's Hospital and University of Sheffield

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 23, 2017
• First Posted: July 5, 2017
• Study Start: April 1, 2017
• Primary Completion: November 2, 2017
• Study Completion: November 2, 2017
• Last Updated: September 7, 2018

Record last verified: 2017-05

Locations

GB (1)