Gaucher Disease Type 1NCT02067247
Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher Disease
A study testing BeamMed Speed-of-Sound bone strength test at forearm in people with Gaucher Disease Type 1, run by Shaare Zedek Medical Center at 1 site in Israel.
Trial summary
The purpose of this study is to compare the accuracy and comparability and secondarily to assess the values achieved by measurement of the forearm BMD by DXA and SOS by BeamMed, relative to standard DXA evaluations at the FN and LS. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Gaucher Disease Type 1
- What is being tested?
- BeamMed Speed-of-Sound bone strength test at forearm
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteIsrael
- Study dates
- Feb 2014to Sep 2014
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Age: >18
- Gaucher disease type 1
- Able to undergo a DEXA test
Who cannot join
- Minors
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Comparison of BMD Measurements by Dual Energy X-Ray Absorptiometry and Multiple Sites to BeamMed Speed-of-Sound Measurements at the Forearm in Patients With Gaucher Disease
The purpose of this study is to compare the accuracy and comparability and secondarily to assess the values achieved by measurement of the forearm BMD by DXA and SOS by BeamMed, relative to standard DXA evaluations at the FN and LS.
Read the detailed description
Dual-energy X-ray absorptiometry (DXA) is the current gold standard for the clinical diagnosis of osteoporosis based on measurement of bone mineral density (BMD) [Baim et al, 2006]. As DXA technology continues to evolve, new instruments and technologies are introduced [Shepherd et al, 2005], making it necessary to document how these advances compare to prior densitometers.
The purpose of this study is to assess bone mineral density (BMD) comparability and precision using a standard Hologic DXA unit and the quantitative ultrasound-driven speed-of-sound (SOS) BeamMed technology. The physical and mechanical properties of bone that are measured by SOS are different than bone density measured by DXA and reflect bone strength and elasticity [Goossens et al, 2008]. The cohorts will be patients with Gaucher disease [Zimran \& Elstein, 2010] who currently are requested to undergo BMD evaluation only bi-annually [Mistry et al, 2011], partly because of the radiation of DXA scans (admittedly low relative to standard X-rays and CT). Patients with Gaucher disease are at added risk for osteonecrosis and pathological fractures but to date no biomarker or technology has been able to predict which patients are imminently at risk. Because the option of disease-specific but very costly enzyme replacement therapy (ERT) [Barton et al, 1991] is predicating on the estimated risk of severe disease, predicting skeletal damage (especially osteonecrosis at the femoral neck, FN) because of osteopenia or osteoporosis has clinically relevant ramifications [Mistry et al, 2011]. Additionally, it has been shown that there is a correlation between low BMD at the lumbar spine (LS) and anemia in patients with Gaucher disease [Khan et al, 2012], so that with correction of anemia with ERT it is hoped that there will be amelioration of BMD values, and this too would be worth performing at closer intervals.
The least inconvenient and shortest evaluation for BMD is at the forearm. For approximately a year, all patients at the Gaucher disease have had DXA forearm evaluations when undergoing routine testing. It is the intention of this study to prospectively compare forearm SOS results [Olszynski et al, 2013] with those from the standard DXA equipment with the possibility of using SOS as a quantitative measure of bone integrity on a more regular basis.
The strategy will be to measure forearm BMD by DXA and SOS by BeamMed for comparison of accuracy and comparability and secondarily to assess the values achieved at the forearm relative to standard DXA evaluations at the FN and LS.
Patients who arrive at the Gaucher Clinic for routine monitoring that includes DXA evaluation of BMD at FN, LS, and forearm will be requested to consent to undergo SunBeam evaluation at the forearm as well. The same DXA hardware and software (Hologic, Bedford, MA) will be used for all examinations as have been employed by the Gaucher Clinic since August 2011.
The SOS technology as developed by the BeamMed Company (originally, the Sunlight Ultrasound Technologies, Israel) is based on the physical property that ultrasound waves travel faster through bone than soft tissue. The bone sonometer measures the time that elapses between the axially transmitted sound generated as an inaudible high-frequency pulsed acoustic signal at a center frequency of 1.25 MHz and the first reception of a signal after it has traveled through the selected bone. This is recorded as the SOS in m/sec. The ultrasonic waves are transmitted and received by transducers embedded in the ultrasound probe. As the ultrasound wave travels, the speed, dispersion, and attenuation of the signal is influenced by the density, elasticity, and cohesiveness of the medium, so that the greater the density and elasticity of the medium, the faster the speed of propagation [Ashman et al, 1984].
The device consists of a desktop main unit and 3 probes of different sizes. The small probe (1.4cm x 2.7cm x 11cm), 900-1000 KHz will be used for this study. Calibration is performed each day using a standardized calibrating plastic provided by the company. The SOS is determined as the mean of 3 scans that are found to be statistically similar (coefficient of variation below 1.2%), as calculated by the software provided with the device. Based on these 3 readings, the 95th percentile of the measured SOS is determined and used for statistical analysis.
- Conditions
- Gaucher Disease Type 1
- Being tested
- BeamMed Speed-of-Sound bone strength test at forearm
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BeamMed Speed-of-Sound bone strength test at forearmDevice
Also called OMNISENSE 7000S ULTRASOUND BONE SONOMETER
BeamMed Speed-of-Sound bone strength test at forearm in addition to the DEXA test
| Study group | Role | What they receive |
|---|---|---|
| SOS forearm test | Experimental | BeamMed Speed-of-Sound bone strength test at forearm Receives: Device: BeamMed Speed-of-Sound bone strength test at forearm |
What is being measured
The primary outcome is the main question the study is designed to answer.
BMD measurements by DEXA and SOS
Measured: 6 months
Secondary outcomes (1)
T-score and Z-score by DEXA and SOS
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2014 (estimated)
- Study startFeb 2014
- Primary completionSep 2014
- Study completionSep 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Gaucher Disease Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shaare Zedek Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ari Zimran, Shaare Zedek Medical Center
- Investigators
- Ari Zimran, Prof., Principal investigator, Shaare Zedek Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02067247
- Sponsor's protocol ID
- DEXA-SOS
- Status last verified
- Nov 2014
- First submitted
- 27 Jan 2014
- Last updated
- 19 Nov 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Baskin E, Dinur T, Lebel E, Tiomkin M, Elstein D, Zimran A. Comparison of Bone Mineral Density by Dual-Energy X-Ray Absorptiometry and Bone Strength by Speed-of-Sound Ultrasonography in Adults With Gaucher Disease. J Clin Densitom. 2016 Oct;19(4):465-470. doi: 10.1016/j.jocd.2015.12.002. Epub 2016 Jan 15.
PubMed 26781431
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparison of BMD Measurements by Dual Energy X-Ray Absorptiometry and Multiple Sites to BeamMed Speed-of-Sound Measurements at the Forearm in Patients With Gaucher Disease
- ClinicalTrials.gov ID
- NCT02067247
- Acronym / sponsor ID
- DEXA-SOS
- Conditions
- Gaucher Disease Type 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Shaare Zedek Medical CenterUniversity, hospital or other
- Study start
- Feb 2014
- Primary completion
- Sep 2014 (actual)
- Study completion
- Sep 2014 (actual)
- First posted
- 20 Feb 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to compare the accuracy and comparability and secondarily to assess the values achieved by measurement of the forearm BMD by DXA and SOS by BeamMed, relative to standard DXA evaluations at the FN and LS.
Detailed description
Dual-energy X-ray absorptiometry (DXA) is the current gold standard for the clinical diagnosis of osteoporosis based on measurement of bone mineral density (BMD) [Baim et al, 2006]. As DXA technology continues to evolve, new instruments and technologies are introduced [Shepherd et al, 2005], making it necessary to document how these advances compare to prior densitometers.
The purpose of this study is to assess bone mineral density (BMD) comparability and precision using a standard Hologic DXA unit and the quantitative ultrasound-driven speed-of-sound (SOS) BeamMed technology. The physical and mechanical properties of bone that are measured by SOS are different than bone density measured by DXA and reflect bone strength and elasticity [Goossens et al, 2008]. The cohorts will be patients with Gaucher disease [Zimran \& Elstein, 2010] who currently are requested to undergo BMD evaluation only bi-annually [Mistry et al, 2011], partly because of the radiation of DXA scans (admittedly low relative to standard X-rays and CT). Patients with Gaucher disease are at added risk for osteonecrosis and pathological fractures but to date no biomarker or technology has been able to predict which patients are imminently at risk. Because the option of disease-specific but very costly enzyme replacement therapy (ERT) [Barton et al, 1991] is predicating on the estimated risk of severe disease, predicting skeletal damage (especially osteonecrosis at the femoral neck, FN) because of osteopenia or osteoporosis has clinically relevant ramifications [Mistry et al, 2011]. Additionally, it has been shown that there is a correlation between low BMD at the lumbar spine (LS) and anemia in patients with Gaucher disease [Khan et al, 2012], so that with correction of anemia with ERT it is hoped that there will be amelioration of BMD values, and this too would be worth performing at closer intervals.
The least inconvenient and shortest evaluation for BMD is at the forearm. For approximately a year, all patients at the Gaucher disease have had DXA forearm evaluations when undergoing routine testing. It is the intention of this study to prospectively compare forearm SOS results [Olszynski et al, 2013] with those from the standard DXA equipment with the possibility of using SOS as a quantitative measure of bone integrity on a more regular basis.
The strategy will be to measure forearm BMD by DXA and SOS by BeamMed for comparison of accuracy and comparability and secondarily to assess the values achieved at the forearm relative to standard DXA evaluations at the FN and LS.
Patients who arrive at the Gaucher Clinic for routine monitoring that includes DXA evaluation of BMD at FN, LS, and forearm will be requested to consent to undergo SunBeam evaluation at the forearm as well. The same DXA hardware and software (Hologic, Bedford, MA) will be used for all examinations as have been employed by the Gaucher Clinic since August 2011.
The SOS technology as developed by the BeamMed Company (originally, the Sunlight Ultrasound Technologies, Israel) is based on the physical property that ultrasound waves travel faster through bone than soft tissue. The bone sonometer measures the time that elapses between the axially transmitted sound generated as an inaudible high-frequency pulsed acoustic signal at a center frequency of 1.25 MHz and the first reception of a signal after it has traveled through the selected bone. This is recorded as the SOS in m/sec. The ultrasonic waves are transmitted and received by transducers embedded in the ultrasound probe. As the ultrasound wave travels, the speed, dispersion, and attenuation of the signal is influenced by the density, elasticity, and cohesiveness of the medium, so that the greater the density and elasticity of the medium, the faster the speed of propagation [Ashman et al, 1984].
The device consists of a desktop main unit and 3 probes of different sizes. The small probe (1.4cm x 2.7cm x 11cm), 900-1000 KHz will be used for this study. Calibration is performed each day using a standardized calibrating plastic provided by the company. The SOS is determined as the mean of 3 scans that are found to be statistically similar (coefficient of variation below 1.2%), as calculated by the software provided with the device. Based on these 3 readings, the 95th percentile of the measured SOS is determined and used for statistical analysis.
Study design
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SOS forearm test | Experimental | Device: BeamMed Speed-of-Sound bone strength test at forearmBeamMed Speed-of-Sound bone strength test at forearm |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Age: >18
- Gaucher disease type 1
- Able to undergo a DEXA test
Key exclusion criteria (1 listed)
- Minors
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BeamMed Speed-of-Sound bone strength test at forearmAlso: OMNISENSE 7000S ULTRASOUND BONE SONOMETERBeamMed Speed-of-Sound bone strength test at forearm in addition to the DEXA test | Device | SOS forearm test |
Primary outcome
BMD measurements by DEXA and SOS
Time frame: 6 months
1 secondary outcome
T-score and Z-score by DEXA and SOS
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2014.
- Derived from this study: PubMed 26781431Baskin E, Dinur T, Lebel E, Tiomkin M, Elstein D, Zimran A. Comparison of Bone Mineral Density by Dual-Energy X-Ray Absorptiometry and Bone Strength by Speed-of-Sound Ultrasonography in Adults With Gaucher Disease. J Clin Densitom. 2016 Oct;19(4):465-470. doi: 10.1016/j.jocd.2015.12.002. Epub 2016 Jan 15.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ari Zimran, Prof. principal investigator
Shaare Zedek Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing BeamMed Speed-of-Sound bone strength test at forearm in people with Gaucher Disease Type 1, run by Shaare Zedek Medical Center at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
BMD measurements by DEXA and SOS (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02067247, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Nov 2014.