Von-Hippel LindauPhase 2CEUS-VHLNCT03907657
Contrast-enhanced Ultrasound as a Screening Tool for Kidney Cancer in Patients With Von-Hippel Lindau
A Phase 2 study testing Perflutren lipid microsphere and Sulfur hexafluoride lipid microspheres in people with Von-Hippel Lindau and Kidney Disease, Chronic, run by UNC Lineberger Comprehensive Cancer Center at 1 site in United States.
Trial summary
The purpose of this study is to determine if contrast-enhanced ultrasound can detect abnormal features of kidney lesions in patients with Von-Hippel Lindau with the same accuracy as conventional ultrasound and contrast-enhanced magnetic resonance imaging (MRI) Read more about this study
- Who is it for?
- Ages 16 to 99 years
- Conditions the study is for
- Von-Hippel Lindau, Kidney Disease, Chronic
- What is being tested?
- Perflutren lipid microsphere and Sulfur hexafluoride lipid microspheres
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Sep 2019to 13 Mar 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 16 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- To be eligible for the present study, patients must meet the following criteria:
- Able to provide written informed consent
- Willing to comply with protocol requirements
- At least 16 years of age
- Carry a diagnosis of VHL and able to undergo routine clinical screening tests for RCC
Who cannot join
- Critically ill or medically unstable or in an intensive care setting and whose critical course during the observation period would be unpredictable
- Known hypersensitivity to sulfur hexafluoride or to any component of perflutren lipid (Definity®) or sulfur hexafluoride (Lumason®)
- Right to left shunt, severe pulmonary hypertension (Pulmonary artery pressure >90mmHg), or adult respiratory distress syndrome
- Active cardiac disease including any of the following:
- Severe congestive heart failure (class IV in accordance with the classification of the New York Heart Association) B.Unstable angina. C.Symptomatic arrhythmia (i.e. tachycardia, bradycardia, supraventricular tachycardia, ventricular fibrillation, ventricular tachycardia, atrial flutter or fibrillation).
- D.Myocardial infarction within 14 days prior to the date of proposed microbubble administration.
Show all 11Show fewer
- Has any other medical condition or other circumstances that would significantly decrease the chances of obtaining reliable data or of achieving the study objectives such as:
- Mental illness
- Drug abuse
- Female patient who is pregnant or lactating
- Obesity that limits obtainment of acceptable images
Trial locations and contacts
1 site in 1 country.
United States1 site
University of North Carolina of Chapel hill
Chapel Hill, North Carolina, 27599-7510
About this study
Official title: Double-Center Cross-Sectional Study of Contrast-Enhanced Ultrasound With Lumason/Definity as a Screening Tool for Kidney Cancer in Patients With Von-Hippel Lindau
The purpose of this study is to determine if contrast-enhanced ultrasound can detect abnormal features of kidney lesions in patients with Von-Hippel Lindau with the same accuracy as conventional ultrasound and contrast-enhanced magnetic resonance imaging (MRI)
Read the detailed description
This is a pilot cross-sectional study that compares contrast enhanced ultrasound to conventional ultrasound and contrasted MRI. Any patient undergoing annual imaging screening is eligible, but the investigators will target inclusion of at least 10 subjects who have at least 1 kidney lesion. Therefore, up to 5 subjects may have no current kidney lesions. Subject participation will be only for the day of CEUS study. There will be no follow-up period for this study. However, if results are encouraging, a longitudinal observational study may follow, and these same subjects would be eligible for enrollment. Eligible subjects will undergo a contrast enhanced ultrasound.
Following completion of imaging, all CEUS, MRI (within 4 months) and B-mode (at time of CEUS) US studies will be de-identified. Blinded radiologists will interpret images and provide an overall assessment of risk of malignancy to each kidney using the Bosniak criteria for each kidney lesion present. The Bosniak criteria places cystic lesions into one of 5 categories (I, II, IIF, III and IV) based on lesion characteristics. CEUS based diagnosis will be compared to the diagnoses on routine B-mode US and contrast-enhanced MRI.
- Conditions
- Von-Hippel Lindau, Kidney Disease, Chronic
- Being tested
- Perflutren lipid microsphere, Sulfur hexafluoride lipid microspheres
- Medical subject headings
- Renal Insufficiency, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Perflutren lipid microsphereDrug
Also called • Definity®, • Microbubble contrast agent
Baseline B-mode US will be performed to confirm the lesions being imaged (unless it is a subject with no lesions, then a basic B-mode US will be performed. Perflutren is a diagnostic drug that is intended to be used for contrast enhancement. The vial contains a clear, colorless, sterile, non-pyrogenic, hypertonic solution which is activated by mechanical agitation with Vialmix®.It will be dispensed in inactivated form to a study team member. The contrast agent will be activated just prior to administration (ideally to be used within 5 minutes of activation). Contrast agent Definity (or Lumason) will be activated according to package insert instructions, including use of VialMix®, the device used to activate Definity. Administration will occur IV in coordination with sonography staff trained specifically in contrast ultrasound imaging.The total imaging time is anticipated to be less than 30 minutes.
Sulfur hexafluoride lipid microspheresDrug
Also called Lumason
Baseline B-mode US will be performed to confirm the lesions being imaged (unless it is a subject with no lesions, then a basic B-mode US will be performed. Lumason will be used as a secondary contrast agent only if Definity(perflutren) is unavailable.This drug will be administered using the dosing range and administration type within the Lumason prescribing information. The contrast agent will be activated just prior to administration (ideally to be used within 5 minutes of activation). Contrast agent Definity (or Lumason) will be activated according to package insert instructions, including use of VialMix®, the device used to activate Definity. Administration will occur IV in coordination with sonography staff trained specifically in contrast ultrasound imaging.The total imaging time is anticipated to be less than 30 minutes.
| Study group | Role | What they receive |
|---|---|---|
| Perflutren Lipid Microsphere or Lumason | Experimental | Patients with Von-Hippel Lindau disease will be imaged using contrast-enhanced ultrasound with either perflutren or Lumason. Receives: Drug: Perflutren lipid microsphere; Drug: Sulfur hexafluoride lipid microspheres |
What is being measured
The primary outcome is the main question the study is designed to answer.
Comparison in Radiologist's Lesion Evaluation: CEUS Versus B-mode Ultrasound
Each reader will evaluate kidney imaging using Contrast-Enhanced Ultrasound (CEUS) versus Brightness Mode Ultrasound (B-mode) in patients at risk for kidney lesions. A dichotomized Bosniak score was used for comparison. Dichotomized Bosniak score: 0= nonmalignant and 1=malignant or possibly malignant. Scores were averaged across all readers. Kidneys were assessed by CEUS and B-mode by three different readers.
Measured: Baseline
Secondary outcomes (1)
Comparison in Radiologist's Lesion Evaluation: CEUS Versus Clinically Performed MRI
Each reader will evaluate kidney imaging using Contrast-Enhanced Ultrasound (CEUS). Potentially malignant lesions were extracted from clinically performed magnetic resonance imaging (MRI) completed within 4 months of CEUS. Dichotomized Bosniak scores from CEUS were compared with MRI-derived malignancy scores.
Dichotomized Bosniak scores: 0= nonmalignant and 1=malignant or possibly malignant. Scores were averaged across all readers.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2019
- Study start23 Sep 2019
- Primary completion13 Mar 2023
- Study completion13 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Von-Hippel LindauStudies of the same condition, open and under way.
- More trials run by UNC Lineberger Comprehensive Cancer CenterThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- UNC Lineberger Comprehensive Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Lantheus Medical Imaging
- Responsible party
- Sponsor
- Investigators
- Emily Chang, MD, Principal investigator, University of North Carolina, Chapel Hill
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03907657
- Sponsor's protocol ID
- LCCC1824
- Acronym
- CEUS-VHL
- Status last verified
- May 2024
- First submitted
- 5 Apr 2019
- Last updated
- 21 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Larcher A, Rowe I, Belladelli F, Fallara G, Raggi D, Necchi A, Montorsi F, Capitanio U, Salonia A; OSR VHL Program. Von Hippel-Lindau disease-associated renal cell carcinoma: a call to action. Curr Opin Urol. 2022 Jan 1;32(1):31-39. doi: 10.1097/MOU.0000000000000950.
PubMed 34783716
Study at a glance
From the registry record, last updated by the sponsor on 21 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-Center Cross-Sectional Study of Contrast-Enhanced Ultrasound With Lumason/Definity as a Screening Tool for Kidney Cancer in Patients With Von-Hippel Lindau
- ClinicalTrials.gov ID
- NCT03907657
- Acronym / sponsor ID
- CEUS-VHL / LCCC1824
- Conditions
- Von-Hippel Lindau, Kidney Disease, Chronic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- UNC Lineberger Comprehensive Cancer CenterUniversity, hospital or other
- Collaborators
- Lantheus Medical Imaging
- Study start
- 23 Sep 2019 (actual)
- Primary completion
- 13 Mar 2023 (actual)
- Study completion
- 13 Mar 2023 (actual)
- First posted
- 9 Apr 2019 (actual)
- Results
- Posted 21 May 2024
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 determine if contrast-enhanced ultrasound can detect abnormal features of kidney lesions in patients with Von-Hippel Lindau with the same accuracy as conventional ultrasound and contrast-enhanced magnetic resonance imaging (MRI)
Detailed description
This is a pilot cross-sectional study that compares contrast enhanced ultrasound to conventional ultrasound and contrasted MRI. Any patient undergoing annual imaging screening is eligible, but the investigators will target inclusion of at least 10 subjects who have at least 1 kidney lesion. Therefore, up to 5 subjects may have no current kidney lesions. Subject participation will be only for the day of CEUS study. There will be no follow-up period for this study. However, if results are encouraging, a longitudinal observational study may follow, and these same subjects would be eligible for enrollment. Eligible subjects will undergo a contrast enhanced ultrasound.
Following completion of imaging, all CEUS, MRI (within 4 months) and B-mode (at time of CEUS) US studies will be de-identified. Blinded radiologists will interpret images and provide an overall assessment of risk of malignancy to each kidney using the Bosniak criteria for each kidney lesion present. The Bosniak criteria places cystic lesions into one of 5 categories (I, II, IIF, III and IV) based on lesion characteristics. CEUS based diagnosis will be compared to the diagnoses on routine B-mode US and contrast-enhanced MRI.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Perflutren Lipid Microsphere or Lumason | Experimental | Drug: Perflutren lipid microsphere, Drug: Sulfur hexafluoride lipid microspheresPatients with Von-Hippel Lindau disease will be imaged using contrast-enhanced ultrasound with either perflutren or Lumason. |
Who may qualify
- Ages
- 16 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Able to provide written informed consent
- Willing to comply with protocol requirements
- At least 16 years of age
- Carry a diagnosis of VHL and able to undergo routine clinical screening tests for RCC
Key exclusion criteria (11 listed)
- Critically ill or medically unstable or in an intensive care setting and whose critical course during the observation period would be unpredictable
- Known hypersensitivity to sulfur hexafluoride or to any component of perflutren lipid (Definity®) or sulfur hexafluoride (Lumason®)
- Right to left shunt, severe pulmonary hypertension (Pulmonary artery pressure >90mmHg), or adult respiratory distress syndrome
- Active cardiac disease including any of the following:
- D.Myocardial infarction within 14 days prior to the date of proposed microbubble administration.
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 |
|---|---|---|
| Perflutren lipid microsphereAlso: • Definity®, • Microbubble contrast agentBaseline B-mode US will be performed to confirm the lesions being imaged (unless it is a subject with no lesions, then a basic B-mode US will be performed. Perflutren is a diagnostic drug that is intended to be used for contrast enhancement. The vial contains a clear, colorless, sterile, non-pyrogenic, hypertonic solution which is activated by mechanical agitation with Vialmix®.It will be dispensed in inactivated form to a study team member. The contrast agent will be activated just prior to administration (ideally to be used within 5 minutes of activation). Contrast agent Definity (or Lumason) will be activated according to package insert instructions, including use of VialMix®, the device used to activate Definity. Administration will occur IV in coordination with sonography staff trained specifically in contrast ultrasound imaging.The total imaging time is anticipated to be less than 30 minutes. | Drug | Perflutren Lipid Microsphere or Lumason |
| Sulfur hexafluoride lipid microspheresAlso: LumasonBaseline B-mode US will be performed to confirm the lesions being imaged (unless it is a subject with no lesions, then a basic B-mode US will be performed. Lumason will be used as a secondary contrast agent only if Definity(perflutren) is unavailable.This drug will be administered using the dosing range and administration type within the Lumason prescribing information. The contrast agent will be activated just prior to administration (ideally to be used within 5 minutes of activation). Contrast agent Definity (or Lumason) will be activated according to package insert instructions, including use of VialMix®, the device used to activate Definity. Administration will occur IV in coordination with sonography staff trained specifically in contrast ultrasound imaging.The total imaging time is anticipated to be less than 30 minutes. | Drug | Perflutren Lipid Microsphere or Lumason |
Primary outcome
Comparison in Radiologist's Lesion Evaluation: CEUS Versus B-mode Ultrasound
Time frame: Baseline
Each reader will evaluate kidney imaging using Contrast-Enhanced Ultrasound (CEUS) versus Brightness Mode Ultrasound (B-mode) in patients at risk for kidney lesions. A dichotomized Bosniak score was used for comparison. Dichotomized Bosniak score: 0= nonmalignant and 1=malignant or possibly malignant. Scores were averaged across all readers. Kidneys were assessed by CEUS and B-mode by three different readers.
1 secondary outcome
Comparison in Radiologist's Lesion Evaluation: CEUS Versus Clinically Performed MRI
Time frame: Baseline
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34783716Larcher A, Rowe I, Belladelli F, Fallara G, Raggi D, Necchi A, Montorsi F, Capitanio U, Salonia A; OSR VHL Program. Von Hippel-Lindau disease-associated renal cell carcinoma: a call to action. Curr Opin Urol. 2022 Jan 1;32(1):31-39. doi: 10.1097/MOU.0000000000000950.
- Individual participant dataSharing planned: No
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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.
Emily Chang, MD principal investigator
University of North Carolina, Chapel Hill
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Perflutren lipid microsphere and Sulfur hexafluoride lipid microspheres in people with Von-Hippel Lindau and Kidney Disease, Chronic, run by UNC Lineberger Comprehensive Cancer Center at 1 site in United States.
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 5 inclusion and 11 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 21 May 2024. 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 United States. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Comparison in Radiologist's Lesion Evaluation: CEUS Versus B-mode Ultrasound (time frame: Baseline). 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 NCT03907657, 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 21 May 2024.