Sickle Cell DiseasePhase 1NCT04925492
PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD
A Phase 1 study testing Positron Emission Tomography and Cu-64]-LLP2A in people with Sickle Cell Disease, run by Enrico Novelli at 1 site in United States.
Trial summary
The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Positron Emission Tomography and Cu-64]-LLP2A
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Nov 2022to 31 Mar 2026
- Phase
- Phase 1
- 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have a confirmed diagnosis of SCD (HbSS, SC, S/β-thalassemia, SD, SE, SO) by hemoglobin electrophoresis/High Performance Liquid Chromatography (HPLC)
- Aged 18 and above
- Ability to understand and provide informed consent.
- If receiving hydroxyurea or L-glutamine, crizanlizumab, voxelotor or erythropoietin stimulating agents, must have been receiving the drug for at least 12 weeks prior to screening and plan to continue taking the drug at the same dose and schedule during the study
- Experienced at least 2 VOCs leading to healthcare visit within the 12 months prior to screening visit as determined by medical history.
Who cannot join
- Active malignancy
- Current pregnancy or breast feeding
- Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted
- Received active treatment on another investigational trial within 30 days (or 5 half-lives of that agent, whichever is greater) prior to screening visit or plans to participate in another investigational drug trial
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Positron Emission Tomography (PET) Imaging of Vaso-occlusive Crisis(VOC) in Sickle Cell Disease (SCD).
The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET).
The ability to see and measure VOC in SCD patients can help patient care, by showing when and how a VOC is occurring or going to occur. Studying this method will also help in future drug research, as it will allow researchers to deliver promising new medications that target hyper-adhesion and sickling in people with SCD.
Read the detailed description
The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with Sickle Cell Disease(SCD). Using information from earlier studies, and work being done at Washington University, a strategy to image vaso-occulusive crisis (VOC) has been developed, using positron emission tomography (PET) for anatomical localization only. 64Cu-LLP2A is the radio tracer used for the study.
Aim: To develop quantitative PET imaging of VOC in patients with SCD. The researchers hypothesize that the radio tracer 64Cu-LLP2A uptake increases proportionally to the intensity of pain in patients with VOC, compared to baseline values. This increase in uptake will be assessed focally in areas of pain as well as globally to reflect heightened systemic inflammation.
Primary and secondary study endpoint: The overarching hypothesis of this study is that PET tracer uptake of intensity of 64Cu-LLP2A is a real time, quantitative measure of hyper adhesion in VOC.
- Conditions
- Sickle Cell Disease
- Being tested
- Positron Emission Tomography, Cu-64]-LLP2A
- Keywords
- PET MRI
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Positron Emission TomographyRadiation
Also called PET Scan
An imaging method that uses radiotracers to view changes in the metabolic process.
Cu-64]-LLP2ADrug
Also called Cu-64]-LLP2A Radiotracer
A radioactive tracer used in PET imaging.
| Study group | Role | What they receive |
|---|---|---|
| Finding Optimal Scan Timing | Experimental | Group A will receive two PET scans after the radiotracer injection to learn the best timing of the scan for the rest of the people in the study during participant's baseline state. Participants will receive another injection of the radiotracer during a sickle cell crisis and have one PET scan. Receive an optional injection and perform another PET scan 12 months after your sickle cell crisis if there were technical problems the previous scans. Receives: Radiation: Positron Emission Tomography; Drug: Cu-64]-LLP2A |
| Scan at Determined Optimal Timepoint | Experimental | Group B participants will receive an injection of the radiotracer and undergo only one PET scan during a baseline state. Participants will receive another injection of the radiotracer during a sickle cell crisis and have one PET scan. Receive an optional injection and perform another PET scan 12 months after your sickle cell crisis if there were technical problems the previous scans. Receives: Radiation: Positron Emission Tomography; Drug: Cu-64]-LLP2A |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in PET tracer uptake in VOC
Intensity of PET tracer uptake in VOC will be measured and compared to uptake at baseline in predefined regions of interest and over the whole body
Measured: Up to five years from first assessment depending on when VOC occurs.
Association of PET tracer uptake with intensity of pain in VOC
Intensity of PET tracer uptake will be compared to intensity of pain by Visual Analog Score (scored from 0-10, with 0 meaning no pain, and 10 meaning the most pain) and pain characteristic assessed by the Painimation assessment tool in specific anatomical areas in the patients during a sickle cell vaso-occlusive event.
Measured: 2 hours during an assessment while in VOC.
Association of PET tracer uptake with clinical VOC markers
Measure of PET tracer uptake will be compared with clinical markers of vaso-occlusive events including length of stay and hematologic markers of hemolysis.
Measured: Up to the length of a hospital visit for treatment of VOC. On average, about 5 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2021
- Study start11 Nov 2022
- Primary completion11 Mar 2026
- Study completion31 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies 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 1
- Primary purpose
- Screening
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Population of adult Sickle Cell patients.
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Enrico Novelli
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor-investigator, Enrico Novelli, University of Pittsburgh
- Investigators
- Enrico Novelli, MD, MS, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04925492
- Sponsor's protocol ID
- STUDY20020135
- Other IDs
- R01HL154629
- Status last verified
- Aug 2026
- First submitted
- 15 Dec 2020
- Last updated
- 18 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No: Information and stored samples may be shared without identifiers with other researchers in the future. These researchers will not receive identifiable information linking data or sample back to individual participants. This access may be granted for the advancement of scientific knowledge. Any future sharing will be conducted under an approved sharing agreement
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Positron Emission Tomography (PET) Imaging of Vaso-occlusive Crisis(VOC) in Sickle Cell Disease (SCD).
- ClinicalTrials.gov ID
- NCT04925492
- Acronym / sponsor ID
- STUDY20020135
- Conditions
- Sickle Cell Disease
- Keywords
- PET MRI
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Enrico NovelliUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 11 Nov 2022 (actual)
- Primary completion
- 11 Mar 2026 (actual)
- Study completion
- 31 Mar 2026 (actual)
- First posted
- 14 Jun 2021 (actual)
- 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 find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET).
The ability to see and measure VOC in SCD patients can help patient care, by showing when and how a VOC is occurring or going to occur. Studying this method will also help in future drug research, as it will allow researchers to deliver promising new medications that target hyper-adhesion and sickling in people with SCD.
Detailed description
The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with Sickle Cell Disease(SCD). Using information from earlier studies, and work being done at Washington University, a strategy to image vaso-occulusive crisis (VOC) has been developed, using positron emission tomography (PET) for anatomical localization only. 64Cu-LLP2A is the radio tracer used for the study.
Aim: To develop quantitative PET imaging of VOC in patients with SCD. The researchers hypothesize that the radio tracer 64Cu-LLP2A uptake increases proportionally to the intensity of pain in patients with VOC, compared to baseline values. This increase in uptake will be assessed focally in areas of pain as well as globally to reflect heightened systemic inflammation.
Primary and secondary study endpoint: The overarching hypothesis of this study is that PET tracer uptake of intensity of 64Cu-LLP2A is a real time, quantitative measure of hyper adhesion in VOC.
Study design
- Primary purpose
- Screening
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Population of adult Sickle Cell patients.
| Arm | Type | Receives |
|---|---|---|
| Finding Optimal Scan Timing | Experimental | Radiation: Positron Emission Tomography, Drug: Cu-64]-LLP2AGroup A will receive two PET scans after the radiotracer injection to learn the best timing of the scan for the rest of the people in the study during participant's baseline state. Participants will receive another injection of the radiotracer during a sickle cell crisis and have one PET scan. Receive an optional injection and perform another PET scan 12 months after your sickle cell crisis if there were technical problems the previous scans. |
| Scan at Determined Optimal Timepoint | Experimental | Radiation: Positron Emission Tomography, Drug: Cu-64]-LLP2AGroup B participants will receive an injection of the radiotracer and undergo only one PET scan during a baseline state. Participants will receive another injection of the radiotracer during a sickle cell crisis and have one PET scan. Receive an optional injection and perform another PET scan 12 months after your sickle cell crisis if there were technical problems the previous scans. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Have a confirmed diagnosis of SCD (HbSS, SC, S/β-thalassemia, SD, SE, SO) by hemoglobin electrophoresis/High Performance Liquid Chromatography (HPLC)
- Aged 18 and above
- Ability to understand and provide informed consent.
- If receiving hydroxyurea or L-glutamine, crizanlizumab, voxelotor or erythropoietin stimulating agents, must have been receiving the drug for at least 12 weeks prior to screening and plan to continue taking the drug at the same dose and schedule during the study
- Experienced at least 2 VOCs leading to healthcare visit within the 12 months prior to screening visit as determined by medical history.
Key exclusion criteria (4 listed)
- Active malignancy
- Current pregnancy or breast feeding
- Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted
- Received active treatment on another investigational trial within 30 days (or 5 half-lives of that agent, whichever is greater) prior to screening visit or plans to participate in another investigational drug trial
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 |
|---|---|---|
| Positron Emission TomographyAlso: PET ScanAn imaging method that uses radiotracers to view changes in the metabolic process. | Radiation | Finding Optimal Scan Timing, Scan at Determined Optimal Timepoint |
| Cu-64]-LLP2AAlso: Cu-64]-LLP2A RadiotracerA radioactive tracer used in PET imaging. | Drug | Finding Optimal Scan Timing, Scan at Determined Optimal Timepoint |
Primary outcome
Change in PET tracer uptake in VOC
Time frame: Up to five years from first assessment depending on when VOC occurs.
Intensity of PET tracer uptake in VOC will be measured and compared to uptake at baseline in predefined regions of interest and over the whole body
Association of PET tracer uptake with intensity of pain in VOC
Time frame: 2 hours during an assessment while in VOC.
Intensity of PET tracer uptake will be compared to intensity of pain by Visual Analog Score (scored from 0-10, with 0 meaning no pain, and 10 meaning the most pain) and pain characteristic assessed by the Painimation assessment tool in specific anatomical areas in the patients during a sickle cell vaso-occlusive event.
Association of PET tracer uptake with clinical VOC markers
Time frame: Up to the length of a hospital visit for treatment of VOC. On average, about 5 days.
Measure of PET tracer uptake will be compared with clinical markers of vaso-occlusive events including length of stay and hematologic markers of hemolysis.
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 18 Aug 2026.
- Individual participant dataSharing planned: No. Information and stored samples may be shared without identifiers with other researchers in the future. These researchers will not receive identifiable information linking data or sample back to individual participants. This access may be granted for the advancement of scientific knowledge. Any future sharing will be conducted under an approved sharing agreement
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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.
Enrico Novelli, MD, MS principal investigator
University of Pittsburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Positron Emission Tomography and Cu-64]-LLP2A in people with Sickle Cell Disease, run by Enrico Novelli 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 4 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 18 Aug 2026. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Change in PET tracer uptake in VOC (time frame: Up to five years from first assessment depending on when VOC occurs.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 Aug 2026.