PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD

NCT04925492 | Recruiting Phase 1 DMC FDA Drug FDA Device

• 2 other identifiers: STUDY20020135R01HL154629
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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). 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.

Conditions

Sickle Cell Disease

Keywords

PET MRI

Outcome Measures

Primary Outcomes (3)

• 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

  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.

  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.

  Up to the length of a hospital visit for treatment of VOC. On average, about 5 days.

Study Arms (2)

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.

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.

Radiation: Positron Emission Tomography; Drug: Cu-64]-LLP2A

Interventions

Positron Emission Tomography RADIATION

An imaging method that uses radiotracers to view changes in the metabolic process.

Also known as: PET Scan

Finding Optimal Scan Timing; Scan at Determined Optimal Timepoint

Cu-64]-LLP2A DRUG

A radioactive tracer used in PET imaging.

Also known as: Cu-64]-LLP2A Radiotracer

Finding Optimal Scan Timing; Scan at Determined Optimal Timepoint

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Enrico Novelli: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (1)

UPMC

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Magnetic Resonance Spectroscopy

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Spectrum Analysis; Chemistry Techniques, Analytical; Investigative Techniques

Study Officials

• Enrico Novelli, MD, MS

  University of Pittsburgh

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jude Jonassaint, RN

CONTACT

919-219-7481; jonas@pitt.edu

Leticia Candra, BA

CONTACT

LEC117@pitt.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: SCREENING
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Model Details: Population of adult Sickle Cell patients.

Study Record Dates

• First Submitted: December 15, 2020
• First Posted: June 14, 2021
• Study Start: November 11, 2022
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: May 7, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)