NCT05583721

Brief Summary

There is limited information on what causes injury to the heart in individuals with Sickle Cell Disease (SCD). Researchers in this study want to see if decreased blood flow to the heart during stress could be causing the heart damage seen in SCD patients. They also want to test people who don't have SCD to see if their hearts react the same way under stress. Primary Objective

  • To estimate the coronary flow reserve (CFR) (also referred to as myocardial perfusion reserve), as measured by PET stress-rest myocardial perfusion imaging, in SCD patients with and without diastolic dysfunction, and healthy controls. Secondary Objectives
  • To investigate the relationship between decreased CFR (quantified with PET stress- rest myocardial perfusion imaging) and presence of abnormal diastolic parameters

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at P25-P50 for phase_2

Timeline
28mo left

Started Oct 2023

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress53%
Oct 2023Sep 2028

First Submitted

Initial submission to the registry

October 13, 2022

Completed
5 days until next milestone

First Posted

Study publicly available on registry

October 18, 2022

Completed
12 months until next milestone

Study Start

First participant enrolled

October 10, 2023

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2027

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2028

Last Updated

May 6, 2026

Status Verified

May 1, 2026

Enrollment Period

3.9 years

First QC Date

October 13, 2022

Last Update Submit

May 1, 2026

Conditions

Keywords

Sickle Cell Disease patientsHealthy controlsAbnormal diastolic heart functionCoronary Flow Reserve (CFR)Coronary microvasculature dysfunctionMyocardial perfusion reservePositron emission tomography (PET) imagingPET (stress-rest) myocardial perfusion imaging

Outcome Measures

Primary Outcomes (2)

  • Mean and standard deviation of coronary flow reserve (CFR)

    We will estimate the mean and standard deviation of CFR, as measured by PET myocardial blood flow reserve imaging, in young adult SCD patients with and without diastolic dysfunction and healthy controls.

    Visit 2, approximately 4 weeks after study entry (procedure takes approximately one hour)

  • CFR difference among Stratum A, Stratum B, and Stratum C

    We will compare whether the CFR is different among 3 predefined strata: Stratum A; SCD with diastolic dysfunction patients , Stratum B; SCD without dysfunction patient control, and Stratum C; normal healthy controls by one-way ANOVA or Kruskal-Wallis one-way analysis of variance, depending on the distribution of the data. If the difference exists, the pairwise comparison will be carried out by two sample t test or Wilcoxon rank sum test between two strata.

    Visit 2, approximately 4 weeks after study entry (procedure takes approximately one hour)

Secondary Outcomes (1)

  • Relationship between decreased CFR (quantified with PET stress-rest myocardial perfusion imaging) and presence of abnormal diastolic parameters

    Visit 2, approximately 4 weeks after study entry (procedure takes approximately one hour)

Study Arms (3)

Stratum A

EXPERIMENTAL

Sickle Cell patients with diastolic dysfunction

Drug: [13N]NH3Drug: LexiscanDiagnostic Test: Positron emission tomography

Stratum B

EXPERIMENTAL

Sickle cell patients without diastolic dysfunction

Drug: [13N]NH3Drug: LexiscanDiagnostic Test: Positron emission tomography

Stratum C

EXPERIMENTAL

Healthy controls

Drug: [13N]NH3Drug: LexiscanDiagnostic Test: Positron emission tomography

Interventions

Ammonia N 13 (a radioactive tracer) will be given per intravenous (IV) injection for diagnostic purposes in conjunction with positron emission tomography (PET) imaging.

Also known as: [N-13] Ammonia, azane, CAS# 34819-78-8
Stratum AStratum BStratum C

Participants will complete a onetime PET (stress-rest) myocardial perfusion scan.

Also known as: PET
Stratum AStratum BStratum C

Given IV prior to PET stress test

Also known as: Regadenoson
Stratum AStratum BStratum C

Eligibility Criteria

Age18 Years - 21 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • to 21 years of age
  • Black
  • Diagnosis of SCD of HbSS and HbSβ0thalassemia genotypes
  • Three or more abnormal diastolic parameters (based on guidelines by American Society of echocardiography)
  • to 21 years of age
  • Black
  • Diagnosis of SCD of HbSS and HbSβ0thalassemia genotypes
  • Two or less abnormal diastolic parameters
  • to 21 years of age
  • Black
  • Two or less abnormal diastolic parameters

You may not qualify if:

  • Recent hospitalization for vaso-occlusive pain crisis or acute chest syndrome in last 4 weeks
  • Blood transfusion in the last 3 months
  • Individuals with signs, symptoms or EKG findings of acute myocardial ischemia, infarction or unstable angina
  • Individuals with history of VT/VF or SVT
  • Previous cardiac surgery
  • Known congenital heart disease (other than patent ductus arteriosus or Atrial septal defect)
  • Stenotic valvular disease or left main coronary artery stenosis
  • History of myo/pericarditis
  • Left ventricle systolic dysfunction
  • Cardiovascular instability/uncontrolled hypertension (h/o hypertensive urgency or emergency)
  • History of sinus node dysfunction or high grade AV nodal block
  • History of aborted sudden cardiac death or cardiac arrest
  • Current seizure disorder on AED
  • Pregnant/Breast-feeding
  • Any medical or social reason, which, in the opinion of the principal investigators would make the participation of the subject ill-advised.
  • +32 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Ammonia1,3-(N'-amidinosulfanilamide)-2,2,2,4,4,4-hexachlorocyclodiphosph(V)azaneregadenosonPositron-Emission Tomography

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

GasesInorganic ChemicalsNitrogen CompoundsTomography, Emission-ComputedImage Interpretation, Computer-AssistedDiagnostic ImagingDiagnostic Techniques and ProceduresDiagnosisImage EnhancementPhotographyRadionuclide ImagingTomographyDiagnostic Techniques, Radioisotope

Study Officials

  • Parul Rai, MD

    St. Jude Children's Research Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
DIAGNOSTIC
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 13, 2022

First Posted

October 18, 2022

Study Start

October 10, 2023

Primary Completion (Estimated)

September 1, 2027

Study Completion (Estimated)

September 1, 2028

Last Updated

May 6, 2026

Record last verified: 2026-05

Data Sharing

IPD Sharing
Will share

Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.

Shared Documents
STUDY PROTOCOL, SAP, ICF
Time Frame
Data will be made available at the time of article publication.
Access Criteria
Data will be provided to researchers following a formal request with the following information: full name of requestor, affiliation, data set requested, and timing of when data is needed. As an informational point, the lead statistician and study principal investigator will be informed that primary results datasets have been requested.

Locations