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NCT02410811

Novel Cardiac Magnetic Resonance Imaging to Define a Unique Restrictive Cardiomyopathy in Sickle Cell Disease

Completed Results posted Last updated 2 November 2020
What this trial tests

trial testing Cardiac magnetic resonance imaging (CMR) in Sickle Cell Disease in 33 participants. Completed in 20 May 2019.

Timeline
31 January 2014
Primary endpoint
29 January 2018
20 May 2019

Quick facts

Lead sponsorChildren's Hospital Medical Center, Cincinnati
StatusCompleted
Study typeOBSERVATIONAL
Enrollment33
Start date31 January 2014
Primary completion29 January 2018
Estimated completion20 May 2019
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Children's Hospital Medical Center, Cincinnati

Who can join

Adults 6 to 65, any sex, with Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Frequency of the Diffuse Myocardial Fibrosis Phenotype Primary · Assessed annually over a 2-year period (3 assessments over 2 years)

The occurrence of an abnormally increased extracellular volume (ECV) measurement \[i.e., the presence of the diffuse myocardial fibrosis phenotype\] as assessed by cardiac magnetic resonance imaging (CMR) using T1 mapping before and after administration of gadolinium. Expressed as number of participants with the diffuse myocardial fibrosis phenotype in each stratum.

GroupValue95% CI
Stratum A (6-13.99 Years)5
Stratum B (14-20.99 Years)10
Stratum C (≥21 Years)10
Stratum D (Early Treatment)1
Stability of the Diffuse Myocardial Fibrosis Phenotype Over Time Secondary · Assessed annually over a 2-year period (3 assessments over 2 years)

The occurrence of a change \[from the baseline assessment\] in the classification \[presence or absence\] of the diffuse myocardial fibrosis phenotype, which is defined as an abnormally increased extracellular volume (ECV) measurement as assessed by cardiac magnetic resonance imaging (CMR) using T1 mapping before and after administration of gadolinium. Expressed as number of participants who had a change in classification of the diffuse myocardial fibrosis phenotype \[e.g., presence to absence, or absence to presence\] during the 2-year study in each stratum.

GroupValue95% CI
Stratum A (6-13.99 Years)0
Stratum B (14-20.99 Years)0
Stratum C (≥21 Years)0

Adverse events — posted to ClinicalTrials.gov

Time frame: AEs were collected for three, separate 30-day intervals over the entire study period of 2 years. Each 30-day interval was defined as starting with the study visit during which annual study activities were performed (e.g., cardiac magnetic resonance imaging [CMR]) and ending 30 days after that study visit. AEs were assessed as they occurred during each study visit, and AEs that occurred in the 30 days after each study visit were collected by scripted phone call from the study team.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Stratum A (6-13.99 Years)
Serious: 0/5 (0%)
Deaths: 0/5
Stratum B (14-20.99 Years)
Serious: 0/10 (0%)
Deaths: 0/10
Stratum C (≥21 Years)
Serious: 0/10 (0%)
Deaths: 0/10
Stratum D (Early Treatment)
Serious: 0/7 (0%)
Deaths: 0/7
Other adverse events (2 terms — click to expand)

ReactionSystemStratum A (6-13.99 Years)Stratum B (14-20.99 Years)Stratum C (≥21 Years)Stratum D (Early Treatment)
Vaso-occlusive pain episodeBlood and lymphatic system disorders
Nausea and vomitingGastrointestinal disorders

Data from ClinicalTrials.gov NCT02410811 adverse events section.

Sponsor's own description

The purpose of this study is to use cardiac magnetic resonance imaging (CMR) and echocardiographic tissue Doppler imaging to demonstrate a unique restrictive cardiomyopathy of sickle cell disease. The investigators will characterize its frequency and how it might change (e.g., presence/absence and severity) over a 2-year period.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Association between diffuse myocardial fibrosis and diastolic dysfunction in sickle cell anemia.
    Niss O, Fleck R, Makue F, Alsaied T, et al · · 2017 · cited 88× · PMID 28507082 · DOI 10.1182/blood-2017-02-767624
  2. Biochemical surrogate markers of hemolysis do not correlate with directly measured erythrocyte survival in sickle cell anemia.
    Quinn CT, Smith EP, Arbabi S, Khera PK, et al · · 2016 · cited 35× · PMID 27648808 · DOI 10.1002/ajh.24562
  3. Diastolic dysfunction is associated with exercise impairment in patients with sickle cell anemia.
    Alsaied T, Niss O, Powell AW, Fleck RJ, et al · · 2018 · cited 20× · PMID 29781568 · DOI 10.1002/pbc.27113
  4. Early initiation of disease-modifying therapy can impede or prevent diffuse myocardial fibrosis in sickle cell anemia.
    Niss O, Detterich J, Wood JC, Coates TD, et al · · 2022 · cited 14× · PMID 35857896 · DOI 10.1182/blood.2021015303
  5. Abnormal submaximal cardiopulmonary exercise parameters predict impaired peak exercise performance in sickle cell anemia patients.
    Powell AW, Alsaied T, Niss O, Fleck RJ, et al · · 2019 · cited 4× · PMID 30848046 · DOI 10.1002/pbc.27703
  6. Longitudinal changes and predictors of cardiac extracellular volume fraction in sickle cell anemia.
    Niss O, Morin CE, Hashemi S, Alsaied T, et al · · 2025 · cited 1× · PMID 41312339 · DOI 10.1016/j.brci.2025.100031

Verify or expand the search:

Other recruiting trials for Sickle Cell Disease

Currently open trials in the same condition.

Other Children's Hospital Medical Center, Cincinnati trials

Trials by the same sponsor.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02410811.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing