Sickle Cell DiseaseNCT00842621
Long Term Effects of Erythrocyte Lysis
An observational study of Sickle Cell Disease and Hemolytic Anemia, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
In this prospective observational trial, participants with chronic hemolysis will be assessed with echocardiogram for elevated tricuspid jet velocity and other evidence of pulmonary hypertension. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Sickle Cell Disease, Hemolytic Anemia
- What is being tested?
- Clinical Evaluations and Laboratory Studies
- Type of treatment
- Surgery or procedure
- How many people
- 390 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2009to Jun 2016
- Phase
- Observational
- 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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Children aged 5 years of age up to 19th birthday with sickle cell disease or other forms of hemolytic anemia; Adults over the age of 18 with non-sickling hemolytic anemia.
Who may be able to join
- Established Diagnosis of Hemolysis
- Sickle Cell Disease (e.g., HbSS, HbS/β-thalassemia, HbSC)
- Other conditions with hemolysis (e.g., RBC membranopathies, enzymopathies, unstable hemoglobinopathies, PNH)
- Age
- SCD participants: 5 years of age up to 19th birthday
- All other participants: 5 years of age and up (no age limit)
Who cannot join
- Previous cardiac surgery
- Known left ventricle dysfunction (i.e. shortening fraction < 28%)
- Known right sided congenital heart defect such as atrial septal defect or pulmonary valve stenosis
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38115
About this study
In this prospective observational trial, participants with chronic hemolysis will be assessed with echocardiogram for elevated tricuspid jet velocity and other evidence of pulmonary hypertension. Participants will have laboratory studies evaluating: severity of hemolysis, splenic function, inflammation, endothelial dysfunction, and hypercoagulability. There will be 3 main categories of participants enrolled in this study: (1) pediatric participants with severe sickle cell disease (SCD) (HbSS, HbS/β° thalassemia ) who are not receiving treatment (e.g., hydroxyurea or chronic transfusions); (2) pediatric participants with other forms of SCD or severe SCD (HbSS, HbS/β° thalassemia) patients being treated with hydroxyurea or chronic transfusions; and (3) pediatric and adult participants with other non-sickling hematological disorders.
Read the detailed description
- The study will investigate the relationship between tricuspid regurgitation jet velocity (TRV) and intravascular hemolysis, as measured by serum lactate dehydrogenase (LDH), in untreated children with severe sickle cell disease (HbSS or Hb S/β°-thalassemia)
- The Study will estimate the prevalence of elevated TRV (≥ 2.5 m/s) in untreated children with severe sickle cell disease (HbSS or Hb S/β°-thalassemia), as measured by echocardiography.
Secondary objectives for this study include the following:
- To estimate the prevalence of elevated TRV in children with severe sickle cell disease (HbSS or Hb S/β°-thalassemia) receiving hydroxyurea or chronic transfusion therapy.
- To estimate the prevalence of elevated TRV in children with other forms of hemolytic anemia, including other sickling disorders (such as HbSC or HbS/β+-thalassemia) and non-sickling hemolytic anemia (such as hereditary spherocytosis).
- To estimate the prevalence of elevated TRV in adults with non-sickling hemolytic anemia, with or without splenic function.
- To investigate the association between TRV and splenic function
- To investigate the associations between TRV and laboratory parameters of inflammation and hypercoagulability, such as white blood cell count, platelet count, serum N-terminal pro-brain natriuretic peptide (NT-proBNP),endothelial dysfunction, and other markers of hemolysis (bilirubin, plasma free hemoglobin, haptoglobin, etc.)
- To evaluate genetic determinants of elevated TRV in children and adults with hemolytic anemia.
- To investigate changes in TRV and hemolysis over time using serial measurements 2 ± 0.5 years after initial enrollment testing.
- Conditions
- Sickle Cell Disease, Hemolytic Anemia
- Being tested
- Clinical Evaluations, Laboratory Studies
- Keywords
- Sickle Cell Disease, Hemolytic Anemia, Hemolysis
- Medical subject headings
- Anemia, Sickle Cell, Anemia, Hemolytic, Hemolysis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clinical EvaluationsProcedure or surgery
All clinical evaluations should be performed greater than 30 days from illness or hospitalization. Participants will have weight, height, body mass index (BMI), heart rate, respiratory rate, blood pressure (systolic and diastolic) and pulse oximetry recorded while at rest on room air by clinical staff. Cardiac examination will be performed by echocardiogram.
Laboratory StudiesOther
Blood for:
Complete Blood Count with Differential, Reticulocyte Count, Micronuclei enumeration, α-globin genotype, G6PD activity, genotype, SNP array,
Serum for:
Soluble VCAM-1, ICAM-1, Soluble CD40L, Arginase, Endothelin-1, Prothrombin Fragment 1+2, IL-6, -8, -10, Soluble E-selectin, NOx, Haptoglobin, Tumor necrosis factor alpha
Plasma for:
Chemistry 18, Cystatin-C, Ferritin, Lactate Dehydrogenase, C-reactive protein, Erythropoietin, NT-proBNP, Haptoglobin, Von Willibrand Factor Antigen, D-dimer, Factor VIII Assay, Quantitative amino acids, Free hemoglobin, Tumor necrosis factor- α, Thrombin-Antithrombin complex, endothelin-1
Urine for:
Urinalysis, Urine spot protein, creatinine, Microalbumin, Urine hemosiderin, Urine hepcidin
| Study group | Role | What they receive |
|---|---|---|
| 1 | Pediatric participants with severe sickle cell disease (HbSS or Hb S/β°-thalassemia) who are not receiving treatment, e.g., hydroxyurea or chronic transfusions Receives: Procedure: Clinical Evaluations; Other: Laboratory Studies | |
| 2 | Pediatric participants with other forms of SCD or severe sickle cell disease patients (HbSS or Hb S/β°-thalassemia) being treated with hydroxyurea or chronic transfusions Receives: Procedure: Clinical Evaluations; Other: Laboratory Studies | |
| 3 | Pediatric and adult participants with other non-sickling hematological disorders Receives: Procedure: Clinical Evaluations; Other: Laboratory Studies |
What is being measured
The primary outcome is the main question the study is designed to answer.
1.To investigate the relationship between tricuspid regurgitation jet velocity (TRV) and intravascular hemolysis, as measured by serum lactate dehydrogenase (LDH), in untreated children with severe sickle cell disease(HbSS or Hb S/β°-thalassemia.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2009 (estimated)
- Study startMar 2009
- Primary completionJun 2016
- Study completionJun 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 390 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jane Hankins, MD, MS, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00842621
- Sponsor's protocol ID
- ELYSIS
- Status last verified
- Jun 2016
- First submitted
- 11 Feb 2009
- Last updated
- 27 Jun 2016
- Results posted
- No
- Data monitoring committee
- No
Publications
Rai P, Okhomina VI, Kang G, Martinez HR, Hankins JS, Joshi V. Longitudinal effect of disease-modifying therapy on left ventricular diastolic function in children with sickle cell anemia. Am J Hematol. 2023 Jun;98(6):838-847. doi: 10.1002/ajh.26911. Epub 2023 Mar 20.
PubMed 36890729Whipple NS, Naik RJ, Kang G, Moen J, Govindaswamy SD, Fowler JA, Dowdy J, Penkert R, Joshi VM, Hankins JS. Ventricular global longitudinal strain is altered in children with sickle cell disease. Br J Haematol. 2018 Dec;183(5):796-806. doi: 10.1111/bjh.15607. Epub 2018 Nov 19.
PubMed 30450553
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Effects of Erythrocyte Lysis
- ClinicalTrials.gov ID
- NCT00842621
- Acronym / sponsor ID
- ELYSIS
- Conditions
- Sickle Cell Disease, Hemolytic Anemia
- Keywords
- Sickle Cell Disease, Hemolytic Anemia, Hemolysis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 390 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- Mar 2009
- Primary completion
- Jun 2016 (actual)
- Study completion
- Jun 2016 (actual)
- First posted
- 12 Feb 2009 (estimated)
- 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.
In this prospective observational trial, participants with chronic hemolysis will be assessed with echocardiogram for elevated tricuspid jet velocity and other evidence of pulmonary hypertension. Participants will have laboratory studies evaluating: severity of hemolysis, splenic function, inflammation, endothelial dysfunction, and hypercoagulability. There will be 3 main categories of participants enrolled in this study: (1) pediatric participants with severe sickle cell disease (SCD) (HbSS, HbS/β° thalassemia ) who are not receiving treatment (e.g., hydroxyurea or chronic transfusions); (2) pediatric participants with other forms of SCD or severe SCD (HbSS, HbS/β° thalassemia) patients being treated with hydroxyurea or chronic transfusions; and (3) pediatric and adult participants with other non-sickling hematological disorders.
Detailed description
- The study will investigate the relationship between tricuspid regurgitation jet velocity (TRV) and intravascular hemolysis, as measured by serum lactate dehydrogenase (LDH), in untreated children with severe sickle cell disease (HbSS or Hb S/β°-thalassemia)
- The Study will estimate the prevalence of elevated TRV (≥ 2.5 m/s) in untreated children with severe sickle cell disease (HbSS or Hb S/β°-thalassemia), as measured by echocardiography.
Secondary objectives for this study include the following:
- To estimate the prevalence of elevated TRV in children with severe sickle cell disease (HbSS or Hb S/β°-thalassemia) receiving hydroxyurea or chronic transfusion therapy.
- To estimate the prevalence of elevated TRV in children with other forms of hemolytic anemia, including other sickling disorders (such as HbSC or HbS/β+-thalassemia) and non-sickling hemolytic anemia (such as hereditary spherocytosis).
- To estimate the prevalence of elevated TRV in adults with non-sickling hemolytic anemia, with or without splenic function.
- To investigate the association between TRV and splenic function
- To investigate the associations between TRV and laboratory parameters of inflammation and hypercoagulability, such as white blood cell count, platelet count, serum N-terminal pro-brain natriuretic peptide (NT-proBNP),endothelial dysfunction, and other markers of hemolysis (bilirubin, plasma free hemoglobin, haptoglobin, etc.)
- To evaluate genetic determinants of elevated TRV in children and adults with hemolytic anemia.
- To investigate changes in TRV and hemolysis over time using serial measurements 2 ± 0.5 years after initial enrollment testing.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 1 | – | Procedure: Clinical Evaluations, Other: Laboratory StudiesPediatric participants with severe sickle cell disease (HbSS or Hb S/β°-thalassemia) who are not receiving treatment, e.g., hydroxyurea or chronic transfusions |
| 2 | – | Procedure: Clinical Evaluations, Other: Laboratory StudiesPediatric participants with other forms of SCD or severe sickle cell disease patients (HbSS or Hb S/β°-thalassemia) being treated with hydroxyurea or chronic transfusions |
| 3 | – | Procedure: Clinical Evaluations, Other: Laboratory StudiesPediatric and adult participants with other non-sickling hematological disorders |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Children aged 5 years of age up to 19th birthday with sickle cell disease or other forms of hemolytic anemia; Adults over the age of 18 with non-sickling hemolytic anemia.
Key inclusion criteria (6 listed)
- Established Diagnosis of Hemolysis
- Age
Key exclusion criteria (3 listed)
- Previous cardiac surgery
- Known left ventricle dysfunction (i.e. shortening fraction < 28%)
- Known right sided congenital heart defect such as atrial septal defect or pulmonary valve stenosis
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 |
|---|---|---|
| Clinical EvaluationsAll clinical evaluations should be performed greater than 30 days from illness or hospitalization. Participants will have weight, height, body mass index (BMI), heart rate, respiratory rate, blood pressure (systolic and diastolic) and pulse oximetry recorded while at rest on room air by clinical staff. Cardiac examination will be performed by echocardiogram. | Procedure or surgery | 1, 2, 3 |
| Laboratory StudiesBlood for: Complete Blood Count with Differential, Reticulocyte Count, Micronuclei enumeration, α-globin genotype, G6PD activity, genotype, SNP array, Serum for: Soluble VCAM-1, ICAM-1, Soluble CD40L, Arginase, Endothelin-1, Prothrombin Fragment 1+2, IL-6, -8, -10, Soluble E-selectin, NOx, Haptoglobin, Tumor necrosis factor alpha Plasma for: Chemistry 18, Cystatin-C, Ferritin, Lactate Dehydrogenase, C-reactive protein, Erythropoietin, NT-proBNP, Haptoglobin, Von Willibrand Factor Antigen, D-dimer, Factor VIII Assay, Quantitative amino acids, Free hemoglobin, Tumor necrosis factor- α, Thrombin-Antithrombin complex, endothelin-1 Urine for: Urinalysis, Urine spot protein, creatinine, Microalbumin, Urine hemosiderin, Urine hepcidin | Other | 1, 2, 3 |
Primary outcome
1.To investigate the relationship between tricuspid regurgitation jet velocity (TRV) and intravascular hemolysis, as measured by serum lactate dehydrogenase (LDH), in untreated children with severe sickle cell disease(HbSS or Hb S/β°-thalassemia.
Time frame: 2 years
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 27 Jun 2016.
- Derived from this study: PubMed 36890729Rai P, Okhomina VI, Kang G, Martinez HR, Hankins JS, Joshi V. Longitudinal effect of disease-modifying therapy on left ventricular diastolic function in children with sickle cell anemia. Am J Hematol. 2023 Jun;98(6):838-847. doi: 10.1002/ajh.26911. Epub 2023 Mar 20.
- Derived from this study: PubMed 30450553Whipple NS, Naik RJ, Kang G, Moen J, Govindaswamy SD, Fowler JA, Dowdy J, Penkert R, Joshi VM, Hankins JS. Ventricular global longitudinal strain is altered in children with sickle cell disease. Br J Haematol. 2018 Dec;183(5):796-806. doi: 10.1111/bjh.15607. Epub 2018 Nov 19.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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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.
Jane Hankins, MD, MS principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease and Hemolytic Anemia, run by St. Jude Children's Research Hospital 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 6 inclusion and 3 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 27 Jun 2016. 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.
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?
1.To investigate the relationship between tricuspid regurgitation jet velocity (TRV) and intravascular hemolysis, as measured by serum lactate dehydrogenase (LDH), in untreated children with severe sickle cell disease(HbSS or Hb S/β°-thalassemia. (time frame: 2 years). 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 NCT00842621, which also lists 2 linked publications.
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 27 Jun 2016.