Sickle Cell DiseaseNCT03958643
Best Noninvasive Predictor of Renal Function in Assessing Adult Sickle Nephropathy
An observational study of Sickle Cell Disease, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Background: Sickle cell disease is a common inherited blood disorder. Kidney disease is a major cause of problems in people with sickle cell disease. In order to identify kidney problems early and stop the progression of kidney disease, doctors need the most accurate tests to check kidney function. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 70 participants
- Where
- 1 siteUnited States
- Study dates
- 24 May 2019to 8 Feb 2022
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study will be open to all eligible subjects based on inclusion and exclusion criteria and who provide informed consent. No patient will be excluded from participation based on gender, race, or ethnicity. Patients may self-refer, be recruited through the NIH office of recruitment, and may include patients participating on NIH Clinical Center Protocols, and NIH employees.
Who may be able to join
- Known diagnosis of Sickle Cell Anemia (Hb SS or HbS-beta0-thal) >=18 years of age
- Willingness and capacity to provide written informed consent
Who cannot join
- Pregnancy
- Uncontrolled/poorly controlled hypertension
- Diabetes
- Dialysis
- GFR <30 ml/min/1.73m2
- HIV positive
Show all 16Show fewer
- HepatitisC
- Hepatitis B
- Prior transplantation
- Uncontrolled infection or acute illness
- Chronic inflammatory disease (e.g. lupus, multiple sclerosis, rheumatoid arthritis)
- Allergy to iodine or iodinated contrast solutions
- Hydroxyurea initiation or dose adjustment <2mo prior
- Initiation of chronic transfusion therapy <2mo prior
- Antihypertensive medication initiation or dose adjustment <1mo prior
- Pain crisis in preceding 4weeks
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Background:
Sickle cell disease is a common inherited blood disorder. Kidney disease is a major cause of problems in people with sickle cell disease. In order to identify kidney problems early and stop the progression of kidney disease, doctors need the most accurate tests to check kidney function. Researchers hope to understand more about how to test for kidney disease in people with sickle cell disease.
Read more
Objective:
To determine which of two different lab tests is the best to measure kidney function in adults with sickle cell disease.
Eligibility:
People 18 years and older who have sickle cell disease
Design:
Participants will be screened with a medical history and blood tests.
Participants will have up to 3 visits.
Participants will collect their urine in a special container over 24 hours.
At the first visit, participants will have blood tests. They will bring their container of urine to the visit. They will have an iothalamate test. For the test, they will get a catheter: a small tube will be inserted into a vein. A special contract agent will be injected into the vein. Blood will be collected over the next 4 hours to test kidney function.
Participants will return the next day for a second visit. They will have blood tests. They will have an MRI. For the MRI, they will like on a table that slides into a machine that takes pictures of the kidneys. They may have the MRI in a third visit.
...
Read the detailed description
The characteristic sickling of red blood cells in hypoxic conditions is the root cause of pathology in sickle cell disease (SCD). When this sickling occurs in the renal microvasculature, and is compounded by chronic vasculopathy related to hemolysis, the result is local infarction, ischemic injury, and interstitial fibrosis. The kidney damage begins in early childhood and is cumulative over time, resulting in sickle cell nephropathy (SCN). Creatinine clearance remains the most commonly used method to evaluate renal function in SCD patients although serum creatinine generally over-estimates the GFR in SCD. Cystatin-C (Cys-C) is freely filtered. Unlike creatinine, it is not secreted by the tubules. Its serum levels correlate with GFR in adults with various kidney diseases as well as in pediatric and adult SCD populations as compared with creatinine-based assessments.
This study seeks to evaluate whether Cys-C is a better noninvasive measure of renal function in the adult sickle cell population than creatinine. Further, this work will elucidate the ability of other markers, including beta 2-microglobulin (beta 2M) and endothelin-1 (ET-1), to predict sickle nephropathy. Finally, renal imaging by MRI will be performed and correlated with measured GFR and renal function markers. The results of this study could help alter clinical practice and thereby ensure the most accurate non-invasive assessment of kidney function by substantiating the role of Cys-C, beta 2M and ET-1 in adults with SCD. Finally, the descriptive analysis including measured GFR with renal MRI, novel biomarkers, markers of hemolysis, and analysis of urinary protein secretion will contribute to a better understanding of the pathophysiology of SCN.
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Nephropathy, Iothalamate, Cystatin-C, Natural History
- 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.
| Study group | Role | What they receive |
|---|---|---|
| 1 | In a population of adult patients with SCD we will comprehensively evaluate renal function. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine whether serum cystatin C or serum creatinine-based GFR methods better estimate renal function in the adult sickle cell population
In a population of patients with sickle cell anemia (including HbSS, HbS-0 thalassemia), who are age 18 and above, we will comprehensively evaluate renal function with the following primary objective:-determine whether serum cystatin C or serum creatinine- based GFR methods better estimate renal function in the adult sickle cell population
Measured: 2 years
Secondary outcomes (1)
Determine whether endothelin-1 or beta-2 microglobulin correlates with measured GFR (mGFR)
Determine whether endothelin-1 or beta-2 microglobulin correlates with measured GFR (mGFR)-establish potential correlation between mGFR, endothelin-1, or beta-2 microglobulin and renal blood flow-characterize the proteinuria associated with sickle cell disease-characterize kidney anatomy in patients with sickle cell disease-ascertain if markers of hemolysis are associated with mGFR or renal iron deposition-quantify renal iron burden in sickle cell disease
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 posted22 May 2019
- Study start24 May 2019
- Primary completion1 May 2021
- Study completion8 Feb 2022
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.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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
- 70 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Courtney F Joseph, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03958643
- Sponsor's protocol ID
- 190100
- Other IDs
- 19-H-0100
- Status last verified
- 10 Jul 2026
- First submitted
- 21 May 2019
- Last updated
- 14 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Best Noninvasive Predictor of Renal Function in Assessing Adult Sickle Nephropathy
- ClinicalTrials.gov ID
- NCT03958643
- Acronym / sponsor ID
- 190100
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Nephropathy, Iothalamate, Cystatin-C, Natural History
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 24 May 2019 (actual)
- Primary completion
- 1 May 2021 (actual)
- Study completion
- 8 Feb 2022 (actual)
- First posted
- 22 May 2019 (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.
Background:
Sickle cell disease is a common inherited blood disorder. Kidney disease is a major cause of problems in people with sickle cell disease. In order to identify kidney problems early and stop the progression of kidney disease, doctors need the most accurate tests to check kidney function. Researchers hope to understand more about how to test for kidney disease in people with sickle cell disease.
Objective:
To determine which of two different lab tests is the best to measure kidney function in adults with sickle cell disease.
Eligibility:
People 18 years and older who have sickle cell disease
Design:
Participants will be screened with a medical history and blood tests.
Participants will have up to 3 visits.
Participants will collect their urine in a special container over 24 hours.
At the first visit, participants will have blood tests. They will bring their container of urine to the visit. They will have an iothalamate test. For the test, they will get a catheter: a small tube will be inserted into a vein. A special contract agent will be injected into the vein. Blood will be collected over the next 4 hours to test kidney function.
Participants will return the next day for a second visit. They will have blood tests. They will have an MRI. For the MRI, they will like on a table that slides into a machine that takes pictures of the kidneys. They may have the MRI in a third visit.
...
Detailed description
The characteristic sickling of red blood cells in hypoxic conditions is the root cause of pathology in sickle cell disease (SCD). When this sickling occurs in the renal microvasculature, and is compounded by chronic vasculopathy related to hemolysis, the result is local infarction, ischemic injury, and interstitial fibrosis. The kidney damage begins in early childhood and is cumulative over time, resulting in sickle cell nephropathy (SCN). Creatinine clearance remains the most commonly used method to evaluate renal function in SCD patients although serum creatinine generally over-estimates the GFR in SCD. Cystatin-C (Cys-C) is freely filtered. Unlike creatinine, it is not secreted by the tubules. Its serum levels correlate with GFR in adults with various kidney diseases as well as in pediatric and adult SCD populations as compared with creatinine-based assessments.
This study seeks to evaluate whether Cys-C is a better noninvasive measure of renal function in the adult sickle cell population than creatinine. Further, this work will elucidate the ability of other markers, including beta 2-microglobulin (beta 2M) and endothelin-1 (ET-1), to predict sickle nephropathy. Finally, renal imaging by MRI will be performed and correlated with measured GFR and renal function markers. The results of this study could help alter clinical practice and thereby ensure the most accurate non-invasive assessment of kidney function by substantiating the role of Cys-C, beta 2M and ET-1 in adults with SCD. Finally, the descriptive analysis including measured GFR with renal MRI, novel biomarkers, markers of hemolysis, and analysis of urinary protein secretion will contribute to a better understanding of the pathophysiology of SCN.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | –In a population of adult patients with SCD we will comprehensively evaluate renal function. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study will be open to all eligible subjects based on inclusion and exclusion criteria and who provide informed consent. No patient will be excluded from participation based on gender, race, or ethnicity. Patients may self-refer, be recruited through the NIH office of recruitment, and may include patients participating on NIH Clinical Center Protocols, and NIH employees.
Key inclusion criteria (2 listed)
- Known diagnosis of Sickle Cell Anemia (Hb SS or HbS-beta0-thal) >=18 years of age
- Willingness and capacity to provide written informed consent
Key exclusion criteria (16 listed)
- Pregnancy
- Uncontrolled/poorly controlled hypertension
- Diabetes
- Dialysis
- GFR <30 ml/min/1.73m2
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Determine whether serum cystatin C or serum creatinine-based GFR methods better estimate renal function in the adult sickle cell population
Time frame: 2 years
In a population of patients with sickle cell anemia (including HbSS, HbS-0 thalassemia), who are age 18 and above, we will comprehensively evaluate renal function with the following primary objective:-determine whether serum cystatin C or serum creatinine- based GFR methods better estimate renal function in the adult sickle cell population
1 secondary outcome
Determine whether endothelin-1 or beta-2 microglobulin correlates with measured GFR (mGFR)
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 14 Jul 2026.
- NIH Clinical Center Detailed Web PageLink 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.
Courtney F Joseph, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
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, run by National Heart, Lung, and Blood Institute (NHLBI) 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 2 inclusion and 16 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 14 Jul 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.
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?
Determine whether serum cystatin C or serum creatinine-based GFR methods better estimate renal function in the adult sickle cell population (time frame: 2 years). 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 14 Jul 2026.