Diabetic RetinopathyLXR and DRNCT03403686
Liver X Receptor (LXR) as a Novel Therapeutic Target in Diabetic Retinopathy (DR)
An observational study of Diabetic Retinopathy, run by University of Alabama at Birmingham at 1 site in United States.
Trial summary
Results from large clinical trials demonstrate a strong association between lipid abnormalities and progression of the most common microvascular complication, diabetic retinopathy (DR). Read more about this study
- Who is it for?
- Ages 21 to 98 yearsHealthy volunteers can take part
- Conditions the study is for
- Diabetic Retinopathy
- What is being tested?
- blood draw
- How many people
- 104 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Jan 2018to 31 Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 21 to 98 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Patients who have retinal abnormalities other than diabetic retinopathy will be excluded. Patients who have systemic conditions that influence hematopoietic stem cell function such as cardiovascular disease, malignant disease, diabetes, hematologic disorder, or estimated glomerular filtration rate less than 60 mL/min or who have undergone treatment with erythropoietin will be excluded. We will record all medications including antihypertensive drug treatment, treatment with statins, Angiotensin-Converting Enzyme (ACE) Inhibitors, Angiotensin Receptor Blockers (ARB) or other pharmacological agents that may influence CD34+ cell function. Baseline characteristics will be recorded, including age, lipid parameters, body mass index (BMI), blood pressure, smoking history, antioxidant intake and use of nutritional supplements.
Who may be able to join
- Any man or woman between the ages of 21- 98 years of age will be eligible to participate. To participate in the study as a study subject we will require: a) the subject must either carry the diagnosis of diabetes or be a healthy aged control and b) the patient be willing and have the ability to cooperate with the protocol.
Who cannot join
- Exclusion criteria: We will apply the following exclusion criteria: a) evidence of ongoing acute or chronic infection (HIV, Hepatitis B or C, tuberculosis); b) ongoing malignancy; c) cerebral vascular accident or cerebral vascular procedure; d) current pregnancy; e) history of organ transplantation; f) presence of a graft (to avoid any effect of the graft on inflammatory parameters; g) uremic symptoms, an estimated glomerular filtration rate of less than 20 cc/min (by Modification of Diet in Renal Disease equation), or an albumin of less than 3.6 (to avoid malnutrition as a confounding variable); h) be unwilling to abstain from drinking alcohol and i) patients with anemia. Subjects with AMD, glaucoma, uveitis, known hereditary degenerations or other significant ocular complications other than diabetic retinopathy will be excluded.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Maria B Grant, MD, 2053258674, jmoorer@uabmc.edu
Maria B Grant, MD
About this study
Official title: LXR as a Novel Therapeutic Target in DR
Results from large clinical trials demonstrate a strong association between lipid abnormalities and progression of the most common microvascular complication, diabetic retinopathy (DR). We found that activation of a master regulator of cholesterol metabolism, the nuclear hormone receptors liver X receptors (LXRα/LXRβ), prevents DR in rodent models. In this application, we seek to understand the mechanisms responsible for the beneficial effects of LXR agonists on retina and on bone marrow (BM) to preserve the function of reparative cells while reducing inflammatory cell.
Read the detailed description
Diabetic retinopathy (DR) is a disabling microvascular complication. Despite recent advances using pharmacotherapy, a cure for DR has yet to be realized. Thus, a conceptual and technical breakthrough to identify novel targets, and a strategy to cure this complication is paramount. We believe that the recent clinical evidence from large clinical trials demonstrating a strong association between lipid abnormalities and DR progression and the discovery that activation of the nuclear hormone receptors liver X receptors (LXRα/LXRβ) prevents DR in rodent models offers such a breakthrough. The detrimental effect of dyslipidemia is not limited to the vasculature but also leads to dysfunction of circulating angiogenic cells (CAC) and of macrophages. The endogenous ligands for LXRs are oxidative metabolites of cholesterol that serve as intracellular cholesterol "sensors". LXR agonists operate, in part, by transcriptional upregulation of genes involved in promoting cholesterol efflux and inhibition of cholesterol uptake; and by inhibiting inflammation. Our published studies and new preliminary data show that pharmacological LXR activation prevents DR development in both T1D and T2D rodent models. In this application, we seek to understand the mechanisms involved in this beneficial effect. We put forth the hypothesis that LXR activation will restore cholesterol homeostasis in the diabetic retina and correct diabetes-induced bone marrow dysfunction to sustain CAC levels and function and to reduce of myeloid cell production.
- Conditions
- Diabetic Retinopathy
- Being tested
- blood draw
- Keywords
- Liver X receptor, Circulating angiogenic cells
- Medical subject headings
- Diabetic Retinopathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
blood drawBiological
Blood sample will be obtained and CD34+ cells will be isolated for functional testing.
| Study group | Role | What they receive |
|---|---|---|
| Controls | Any man or woman between the ages of 21- 98 years of age will be eligible to participate. To participate in the study as a study subject we will require that the subject must carry the diagnosis of healthy control. Receives: Biological: blood draw | |
| Diabetic no retinopathy | Patients with diabetes but with no evidence of diabetic retinopathy Receives: Biological: blood draw | |
| Diabetic with mild retinopathy | Diabetics with mild non proliferative diabetic retinopathy (NPDR). Receives: Biological: blood draw | |
| Diabetic with moderate retinopathy | Diabetics with moderate NPDR Receives: Biological: blood draw | |
| Diabetics with severe retinopathy | Diabetic with severe NPDR. Receives: Biological: blood draw | |
| Diabetics with proliferative diabetic retinopathy (PDR) | Diabetics with proliferative diabetic retinopathy (PDR) Receives: Biological: blood draw |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessing CD34+ cells function
We are isolating CD34+ cells from peripheral blood and then examining the cell membrane characteristics of CD34+ cells and their in vitro function.
Measured: from blood draw to 48 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2018
- Study start11 Jan 2018
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetic RetinopathyStudies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe 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
- Other
- Enrolment
- 104 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Maria Grant, University of Alabama at Birmingham
- Investigators
- Maria B Grant, MD, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03403686
- Sponsor's protocol ID
- IRB-300000068
- Acronym
- LXR and DR
- Status last verified
- Sep 2026
- First submitted
- 11 Jan 2018
- Last updated
- 28 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- LXR as a Novel Therapeutic Target in DR
- ClinicalTrials.gov ID
- NCT03403686
- Acronym / sponsor ID
- LXR and DR / IRB-300000068
- Conditions
- Diabetic Retinopathy
- Keywords
- Liver X receptor, Circulating angiogenic cells
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 104 participants (estimated)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Study start
- 11 Jan 2018 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 19 Jan 2018 (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.
Results from large clinical trials demonstrate a strong association between lipid abnormalities and progression of the most common microvascular complication, diabetic retinopathy (DR). We found that activation of a master regulator of cholesterol metabolism, the nuclear hormone receptors liver X receptors (LXRα/LXRβ), prevents DR in rodent models. In this application, we seek to understand the mechanisms responsible for the beneficial effects of LXR agonists on retina and on bone marrow (BM) to preserve the function of reparative cells while reducing inflammatory cell.
Detailed description
Diabetic retinopathy (DR) is a disabling microvascular complication. Despite recent advances using pharmacotherapy, a cure for DR has yet to be realized. Thus, a conceptual and technical breakthrough to identify novel targets, and a strategy to cure this complication is paramount. We believe that the recent clinical evidence from large clinical trials demonstrating a strong association between lipid abnormalities and DR progression and the discovery that activation of the nuclear hormone receptors liver X receptors (LXRα/LXRβ) prevents DR in rodent models offers such a breakthrough. The detrimental effect of dyslipidemia is not limited to the vasculature but also leads to dysfunction of circulating angiogenic cells (CAC) and of macrophages. The endogenous ligands for LXRs are oxidative metabolites of cholesterol that serve as intracellular cholesterol "sensors". LXR agonists operate, in part, by transcriptional upregulation of genes involved in promoting cholesterol efflux and inhibition of cholesterol uptake; and by inhibiting inflammation. Our published studies and new preliminary data show that pharmacological LXR activation prevents DR development in both T1D and T2D rodent models. In this application, we seek to understand the mechanisms involved in this beneficial effect. We put forth the hypothesis that LXR activation will restore cholesterol homeostasis in the diabetic retina and correct diabetes-induced bone marrow dysfunction to sustain CAC levels and function and to reduce of myeloid cell production.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Controls | – | Biological: blood drawAny man or woman between the ages of 21- 98 years of age will be eligible to participate. To participate in the study as a study subject we will require that the subject must carry the diagnosis of healthy control. |
| Diabetic no retinopathy | – | Biological: blood drawPatients with diabetes but with no evidence of diabetic retinopathy |
| Diabetic with mild retinopathy | – | Biological: blood drawDiabetics with mild non proliferative diabetic retinopathy (NPDR). |
| Diabetic with moderate retinopathy | – | Biological: blood drawDiabetics with moderate NPDR |
| Diabetics with severe retinopathy | – | Biological: blood drawDiabetic with severe NPDR. |
| Diabetics with proliferative diabetic retinopathy (PDR) | – | Biological: blood drawDiabetics with proliferative diabetic retinopathy (PDR) |
Who may qualify
- Ages
- 21 to 98 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Patients who have retinal abnormalities other than diabetic retinopathy will be excluded. Patients who have systemic conditions that influence hematopoietic stem cell function such as cardiovascular disease, malignant disease, diabetes, hematologic disorder, or estimated glomerular filtration rate less than 60 mL/min or who have undergone treatment with erythropoietin will be excluded. We will record all medications including antihypertensive drug treatment, treatment with statins, Angiotensin-Converting Enzyme (ACE) Inhibitors, Angiotensin Receptor Blockers (ARB) or other pharmacological agents that may influence CD34+ cell function. Baseline characteristics will be recorded, including age, lipid parameters, body mass index (BMI), blood pressure, smoking history, antioxidant intake and use of nutritional supplements.
Key inclusion criteria (1 listed)
- Any man or woman between the ages of 21- 98 years of age will be eligible to participate. To participate in the study as a study subject we will require: a) the subject must either carry the diagnosis of diabetes or be a healthy aged control and b) the patient be willing and have the ability to cooperate with the protocol.
Key exclusion criteria (1 listed)
- Exclusion criteria: We will apply the following exclusion criteria: a) evidence of ongoing acute or chronic infection (HIV, Hepatitis B or C, tuberculosis); b) ongoing malignancy; c) cerebral vascular accident or cerebral vascular procedure; d) current pregnancy; e) history of organ transplantation; f) presence of a graft (to avoid any effect of the graft on inflammatory parameters; g) uremic symptoms, an estimated glomerular filtration rate of less than 20 cc/min (by Modification of Diet in Renal Disease equation), or an albumin of less than 3.6 (to avoid malnutrition as a confounding variable); h) be unwilling to abstain from drinking alcohol and i) patients with anemia. Subjects with AMD, glaucoma, uveitis, known hereditary degenerations or other significant ocular complications other than diabetic retinopathy will be excluded.
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 |
|---|---|---|
| blood drawBlood sample will be obtained and CD34+ cells will be isolated for functional testing. | Biological | Controls, Diabetic no retinopathy, Diabetic with mild retinopathy, Diabetic with moderate retinopathy, Diabetics with proliferative diabetic retinopathy (PDR), Diabetics with severe retinopathy |
Primary outcome
Assessing CD34+ cells function
Time frame: from blood draw to 48 hours
We are isolating CD34+ cells from peripheral blood and then examining the cell membrane characteristics of CD34+ cells and their in vitro function.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2026.
- Individual participant dataSharing planned: No
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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.
Maria B Grant, MD principal investigator
University of Alabama at Birmingham
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 Diabetic Retinopathy, run by University of Alabama at Birmingham 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 1 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 28 Sep 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Maria B Grant, MD (3528714314, jmoorer@uabmc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessing CD34+ cells function (time frame: from blood draw to 48 hours). 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 28 Sep 2026.