CystinuriaPhase 2NCT05058859
Long Term Clinical Efficacy of Sodium-glucose Cotransporter-2 (SGLT-2) Inhibitor in Cystinurics
A Phase 2 study testing Dapagliflozin in people with Cystinuria, run by University of California, San Francisco at 1 site in United States.
Trial summary
Cystinuria is an inherited autosomal recessive disorder of the kidney that is the result of an inability to reabsorb dibasic amino acids, including cystine, from the urine. Supersaturation of cystine in the urine produces crystals that precipitate and form stones in the kidney, which can be a cause of obstruction… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystinuria
- What is being tested?
- Dapagliflozin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Sep 2023to 1 Nov 2025
- Phase
- Phase 2
- 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
Who may be able to join
- males and females age 18 or older
- documented cystinuria on prior 24-hour urine collection and/or stone analysis
- history of previous cystine kidney stones
- able and willing to provide consent
Who cannot join
- prior diagnosis of diabetes mellitus (type I or type II)
- vulnerable populations including incarceration status
- anticipation of pregnancy during the study duration
- unable to give informed consent
- non-English primary language
- pregnancy, lactation, or child- bearing age without birth control devices
- serious illness likely to cause death within the next 5 years
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, San Francisco
San Francisco, California, 94143
About this study
Cystinuria is an inherited autosomal recessive disorder of the kidney that is the result of an inability to reabsorb dibasic amino acids, including cystine, from the urine. Supersaturation of cystine in the urine produces crystals that precipitate and form stones in the kidney, which can be a cause of obstruction, infection, and chronic kidney disease. Cystine stones constitute a major health challenge for affected individuals with cystinuria because of the frequent recurrence of painful symptoms and the current absence of effective, patient-accepting treatment.
A mainstay of therapy is breaking or preventing the cystine bond on the molecular level such that cystine (which is formed from the joining of two cysteine amino acids and their corresponding sulfur atoms) cannot precipitate in the urine. It is hypothesized that a glucose molecule may be able to do this if introduced into the urine. SGLT-2 inhibitors are a class of drug that are FDA approved to treat diabetes mellitus (DM) and heart failure by inhibiting an enzyme in the kidney that allows for reabsorption of glucose from the urine. This effectively increases the concentration of glucose in the urine. The hypothesis suggests that administration of this drug to patients with cystinuria will introduce sufficient glucose into the urine to prevent or reverse the formation of cystine stones. To date, there has been no published data on the effectiveness of this therapy for this indication, although the dosage and administration would be identical to that already approved by the FDA for the treatment of DM and heart failure.
Read the detailed description
This is a single center, prospective cohort trial designed to assess the effect of daily oral administration of dapagliflozin 10 mg on cystine stone formation across routine imaging obtained during management of this disease. 25 subjects are planned, each with previously diagnosed cystinuria and without current treatment except with potassium citrate medication.
Each patient identified in the clinic as a potential participant will be screened to determine subject eligibility. Subjects who meet all inclusion criteria and none of the exclusion criteria will be entered into the study. Consent will be obtained. The therapy under investigation, dapagliflozin 10 mg, will then be administered orally to each participant daily for one year. Each subject will be contacted 1 week by the study team after treatment begins to check compliance, tolerability, and side effects with SGLT-2 inhibitor therapy. Each subject will then be contacted every 8 weeks by the study team for follow-up and to continue checks on compliance, tolerability, and side effects with SGLT-2 inhibitor therapy. Each subject will subsequently undergo routine care with no further alterations or interruptions to their typical care with routine follow up appointments with a study doctor every 3-4 months.
Routine standard-of-care surveillance imaging for their cystinuria and formation of cystine stones will also occur as part of the treatment and management of each participant's kidney stone disease. This routine care will continue to be performed during the study period, the only difference being the collection of data with regard to ongoing stone burden for the cystinuria patients receiving treatment with daily oral dapagliflozin 10 mg on each routine imaging scan. Tolerability of the study therapy will be assessed at each routine visit during the participant's usual care. Participants who require operative intervention for their kidney stones during the treatment period will be removed from the study. No placebo will be used during this study.
Total duration of subject participation with be up to 1 year and 3 months. Total duration of the study is expected to be up to 1 year and 3 months.
- Conditions
- Cystinuria
- Being tested
- Dapagliflozin
- Medical subject headings
- Cystinuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DapagliflozinDrug
Also called FARXIGA
Dapagliflozin is to lower blood sugar levels in adults with type 2 diabetes
| Study group | Role | What they receive |
|---|---|---|
| Study Drug | Experimental | The study drug is Dapagliflozin Receives: Drug: Dapagliflozin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in cystine stone size over time (1 year and 3 months)
The change in cystine stone size in mm will be measured over time using routine, standard-of-care imaging obtained during the management of patients with cystinuria.
Measured: 1 year and 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2021
- Study start1 Sep 2023 (estimated)
- Primary completion31 Oct 2024 (estimated)
- Study completion1 Nov 2025 (estimated)
Why it stopped: Funding was never secured so clinical trial was never started; this study was abandoned
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CystinuriaStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Marshall Stoller, MD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05058859
- Sponsor's protocol ID
- SGLT2 1 Year
- Status last verified
- Aug 2025
- First submitted
- 17 Sep 2021
- Last updated
- 29 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Clinical Efficacy of Sodium-glucose Cotransporter-2 (SGLT-2) Inhibitor in Cystinurics
- ClinicalTrials.gov ID
- NCT05058859
- Acronym / sponsor ID
- SGLT2 1 Year
- Conditions
- Cystinuria
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Funding was never secured so clinical trial was never started; this study was abandoned
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Study start
- 1 Sep 2023 (estimated)
- Primary completion
- 31 Oct 2024 (estimated)
- Study completion
- 1 Nov 2025 (estimated)
- First posted
- 28 Sep 2021 (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.
Cystinuria is an inherited autosomal recessive disorder of the kidney that is the result of an inability to reabsorb dibasic amino acids, including cystine, from the urine. Supersaturation of cystine in the urine produces crystals that precipitate and form stones in the kidney, which can be a cause of obstruction, infection, and chronic kidney disease. Cystine stones constitute a major health challenge for affected individuals with cystinuria because of the frequent recurrence of painful symptoms and the current absence of effective, patient-accepting treatment.
A mainstay of therapy is breaking or preventing the cystine bond on the molecular level such that cystine (which is formed from the joining of two cysteine amino acids and their corresponding sulfur atoms) cannot precipitate in the urine. It is hypothesized that a glucose molecule may be able to do this if introduced into the urine. SGLT-2 inhibitors are a class of drug that are FDA approved to treat diabetes mellitus (DM) and heart failure by inhibiting an enzyme in the kidney that allows for reabsorption of glucose from the urine. This effectively increases the concentration of glucose in the urine. The hypothesis suggests that administration of this drug to patients with cystinuria will introduce sufficient glucose into the urine to prevent or reverse the formation of cystine stones. To date, there has been no published data on the effectiveness of this therapy for this indication, although the dosage and administration would be identical to that already approved by the FDA for the treatment of DM and heart failure.
Detailed description
This is a single center, prospective cohort trial designed to assess the effect of daily oral administration of dapagliflozin 10 mg on cystine stone formation across routine imaging obtained during management of this disease. 25 subjects are planned, each with previously diagnosed cystinuria and without current treatment except with potassium citrate medication.
Each patient identified in the clinic as a potential participant will be screened to determine subject eligibility. Subjects who meet all inclusion criteria and none of the exclusion criteria will be entered into the study. Consent will be obtained. The therapy under investigation, dapagliflozin 10 mg, will then be administered orally to each participant daily for one year. Each subject will be contacted 1 week by the study team after treatment begins to check compliance, tolerability, and side effects with SGLT-2 inhibitor therapy. Each subject will then be contacted every 8 weeks by the study team for follow-up and to continue checks on compliance, tolerability, and side effects with SGLT-2 inhibitor therapy. Each subject will subsequently undergo routine care with no further alterations or interruptions to their typical care with routine follow up appointments with a study doctor every 3-4 months.
Routine standard-of-care surveillance imaging for their cystinuria and formation of cystine stones will also occur as part of the treatment and management of each participant's kidney stone disease. This routine care will continue to be performed during the study period, the only difference being the collection of data with regard to ongoing stone burden for the cystinuria patients receiving treatment with daily oral dapagliflozin 10 mg on each routine imaging scan. Tolerability of the study therapy will be assessed at each routine visit during the participant's usual care. Participants who require operative intervention for their kidney stones during the treatment period will be removed from the study. No placebo will be used during this study.
Total duration of subject participation with be up to 1 year and 3 months. Total duration of the study is expected to be up to 1 year and 3 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study Drug | Experimental | Drug: DapagliflozinThe study drug is Dapagliflozin |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- males and females age 18 or older
- documented cystinuria on prior 24-hour urine collection and/or stone analysis
- history of previous cystine kidney stones
- able and willing to provide consent
Key exclusion criteria (7 listed)
- prior diagnosis of diabetes mellitus (type I or type II)
- vulnerable populations including incarceration status
- anticipation of pregnancy during the study duration
- unable to give informed consent
- non-English primary language
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 |
|---|---|---|
| DapagliflozinAlso: FARXIGADapagliflozin is to lower blood sugar levels in adults with type 2 diabetes | Drug | Study Drug |
Primary outcome
Change in cystine stone size over time (1 year and 3 months)
Time frame: 1 year and 3 months
The change in cystine stone size in mm will be measured over time using routine, standard-of-care imaging obtained during the management of patients with cystinuria.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2025.
- 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. This study is not recruiting at the moment.
Marshall Stoller, MD principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dapagliflozin in people with Cystinuria, run by University of California, San Francisco 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 4 inclusion and 7 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: withdrawn, as of the sponsor's last update on 29 Aug 2025. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Change in cystine stone size over time (1 year and 3 months) (time frame: 1 year and 3 months). 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 29 Aug 2025.