Long Term Clinical Efficacy of Sodium-glucose Cotransporter-2 (SGLT-2) Inhibitor in Cystinurics

NCT05058859 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: SGLT2 1 Year
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief 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, 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.

Conditions

Cystinuria

Outcome Measures

Primary Outcomes (1)

• 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.

  1 year and 3 months

Study Arms (1)

Study Drug

EXPERIMENTAL

The study drug is Dapagliflozin

Drug: Dapagliflozin

Interventions

Dapagliflozin DRUG

Dapagliflozin is to lower blood sugar levels in adults with type 2 diabetes

Also known as: FARXIGA

Study Drug

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of California, San Francisco: lead

Study Sites (1)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

MeSH Terms

Conditions

Cystinuria

Interventions

dapagliflozin

Condition Hierarchy (Ancestors)

Renal Aminoacidurias; Renal Tubular Transport, Inborn Errors; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Marshall Stoller, MD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 17, 2021
• First Posted: September 28, 2021
• Study Start: September 1, 2023
• Primary Completion: October 31, 2024
• Study Completion: November 1, 2025
• Last Updated: August 29, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)