Long Term Clinical Efficacy of Sodium-glucose Cotransporter-2 (SGLT-2) Inhibitor in Cystinurics
Status:
Not yet recruiting
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
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.