TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study

• Status: Completed
• Trial end date: 2018-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The goal of this research project is to establish that in short-term use, tolvaptan is a safe and potentially effective new therapy for cystinuria, by conducting a short-term pilot study of the safety and tolerability of this drug, and assess impact on urinary stone risk parameters, among adolescent and young adult patients with clinical cystinuria.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Caleb Nelson
• Collaborator: Otsuka America Pharmaceutical
• Treatments: Tolvaptan

Criteria

Inclusion Criteria:

• Males and females age 12 - 29 years

• Weight ≥ 25kg (55 lbs)

• Confirmed cystinuria diagnosis

• Specific blood test levels (done within the past 6 months)

Exclusion Criteria:

• Concurrent non-renal disease that might increase risk of complications due to aquaresis

• Liver or biliary disease (chronic or acute)

• Malabsorption syndrome or other gastrointestinal condition that may interfere with response to therapy

• Non-cutaneous malignancy within last 5 years

• History of adverse reaction or allergy to Tolvaptan or other arginine vasopressin V2-receptor antagonists