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

Caleb Nelson

Status: Completed

Conditions: Cystinuria

Treatments: Drug: Tolvaptan

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02538016

16726

Details and patient eligibility

About

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.

Enrollment: 4 patients

Sex: All

Ages: 12 to 29 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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4 participants in 1 patient group

Tolvaptan

Other group

Treatment:

Drug: Tolvaptan