N/A
N=4
TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study
Cystinuria
Bottom Line
View on ClinicalTrials.gov: NCT02538016 ↗Enrolled (actual)
4
Serious AEs
0.0%
Results posted
Jun 2020
Primary outcome: Primary: Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8) — 70 mg/L
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Tolvaptan (Drug)
- Age
- Pediatric, Adult · 12+ yrs
- Sex
- All
- Sponsor
- Caleb Nelson
- Primary completion
- Dec 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8) |
70 | — |
| SECONDARY Urine Osmolality at High Dose (Day 8) |
141; 4.05; 102 | — |
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.
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT02538016). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.