Bucillamine Phase 2 Trial in Patients With Cystinuria
Status:
Unknown status
Trial end date:
2018-03-01
Target enrollment:
Participant gender:
Summary
Subjects on a standard regimen of tiopronin (cystine binding thiol drug; CBTD) plus
prescribed first-line therapy (i.e. on a hydration, alkali therapy and dietary restriction)
who are failing therapy will be selected for this trial.
After completing informed consent, the subject will have Screening consisting of medication
history and physical examination with vital signs. Samples of blood and urine will be taken
for clinical laboratory and urinalysis. Patients will undergo a 12-lead ECG test. A history
of side effects with current CBTD as well as laboratory recordings of abnormalities
attributable to treatment will also be recorded.
Subjects will be dosed in a sequential manner, starting with the low dose group (300 mg/day),
then proceeding to the 600 mg.day dose group.. Safety and tolerability will be monitored
closely by an Independent Medical Monitor (IMM) and based on the IMM's assessment that it is
safe to proceed to the higher dose (600 mg/day), subsequent subjects will be enrolled into
that group. Up to 15 subjects each will be enrolled into either Group A or Group B.
After 7 days on the assigned bucillamine dose, a 24-hour urine sample will be taken and after
completing the Day 8 safety visit, subjects will undergo a 7 day washout where no CBTDs will
be taken. Thereafter, subjects will be allowed to resume their originally prescribed CBTDs
under Investigator's supervision.
One week following study drug discontinuation, subjects will return to the clinic for
follow-up safety assessments.