Bucillamine Phase 2 Trial in Patients With Cystinuria (NCT02942420) | Clinical Trial Compass
UnknownPhase 2
Bucillamine Phase 2 Trial in Patients With Cystinuria
United States30 participantsStarted 2017-05-01
Plain-language 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.
Who can participate
Age range
18 Years – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Subjects of any gender and of any race ≥18 and ≤80 years of age
. Subjects with proven cystinuria who are failing their standard drug therapy of tiopronin plus first-line therapy (hydration, alkali and diet restriction) and who meet the following criteria.
. Subjects must be able to reliably urinate in a collection vessel and measure urine volume
. Subjects must have documentation of a stable complete blood count (CBC) and urinalysis (UA) in the 6 months prior to date of enrollment
. Subjects may have a history of but not currently active CNS disorders or symptoms/effects (e.g., headache)
. Subjects must have adequate organ function, evidenced by the following laboratory results within 30 days prior to enrollment:
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Timeframe: Days 0, 2, 3, 7, 8 and + 1 wk post study
. Female subject who has been post-menopausal for at least 24 consecutive months, or women who have undergone surgical sterilization, (e.g. hysterectomy, bilateral oophorectomy, tubal ligation or salpingectomy) is eligible without requiring the use of a contraceptive methods described in Inclusion #8
. For women of childbearing potential and men with partners of childbearing potential, agreement to use a highly effective, non-hormonal form of contraception:
Exclusion criteria
. Subjects with renal colic
. Subjects who are scheduled to undergo a surgical procedure
. Subjects on D-penicillamine (see page 35 for explanation)
. Subjects with cancer
. Subjects with acute or chronic infections including HIV, tuberculosis, hepatitis B or hepatitis C
. Patients with proteinuria ≥30 mg that is confirmed on repeat laboratory assessment within 24 hours
. Subjects with QTc interval \>450 ms
. A history of, hypokalemia and family history of Long QT syndrome