Trial of Venetoclax (ABT-199) and Dexamethasone for Relapsed or Refractory Systemic AL Amyloidosis (NCT03000660) | Clinical Trial Compass
TerminatedPhase 1
Trial of Venetoclax (ABT-199) and Dexamethasone for Relapsed or Refractory Systemic AL Amyloidosis
Stopped: partial clinical hold per FDA
United States3 participantsStarted 2017-01
Plain-language summary
This is a study to determine the safety, tolerability and maximum tolerated dose of Venetoclax (ABT-199) and dexamethasone in relapsed or refractory amyloid light chain (AL) amyloidosis patients.
Who can participate
Age range
18 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
. Renal: albuminuria higher than 0.5 g/day in a 24-hour urine collection.
. Cardiac: involvement is defined as the presence of a mean left ventricular wall thickness on echocardiogram more than 12 mm in the absence of a history of hypertension or valvular heart disease, or unexplained low voltage (\< 0.5 mV) on electrocardiogram; or an NT-proBNP \> 332 ng/L in CKD 1 or 2 patients or a BNP \> 100ng/L in those who are CKD3.
. Hepatic: hepatomegaly on physical examination with alkaline phosphatase \> 1.5 X the upper limit of normal (ULN).
. Autonomic or peripheral neuropathy: based on clinical history, autonomic dysfunction with orthostasis, symptoms of nausea or dysgeusia, gastric atony by gastric emptying scan, diarrhea or constipation, or abnormal sensory and/or motor findings on neurologic examination.
. Stage I, both under threshold;
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
Participants with treatment related adverse events using NCI CTCAE version 4.03.
Timeframe: Up to 8 months after beginning study drug
. Stage II, either troponin or NT-proBNP \[but not both\] over threshold;
. Stage III, both over threshold;
. Stage IIIa, both over threshold but NT-proBNP \< 8500 pg/ml.
Exclusion criteria
. QTc \> 470 milliseconds (msec) on a 12 lead ECG obtained during the Screening period. If a machine reading is above this value, the ECG should be reviewed by a qualified reader and confirmed on a subsequent ECG.
. AL Amyloidosis Risk Stage IIIb disease. Stage IIIb is defined by NT-proBNP \> 8500 pg/mL and troponin I \> 0.10 ng/mL.
. New York Heart Association (NYHA) classification III or IV.
. Enzyme-documented myocardial infarction within 6 months before enrollment.
. Chronic atrial fibrillation.
. Grade 2 or 3 atrioventricular (AV) block (Mobitz, Type I permitted).
. Supine systolic blood pressure \< 90 mmHg, or symptomatic orthostatic hypotension, or a decrease in systolic blood pressure on standing of \> 20 mm Hg in spite of being treated for orthostatic hypotension.
. History of a bleeding diathesis or currently receiving treatment with warfarin. Patients are allowed to take aspirin.