We are conducting the ELiSA Study (Evaluation of Lixivaptan In Subjects with ADPKD), a Phase 2 clinical trial that will evaluate the safety, pharmacokinetics and pharmacodynamics of multiple doses of lixivaptan in patients with ADPKD with relatively preserved kidney function (chronic kidney disease stages CKD1 and CKD2) and moderately impaired renal function (CKD3).
This study will look at how well two different doses of the study drug, lixivaptan, work in preserving kidney function. It will also look at how well adult subjects with ADPKD tolerate the study drug, which will be given by mouth twice per day for seven days. Blood and urine samples will be taken to evaluate what happens to the study drug in the body, for example how long it remains in the blood and how quickly it is removed, as well as to examine possible other measures such as liver function tests and urine concentration.
Study participants must be able to stay overnight at the clinical research unit on two separate occasions. Participants will be eligible to receive a stipend as well as reimbursement for travel and certain other expenses for themselves and a companion. Additional details will be provided by the clinical study site.
You may be able to take part in the ELiSA Study if you:
- Have been diagnosed with ADPKD by the modified Ravine criteria
- Are 18 to 65 years of age
- Have chronic kidney disease (CKD) in stages 1, 2, or 3
- Have an estimated glomerular filtration rate (eGFR) ≥ 30mL/min/1.73m2
- Are considered by the Study Investigator to be in good health relative to underlying CKD status and clinically stable with respect to underlying CKD
You will not be able to participate in the study if you:
- Have had an allergic or bad reaction (known sensitivity or idiosyncratic reaction) to lixivaptan or its related compounds, such as benzazepines (e.g., tolvaptan, conivaptan, benazepril, fenoldopam, or mirtazapine)
- Are pregnant or breastfeeding
- Have taken tolvaptan, oral or intravenous antibiotics, or any investigational drug or used an investigational device within 30 days prior to first study dose
- Have a transplanted kidney, or absence of a kidney
- Have clinically significant incontinence, overactive bladder, or urinary retention (for example, benign prostatic hyperplasia)
- Have clinically significant liver disease, or clinically significant liver function abnormalities other than that expected for ADPKD with cystic liver disease at baseline
- Have any other clinically significant disease or condition in addition to ADPKD (including treatment for such conditions) that, in the opinion of the Study Investigator, could either interfere with the study drug or pose an unacceptable risk to you as the subject
The study is currently enrolling and is expected to include up to 35 patients at approximately 15 sites in California, Florida, Kansas, Maryland, Minnesota, Missouri, New York, Pennsylvania, Tennessee, and Utah.
For more information about the ELiSA study, including contact information for sites that are actively recruiting, please visit the study record at the Clinicaltrials.gov website at this link.
Clinical Trials / Compassionate Use
Palladio Biosciences is committed to improving the lives of patients by bringing forward new therapies for orphan and rare diseases of the kidney. The conduct of clinical studies is the primary route to scientifically understanding the value of new drugs in the treatment of kidney disease. Palladio Biosciences will pursue such clinical trials to provide the safety and efficacy data necessary to obtain approval from regulatory authorities to market such drugs.
The most productive route for patients to gain access to new medications before they are approved is by participating in clinical trials. It is recognized, however, that under certain rare and particular situations there will be individuals with a bona fide need to gain access to a new therapy who do not meet the traditional entry criteria for an available clinical trial. Physicians caring for such patients with serious or life-threatening conditions can seek special access to investigational medicines through expanded access pathways (also, referred to as compassionate use) outside of the clinical trial process. Palladio will consider such requests from a physician when the following circumstances are satisfied:
- The patient(s) for whom expanded access or compassionate use is sought must meet all of the following conditions:
- The patient is suffering from a serious, disabling, painful, or life-threatening disease or condition;
- The patient has undergone appropriate standard treatments without success or tolerability; no comparable or satisfactory alternative treatment is available or no standard treatment exists for the disease or condition;
- The patient is ineligible for participation in any ongoing clinical study of the unapproved product; and
- The patient meets any other relevant medical criteria for expanded access or compassionate use of the investigational product, as established by Palladio Biosciences.
- There are meaningful human clinical data to support an assessment that the potential benefits to the patient outweigh the risks to the patient.
- Providing the unapproved product for expanded access or compassionate use will not interfere with development of the product.
- Expanded access or compassionate use is allowed under all applicable legal and regulatory requirements.
The requesting licensed physician will submit the following information to Palladio Biosciences:
- Demographic information on the proposed patient seeking expanded access;
- Medical history, including all treatments administered for the relevant use and the clinical course with those treatments;
- Description of the patient’s prognosis;
- Reason for requesting expanded access;
- Plan of treatment if expanded access is granted, including safety monitoring and periodic assessment of benefit/risk ratio
A team of Palladio Biosciences scientists and physicians will review the information with the following points in mind:
- Is the scientific/clinical rationale for the proposed use valid?
- Is there the potential of including the patient in an ongoing Palladio clinical trial?
- Is the proposed use through expanded access compatible with Palladio’s research objectives?
- Can clinical drug supplies be made available for this expanded access request?
- What experience does the physician/investigator have with the requested drug and clinical investigation?
If the Palladio Biosciences team makes a recommendation for expanded access, the Board of Directors of Palladio Biosciences will review the request and determine whether to approve it or not. The results of that review will be communicated to the physician submitting the expanded access request. If the request has been denied, no further appeal will be allowed.
If the Palladio Biosciences team denies the request for expanded access, it will communicate that result to the requesting physician. Again, denial of the request by the Palladio Biosciences team cannot be appealed.
Requests for expanded access or to discuss eligibility or answer other questions may be submitted by a treating physician to the attention of Dr. Neil Shusterman at firstname.lastname@example.org. Acknowledgement of the request will be within 5 business days after receipt.