Clinical Trials

Phase 1: - Primary purpose is to determine the safety of KD019. - 28-days, daily dosing study with option to continue through six months of KD019 dosing. - All participants receive active KD019 study drug. - KD019 is an oral once daily tablet. Tablets are 50 mg in strength. Participants will enroll into three sequential dosing cohort levels (50 mg, 100 mg and 150 mg. - Study participants will have MRI of the abdomen (kidneys) at Screening and Month 6 visit to explore effects of KD019. - Echocardiogram will be performed at Screening, Day 28, and Months 2-6 if option for continuation past Day 28 is accepted. Phase 2: - Primary purpose is to compare the annualized change in glomerular filtration rate (GFR) in subjects with ADPKD when treated with KD019. - Dosing is 28-days of daily. Subjects may, if they desire and at the discretion of the investigator, continue to receive study treatment for 12 months from their first dose or until the development of unacceptable toxicity, noncompliance, or withdrawal of consent by the subject, or investigator decision. - All participants receive active KD019 study drug. - KD019 is an oral once daily tablet. Tablets are 50 mg in strength. Participants in Phase 2 will be given the maximum tolerated dose. - Study participants will have MRI of the abdomen (kidneys) at Screening and Month 6 visit and every 6 months after to explore effects of KD019. - Echocardiogram will be performed at Screening, Day 28, and Months 2-6 if option for continuation past Day 28 is accepted.

Some patients with autosomal dominant polycystic kidney disease (ADPKD) have intractable disabling chronic kidney pain.  Among methods used to manage these patients, removal of the nerve supply to the kidney by Videothoracoscopic excision of Splanchnic nerve (videothoracoscopic splanchnicectomy — VSPL) is one of the most promising procedures.  Few of these procedures have been performed in ADPKD patients (and in only one patient using the less invasive thoracic approach).  We recently performed VSPL on two patients who had chronic pain.  Both had initial improvement but have had very short follow up. This study should improve understanding of the molecular mechanisms and mediators of kidney pain and provide the hope of an effective procedure to ADPKD patients with chronic kidney pain.

     We are now enrolling patients to study the effectiveness of the VSPL operation, collecting information on pain intensity, quality of life, kidney blood flow, kidney/cyst volume and also to study the involved pain pathways.

     Baseline Visit - The patient will also have a complete physical exam and have  the kidney's filtration rate measured. Urine and blood tests are performed at each evaluation. You will have an MRI performed of your kidneys. There is no radiation exposure associated with this procedure. You will also be asked to complete Quality of Life (QOL), HALT Pain, and Depression Score questionnaires. These will take approximately 15 minutes to complete and will be repeated at each evaluation.

     The VSPL operation will be done within approximately 1 week of your Baseline Visit and is considered part of your clinical care and not part of the research.In this procedure the surgeon approaches the nerves supplying the kidneys through the chest wall, using 3 small incisions. He locates these nerves, then cuts a piece of them and removes it from the chest cavity.You will be released from the hospital when your pain is controlled on oral medications.

     1 Month Visit-You will be asked to return to the Mayo Clinic one month after your VSPL procedure for a physical exam and completion of the questionnaires.If unable to return to the Mayo Clinic this evaluation can be done with your local doctor.

     3 Month Visit-The 3 Month Visit will be identical to the Baseline visit. You will have a physical exam, blood and urine samples, MRI of your kidneys, Iothalamate Clearance, and the three questionnaires to complete.

     6 Months: The questionnaires will be mailed to you for completion.

     12 Month Visit-The 12 Month Visit is identical to the 1 Month visit. You will be asked to return to the Mayo Clinic for a physical exam and completion of the questionnaires.

     2 Year Visit-The 2 Year Visit will be identical to the Baseline visit. You will have a physical exam, blood and urine samples, MRI of your kidneys, measurement of kidney filtration and complete the three questionnaires.