A Phase 1b/2a, Safety, Pharmacokinetic and Dose-Escalation Study of KD019 in Subjects With Autosomal Dominant Polycystic Kidney Disease

Location:

Rochester, Minn.

Trial status:

Open for Enrollment

Why is this study being done?

Phase 1: - Primary purpose is to determine the safety of KD019. - Dosing is for 28 days daily. After the 28-day treatment period, subjects will, at the discretion of the investigator, continue to receive study treatment for 24 months from their first dose or until the development of unacceptable toxicity, noncompliance, or withdrawal of consent by the subject, or investigator decision. Subjects may continue beyond 24 months at the discretion of the investigator after consultation with the medical monitor. - 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.). Participants in Phase 1b will have their dose increased or decreased to the MTD. - Study participants will have MRI of the abdomen (kidneys) at Screening and 6 months thereafter to explore effects of KD019. - Echocardiogram will be performed at Screening, Day 28, months 3 and 6 and every 6 months thereafter. Phase 2: - Primary purpose is to compare the annualized change in glomerular filtration rate (GFR) in subjects with ADPKD when treated with KD019. - KD019 will be dosed on Monday, Wednesday and Friday of each week. Subjects will receive study treatment for 24 months from their first dose or until the development of unacceptable toxicity, noncompliance, or withdrawal of consent by the subject, or investigator decision. Subjects may continue beyond 24 months at the discretion of the investigator after consultation with the sponsor. - This alternate dosing schedule will be explored to determine if it is more tolerable than daily dosing when used chronically in subjects with ADPKD. - All participants receive active KD019 study drug. - KD019 is an oral tablet dosed on Monday, Wednesday and Friday every week. Tablets are 50 mg in strength. - 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 3 and 6 and 6 months thereafter.

Who is eligible to participate?

Inclusion Criteria: - The subject has a confirmed diagnosis of ADPKD. - The subject has a GFR ≥ 50 mL/min/1.73 m2. - Cysts must be at least 1 cm in size. - Adequate bone marrow, kidney, and liver function. - Must agree to use two forms of birth control for those of child bearing potential Exclusion Criteria: - The subject has had a previous partial or total nephrectomy. - The subject has tuberous sclerosis, Hippel-Lindau disease, or acquired cystic disease. - The subject has congenital absence of one kidney and/or need for dialysis. - Presence of renal or hepatic calculi (stones) causing symptoms. - The subject has received any investigational therapy within 30 days prior to study entry. - Active treatment (within 4 weeks of study entry) for urinary tract infection. - Subject is known to be immunocompromised - Subject is pregnant or nursing.

Last updated:

6/6/2014

NCT ID:

NCT01559363

IRB Number:

11-006482