A Safety, Pharmacokinetic and Dose-Escalation Study of KD019 in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)


  • Study type

  • Study phase

  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 11-006482
    NCT ID: NCT01559363
    Sponsor Protocol Number: KD019-101

About this study

The primary objective of this study is to determine the safety, plasma pharmacokinetics, and maximum tolerated dose (MTD) of KD019 when administered to subjects with ADPKD.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

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
  • Subject is unwilling to do testing required for study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Ziad El-Zoghby, M.D.

Closed for enrollment


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