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A Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Intravenously Administered PYC-003, a Peptide-phosphorodiamidate Morpholino Oligonucleotide Conjugate, in Healthy Adult Participants and Adult Participants With Confirmed PKD1 Mutation-associated Autosomal Dominant Polycystic Kidney Disease

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Trial Profile

A Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Intravenously Administered PYC-003, a Peptide-phosphorodiamidate Morpholino Oligonucleotide Conjugate, in Healthy Adult Participants and Adult Participants With Confirmed PKD1 Mutation-associated Autosomal Dominant Polycystic Kidney Disease

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 07 Mar 2026

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At a glance

  • Drugs PYC 003 (Primary)
  • Indications Polycystic kidney disease; Telomeric 22q13 Monosomy Syndrome
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors PYC Therapeutics

Most Recent Events

  • 27 Feb 2026 Treatments section is updated to add "Active Comparator: Part C (MAD - ADPKD)" arm, and 4 new safety primary end-points are added.
  • 27 Feb 2026 Number of treatment arms are increased from 2 to 3 by the addition of "Active Comparator: Part C (MAD - ADPKD) arm".
  • 27 Feb 2026 Planned number of patients changed from 56 to 116.

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