NCT06714006

Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003

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: Phase 1
• Study type: Interventional
• Enrollment: 116 (estimated)
• Sponsor: PYC Therapeutics · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Accepted

Summary

This is a Phase 1, First-in-Human study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and immunogenicity of PYC-003 in healthy adult participants and adult participants with confirmed PKD1 mutation-associated Autosomal Dominant Polycystic Kidney Disease (ADPKD) There are 3 parts in this study, i.e. Part A, Part B and Part C

Detailed description

Part A (SAD - Healthy) will be conducted as a randomized, double-blind, placebo-controlled, SAD study to assess the safety, tolerability, PK, PD, and immunogenicity of PYC-003 in healthy adult participants. The anticipated number of participants across 4 Part A (SAD - Healthy) cohorts is approximately 32 participants. On Day 1, each participant will receive the investigational product (IP; ie, PYC-003 or placebo), as a single intravenous (IV) infusion. All Part A (SAD - Healthy) cohorts will first dose 2 sentinel participants in a blinded manner on Day 1. Part B (SAD - ADPKD) will be conducted as an open-label single ascending dose (SAD) study to assess the safety, tolerability, PK, PD, and immunogenicity of PYC-003 in adult participants with confirmed PKD1 mutation-associated ADPKD. The anticipated number of participants across 3 Part B (SAD - ADPKD) cohorts is approximately 18 participants. On Day 1, each participant will receive PYC-003 as a single IV infusion. Part C (MAD-ADPKD) will be conducted as an open label multiple ascending dose (MAD) study to assess the safety, tolerability, PK, PD, and immunogenicity of PYC-003 in adult participants with confirmed PKD1 mutation-associated ADPKD. The anticipated number of participants across 2 Part C (MAD - ADPKD) cohorts is approximately 24 participants. Each participant will receive PYC-003 as an IV infusion either once every 6 weeks for 13 weeks (i.e., dosing on Day 1, Day 43, and Day 85) or once every 8 weeks for 17 weeks (i.e., dosing on Day 1, Day 57, and Day 113).

Conditions

• Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Interventions

Type	Name	Description
DRUG	PYC-003	A peptide-phosphorodiamidate morpholino oligonucleotide conjugate administered as a single intravenous infusion

Timeline

• Start date: 2025-04-07
• Primary completion: 2026-09-01
• Completion: 2027-03-01
• First posted: 2024-12-03
• Last updated: 2026-02-27

Locations

9 sites across 2 countries: Australia, New Zealand