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
Location: See all (6) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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 2 parts in this study, i.e. Part A and Part B.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: t
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• Male or female aged 18 to 65 years (inclusive) at the time of informed consent.

• ADPKD diagnosis as confirmed by the presence of genetic mutations associated with ADPKD, including, but not limited to, the presence of PKD1 mutation. Note: Where genotyping is not included the medical history for a participant, genotyping may be completed at Pre-Screening.

• Class 1C, 1D, or 1E per Mayo Imaging Classification System for Predicting Kidney Outcomes in ADPKD (Irazabal et al. 2015) (based upon prior magnetic resonance imaging \[MRI\] or computed tomography \[CT\] scan obtained \> 6 months prior to Screening, or MRI obtained during Pre-Screening).

• BMI ≥ 18.0 and ≤ 32.0 kg/m2 and weight ≥ 50 kg.

• Non-smoker and must not have used any tobacco or nicotine products within 2 months prior to Screening.

• Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73 m2 via the CKD EPI 2021 calculation (Inker et al. 2021).

⁃ 8\. Hematology and serum chemistry results at Screening that meet the following criteria:

• Platelets \> 150 × 10\^9/L

• Total white blood cell count \> 3.0 × 10\^9/L

• Absolute neutrophil count \> 1.5 × 10\^9/L

• Hemoglobin \> 110 g/L for females and \> 120 g/L for males

• Total and direct bilirubin \< 1.5 × ULN, unless elevated bilirubin is associated with a known benign condition (e.g., Gilbert's syndrome)

• Alanine aminotransferase (ALT) \< 1.5 × ULN

• Aspartate aminotransferase (AST) \< 1.5 × ULN

• Alkaline phosphatase (ALP) \< 1.5 × ULN

• Gamma-glutamyl transferase \< 2 × ULN Note: Screening laboratory testing may be repeated once at the discretion of the PI or designee to confirm out-of-range(exclusionary) results.

Locations
Other Locations
Australia
Linear Clinical Research
RECRUITING
Joondalup
Liverpool Hospital, Clinic G-Reception 133, Level 1, Clinical Building, Burnside Drive
RECRUITING
Liverpool
Sunshine Hospital, Western Centre for Health Research and Education, Level 3, 176-190 Furlong Road
RECRUITING
Saint Albans
Mater Hospital Brisbane
RECRUITING
South Brisbane
South Coast Renal, Brockway House, Level 1, Suite 8, 82-86 Queen Street,
RECRUITING
Southport
Westmead Hospital, Clinical Research Unit, Level 6, B Wing/Building, Hawkesbury Road
RECRUITING
Westmead
Contact Information
Primary
Sreenivasu Mudumba Chief Research & Development Officer, PhD
pkd@pyctx.com
510-423-2680
Time Frame
Start Date: 2025-04-07
Estimated Completion Date: 2026-01
Participants
Target number of participants: 56
Treatments
Placebo_comparator: Part A (SAD - Healthy)
Part A will be conducted as a randomized, double-blend, placebo-controlled, Single Ascending Dose Study to assess the safety, tolerability, Pharmacokinetic, Pharmacodynamic, and immunogenicity of PYC-003 in healthy adult participants
Active_comparator: Part B (SAD - ADPKD)
Part B will be conducted as an open-label Single Ascending Dose study to assess the safety, tolerability, Pharmacokinetic, Pharmacodynamic, and immunogenicity of PYC-003 in adult participants with confirmed PKD1 mutation-associated ADPKD.
Sponsors
Leads: PYC Therapeutics

This content was sourced from clinicaltrials.gov

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