Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003
NCT ID: NCT06714006
Last Updated: 2025-11-28
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
56 participants
INTERVENTIONAL
2025-04-07
2026-01-31
Brief Summary
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Detailed Description
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The anticipated number of participants across 3 Part A (SAD - Healthy) cohorts is approximately 24 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 SAD study to assess the safety, tolerability, PK, PD, and immunogenicity of PYC-003 in adult participants with confirmed PKD1mutation-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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
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. On Day 1, each participant will receive either PYC-003 or placebo, as a single intravenous infusion.
Part B (SAD - ADPKD) will be conducted as an open-label SAD study to assess the safety, tolerability, PK, PD, and immunogenicity of PYC-003 in adult participants with confirmed PKD1 mutation-associated ADPKD. On Day 1, each participant will receive PYC-003 as a single IV infusion.
TREATMENT
TRIPLE
Part B will be open-label study.
Study Groups
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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
PYC-003
A peptide-phosphorodiamidate morpholino oligonucleotide conjugate administered as a single intravenous infusion
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.
PYC-003
A peptide-phosphorodiamidate morpholino oligonucleotide conjugate administered as a single intravenous infusion
Interventions
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PYC-003
A peptide-phosphorodiamidate morpholino oligonucleotide conjugate administered as a single intravenous infusion
Eligibility Criteria
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Inclusion Criteria
2. 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.
3. 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).
4. BMI ≥ 18.0 and ≤ 32.0 kg/m2 and weight ≥ 50 kg.
5. Non-smoker and must not have used any tobacco or nicotine products within 2 months prior to Screening.
6. 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:
1. Platelets \> 150 × 10\^9/L
2. Total white blood cell count \> 3.0 × 10\^9/L
3. Absolute neutrophil count \> 1.5 × 10\^9/L
4. Hemoglobin \> 110 g/L for females and \> 120 g/L for males
5. Total and direct bilirubin \< 1.5 × ULN, unless elevated bilirubin is associated with a known benign condition (e.g., Gilbert's syndrome)
6. Alanine aminotransferase (ALT) \< 1.5 × ULN
7. Aspartate aminotransferase (AST) \< 1.5 × ULN
8. Alkaline phosphatase (ALP) \< 1.5 × ULN
9. 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.
Exclusion Criteria
2. Use of (or anticipated use of) Tolvaptan and/or metformin administration within 30 days prior to the first administration of IP until study completion.
3. Any renal or systemic pathology other than ADPKD or any other condition or prior therapy that in the opinion of the PI or designee would make the participant unsuitable for this study.
4. Has only 1 kidney or has a kidney transplant.
5. History of borderline to low blood magnesium and potassium levels and/or Screening or Day -1 blood magnesium level \< 0.7 mmol/L and potassium levels \< 3.5 mmol/L.
Note: Repeat testing (i.e., 1 repeat per parameter) at Screening or Day -1 is permitted for out-of-range values following approval by the PI or designee.
6. Proteinuria \> 500 milligrams per 24 hours.
7. Hematuria (urine albumin:creatinine ratio \> 30 mg/mmoL, or hematuria \> ++ on dipstick, or \> 100 cells per high-power field on microscopy)and/or urinary abnormalities at Screening deemed by the PI or designee to be of moderate or higher severity.
18 Years
65 Years
ALL
Yes
Sponsors
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PYC Therapeutics
INDUSTRY
Responsible Party
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Locations
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South Coast Renal, Brockway House, Level 1, Suite 8, 82-86 Queen Street,
Southport, Gold Coast, Australia
Liverpool Hospital, Clinic G-Reception 133, Level 1, Clinical Building, Burnside Drive
Liverpool, New South Wales, Australia
Westmead Hospital, Clinical Research Unit, Level 6, B Wing/Building, Hawkesbury Road
Westmead, New South Wales, Australia
Mater Hospital Brisbane
South Brisbane, Queensland, Australia
Sunshine Hospital, Western Centre for Health Research and Education, Level 3, 176-190 Furlong Road
Saint Albans, Victoria, Australia
Linear Clinical Research
Joondalup, Western Australia, Australia
Countries
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Central Contacts
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Sreenivasu Mudumba Chief Research & Development Officer, PhD
Role: CONTACT
Facility Contacts
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Other Identifiers
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PYC-003-CL-001
Identifier Type: -
Identifier Source: org_study_id
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