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

PHASE1RECRUITING

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.

Study details:

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 5 Part A (SAD - Healthy) cohorts is approximately 40 participants. On Day 1, each participant will receive the investigational product (IP; ie, PYC-003 or placebo), as a single intravenous (IV) infusion.

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 5 Part B (SAD - ADPKD) cohorts is approximately 30 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 4 Part C (MAD - ADPKD) cohorts is approximately 48-96 participants. Each participant will receive PYC-003 as an IV infusion either once every 6 weeks for 13 weeks or once every 8 weeks for 17 weeks.

Part D will be an extension study for participants that complete Part C where participants will continue receiving doses for up to 96 weeks.

Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

  1. Male or female aged 18 to 60 years (inclusive) at the time of informed consent.
  2. At the discretion of the PI or designee, in good general health, with no significant medical history, and have no clinically significant abnormalities on physical examination at Screening and/or before the first administration of IP
  3. BMI between ≥ 18.0 and ≤ 32.0 kg/m2 and weight ≥ 50 kg
  4. Nonsmoker and must not have used any tobacco products within 2 months prior to Screening.
  5. Clinical laboratory values within normal range as specified by the testing laboratory, unless deemed not clinically significant by the PI or designee
  6. Estimated glomerular filtration rate (eGFR) ≥ 80 mL/min/1.73 m2 via the Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) 2021 calculation
  7. Women of childbearing potential (WOCBP) must agree to use an acceptable, highly effective, double barrier method of contraception from the start of Screening until study completion.
  8. Males must be surgically sterile or, if engaged in sexual relations with a WOCBP, must agree to use an acceptable, highly effective, double barrier method of contraception from the start of Screening until study completion
  9. Females must agree not to donate ova from the first administration of IP until 30 days following study completion
  10. Males must agree not donate sperm from the first administration of IP until 90 days following study completion
  11. Able and willing to attend the necessary visits to the study site
  12. Able and willing to adhere to caffeine, alcohol, and nicotine-containing product restrictions
  13. Able and willing to provide written informed consent after the nature of the study has been explained and prior to the commencement of any study procedures.

Exclusion criteria

  1. Females who are pregnant, breastfeeding, or plan to become pregnant during the course of the study
  2. Underlying physical or psychological medical condition that, in the opinion of the PI or designee, would make the participant unlikely to comply with the protocol or complete the study per protocol
  3. Has only 1 kidney or has received a kidney transplant
  4. Blood donation or has significant blood loss (> 500 mL) within 30 days prior to the first administration of IP
  5. Plasma donation within 7 days prior to the first administration of IP
  6. Fever (body temperature >38°C) or symptomatic viral or bacterial infection within 2 weeks prior to first administration of IP
  7. Infections requiring parenteral antibiotics within 6 months prior to Screening
  8. Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), HIV antibody.
  9. History of life threatening infection (eg, meningitis).
  10. Vaccination with a live vaccine within 4 weeks prior to the first administration of IP.
  11. Poor venous access.
  12. History of severe allergic or anaphylactic reactions, or sensitivity to the IP or its constituents.
  13. History of malignancy, except for non-melanoma skin cancer, excised more than 1 year prior to screening and cervical intraepithelial neoplasia that has been successfully cured more than 2 years prior to Screening.
  14. Abnormal ECG findings at Screening, Day -1, or predose that are considered by the PI or designee to be clinically significant.
  15. History or presence of a condition associated with significant immunosuppression
  16. Exposure to any drugs that cause significant immunosuppression (including experimental therapies as part of a clinical study) within 4 months or 5 half lives (whichever is longer), prior to Screening.
  17. ALP, AST, and ALT >1.5 × ULN at Screening or Day -1
  18. History of borderline to low blood magnesium and potassium levels and/or Screening or Day -1 blood magnesium and potassium levels <0.7mmol/L and potassium levels < 3.5mmol/L
  19. Active infection of the urinary tract (ie, kidney, bladder).
  20. Positive toxicology screening panel (urine test including qualitative identification of amphetamines, barbiturates, benzodiazepines, cocaine, methamphetamine, methadone, opiates, phencyclidine, tetrahydrocannabinol [THC], tricyclic antidepressants), or alcohol breath test.
  21. History of substance abuse or dependency or history of recreational IV drug use over the last 5 years (by self-declaration).
  22. Regular alcohol consumption defined as > 14 standard drinks per week for females and > 21 standard drinks for males (where 1 standard drink = 375 mL of mid-strength beer [3.5% alcohol/volume], 100 mL wine [13.5% alcohol/volume] or 30 mL of spirits [40% alcohol/volume]) or > 4 standard drinks on any single day.
  23. Unwilling to abstain from alcohol for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.
  24. Use of any investigational medical device or investigational drug within 30 days or 5 half lives of the investigational drug (whichever is longer) prior to the first administration of IP.
  25. Use of (or anticipated use of) the following: Any prescription drugs (other than hormonal contraception; oral contraceptive pills, long-acting implantable hormones, injectable hormones, a vaginal ring, or an intrauterine device [IUD]) within 14 days prior to the first administration of IP and during the course of the study without prior approval of the PI and MM. Any over-the-counter medication, herbal remedies, supplements or vitamins within 7 days prior to the first administration of IP and during the course of the study without prior approval of the PI and MM.
  26. Unwilling to refrain from strenuous exercise (including weightlifting) for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.
  27. Anything that the PI considers would jeopardize the safety of the participant, prevent complete participation in the study, or compromise interpretation of study data.

Eligibility

Age eligible for study : NA

Healthy volunteers accepted : No

Gender eligible for study: ALL

Things to know

Study dates

Study start:

Primary completion:

Study completion finish:

study type

Study type

TREATMENT

phase

Phase

    PHASE1

trial

Study ID

NCT06714006

Intervention or treatment

DRUG: PYC-003

Conditions

  • Autosomal Dominant Polycystic Kidney Disease (ADPKD)

  • Condition: Autosomal Dominant Polycystic Kidney Disease (ADPKD)

  • DRUG: PYC-003

  • Southport, Gold Coast, Australia and more

  • Sponsor: PYC Therapeutics

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Research sites nearby

Closest Location:

South Coast Renal, Brockway House, Level 1, Suite 8, 82-86 Queen Street,

Select from list below to view details:

  • 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

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Who is running the clinical trial?

PT

PYC Therapeutics

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/ArmIntervention/Treatment
PLACEBO_COMPARATOR: Part A (SAD - Healthy)
  • Part A will be conducted as a randomized, double-blend, placebo-controlled, Single Ascending Dose Study in healthy adult participants. On Day 1, each participant will receive the investigational product (IP; i.e., 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.
Drug: PYC-003
    EXPERIMENTAL: Part B (SAD - ADPKD)
    • Part B will be conducted as an open-label Single Ascending Dose study in adult participants with confirmed PKD1 mutation-associated ADPKD. On Day 1, each participant will receive PYC-003 as a single IV infusion.
    Drug: PYC-003
      EXPERIMENTAL: Part C (MAD - ADPKD)
      • Part C (MAD - ADPKD) will be conducted as an open-label MAD study in adult participants with confirmed PKD1 mutation-associated ADPKD. 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).
      Drug: PYC-003
        EXPERIMENTAL: Part D - Open Label Extension
        • Part D will be an extension study for participants that complete Part C. Participants will receive doses either every 6 or 8 weeks for 96 weeks .
        Drug: PYC-003

          What is the study measuring?

          Primary Outcome

          MeasureDescriptionTime Frame
          (Part A and B) Number of participants with treatment-related adverse events as assessed by CTCAE V5.0To evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD. The incidence, severity, and relatedness of TEAEs and treatment-emergent SAEs will be recorded24 weeks
          (Part A and B) To evaluate the changes in Heart rate via 12-lead ECG (local) following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes in ECG PR interval from baseline via 12-lead ECG (local) following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes in ECG QRS duration from baseline via 12-lead ECG (local) following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes in ECG QT interval from baseline via 12-lead ECG (local) following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes in ECG QTcF interval from baseline via 12-lead ECG (local) following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes in baseline body temperature following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes in baseline systolic and diastolic blood pressure following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes in baseline pulse rate following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes in baseline respiratory rate following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes physically via complete and symptom-directed physical examination following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD Complete and symptom-directed physical examinations are to be performed by a licensed physician. Complete physical examinations include general appearance, head, ears, eyes, nose, throat, dentition, thyroid, chest (heart, lungs), abdomen, skin, neurological, extremities, back, neck, musculoskeletal, and lymph nodes.24 weeks
          (Part A and B) To evaluate the changes from baseline in serum potassium, serum magnesium, serum sodium, serum osmolality, serum cystatin C, and serum creatinine following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes from baseline in urine potassium, urine magnesium, urine creatinine, and urine osmolality following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) To evaluate the changes in standard renal and hepatic clinical chemistry parameters following a single dose of PYC-003 administered intravenouslyTo evaluate the safety and tolerability of PYC-003 following a single dose administered intravenously to healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks

          Secondary Outcome

          MeasureDescriptionTime Frame
          (Part A and B) The peak plasma concentration, Cmax of PYC-003 following a single dose administered intravenouslyThis is done to characterize the plasma PK of PYC-003 in participants following a single dose administered intravenously, for both healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) The peak plasma concentration, Tmax (time to maximum observed plasma drug concentration) of PYC-003 following a single dose administered intravenouslyThis is done to characterize the plasma PK of PYC-003 in participants following a single dose administered intravenously, for both healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) The Plasma PK parameter, t½ (half life) of PYC-003 will be determined following a single dose administered intravenously to adult participantsThis is done to characterize the plasma PK of PYC-003 in participants following a single dose administered intravenously, for both healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) The Plasma PK parameter, AUC0-24 (Area under the plasma concentration versus time curve over the last 24 hour) of PYC-003 will be determined following a single dose administered intravenouslyThis is done to characterize the plasma PK of PYC-003 in participants following a single dose administered intravenously, for both healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) Plasma PK parameter, AUC0-inf (extrapolated area under concentration-time curve) of PYC-003 will be determined following a single dose administered intravenously to adult participantsThis is done to characterize the plasma PK of PYC-003 in participants following a single dose administered intravenously, for both healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) The Plasma PK parameter, AUC%extrap (the area under the curve) of PYC-003 will be determined following a single dose administered intravenouslyThis is done to characterize the plasma PK of PYC-003 in participants following a single dose administered intravenously, for both healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) The Plasma PK parameter, kel (elimination rate constant) of PYC-003 will be determined following a single dose administered intravenouslyThis is done to characterize the plasma PK of PYC-003 in participants following a single dose administered intravenously, for both healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) The Plasma PK parameter, CL (Total drug clearance from plasma) of PYC-003 will be determined following a single dose administered intravenouslyThis is done to characterize the plasma PK of PYC-003 in participants following a single dose administered intravenously, for both healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
          (Part A and B) The Plasma PK parameter, Vz (Volume of distribution) of PYC-003 will be determined following a single dose administered intravenouslyThis is done to characterize the plasma PK of PYC-003 in participants following a single dose administered intravenously, for both healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD24 weeks
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          References

          Clinical Trials Gov: Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003

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