Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorPYC Therapeutics
About this trial
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 4 parts in this study, i.e. Part A, Part B, Part C and Part D.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
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
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.
Clinical laboratory values within normal range or deemed not clinically significant by the PI
Disqualifiers
Females who are pregnant, breastfeeding, or plan to become pregnant during the course of the study.
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.
Has only 1 kidney or has received a kidney transplant.
Blood donation or had significant blood loss (> 500 mL) within 30 days prior to the first administration of IP.
Trial design
Design model
Parallel
Treatments tested in this trial
PYC-003
DrugA peptide-phosphorodiamidate morpholino oligonucleotide conjugate administered as a single intravenous infusion
Treatment groups
166 Participants
are divided into 4 treatment groupsGroup A: Part A (SAD - Healthy)Placebo comparator 1 intervention
Group B: Part B (SAD - ADPKD)Experimental treatment 1 intervention
Group C: Part C (MAD - ADPKD)Experimental treatment 1 intervention
Group D: Part D - Open Label ExtensionExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
[Part A, B, C and D] Number of participants experiencing treatment emergent adverse events (AE) as assessed by CTCAE V5.0
The incidence, severity, and relatedness of treatment emergent adverse events and treatment-emergent Serious Adverse Events will be recorded An AE is any event, side-effect, or other untoward medical occurrence that occurs in conjunction with the use of a medicinal product in humans, whether or not considered to have a causal relationship to this treatment. An AE can, therefore, be any unfavourable and unintended sign (that could include a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
Time frame
Up to 98 weeks2
[Part A, B, C and D] Changes from baseline in vital signs (body temperature)
Time frame
Up to 98 weeks3
[Part A, B, C and D] Changes from baseline in vital signs (systolic and diastolic blood pressure)
Time frame
Up to 98 weeks4
[Part A, B, C and D] Changes from baseline in vital signs (pulse rate)
Time frame
Up to 98 weeksSecondary outcomes
1
[Part A, B, C and D] Peak plasma concentration (Cmax) of PYC003
Time frame
Up to 98 weeks2
[Part A, B, C and D] Time to maximum observed plasma drug concentration (Tmax) of PYC003
Time frame
Up to 98 weeks3
[Part A, B and C] Area under the plasma concentration-time curve, from time zero to 24 hours post dose of PYC-003 (AUC0-24)
Time frame
Up to 36 weeks4
[Part A, B and C] Area under the plasma concentration-time curve, from time zero to the last time point with measurable analyte concentration after PYC-003 dose (AUC0-last)
Time frame
Up to 36 weeksSponsors and contacts
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Source ClinicalTrials.gov