Study to Evaluate CAY001 in Healthy Volunteers

NCT06845839 | Recruiting Phase 1 DMC FDA Drug

• 3 other identifiers: CAY001-01MT21006.078MTEC-21-06-MPAI-078
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase 1, first-in-human, randomized, double-blind, placebo-controlled, single ascending dose, sequential group study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of CAY001 when administered to healthy male and female subjects. Three dose levels will be evaluated with a total of approximately 24 subjects.

Conditions

Healthy

Keywords

hemorrhage; polyphosphate; hemostatic drug; coagulation; clotting

Outcome Measures

Primary Outcomes (22)

• Incidence and severity of serious adverse events from Baseline through Day 28.

  28 days

• Incidence and severity of treatment related adverse events from Baseline through Day 28

  28 days

• Changes in physical examination findings from Baseline through Day 28

  Physical examination includes general appearance, HEENT (head, eyes, ears, nose, throat), chest/lungs, heart, abdominal, neurological, extremities, skin, and palpable lymph nodes. Physical examination will be conducted at screening, at multiple timepoints during the study (symptom-directed).

  28 days

• Changes in electrocardiograms (ECGs) from Baseline through Day 28

  A standard 12-lead ECG will be performed after at least 10 minutes in the supine position. ECG data collected includes PR, QT interval, QRS duration, QTcF and heart rate.

  28 days

• Changes in systolic and diastolic blood pressure in mmHg from Baseline through Day 28

  Vital signs, including blood pressure will be collected with the subject supine and after resting for 10 minutes.

  28 days

• Changes in blood chemistry parameters (calcium, phosphorus, glucose, BUN, uric acid, bilirubin, creatinine and magnesium) in mg/dL, from Baseline through Day 28

  28 days

• Changes in hematology parameters (white blood cell, red blood cell, platelets) in x10E3/uL, from Baseline through Day 28

  28 days

• Changes in coagulation parameters (aPTT and PT) in seconds, from Baseline through Day 28

  28 days

• Changes in urinalysis from Baseline through Day 28

  Urinalysis evaluations are microscopic examination and specific gravity, pH, protein, glucose, ketones, blood, urobilinogen, bilirubin, nitrites and leucocyte values.

  28 days

• Changes in temperature in degrees Celsius from Baseline through Day 28

  Vital signs, including temperature, will be collected with the subject supine and after resting for 10 minutes.

  28 days

• Changes in pulse in beats per minute, from Baseline through Day 28

  Vital signs, including pulse, will be collected with the subject supine and after resting for 10 minutes.

  28 days

• Changes in respiratory rate in breaths per minute, from Baseline through Day 28

  Vital signs, including respiratory rate, will be collected with the subject supine and after resting for 10 minutes.

  28 days

• Changes in blood chemistry (total protein, albumin) in g/dL, from Baseline through Day 28

  28 days

• Changes in blood chemistry (alkaline phosphatase, AST, ALT, GGT ) in IU/dL, from Baseline through Day 28

  28 days

• Changes in blood chemistry (creatinine kinase, ) in U/L, from Baseline through Day 28

  28 days

• Changes in blood chemistry (potassium, sodium, chloride) in mmol/L, from Baseline through Day 28

  28 days

• Changes in hematology parameters (hemoglobin, MCHC) in g/dL, from Baseline through Day 28

  28 days

• Changes in hematocrit in % from Baseline through Day 28

  28 days

• Changes in hematology parameter, MCV, in fL from Baseline through Day 28

  28 days

• Changes in hematology parameter, MCH in pg from Baseline through Day 28

  28 days

• Changes in coagulation parameter, D-Dimer, in mg/L FEU, from Baseline through Day 28

  28 days

• Changes in coagulation parameter, INR, in NA, from Baseline through Day 28

  28 days

Secondary Outcomes (9)

• Change in calibrated automated thrombogram-thrombin generation assay (CAT-TGA) from Baseline through Day 28

  28 days

• Determine the half-life (t½) (terminal)

  Pre-dose through 24 hours post-dose

• Determine the time to maximum concentration (Tmax)

  Pre-dose through 24 hours post-dose

• Determine the maximum observed concentration (Cmax)

  Pre-dose through 24 hours post-dose

• Determine the area under the curve (AUC)

  Pre-dose through 24 hours post-dose

Study Arms (2)

CAY001

EXPERIMENTAL

Drug: CAY001

Placebo

PLACEBO COMPARATOR

Other: Placebo

Interventions

CAY001 DRUG

Polyphosphate (PolyP) - silica nanoparticle (SNP) complex

CAY001

Placebo OTHER

Vehicle - aqueous solution

Placebo

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• The subject is able to provide written informed consent
• The subject is male or female (based on gender assigned at birth), 18-50 years of age inclusive
• The subject has a body mass index ≥ 18 and ≤ 30.0 kg/m2 and weighs at least 61 kg for males and females
• The subject is in good general health per Investigator evaluation for age as determined by medical history, vital signs, physical examination findings, screening laboratory test results, and 12-lead ECG results.
• Aspartate transaminase (AST), alanine transaminase (ALT) \< 1.5 x upper limit of normal (ULN)
• Total serum bilirubin and alkaline phosphatase levels \< 1.2 x ULN
• White blood cell (WBC) count, platelet count, and hemoglobin level within the normal range; out of range values are allowed if per the Investigator they are not deemed clinically significant
• PT/INR, partial thromboplastin time (PTT), and D-dimer level (age-adjusted) within the normal range
• Negative drug and alcohol tests at Screening and check-in (Day -1) and willing to abstain from alcohol and recreational drug use from the screening visit until the EOS/ET Visit;
• No use of any tobacco or nicotine-containing products within 3 months, negative cotinine test at Screening and check-in (Day -1), and willing to abstain from tobacco or nicotine use from the screening visit until the EOS/ET Visit;
• Male and female (women of childbearing potential \[WOCBP\]) subjects of childbearing potential must agree to the double-barrier method (i.e. male condom and spermicide or diaphragm and spermicide) or abstinence and refrain from sperm/egg donation throughout the study starting with the first dose of study treatment and for at least 3 months after the last dose of study drug. Hormonal contraceptives and intrauterine devices \[IUD\] must be stopped at least 3 months prior to the first dose of study treatment and for at least 3 months after the last dose of study drug. ▪ Note: Non-WOCBP includes healthy postmenopausal women who have undergone surgical menopause (hysterectomy, oophorectomy) or have been naturally menopausal, with no menstrual cycle for at least 24 months prior to Day 1; In the case of females, a negative serum pregnancy test (SPT) at Screening and a negative urine pregnancy test at check-in on Day -1.
• Able to communicate adequately with the Investigator and to comply with the requirements for the entire study

You may not qualify if:

• Any clinically significant acute or chronic medical condition that in the evaluating Investigator's opinion could interfere with the study
• Any history of plastic surgery involving silicone, any clinically significant illness, medical/surgical procedure, or trauma within 4 weeks of the first administration of the study drug or any planned surgical procedure that will occur during the study and cannot be delayed (from screening through the Day 28 EOS/ET Visit);
• Any of the following vital signs occurring after 10 minutes of supine rest at screening or check-in: ▪ Systolic blood pressure \>140 mm Hg ▪ Diastolic blood pressure \>90 mmHg ▪ Heart rate \<45 or \>100 beats per minute
• Any of the following ECG parameters at screening or check-in:
• Prolonged Fridericia-corrected QT interval (QTcF) \>450 msec for males and \> 460 msec for females, shortened QTcF \<340 msec, or pause \>3 seconds;
• Prolonged PR (PQ) interval \>200 msec, intermittent second- or third-degree atrioventricular (AV) block or AV dissociation
• Complete right or left bundle branch block (QRS \> 120 msec); left ventricular hypertrophy
• Family history of long QT syndrome or sudden cardiac death
• Have a known hypersensitivity or allergy to CAY001 components polyP or SNP, or to any ingredients in medication(s) to be received in this study;
• Any history of significant liver, spleen, or kidney conditions
• Any history of arterial or venous thrombosis or hypercoagulable or thrombotic condition, including any of the following
• History of transient ischemic attack, cardiovascular accident, stroke (ischemic or hemorrhagic), unstable angina, myocardial infarction, or peripheral arterial disease
• History of deep venous thrombosis, pulmonary embolus, thrombophlebilitis or arterial thrombosis
• History of known clotting disorders like factor V leiden syndrome, protein C or S deficiency, or antiphospholipid syndrome;
• An increased risk of bleeding, including but not limited to the following:

Sponsors & Collaborators

• Cayuga Biotech, Inc.: lead

Study Sites (1)

TKL Research

Fair Lawn, New Jersey, 07410, United States

RECRUITING

MeSH Terms

Conditions

Hemorrhage; Thrombosis

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms; Embolism and Thrombosis; Vascular Diseases; Cardiovascular Diseases

Study Officials

• Charles Pollack, MD

  Cayuga Biotech, Inc.

  STUDY DIRECTOR

Central Study Contacts

Iris Moscoso, CCRC

CONTACT

201-587-0500; irios@tklresearch.onmicrosoft.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 8, 2024
• First Posted: February 25, 2025
• Study Start: December 2, 2025
• Primary Completion (Estimated): October 30, 2026
• Study Completion (Estimated): October 30, 2026
• Last Updated: May 5, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)