CAY001 for Healthy Volunteers

Overseen By Iris Moscoso, CCRC

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Cayuga Biotech, Inc.

Must not be taking: Prescription drugs, Illicit drugs

Disqualifiers: Thrombosis, Bleeding disorders, Hypertension, others

Compensation: Varies

Number of Visits: 1

Duration: 1 day

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to assess the safety of a new treatment called CAY001. Researchers seek to understand how CAY001 behaves in the body and its safety for human use. The study involves healthy men and women who do not smoke or use drugs and are in good health, as confirmed by a doctor. Participants will receive either CAY001 or a placebo (a harmless pill without the treatment) to compare effects. Those who feel healthy and have no major health issues might be suitable for this trial. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

Yes, you will need to stop taking any prescription, investigational, or illicit drugs 30 days before the trial, and over-the-counter products, dietary supplements, and herbal products 14 days before the trial.

Is there any evidence suggesting that CAY001 is likely to be safe for humans?

Research shows that CAY001 remains in the early stages of human testing. This treatment is under investigation for its potential to address bleeding issues. However, detailed safety information for CAY001 is not yet available. Some reports suggest that side effects are common, meaning some participants might experience them.

As a Phase 1 trial, this marks the first time CAY001 is tested in humans. Phase 1 trials primarily focus on assessing the safety of a treatment. Researchers closely monitor for any side effects, but there isn't enough information yet to fully understand the safety profile of CAY001. Prospective participants should discuss any concerns with the research team. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

CAY001 is unique because it introduces a novel active ingredient that targets pathways not addressed by current treatments. While existing options for similar conditions might rely on conventional mechanisms like inhibition of specific enzymes, CAY001 works by modulating a different set of biological processes, potentially offering a new approach to managing symptoms or conditions. Researchers are excited about CAY001 because its innovative mechanism may lead to more effective outcomes with possibly fewer side effects, offering hope for improved patient experiences.

What evidence suggests that CAY001 might be an effective treatment?

Research has shown that CAY001 has potential in early studies for treating blood clotting problems, especially in serious injuries. In animal tests, those given CAY001 had a better chance of survival, with none dying early from blood loss compared to those not treated. This suggests CAY001 might help the body stop dangerous bleeding. Although the current study involves healthy volunteers, these early results highlight CAY001's potential to improve blood clotting in critical situations. Human trials are just starting, so more information is needed to confirm these effects in people. ^{1 2 4 6 7}

Who Is on the Research Team?

Charles Pollack, MD

Principal Investigator

Cayuga Biotech, Inc.

Are You a Good Fit for This Trial?

This trial is for healthy men and women who want to participate in a study testing a new medication, CAY001. Specific eligibility criteria are not provided, but typically participants must meet certain health standards.

Inclusion Criteria

The subject is able to provide written informed consent

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. Specific inclusionary laboratory values prior to randomization require the following: 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. 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

My BMI is between 18 and 30, and I weigh at least 61 kg.

Exclusion Criteria

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 clinically significant abnormal findings in physical examination, vital signs, laboratory assessments (other than those discussed in the inclusion criteria), and ECG parameters identified during screening or check-in that in the evaluating Investigator's opinion could interfere with the study. Note: abnormal test results may be repeated once for confirmation; 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 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: Recent history (within 6 months preceding the first dose of study drug) of gastrointestinal (GI) bleeding that required unscheduled medical evaluation; History of intracranial, intraocular, retroperitoneal, or spinal bleeding; Recent major trauma (within 6 months preceding the first dose of the study drug), History of hemorrhagic disorders, e.g. hemophilia, von Willebrand's disease, Hermansky-Pudlak syndrome, History of severe head trauma, intracranial hemorrhage, intracranial neoplasm, arteriovenous malformation, aneurysm, or proliferative retinopathy; Subject reported history of blood in the stool, Use of any prescription, investigational or illicit drugs within 30 days (or 5 half-lives whichever is longer) prior to dosing; Use of any over-the-counter (OTC) products, dietary supplements and herbal products (ie. St. John's Wort or fish oil) within 14 days of dosing through the Day 7 Safety Follow-up Visit; Known active hepatitis B or C infection, human immunodeficiency virus (HIV) infection, or known other immune deficiency disease at screening; Females who are pregnant, plan to become pregnant within 3 months of the last dose of study drug, or are breastfeeding a child; History of or treatment for alcoholism or drug addiction within 1 year; Receipt of blood transfusion, blood-derived products, or coagulation factors within 1 month prior to Day 1; Prior exposure to CAY001; An employee, family member, or student of the Sponsor, Investigator, or clinical site(s); Unable to adhere to or understand the requirements of the protocol; Consume caffeine containing food or beverages 48 hours prior to check in and during study confinement period; Females who are actively menstruating on the day of dosing (Day 1); Subject meets eligibility criteria, but study is filled.

I don't have any health issues that could affect the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single ascending dose of CAY001 or placebo to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including changes in vital signs, physical examination, ECGs, and laboratory tests

28 days

Multiple visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• CAY001

Trial Overview The trial is testing the safety and effects of different doses of CAY001 compared to a placebo. It's the first time this drug is being given to humans, with around 48 people participating in several groups receiving increasing doses.

How Is the Trial Designed?

Group I: CAY001 Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Cayuga Biotech, Inc.

Lead Sponsor

Trials

1

Recruited

50+

Published Research Related to This Trial

In a review of 22 First-in-Human trials involving 1,250 healthy volunteers, the overall risk of severe adverse events (AEs) was low, with only 0.4% of subjects experiencing serious AEs related to the active drug or procedure.

Participants receiving active treatment experienced a higher incidence of AEs compared to those on placebo, with 88.2% of reported AEs being mild, indicating that while there are risks, most side effects were not severe.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

How safe are our studies? Analysis of adverse events in Bayer First-in-Human trials from 2006 to 2016 . Jung, D., Boettcher, MF., Wensing, G.[2020]

In a comprehensive review of 54 phase-I studies involving 1015 healthy volunteers over 10 years, the overall incidence of adverse events was found to be 12.8%, with a higher rate for active drugs (13.7%) compared to placebo (7.9%).

Most adverse events were minor, with only 3% classified as severe, and no deaths or life-threatening events reported, highlighting that while adverse events are common in phase-I trials, they are typically not serious.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse events in phase-I studies: a report in 1015 healthy volunteers. Sibille, M., Deigat, N., Janin, A., et al.[2019]

In a five-year analysis of 1,559 healthy volunteers across 142 phase I studies, the overall incidence of adverse events (AEs) was 8.8%, with most AEs being mild or moderate in severity, indicating that while AEs are common, they are generally not severe.

The incidence of AEs was significantly higher in multiple-dose studies compared to single-dose trials, highlighting the importance of study design in assessing the safety profile of active drugs, with a notable increase in AEs on the first day of drug administration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse events in volunteers participating in phase I clinical trials: a single-center five-year survey in 1,559 subjects. Lutfullin, A., Kuhlmann, J., Wensing, G.[2019]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06845839?aggFilters=status%3Anot%2Cphase%3A

Study to Evaluate CAY001 in Healthy Volunteers This is a Phase 1, first-in-human, randomized, double-blind, placebo-controlled, single ascending dose, sequential group study to evaluate the safety, ...

2. ctv.veeva.com ctv.veeva.com/study/study-to-evaluate-cay001-in-healthy-volunteers

Study to Evaluate CAY001 in Healthy Volunteers This is a Phase 1, first-in-human, randomized, double-blind, placebo-controlled, single ascending dose, sequential group study to evaluate ...

3. withpower.com withpower.com/trial/phase-1-healthy-subjects-hs-3-2025-5070f

CAY001 for Healthy Volunteers · Info for Participants This is a Phase 1, first-in-human, randomized, double-blind, placebo-controlled, single ascending dose, sequential group study to evaluate the safety, ...

4. whnt.com whnt.com/business/press-releases/ein-presswire/864202165/full-data-on-cayugas-novel-synthetic-compound-in-stopping-life-threatening-hemorrhage-shows-promise

Full Data on Cayuga's Novel Synthetic Compound in ... PolyP-SNP Complex Has Potential to Restore Blood Clotting Under Most Dangerous Conditions of Traumatic Injury.

5. firstwordpharma.com firstwordpharma.com/story/5945973

Cayuga's CAY001 Significantly Increases Survival and ... CAY001 data demonstrated that no animal treated with CAY001 died prematurely due to blood loss, compared to untreated control animals. CAY001 ...

6. news10.com news10.com/business/press-releases/ein-presswire/864202165/full-data-on-cayugas-novel-synthetic-compound-in-stopping-life-threatening-hemorrhage-shows-promise

Full Data on Cayuga's Novel Synthetic Compound in ... Cayuga Biotech is developing novel therapeutics to address life-threatening hemorrhage by harnessing the body's innate ability to stop bleeding.

7. trial.medpath.com trial.medpath.com/clinical-trial/98b1dfc85fac58fe/nct06845839-phase1-safety-cay001-healthy-volunteers

Study to Evaluate CAY001 in Healthy Volunteers | MedPath This is a Phase 1, first-in-human, randomized, double-blind, placebo-controlled, single ascending dose, sequential group study to evaluate ...