KP001 for Safety Evaluation

No longer recruiting at 1 trial location

Overseen By Bruno Francoeur, MD

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Kokua Pharma Inc.

Must not be taking: MAO inhibitors

Disqualifiers: Drug abuse, Alcohol abuse, Anaphylaxis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

Duration: 5 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial evaluates the safety and metabolism of a new treatment, KP001 (an experimental treatment), compared to existing epinephrine injections. The study tests various inhalation methods for KP001 to determine the safest and most effective approach. It involves healthy, non-smoking adults who can hold their breath for at least 30 seconds. Participants will visit the clinic multiple times for treatment and check-ups to monitor any effects and how their bodies process the medication. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive it.

Will I have to stop taking my current medications?

Yes, you will need to stop taking any prescription medications 14 days before joining the trial and any over-the-counter medications 7 days before, except for occasional use of acetaminophen (up to 2 grams daily).

Is there any evidence suggesting that this trial's treatments are likely to be safe?

A previous study did not provide specific safety data for KP001. As this trial is in Phase 1, researchers are beginning to test KP001 in humans. At this stage, the focus is on assessing the safety and tolerability of the treatment. Typically, Phase 1 trials involve treatments tested in labs and often shown to be safe in animals before human testing.

Participants will receive KP001 through inhalation, with three different methods available, all involving inhalation. As KP001 is a new treatment, researchers will closely monitor any side effects or adverse reactions. If KP001 resembles existing treatments, it might have similar safety profiles, but this is not guaranteed.

For those considering joining this trial, safety remains the top priority for researchers. They will closely monitor participants to identify any issues and ensure the treatment's safety. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about KP001 because it offers a novel inhalation method and flexible dosing options that could improve drug delivery for respiratory conditions. Unlike traditional treatments that often rely on oral or injectable routes, KP001 is administered via inhalation, allowing for direct and potentially faster action in the lungs. The trial explores several dosing strategies: Treatment Arm A provides two doses over separate visits, Treatment Arm B offers repeated dosing for a cumulative effect, and Treatment Arm C involves a breath-hold technique to maximize lung absorption. These innovative approaches might offer enhanced efficacy and better patient adherence compared to current standards.

What evidence suggests that this trial's treatments could be effective?

Research has shown that KP001 is being developed as a potential new treatment for severe allergic reactions. Although direct evidence on KP001's effectiveness is limited, it is designed to mimic injected epinephrine, a well-known treatment that quickly alleviates allergy symptoms. KP001 is administered through inhalation, potentially offering a quicker and less invasive alternative to injections. In this trial, participants will join different treatment arms to evaluate the safety and absorption of KP001. Early studies are examining its safety and how the body absorbs and utilizes it. These studies aim to ensure it performs as well as current epinephrine products. ^{1 2 6 7 8}

Who Is on the Research Team?

Bruno Francoeur, MD

Principal Investigator

Syneos HealthClinique Inc.

Are You a Good Fit for This Trial?

This trial is for healthy adults who can participate in a study to test KP001, a potential new treatment for severe allergic reactions. Participants will need to stay at the research unit overnight and visit the clinic multiple times. Specific eligibility criteria are not provided.

Inclusion Criteria

Demonstrated ability to successfully complete pressurized metered dose inhaler (pMDI) training

My lung function tests are normal.

My BMI is between 18.5 and 30, and my weight is over 50 kg if male, or over 45 kg if female.

Exclusion Criteria

Positive urine drug screen, urine cotinine test, or alcohol breath test, at screening

Known reaction or sensitivity to sympathomimetic amines, or idiosyncratic reaction to epinephrine or any of the ingredients of KP001, placebo

History of anaphylaxis or other severe allergic reactions (e.g., angioedema)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 6 weeks

Treatment

Participants receive various doses of KP001 or placebo in a crossover design to evaluate safety, tolerability, and pharmacokinetics

5 weeks

5 in-patient dosing visits, separated by 1 week

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 week

1 safety follow-up visit approximately 1 week from last dose administered

What Are the Treatments Tested in This Trial?

Interventions

• KP001

Trial Overview KP001, which might be an alternative to injected epinephrine for severe allergies, is being tested against a placebo. The study looks at how safe it is and how the body processes it with different dosing schedules, including one without inhalation.

How Is the Trial Designed?

Group I: Treatment Arm C: Breath Hold Experimental Treatment 2 Interventions

Subjects will receive 0.5 mg of KP001 (or matching placebo) delivered as 4 rapidly administered sequential inhalations (4 total), dosed approximately 5 seconds apart, spaced over approximately 15 seconds while holding their breath during the entire dosing treatment and after treatment for a total minimum 30 seconds (or longer) before exhaling. Total dose in mg for Arm = 0.5 mg.

Group II: Treatment Arm B: Four Treatments Experimental Treatment 2 Interventions

Subjects will receive four sets of treatments of 0.25 mg (2 inhalations of 0.125 mg each time), totaling 1.0mg of KP001 (or matching placebo) over 2 visits separated by a 1-week washout period. IP will be delivered as 4 sets of 2 inhalations (8 total) spaced approximately 10 seconds apart. Each set of 2 inhalations will be spaced approximately 2 minutes apart. Subjects will repeat Arm therefore total dose in mg for Arm = 2.0 mg.

Group III: Treatment Arm A: Two Treatments Experimental Treatment 2 Interventions

Subjects will receive two sets of treatments of 0.25 mg (2 inhalations of 0.125 mg each time) of KP001 (or matching placebo) over 2 visits separated by a 1-week washout period. Investigational Product (IP) will be delivered as 1 set of 2 inhalations (2 total) spaced approximately 10 seconds apart. Subjects will repeat Arm therefore total dose in mg for Arm = 0.5 mg.

Who Is Running the Clinical Trial?

Kokua Pharma Inc.

Lead Sponsor

Published Research Related to This Trial

The University of Connecticut developed a customized online system for tracking and reviewing adverse events in clinical research, which allows for efficient classification and timely reporting of serious incidents.

In the previous academic year, the system reviewed 371 serious adverse events from over 900 studies, demonstrating its effectiveness in ensuring participant safety and providing recommendations for study modifications or suspensions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Systematic identification and classification of adverse events in human research. Silverman, DI., Cirullo, L., DeMartinis, NA., et al.[2006]

The recent fatal adverse event in the BIA-102474-101 clinical trial highlights significant limitations in the current safety review process for first-into-human trials, despite previous trials with similar compounds being conducted without incident.

To improve safety in future trials, it is crucial to implement sequential dosing strategies, thorough reviews of preclinical toxicity studies, and systematic risk assessments, especially for novel compounds that may pose higher risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Implications of the BIA-102474-101 study for review of first-into-human clinical trials. Eddleston, M., Cohen, AF., Webb, DJ.[2021]

The first-in-human clinical trial of BIA 10-2474 resulted in serious adverse events for 6 healthy volunteers, including 1 death, highlighting the heightened risks associated with novel investigational drugs transitioning from animal studies to human trials.

The symposium emphasized the need for careful planning and communication in clinical trials to minimize risks to participants, suggesting that a complex matrix of factors must be considered to ensure safety and integrity in first-in-human studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety in FIH Trials: A Summary of the Symposium "Fatal Drug Trial in Phase 1: Understanding Risk, Subject Safety, Timelines, and Cost". Greenberg, HE., van Iersel, MT., Westrick, ML., et al.[2020]

The safety of marketed drugs is a significant concern, as some commonly prescribed medications can lead to serious or life-threatening side effects in patients.

The ChEMBL resource will provide a curated drug safety data set, including toxicity classifications and black box warnings, which will be freely available and regularly updated to aid in drug safety research and discovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug Safety Data Curation and Modeling in ChEMBL: Boxed Warnings and Withdrawn Drugs. Hunter, FMI., Bento, AP., Bosc, N., et al.[2023]

In a clinical development program involving 1684 subjects and 2038 injections, OptiMARK demonstrated a safety profile comparable to Magnevist, with 31% of its injections associated with adverse events.

OptiMARK was found to be safe and well-tolerated, showing fewer adverse events compared to Magnevist (35%) and placebo (48%), indicating its potential as a reliable imaging agent.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The OptiMARK clinical development program: summary of safety data. Brown, JJ., Kristy, RM., Stevens, GR., et al.[2019]

Citations

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NCT06963411 | Phase 1 Crossover Study Evaluating the ... This study is a 5-period crossover design to evaluate the safety, tolerability, and PK of KP001 compared to placebo and to evaluate for the potential for ...

2. withpower.com withpower.com/trial/phase-1-anaphylaxis-4-2025-121ef

KP001 for Safety Evaluation · Info for Participants This trial is for healthy adults who can participate in a study to test KP001, a potential new treatment for severe allergic reactions.

3. mdpi.com mdpi.com/2305-6304/13/1/35

A Proof-of-Concept for Safety Evaluation of Inhalation ... This proof-of-concept study provides a means for practical application of the in vitro and in silico methods towards inhalation exposure safety assessment.

4. trial.medpath.com trial.medpath.com/clinical-trial/58e4376ddc352c12/safety-tolerability-efficacy-crg003-pompe-disease

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy ... This is a single-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of CRG003 injection ...

5. bmcpulmmed.biomedcentral.com bmcpulmmed.biomedcentral.com/articles/10.1186/s12890-023-02566-6

Identifying critical inhalation technique errors in Dry Powder ... In this study, we identified multiple critical inhalation technique errors in COPD patients using DPIs each associated with poorer outcomes.

6. fishersci.com fishersci.com/content/dam/fishersci/en_US/documents/programs/education/regulatory-documents/sds/chemicals/chemicals-p/S25493D.pdf

Potassium Iodide After inhalation: Loosen clothing as necessary and position individual in a comfortable position.Seek medical advice if discomfort or ...

7. nj.gov nj.gov/health/eoh/rtkweb/documents/fs/0372.pdf

CHLOROACETALDEHYDE HAZARD SUMMARY. * Chloroacetaldehyde can affect you when breathed in and may be absorbed through the skin. * Chloroacetaldehyde may cause mutations.

8. medicomart.com medicomart.com/images/document/Comirnaty%202025-2026%20Safety%20Data%20Sheet.pdf

SAFETY DATA SHEET 0.5 - 1.0 % of the mixture consists of ingredient(s) of unknown acute inhalation toxicity (dust/mist). Unknown aquatic toxicity. Contains 0.5 ...