JZP441 Safety Study in Healthy Participants

Overseen By Clinical Trial Disclosure & Transparency

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Jazz Pharmaceuticals

Must not be taking: Antidepressants, Benzodiazepines, Kratom, others

Disqualifiers: Cardiovascular disease, Depression, Bipolar, Schizophrenia, Substance use, others

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, JZP441, to determine its effectiveness in promoting wakefulness and reducing cataplexy (sudden muscle weakness) in people with narcolepsy and other sleep disorders. As a Phase 1 study, it examines how the drug behaves in the body and assesses its safety in healthy individuals. Participants will receive either JZP441 or a placebo (a pill with no active drug) to compare effects. This trial is for healthy adults without a history of significant health issues, particularly related to heart, liver, or kidney functions, and who are not currently using tobacco or certain medications. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in humans.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it excludes participants who are taking medications or supplements that lower seizure threshold or have recently stopped such medications. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that JZP441 is likely to be safe for humans?

Research has shown that JZP441 has been tested on healthy individuals, particularly those who are sleep-deprived. In these studies, JZP441 aimed to help people stay awake and prevent sudden muscle weakness, common issues in narcolepsy. However, some participants reported vision changes during the trials. As a result, some trials have paused to better understand these side effects.

Since this trial is in its early phase, it primarily focuses on assessing the safety of JZP441 for humans. This early stage means that while there is hope for its benefits, more information is needed to fully understand its safety. Participants might experience side effects that researchers are still investigating. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

JZP441 is unique because it offers a new approach by being administered orally, which can make it more convenient compared to existing treatments that might require injections or infusions. Researchers are excited about JZP441 because it potentially introduces a novel way of targeting the condition, although specific details about its mechanism are still under wraps. This treatment could provide a more comfortable experience for patients and possibly improve adherence to medication routines.

What evidence suggests that JZP441 might be an effective treatment for narcolepsy?

Research has shown that JZP441 has potential in early studies for treating narcolepsy. In non-human tests, it reduced sudden muscle weakness, known as cataplexy, and helped maintain wakefulness. These results suggest that JZP441 could become a new treatment option for people with narcolepsy and excessive sleepiness disorders. It effectively reaches the brain and can be taken orally. Although more research is needed to confirm these benefits in humans, the initial findings are promising. In this trial, researchers will randomize healthy participants to receive either JZP441 or a placebo to further assess its safety. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for healthy adults aged 18-50 with no gastrointestinal, liver, or kidney diseases that could affect drug processing. They must not have a history of substance abuse in the past two years and should not be using any medications that lower seizure threshold.

Inclusion Criteria

I am generally healthy as confirmed by a full medical check-up.

Exclusion Criteria

I have used tobacco or nicotine products within the last 90 days or heavily in the past year.

My ECG shows a QT interval longer than normal or other significant issues.

My kidney function is reduced with an eGFR below 80 mL/min.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple ascending doses of JZP441 or placebo for up to 4 weeks

4 weeks

Pre-dose and multiple post-dose timepoints

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• JZP441

Trial Overview The study tests JZP441's safety and how it's processed by the body when given in increasing doses to healthy participants. It compares JZP441 against a placebo to see if it can help people with narcolepsy without causing harm.

How Is the Trial Designed?

Group I: JZP441 Experimental Treatment 1 Intervention

Healthy participants who will be randomized to receive an oral dose of JZP441.

Group II: Placebo Placebo Group 1 Intervention

Healthy participants who will be randomized to receive placebo.

Who Is Running the Clinical Trial?

Jazz Pharmaceuticals

Lead Sponsor

Trials

252

Recruited

35,100+

Bruce C. Cozadd

Jazz Pharmaceuticals

Chief Executive Officer since 2009

BA in Economics from Yale University, MBA from Stanford University

Dr. Austin

Jazz Pharmaceuticals

Chief Medical Officer since 2023

MD from the Royal College of Surgeons in Ireland

Published Research Related to This Trial

In a phase III study involving 507 patients with rheumatoid arthritis, peficitinib (100 mg and 150 mg once daily) significantly improved symptoms compared to placebo, with ACR20 response rates of 57.7% and 74.5% respectively at week 12.

Peficitinib was well tolerated overall, although there was a higher incidence of serious infections and herpes zoster compared to placebo, indicating a need for monitoring but no clear dose-dependent increase in adverse events.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of peficitinib (ASP015K) in patients with rheumatoid arthritis and an inadequate response to conventional DMARDs: a randomised, double-blind, placebo-controlled phase III trial (RAJ3). Tanaka, Y., Takeuchi, T., Tanaka, S., et al.[2020]

Janus kinase inhibitors, including new compounds like filgotinib and upadacitinib, show promising fast and dose-dependent clinical improvements in treating rheumatoid arthritis, comparable to existing treatments like tofacitinib and baricitinib.

While these new drugs have similar adverse events, some side effects are specific to individual drugs, highlighting the need for thorough phase III trials and safety evaluations to optimize their use in clinical practice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical efficacy of new JAK inhibitors under development. Just more of the same? Westhovens, R.[2023]

In a study of 10 patients with juvenile dermatomyositis (JDM) treated with Janus kinase inhibitors (JAKis) for at least 6 months, 5 patients achieved clinically inactive disease, indicating that JAKis can be effective for refractory cases.

While JAKis showed promise in improving muscle vasculopathy and reducing steroid use, there was a notable risk of herpes zoster infections in some patients, highlighting the need for careful monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

JAK inhibitors are effective in a subset of patients with juvenile dermatomyositis: a monocentric retrospective study. Le Voyer, T., Gitiaux, C., Authier, FJ., et al.[2021]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06961266

NCT06961266 | A Study to Investigate Efficacy, Safety ... JZP441 is currently being developed for the treatment of narcolepsy type 1 (NT1). This study will assess the safety of efficacy of JZP441 in adult patients with ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05651152

NCT05651152 | A Study to Investigate The Safety ... This Phase 1, double-blind, randomized, placebo-controlled study will characterize the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD)

3. withpower.com withpower.com/trial/phase-1-healthy-subjects-hs-2023-21627

JZP441 Safety Study in Healthy Participants This trial is testing a new drug called JZP441, which helps people stay awake and prevents sudden muscle weakness. It is aimed at people with narcolepsy and ...

4. withpower.com withpower.com/trial/phase-1-healthy-subjects-hs-10-2022-339ec

JZP441 for Sleep Deprivation · Info for Participants This trial tests a new drug called JZP441 to see if it is safe and how it works in the body. The study involves healthy people who haven't had enough sleep.

5. centerwatch.com centerwatch.com/clinical-trials/listings/NCT05651152/a-study-to-investigate-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-jzp441-in-sleep-deprived-healthy-participants

A Study to Investigate The Safety, Tolerability ... This Phase 1, double-blind, randomized, placebo-controlled study will characterize the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD)

6. app.trialscreen.org app.trialscreen.org/trials/phase-1-healthy-participants-to-investigate-safety-tolerability-pharmacokinetics-trial-nct05651152

A Study to Investigate The Safety, Tolerability ... This study aims to analyze JZP441's safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) in sleep-deprived healthy ...

7. fiercebiotech.com fiercebiotech.com/biotech/jazz-halts-recently-licensed-sleep-disorder-med-after-clocking-visual-disturbances-and-cv

Jazz halts sleep disorder med trial over 'visual disturbances' Tan said that the drug, JZP441, demonstrated proof-of-concept in healthy volunteers as measured by the Maintenance of Wakefulness Test (MWT).