NBI-1117568 for Schizophrenia

No longer recruiting at 15 trial locations

Overseen By Neurocrine Medical Information Call Center

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Neurocrine Biosciences

Must not be taking: Antipsychotics

Disqualifiers: Unstable condition, Suicide risk, Substance use, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 6 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called NBI-1117568 for individuals with schizophrenia experiencing a flare-up of symptoms and requiring hospitalization. The trial aims to evaluate the treatment's effectiveness, safety, and how the body processes it compared to a placebo (a pill with no active medicine). Participants will join one of several groups, receiving different doses of the treatment or a placebo for six weeks. Suitable candidates have schizophrenia, are currently experiencing worsening symptoms, and can remain in the hospital for the study period. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in schizophrenia treatment.

Will I have to stop taking my current medications?

Yes, if you are taking certain medications, including antipsychotics, you must stop them before joining the study.

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

Research has shown that NBI-1117568 is generally safe and well-tolerated. Studies found that the number of people who stopped taking it due to side effects was similar to those who took a placebo (a pill with no active medicine). This indicates that people taking NBI-1117568 did not experience more severe side effects than those taking a placebo. So far, the treatment appears to be as safe as expected for people with schizophrenia. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Most treatments for schizophrenia focus on balancing neurotransmitters like dopamine through antipsychotics, such as risperidone or olanzapine. But NBI-1117568 works differently, targeting a unique pathway that may offer benefits beyond symptom control. Researchers are excited because it could lead to fewer side effects and potentially improve cognitive functions in patients. This novel approach could transform how we manage schizophrenia, moving beyond just alleviating symptoms to improving overall brain health.

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

Research has shown that NBI-1117568 can help reduce symptoms of schizophrenia. In studies, patients taking a 20 mg dose showed noticeable improvement in their symptoms by the third week, which continued through the sixth week. PANSS scores, a tool for assessing symptom severity, measured this improvement. The data indicated a significant reduction in both positive symptoms (like hallucinations) and negative symptoms (like lack of emotion). Overall, NBI-1117568 was well tolerated by patients, suggesting it could effectively treat schizophrenia. Participants in this trial will receive one of several dose levels of NBI-1117568 or a placebo to further evaluate its effectiveness and safety. ^{1 3 4 5 6}

Who Is on the Research Team?

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Are You a Good Fit for This Trial?

Adults with schizophrenia who are currently experiencing a worsening of symptoms and need hospitalization can join. They must stop taking any disallowed medications, agree to stay in the hospital for the study, and follow all instructions. People using certain substances or with unstable health conditions, a risk of self-harm, or recent substance abuse issues cannot participate.

Inclusion Criteria

I am not taking any medications that are not allowed in the study.

I have been diagnosed with schizophrenia.

Completed informed consent

Exclusion Criteria

I do not have any unstable health conditions or other cancers.

I have always followed the rules in clinical studies.

Positive alcohol test or drug screen for disallowed substances

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either NBI-1117568 or placebo for 6 weeks

6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• NBI-1117568
• Placebo

Trial Overview The trial is testing NBI-1117568's effectiveness and safety against a placebo in adults with schizophrenia during an acute episode. It's well-controlled: participants don't know if they're getting the real drug or placebo, nor do the researchers (double-blind).

How Is the Trial Designed?

Group I: Dose Level D Experimental Treatment 1 Intervention

Participant administered Dose Level D (6 weeks)

Group II: Dose Level C Experimental Treatment 1 Intervention

Participant administered Dose Level C (6 weeks)

Group III: Dose Level B Experimental Treatment 1 Intervention

Participant administered Dose Level B (6 weeks)

Group IV: Dose Level A Experimental Treatment 1 Intervention

Participant administered Dose Level A (6 weeks)

Group V: Placebo Schedule Placebo Group 1 Intervention

Participant administered placebo (6 weeks)

Who Is Running the Clinical Trial?

Neurocrine Biosciences

Lead Sponsor

Trials

78

Recruited

6,600+

Kyle W. Gano

Neurocrine Biosciences

Chief Executive Officer since 2024

PhD in Pharmacology

Dr. Sanjay Keswani

Neurocrine Biosciences

Chief Medical Officer

MD

Published Research Related to This Trial

In a meta-regression analysis of 18 double-blind, randomized, placebo-controlled trials involving 998 patients with stable schizophrenia, drugs were found to be more effective than placebo in reducing negative symptoms, although the effect size was small (Cohen's d: 0.208).

The study revealed a significant placebo response (Cohen's d: 2.909), indicating that factors like the number of trial arms, study sites, and industry sponsorship can influence the placebo effect, suggesting a need for improved trial designs to accurately assess treatment efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Predictors of Placebo Response in Pharmacological Clinical Trials of Negative Symptoms in Schizophrenia: A Meta-regression Analysis. Fraguas, D., Díaz-Caneja, CM., Pina-Camacho, L., et al.[2022]

In a pooled analysis of two 6-week trials involving 422 patients with schizophrenia, early response to antipsychotic treatment (low-dose olanzapine or haloperidol) significantly distinguished active drug from placebo, with a greater mean reduction in psychiatric symptoms observed at 2 weeks.

The study suggests that shortening the duration of placebo-controlled trials from 6 weeks to 2-4 weeks is feasible for identifying efficacy in acutely ill patients, as early treatment response was predictive of overall treatment success.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Early onset of antipsychotic action in schizophrenia: evaluating the possibility of shorter acute efficacy trials. Kinon, BJ., Chen, L., Stauffer, VL., et al.[2021]

The placebo effect in schizophrenia clinical trials is becoming a significant challenge, making it harder to detect the effectiveness of new treatments and increasing development costs.

Understanding the central nervous system mechanisms behind the placebo effect and improving study designs could help mitigate this issue and enhance the reliability of clinical trial results.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo-related effects in clinical trials in schizophrenia: what is driving this phenomenon and what can be done to minimize it? Alphs, L., Benedetti, F., Fleischhacker, WW., et al.[2021]

Placebo-controlled studies are essential for demonstrating the efficacy of treatments in schizophrenia, as they provide a clearer picture of responder rates compared to non-placebo-controlled studies.

The study found that responder rates in the treatment arms of non-placebo-controlled studies were similar to those in placebo arms, highlighting the variability in placebo responses and the need for standardized definitions of responders in future research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Schizophrenia: do we really need placebo-controlled studies? Storosum, JG., Elferink, AJ., van Zwieten, BJ.[2019]

Placebo responses in clinical trials for schizophrenia are similar in magnitude and impact to those seen in major depression trials, and these responses have increased in recent years.

To address the challenge of high placebo responses in schizophrenia trials, a multipronged strategy is recommended, as no single factor has been identified as the primary cause.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo response in clinical trials with schizophrenia patients. Kinon, BJ., Potts, AJ., Watson, SB.[2011]

Citations

1. neurocrine.gcs-web.com neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-presents-new-positive-data-phase-2-study

Release Details - Neurocrine Biosciences The study showed statistically significant improvements in PANSS total score with 20 mg of NBI-1117568 once daily by Week 3 and at all ...

2. prnewswire.com prnewswire.com/news-releases/neurocrine-biosciences-presents-new-positive-data-from-phase-2-study-of-nbi-1117568-in-adults-with-schizophrenia-at-american-society-of-clinical-psychopharmacology-2025-302467554.html

Neurocrine Biosciences Presents New Positive Data from ... The primary endpoint was the change in total Positive and Negative Syndrome Scale (PANSS) score from baseline to Week 6. The study showed ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06963034?term=NCT06963034&rank=1

Study Details | NCT06963034 | NBI-1117568-SCZ3029 ... The primary objective for this study is to evaluate the efficacy of NBI-1117568 compared with placebo on improving behavioral and psychological symptoms of ...

4. psychiatrictimes.com psychiatrictimes.com/view/positive-phase-2-results-on-nbi-1117568-for-schizophrenia-presented-at-2025-ascp-annual-meeting

Positive Phase 2 Results on NBI-1117568 for ... NBI-1117568 showed significant symptom improvement in schizophrenia, particularly with the 20 mg dose, in a phase 2 study. The study ...

5. neurocrine.gcs-web.com neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-reports-positive-phase-2-data-nbi-1117568

Neurocrine Biosciences Reports Positive Phase 2 Data for ... "NBI-1117568 demonstrated a clinically meaningful and statistically significant reduction in PANSS scores and was well tolerated, importantly ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05545111

Study Details | NCT05545111 | Efficacy, Safety, Tolerability ... ... Safety, Tolerability, and Pharmacokinetics of NBI-1117568 in Adults With Schizophrenia Who Warrant Inpatient Hospitalization. Conditions. Schizophrenia.