Lumateperone for Irritability in Autism

Recruiting at 33 trial locations

Overseen By ITI Clinical Trials

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Intra-Cellular Therapies, Inc.

Must be taking: ADHD medications

Disqualifiers: Rett syndrome, Fragile X, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

174 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a treatment called lumateperone to determine if it can reduce irritability in teens with Autism Spectrum Disorder (ASD). It compares two different doses of lumateperone against a placebo (a pill with no active medicine) to understand its effects. Teens aged 13 to 17 with ASD who often feel irritable might be suitable for this study. The goal is to find a safe and effective way to manage irritability, making daily life easier for those affected. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but if you are taking medication for ADHD, you must be on a stable treatment plan for 30 days before the trial and continue it during the study.

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

Research has shown that lumateperone is generally well-tolerated in young people. Studies with children and teens who have schizophrenia and bipolar disorder found that lumateperone can be used safely for several weeks, with no major safety concerns reported.

In studies with children who have Autism Spectrum Disorder (ASD), lumateperone also demonstrated promising safety results. Some children experienced side effects like drowsiness or sleepiness, but these were usually mild. The FDA has already approved the treatment for adults with certain mental health conditions, indicating it has passed thorough safety checks for those uses.

Since this is a Phase 3 trial, earlier studies have shown lumateperone is safe enough to reach this stage. Phase 3 trials usually focus more on how well the treatment works rather than just safety, suggesting that previous safety data is strong. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for irritability in autism?

Researchers are excited about lumateperone for treating irritability in autism because it offers a novel approach compared to traditional medications. While common treatments like antipsychotics work by blocking dopamine receptors, lumateperone acts on multiple neurotransmitter systems, including serotonin and glutamate, potentially offering a more balanced effect with fewer side effects. Additionally, it may provide a more targeted and effective option for managing irritability without the sedation and weight gain often associated with existing treatments. This multi-faceted mechanism of action is what sets lumateperone apart and fuels the hope for a new, effective treatment for young individuals with autism.

What evidence suggests that Lumateperone might be an effective treatment for irritability in Autism?

Studies have shown that lumateperone can help reduce irritability in children and teens with autism spectrum disorder (ASD). Research suggests it may help manage symptoms of irritability in this group. Participants in this trial will receive either a low dose or high dose of lumateperone, or a placebo. In previous trials, those who took lumateperone demonstrated better behavior compared to those who took a placebo. This medication works by balancing chemicals in the brain, such as serotonin, dopamine, and glutamate, which affect mood and behavior. Early findings suggest lumateperone could be a promising option for managing irritability linked to ASD. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

This trial is for children aged 5 to 17 with irritability linked to Autism Spectrum Disorder (ASD), as defined by the DSM-5-TR and confirmed by K-SADS-PL. Specific eligibility details are not provided, but typically include a clear ASD diagnosis and stable health.

Inclusion Criteria

CGI-S score > 4 with respect to irritability associated with ASD at Screening and Baseline

I am between 13 and 17 years old.

All patients must have an LAR (eg, parent or legal guardian) who is willing and able to be responsible for the safety and well-being of the patient, provide information about the patient's condition, and accompany the patient to study visits

Exclusion Criteria

I have a psychiatric condition other than ASD, but ADHD is allowed if my treatment has been stable for 30 days.

History or current diagnosis of Rett syndrome or Fragile X syndrome

Mild and moderate intellectual disability based on Investigator judgment and DSM-5 criteria (severe and profound intellectual disability are excluded)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

Double-blind Treatment

Participants are randomized to receive either lumateperone high dose, lumateperone low dose, or placebo once daily for 6 weeks

6 weeks

Safety Follow-up

Participants return to the clinic for a safety follow-up visit approximately 1 week after the last dose of study drug

1 week

What Are the Treatments Tested in This Trial?

Interventions

• Lumateperone

Trial Overview The study tests Lumateperone at high and low doses against a placebo in pediatric patients. It's randomized, meaning participants are assigned randomly to one of these treatments, and double-blind, so neither the researchers nor participants know who gets what.

How Is the Trial Designed?

Group I: Lumateperone low dose Experimental Treatment 1 Intervention

Lumateperone 21 mg for patients ages 13-17 years old

Group II: Lumateperone high dose Experimental Treatment 1 Intervention

Lumateperone 42 mg for patients ages 13-17 years old

Group III: Placebo Placebo Group 1 Intervention

Placebo

Lumateperone is already approved in United States for the following indications:

Approved in United States as Caplyta for:

• Schizophrenia
• Bipolar I or II disorder (bipolar depression)

Who Is Running the Clinical Trial?

Intra-Cellular Therapies, Inc.

Lead Sponsor

Trials

42

Recruited

10,700+

Published Research Related to This Trial

Aripiprazole is one of the few medications approved in the USA for treating irritability in individuals with autism spectrum disorder (ASD), showing clear benefits in multiple studies compared to placebo.

While effective, aripiprazole can have metabolic side effects, highlighting the importance of monitoring and periodic reassessment of treatment needs for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aripiprazole for treating irritability associated with autism spectrum disorders. Bartram, LA., Lozano, J., Coury, DL.[2019]

Risperidone and aripiprazole are the most supported pharmacological treatments for irritability in autism, with multiple randomized controlled trials (RCTs) showing significant improvements in symptoms.

While several alternative treatments like clonidine and methylphenidate also showed efficacy, they are less established than the FDA-approved antipsychotics, highlighting the need for behavioral therapy alongside medication for optimal management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Review of the pharmacotherapy of irritability of autism. Elbe, D., Lalani, Z.[2021]

In a 14-week study involving 25 children and adolescents with PDD-NOS or Asperger's disorder, aripiprazole significantly reduced irritability symptoms, with 88% of participants showing improvement.

Aripiprazole was well tolerated, with only mild side effects reported, and no participants left the study due to drug-related adverse events, suggesting it is a safe option for managing irritability in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aripiprazole in pervasive developmental disorder not otherwise specified and Asperger's disorder: a 14-week, prospective, open-label study. Stigler, KA., Diener, JT., Kohn, AE., et al.[2021]

In a study of 45 children and adolescents with autism spectrum disorders, low-dose liquid fluoxetine was found to be more effective than placebo in reducing repetitive behaviors, with a moderate to large effect size.

Liquid fluoxetine did not show significant differences in side effects compared to placebo, indicating it may be a safe option for treating repetitive behaviors in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A placebo controlled crossover trial of liquid fluoxetine on repetitive behaviors in childhood and adolescent autism. Hollander, E., Phillips, A., Chaplin, W., et al.[2022]

MPEP, an mGluR5 receptor antagonist, effectively reduced repetitive self-grooming behaviors in BTBR mice, a model for autism, without causing sedation, suggesting its potential as a targeted treatment for such behaviors.

Risperidone also decreased repetitive self-grooming in BTBR mice, but only at sedating doses, indicating that while it may help with certain symptoms, it could also lead to unwanted side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Repetitive self-grooming behavior in the BTBR mouse model of autism is blocked by the mGluR5 antagonist MPEP. Silverman, JL., Tolu, SS., Barkan, CL., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06690398

NCT06690398 | Multicenter Study of Lumateperone for the ... This is a multicenter, randomized, double-blind, placebo-controlled study in pediatric patients aged 5 to 17 years with a primary diagnosis of irritability ...

2. jnjmedicalconnect.com jnjmedicalconnect.com/products/caplyta/medical-content/caplyta-use-in-comorbid-autism-spectrum-disorder

CAPLYTA - Use in Comorbid Autism Spectrum Disorder A summary of the clinical data regarding the use of CAPLYTA® (lumateperone) in patients with comorbid autism spectrum disorder.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06229210

Study Details | NCT06229210 | Safety and Tolerability Trial ... Patients with irritability associated with autism spectrum disorder who are enrolling directly from a lead-in efficacy study. This study will be conducted as ...

4. siumed.edu siumed.edu/trials?f%5B0%5D=treatment%3A392&f%5B1%5D=treatment%3A954&f%5B2%5D=treatment%3A977&f%5B3%5D=treatment%3A1494&f%5B4%5D=treatment%3A148351&page=0

Clinical trials and research studies ... Efficacy and Safety of Lumateperone in the Treatment of Irritability Associated with Autism Spectrum Disorder in Pediatric Patients 5 to 17 Years of Age.

5. withpower.com withpower.com/trial/phase-3-autism-spectrum-disorder-10-2024-82c41

Lumateperone for Irritability in Autism In a 14-week study involving 25 children and adolescents with PDD-NOS or Asperger's disorder, aripiprazole significantly reduced irritability symptoms, with 88% ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06557902?term=LUMATEPERONE&rank=10

Safety, Tolerability and Pharmacokinetics of Lumateperone ... ITI-007-035 is a Phase 1b, multicenter, open-label study to evaluate the safety, tolerability, and PK of lumateperone for pediatric patients with Autism ...

7. trialx.com trialx.com/clinical-trials/listings/323819/safety-tolerability-and-pharmacokinetics-of-lumateperone-in-pediatric-patients-with-autism-spectrum-disorder/

Safety, Tolerability and Pharmacokinetics of Lumateperone ... Clinical trial for Autism Spectrum Disorder , Safety, Tolerability and Pharmacokinetics of Lumateperone in Pediatric Patients With Autism ...

8. research-studies.allinforhealth.info research-studies.allinforhealth.info/us/en/listing/12697/safety-and-tolerability-trial/

Safety and Tolerability Trial of Lumateperone in Pediatric This study investigates the safety and tolerability of an investigational medication in young people with schizophrenia or bipolar disorder.