Lumateperone for Autism

Recruiting at 35 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, suicidal risk, 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 evaluates the effectiveness of lumateperone, an antipsychotic medication, in treating irritability in children and teenagers with Autism Spectrum Disorder (ASD). Participants will receive either a high dose, a low dose of lumateperone, or a placebo to compare outcomes. It targets individuals aged 13 to 17 years diagnosed with ASD who experience significant irritability affecting their daily lives. The study seeks participants who can attend regular visits with a parent or guardian. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for ASD-related irritability.

Will I have to stop taking my current medications?

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

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

Research has shown that lumateperone has been tested for safety in children with conditions like autism and schizophrenia. One study used it with children who have autism and found it generally well-tolerated. Most side effects were mild or moderate, such as drowsiness and dry mouth, and not severe.

Another study tested lumateperone in children with schizophrenia and bipolar disorder. This study also found lumateperone safe to use, with similar mild side effects that were manageable.

These studies are early trials and provide some evidence that lumateperone is safe for children. However, ongoing trials will provide more information about its safety. ^{1 2 3 4 5}

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

Lumateperone is unique because it offers a new approach to treating autism, especially for teenagers aged 13 to 17. Unlike standard treatments that often focus on behavioral therapies or medications like antipsychotics and antidepressants, lumateperone has a distinct mechanism of action. It modulates serotonin, dopamine, and glutamate systems, which are all key in brain function and behavior. Researchers are excited because this could potentially address some of the core symptoms of autism more effectively and with fewer side effects than current options.

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

Studies have shown that lumateperone may help reduce irritability in children with autism. Earlier research linked lumateperone to improvements in managing aggression and mood issues, common in autism. Although specific data on lumateperone for autism-related irritability is limited, its success in treating similar symptoms in other conditions suggests potential benefits. Lumateperone affects serotonin and dopamine, brain chemicals involved in mood and behavior, which might explain its impact on irritability. While more research is needed, early findings suggest that lumateperone could be an effective treatment option for irritability in autism. Participants in this trial will receive either a high dose or low dose of lumateperone, or a placebo, to further evaluate its effectiveness. ^{1 4 6 7 8}

Are You a Good Fit for This Trial?

This trial is for children aged 5 to 17 with a diagnosis of irritability associated with Autism Spectrum Disorder (ASD), confirmed by specific psychiatric assessments. Details on who can or cannot participate are not fully provided.

Inclusion Criteria

Able to provide consent as follows: The patient's LAR must provide written, informed consent. When developmentally appropriate based on Investigator judgment, the patient should provide written assent.

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

ABC-I subscale score of >18 at Screening and Baseline;

Exclusion Criteria

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).

In the opinion of the Investigator, the patient has a significant risk for suicidal behavior during their participation in the study or: At Screening, the patient scores 'yes' on Suicidal Ideation Items 3, 4, or 5 of the Columbia-Suicide Severity Rating Scale (CSSRS) within 6 months prior to Screening or, at Baseline, the patient scores 'yes' on Suicidal Ideation Items 3, 4, or 5 since the Screening Visit; At Screening, the patient has had 1 or more suicidal attempts within the 2 years prior to Screening; or The patient is considered to be an imminent danger to themselves or others.

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 two doses of Lumateperone against a placebo in pediatric patients. It's designed to be randomized and double-blind, meaning neither the researchers nor participants know who receives the actual drug or placebo.

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+

Citations

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

NCT06690398 | Multicenter Study of Lumateperone for the ... Multicenter Study of Lumateperone for the Treatment of Irritability Associated With Autism Spectrum Disorder in Pediatric Patients. ClinicalTrials.gov ID ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06229210

Study Details | NCT06229210 | Safety and Tolerability Trial ... This is a multicenter, global, 26-week, open-label study to assess the safety and tolerability of lumateperone in pediatric patients with schizophrenia, bipolar ...

3. 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.

4. sciencedirect.com sciencedirect.com/science/article/pii/S2773021223000202

Lumateperone treatment of intermittent explosive disorder ... The use of lumateperone in reducing aggression in patients with autism spectrum disorder (ASD) and intermittent explosive disorder (IED) has not heretofore ...

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. 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 ...

7. 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.

8. 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 ...