Lumateperone for Irritability in Autism
Palo Alto (17 mi)Age: < 18
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Intra-Cellular Therapies, Inc.
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 1 jurisdiction
Trial Summary
What is the purpose of this trial?This is a multicenter, randomized, double-blind, placebo-controlled study in pediatric patients aged 5 to 17 years with a primary diagnosis of irritability associated with Autism Spectrum Disorder (ASD) based on Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition Text Revision (DSM-5-TR) and confirmed by the Kiddie Schedule for Affective Disorders and Schizophrenia Present and Lifetime Version (K-SADS-PL).
What safety data exists for Lumateperone in treating irritability in autism?The provided research does not contain specific safety data for Lumateperone (also known as Caplyta, ITI-007, ITI-722) in treating irritability in autism. The studies focus on other medications like risperidone, fluoxetine, palmitoylethanolamide, and valproate. Further research is needed to determine the safety and efficacy of Lumateperone for this purpose.345910
What data supports the idea that Lumateperone for Irritability in Autism is an effective drug?The available research does not provide specific data on Lumateperone for treating irritability in autism. Instead, it highlights other drugs like risperidone, which is currently the only FDA-approved medication for this purpose. Studies on other medications, such as fluoxetine and haloperidol, show varying levels of effectiveness for related symptoms in autism, but Lumateperone is not mentioned in these studies.12358
Is the drug Lumateperone a promising treatment for irritability in autism?The provided research articles do not mention Lumateperone or its effects on irritability in autism, so we cannot determine if it is a promising treatment based on this information.367811
Do I have to stop taking my current medications for the trial?The trial protocol 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 regimen for 30 days before screening and continue it throughout the study.
Eligibility Criteria
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
I am between 13 and 17 years old.
I have been diagnosed with ASD according to the DSM-5-TR criteria.
Treatment Details
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.
3Treatment groups
Experimental Treatment
Placebo Group
Group I: Lumateperone low doseExperimental Treatment1 Intervention
Lumateperone 21 mg for patients ages 13-17 years old
Group II: Lumateperone high doseExperimental Treatment1 Intervention
Lumateperone 42 mg for patients ages 13-17 years old
Group III: PlaceboPlacebo Group1 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)
Find a clinic near you
Research locations nearbySelect from list below to view details:
Clinical SiteDothan, AL
Clinical SiteWest Palm Beach, FL
Clinical SiteSavannah, GA
Clinical SiteGarfield, OH
More Trial Locations
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Who is running the clinical trial?
Intra-Cellular Therapies, Inc.Lead Sponsor
References
Long-term efficacy of haloperidol in autistic children: continuous versus discontinuous drug administration. [2013]The aim of this study was to evaluate the long-term efficacy of haloperidol in autistic children and to determine whether discontinuous drug administration was as effective as continuous drug administration. Sixty children, 48 males and 12 females, ages 2.3 to 7.9 years (X 5.1) completed the study. They received haloperidol over a period of 6 months followed by a 4-week drug withdrawal/placebo period. Haloperidol remained effective, and the discontinuous treatment schedule did not diminish its efficacy. Children with prominent symptoms of irritability, angry and labile affect, and uncooperativeness were the best responders to haloperidol.
Clomipramine versus haloperidol in the treatment of autistic disorder: a double-blind, placebo-controlled, crossover study. [2019]Clomipramine, haloperidol, and placebo were compared with baseline in the treatment of autism, and overall outcome, specific symptoms, and side effects were examined. It was hypothesized that clomipramine would be better tolerated than haloperidol and prove superior on a measure of stereotypy. Individuals with a DSM-IV diagnosis of autistic disorder (mean age, 16.3 years; range, 10-36 years) were randomly assigned, by using a Latin square design, to the following 7-week trials: placebo, clomipramine (mean daily dose, 128.4 mg; range, 100-150 mg), or haloperidol (mean daily dose, 1.3 mg; range, 1-1.5 mg). Data on 36 subjects were analyzed and taken together; the results favored haloperidol. In those patients who were able to complete a full therapeutic trial, clomipramine proved comparable to haloperidol in terms of improvement compared with baseline. However, significantly fewer individuals receiving clomipramine versus haloperidol were able to complete the trial (37.5% vs. 69.7%, respectively) for reasons related to both side effects and efficacy or behavior problems. In the intent-to-treat sample, which is perhaps more clinically relevant, only haloperidol proved superior to baseline on a global measure of autistic symptom severity, as well as specific measures for irritability and hyperactivity. Clomipramine did not seem more effective on a measure of stereotypy, nor was it better tolerated.
A placebo controlled crossover trial of liquid fluoxetine on repetitive behaviors in childhood and adolescent autism. [2022]Repetitive behaviors are a core symptom domain in autism that has been linked to alterations in the serotonin system. While the selective serotonin-receptive inhibitor fluvoxamine has been shown to be effective in adults with autism, as yet no published placebo controlled trials with these agents document safety and efficacy in children with autism. This study examines the selective serotonin reuptake inhibitor liquid fluoxetine in the treatment of repetitive behaviors in childhood and adolescent autism spectrum disorders (ASDs). In total, 45 child or adolescent patients with ASD were randomized into two acute 8-week phases in a double-blind placebo-controlled crossover study of liquid fluoxetine. Study design included two randomized 8-week fluoxetine and placebo phases separated by a 4-week washout phase. Outcome measures included measures of repetitive behaviors and global improvement. Low-dose liquid fluoxetine (mean final dose: 9.9+/-4.35 mg/day) was superior to placebo in the treatment of repetitive behaviors by CY-BOCS compulsion scale. The effect size was in the moderate to large range, and the doses used were low. Liquid fluoxetine was only slightly, and not significantly, superior to placebo on CGI autism score partially due to a phase order effect. However, fluoxetine was marginally superior to placebo on a composite measure of global effectiveness. Liquid fluoxetine did not significantly differ from placebo on treatment emergent side effects. Liquid fluoxetine in low doses is more effective than placebo in the treatment of repetitive behaviors in childhood autism. Limitations include small sample size and the crossover design of the study. Further replication and long-term maintenance trials are needed.
A double-blind, placebo-controlled study of valproate for aggression in youth with pervasive developmental disorders. [2013]The aim of this study was to study valproate efficacy and safety for aggression in children and adolescents with pervasive developmental disorders (PDD).
Pharmacotherapy of irritability in pervasive developmental disorders. [2013]Children and adolescents diagnosed with autism and related pervasive developmental disorders (PDDs) often sustain irritability, including aggression, self-injurious behavior, and tantrums. Research to date supports the use of the atypical antipsychotics as a first-line pharmacologic treatment for this target symptom domain in PDDs. Currently, the atypical antipsychotic risperidone is the only medication approved by the US Food and Drug Administration for irritability in youth with autism. Additional large-scale, placebo-controlled studies of other medications are needed to determine their efficacy for the treatment of irritability in this diagnostic group.
Aripiprazole in pervasive developmental disorder not otherwise specified and Asperger's disorder: a 14-week, prospective, open-label study. [2021]The aim of this study was to determine the effectiveness and tolerability of aripiprazole for irritability in pervasive developmental disorder not otherwise specified (PDD-NOS) and Asperger's disorder.
Repetitive self-grooming behavior in the BTBR mouse model of autism is blocked by the mGluR5 antagonist MPEP. [2022]Autism is a neurodevelopmental disorder characterized by abnormal reciprocal social interactions, communication deficits, and repetitive behaviors with restricted interests. BTBR T+tf/J (BTBR) is an inbred mouse strain that shows robust behavioral phenotypes with analogies to all three of the diagnostic symptoms of autism, including well-replicated deficits in reciprocal social interactions and social approach, unusual patterns of ultrasonic vocalization, and high levels of repetitive self-grooming. These phenotypes offer straightforward behavioral assays for translational investigations of pharmacological compounds. Two suggested treatments for autism were evaluated in the BTBR mouse model. Methyl-6-phenylethynyl-pyridine (MPEP), an antagonist of the mGluR5 metabotropic glutamate receptor, blocks aberrant phenotypes in the Fmr1 mouse model of Fragile X, a comorbid neurodevelopmental disorder with autistic features. Risperidone has been approved by the United States Food and Drug Administration for the treatment of irritability, tantrums, and self-injurious behavior in autistic individuals. We evaluated the actions of MPEP and risperidone on two BTBR phenotypes, low sociability and high repetitive self-grooming. Open field activity served as an independent control for non-social exploratory activity and motor functions. C57BL/6J (B6), an inbred strain with high sociability and low self-grooming, served as the strain control. MPEP significantly reduced repetitive self-grooming in BTBR, at doses that had no sedating effects on open field activity. Risperidone reduced repetitive self-grooming in BTBR, but only at doses that induced sedation in both strains. No overall improvements in sociability were detected in BTBR after treatment with either MPEP or risperidone. Our findings suggest that antagonists of mGluR5 receptors may have selective therapeutic efficacy in treating repetitive behaviors in autism.
Review of the pharmacotherapy of irritability of autism. [2021]To review the randomized controlled trial data regarding pharmacotherapy of irritability of autism.
An open-label extension study of the safety and efficacy of risperidone in children and adolescents with autistic disorder. [2021]The purpose of this study was to evaluate the long-term safety and efficacy of risperidone in treating irritability and related behaviors in children and adolescents with autistic disorders.
Palmitoylethanolamide as adjunctive therapy for autism: Efficacy and safety results from a randomized controlled trial. [2020]Inflammation as well as glutamate excitotoxicity have been proposed to participate in the propagation of autism. Palmitoylethanolamide (PEA) is an endocannabinoid proven to prevent glutamatergic toxicity and inhibit inflammatory responses simultaneously. The present randomized, parallel group, double-blind placebo-controlled trial is the first study depicted to probe the efficacy of co-treatment with risperidone and PEA over 10 weeks in children with autism. Seventy children (aged 4-12 years) with autism and moderate to severe symptoms of irritability were randomly assigned to two treatment regimens. The study outcomes were measured using the Aberrant Behavior Checklist-Community Edition (ABC-C). At trial endpoint (week 10), combination of PEA and risperidone had superior efficacy in ameliorating the ABC-irritability and hyperactivity/noncompliance symptoms (Cohen's d, 95% confidence interval (CI) = 0.94, 0.41 to 1.46, p = 0.001) compared with a risperidone plus placebo regimen. Interestingly, effect of combination treatment on hyperactivity symptoms was also observed at trial midpoint (week 5) but with a smaller effect size (d = 0.53, p = 0.04) than that at the endpoint (d = 0.94, p = 0.001). Meanwhile, there was a trend toward significance for superior effect of risperidone plus PEA over risperidone plus placebo on inappropriate speech at trial endpoint (d = 0.51, p = 0.051). No significant differences existed between the two treatment groups for the other two ABC-C subscales (lethargy/social withdrawal and stereotypic behavior). The findings suggest that PEA may augment therapeutic effects of risperidone on autism-related irritability and hyperactivity. Future studies are warranted to investigate whether PEA can serve as a stand-alone treatment for autism.
Aripiprazole for treating irritability associated with autism spectrum disorders. [2019]Introduction: Autism spectrum disorder (ASD) is a neurodevelopmental disorder with a reported prevalence of 1 in 59 people. Its core features are persistent deficits in social communication and restricted, repetitive patterns of behavior or interests. Individuals with ASD have a high incidence of secondary problems with mood lability, tantrums, self-injurious behavior and aggressiveness toward others. Collectively, these behaviors are often referred to as irritability. Many medications have been used to treat irritability in autism, with aripiprazole one of only two medications approved in the USA for this purpose. Areas covered: Herein, the authors review the evidence supporting the use of aripiprazole for treating irritability in autism, including the pivotal trials leading to regulatory approval and long-term studies conducted post-approval. They utilized PubMed, searching all English language publications since 2000, using the terms aripiprazole, autism, autism spectrum disorder, pervasive developmental disorder, Asperger's disorder, and irritability, and focused on clinical trials and review articles. Expert opinion: Multiple studies have shown the clear benefit of aripiprazole in the treatment of irritability in autism disorders compared to placebo. Often underemphasized are the metabolic effects, the proper monitoring for these effects, and the need for periodic reassessment to determine if ongoing treatment is needed.