CTRI/2025/11/097254尚未招募3 期
A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Lumateperone in the Treatment of Irritability Associated with Autism Spectrum Disorder in Pediatric Patients 5 to 17 Years of Age
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 174
- 试验地点
- 7
- 主要终点
- The primary efficacy objective of this study is to evaluate the efficacy of high and low doses of lumateperone vs placebo for the treatment of irritability associated with ASD in pediatric patients aged 5 to 17 years as measured by the change from baseline to end of Week 6 in the ABC Irritability (ABC-I) subscale score
研究概览
简要总结
This is a global, randomized, double-blind, placebo-controlled study in pediatric patients aged 5 to 17 years with a primary diagnosis of irritability associated with 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). Patients aged 13 to 17 years are eligible for enrollment. Enrollment of patients aged 5 to 12 years will be initiated when PK data supporting dose selection for this age group are available.The study will be conducted in 3 phases:
• Screening Period (up to 14 days) during which patient eligibility will be assessed.
• Double-blind Treatment Period (DBTP) (6 weeks) during which all patients will be randomized in a 1:1:1 ratio to receive either lumateperone high dose, lumateperone low dose, or placebo as a once daily dose, stratified by country and age category.
o Patients who discontinue study drug prior to Visit 8 (Day 43) but agree to continue participation in the DBTP should be seen at all subsequent protocol-defined visits through Visit 8 (Day 43) and should also return for the Safety Follow-up (SFU) Visit. Study drug will not be dispensed to these patients after discontinuation. Clinical management of these patients will be at the discretion of the Investigator.
o Patients who discontinue study drug prior to Visit 8 (Day 43) and do not agree to continue participation in the DBTP should be seen for an early termination (ET) Visit as soon as possible and should return for the SFU Visit 1 week after the ET Visit. Study drug will not be dispensed to these patients after discontinuation. Clinical management of these patients will be at the discretion of the Investigator.
• Safety Follow-up Period (1 week) during which all patients will return to the clinic for a SFU Visit approximately 1 week after the last dose of study drug
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 盲法
- Participant and Investigator Blinded
入排标准
- 年龄范围
- 5.00 Year(s) 至 17.00 Year(s)（—）
- 性别
- All
入选标准
- •To be eligible to participate in the study, patients must meet the following inclusion criteria:
- •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.
- •NOTE: Patients who turn 18 years of age during participation in this study will be allowed to continue study participation for the duration of this trial.
- •Able to provide consent as follows: a.
- •The patient’s LAR must provide written, informed consent.
- •If a patient turns the age of majority (18 years of age in most jurisdictions) during study participation, they should sign the informed consent at the visit following their birthday if developmentally appropriate.
- •When developmentally appropriate based on Investigator judgment, the patient should provide written assent.
- •Male or female patients 5 to 17 years of age.
- •Currently, only patients aged 13 to 17 years will be eligible for enrollment.
- •NOTE: Patients aged 5 to 12 years will be eligible for enrollment when PK data supporting dose selection for this age group are available.
- •The protocol will be amended in the future to allow enrollment of these younger patients.
- •Meet Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition Text Revision (DSM-5-TR) primary diagnosis of ASD as confirmed by the Kiddie Schedule for Affective Disorders and Schizophrenia for School-Age Children-Present and Lifetime Version (K-SADS-PL)
- •ABC-I subscale score of more than 18 at Screening and Baseline.
- •CGI-S score more than 4 with respect to irritability associated with ASD at Screening and Baseline.
- •Patients who require inpatient washout will be evaluated by the Investigator for suitability to transition to outpatient status at the end of the Screening Period.
- •Female patients of childbearing potential must have negative serum pregnancy test at Screening and negative urine pregnancy test at Baseline (Visit 2) and a.
- •Agree to use highly effective methods of birth control (including but not limited to combined and progesterone-only hormonal contraception associated with inhibition of ovulation, intrauterine device or hormone-releasing system, vasectomized partner, bilateral tubal occlusion) from the time informed consent (and assent if developmentally appropriate) is provided through the end of the SFU period.
- •Sexual abstinence may be an acceptable form of birth control based on the Investigators judgment and familiarity with the patients preferred and usual lifestyle.
- •NOTE: Females of non-childbearing potential (defined as either not achieved menarche or permanently sterilized) are exempt from the birth control requirement.
- •If a female patient reaches menarche during the study, the Investigator should have an age-appropriate discussion with the patient and LAR to determine if contraceptive requirements (as noted above) are met.
- •It is the responsibility of Investigator to monitor and reassess the contraception requirement during the course of the study.
- •Sexual abstinence may be an acceptable form of birth control based on the Investigators judgment and familiarity with the patients preferred and usual lifestyle NOTE: If a male patient experiences spermarche during study participation, the Investigator should have an age-appropriate discussion with the patient and LAR to determine if contraceptive requirements (as noted above) are met.
- •Body mass index (BMI) greater than the 5th percentile, according to age- and gender-specific CDC Clinical Growth Charts (2000) at Screening.
- •Ability to swallow capsules for patients aged 13 to 17 years.
- •In the opinion of the Investigator, the LAR and patient are willing to comply with all Investigator and staff instructions.
排除标准
- •Patients who meet any of the following exclusion criteria will not be eligible to participate in the study.
- •Psychiatric and Neurological Exclusion Criteria:
- •Has a primary psychiatric diagnosis other than ASD.
- •Exceptions include: a.
- •attention deficit hyperactivity disorder (ADHD).
- •If a patient is taking medication(s) for ADHD, they must be on a stable treatment regimen of these medication(s) for 30 days prior to screening and the treatment regimen is expected to remain stable throughout the study.
- •This must be confirmed by the Investigator and noted in the source records.
- •Mild and moderate intellectual disability based on Investigator judgment and DSM-5 criteria (severe and profound intellectual disability are excluded).
- •History or current diagnosis of Rett syndrome or Fragile X syndrome
- •In the opinion of the Investigator, the patient has a significant risk for suicidal behavior during their participation in the study or a.
- •At Screening (Visit 1), the patient scores “yes” on Suicidal Ideation Items 3, 4, or 5 of the Columbia–Suicide Severity Rating Scale (C-SSRS) within 6 months prior to Screening or, at Baseline (Visit 2), the patient scores “yes” on Suicidal Ideation Items 3, 4, or 5 since the Screening Visit b.
- •At Screening (Visit 1), the patient has had 1 or more suicidal attempts within the 2 years prior to Screening; or c.
- •The patient is considered to be an imminent danger to him per herself or others.
- •Treatment-related Exclusion Criteria:
- •Electroconvulsive therapy (ECT), vagal nerve stimulation, repetitive trans-cranial magnetic stimulation, or any other neuromodulation therapies for any central nervous system and psychiatry indications within 6 months prior to Screening
- •Likely allergy or sensitivity to lumateperone or its excipients, based on known allergies or hypersensitivities to drugs with shared pharmacology which may be suggestive of an increased potential for an adverse reaction to lumateperone;
- •Use of any strong or moderate cytochrome P450 3A4 inhibitor or any cytochrome P450 3A4 inducer as described in Table 7-3–Restricted and Prohibited Medications;
- •Use of monoamine oxidase inhibitors within 14 days prior to Baseline (Visit 2);
- •Unable or unwilling to discontinue other antipsychotics prior to randomization (Baseline per Visit 2) as described in Table 7-3–Restricted and Prohibited Medications.
- •Use of benzodiazepines and other sleep aids (see Table 7-2–Guidance for Concomitant Medications for permitted treatments for insomnia).
- •The patient has a positive test for alcohol or drugs of abuse (eg, amphetamines, barbiturates, benzodiazepines, cannabinoids, cocaine, methadone, opioids opiates) at Screening (Visit 1).
- •Exceptions may include appropriate prescription treatments (eg, opioids, benzodiazepines) if the use is not chronic and is able to be discontinued restricted as per the Investigator with the concurrence of the Sponsor or designee.
- •Use of a depot per long-acting injectable antipsychotic medication within 2 treatment cycles prior to Screening (Visit 1)
- •Is unable to be safely discontinued from prohibited medications (in the opinion of the Investigator).
- •Use of dietary supplements and medical foods unless approved by the Sponsor or designee.
- •Daily multivitamin use is not excluded.
- •Has had exposure to any investigational product within 3 months of Baseline (Visit 2) (except for those patients who had participated in a lumateperone PK study) or has participated in the past 3 years in more than 2 clinical studies of an investigational product with a central nervous system indication.
- •Other Medical Exclusion Criteria
- •Female patient who is currently pregnant or breastfeeding
- •The patient has abnormal laboratory values or clinical findings at Screening (Visit 1) that are judged to be clinically significant including, but not limited to: a.
- •Alanine aminotransferase (ALT) and or aspartate aminotransferase (AST) more than 2 times the upper limit of normal (ULN) b.
- •Total bilirubin more than ULN c.
- •Hemoglobin less than 8 g per dL (80 g per L) for females and less than 9 g per dL (90 g per L) for males d.
- •Absolute neutrophil count (ANC) less than 1200 cells per micro-L (1.2 × 109 per L) e.
- •Thyroid-stimulating hormone (TSH) outside of the normal reference range and clinically significant, as determined by the Investigator.
- •Free T3 and free T4 will be measured if TSH level is out of range.
- •HbA1c more than 6.5 percent (more than 48 mmol per mol) g.
- •Positive test for hepatitis B surface antigen and per or hepatitis B core antibody immunoglobulin M at screening; positive hepatitis C antibody at Screening (Visit 1), with the exception of a patient for whom the reflex HCV RNA test is negative.
- •Any other clinically significant abnormal laboratory result obtained at Screening (Visit 1) or Baseline (Visit 2)
- •History of human immunodeficiency (HIV) infection.
- •History of a clinically significant cardiac disorder and or abnormal screening electrocardiogram (ECG) or a QT interval corrected for heart rate using Fridericia formula more than 460 msec;
- •Clinically significant abnormality within 2 years of Screening that in the Investigators opinion may place the patient at risk or interfere with study outcome variables; this includes, but is not limited to, history of other clinically significant neurologic, hepatic (including history of Child-Pugh score more than 5), renal, gastrointestinal, respiratory, hematologic, endocrine, or immunologic disease or history of malignancy.
- •Patients with a history of orthostatic hypotension or who have orthostatic hypotension (defined as reduction of more than equals to 20 mm Hg in systolic blood pressure [SBP] or a reduction of more than equals to 10 mm Hg in diastolic blood pressure [DBP] while changing from the supine to standing position) at Screening or Baseline (see Section 8.2.5 for details on vital sign measurements);
- •History of seizures, with the exception of febrile seizures.
- •History of significant head trauma, history of tumor of the CNS, or any other condition that predisposes to seizures.
- •Additional Exclusions:
- •The patient or LAR is an employee of the Investigator or study site, or immediate family ( child, or sibling, whether biological or legally adopted) of such employees, the Investigator, the Sponsor, or contract research organizations (CROs) conducting the study.
结局指标
主要结局
The primary efficacy objective of this study is to evaluate the efficacy of high and low doses of lumateperone vs placebo for the treatment of irritability associated with ASD in pediatric patients aged 5 to 17 years as measured by the change from baseline to end of Week 6 in the ABC Irritability (ABC-I) subscale score
时间窗: Change from baseline to the end of Week 6 in the ABC-I subscale score
次要结局
- The key secondary efficacy objective of this study is to evaluate the efficacy of high and low doses of lumateperone vs placebo for the treatment of irritability associated with ASD in pediatric patients aged 5 to 17 years as measured by the change from baseline to end of Week 6 in the Clinical Global Impression-Severity (CGI-S) score with respect to irritability(Change from baseline to the end of Week 6 in the CGI-S score)
研究者
研究点 (7)
Loading locations...
相似试验
招募中
2 期
A multicenter, randomized, blinded, placebo controlled, phase II study to evaluate the safety and efficacy of cell therapy based with artificially expanded CD4+CD25+CD127- regulatory lymphocytes and anti-CD20 antibody in pediatric patients with presymptomatic diabetes type 1 (stage 1)2023-505226-33-00Poltreg S.A.150
尚未招募
3 期
A Phase III Clinical Trial on the Efficacy and Safety of Deuremidevir Hydrobromide Dry Suspension for the Treatment of RSV Infection in Infants and Young ChildrenRespiratory Syncytial Virus InfectionNCT07402512Simcere Pharmaceutical Co., Ltd498
已完成
不适用
A study to evaluate the effect of TruBlk™ Shilajit Resin on improving sexual health in malesCTRI/2025/07/090919Amaara Ayurveda100
招募中
不适用
A study to evaluate the effect of TruBlk™ Shilajit Resin on improving exercise performance, endurance, muscle strength, and recovery in males.CTRI/2025/07/091323Amaara Ayurveda100
招募中
2 期
Epetraborole in Patients With Mycobacterium Abscessus Lung DiseaseNCT07301320Kevin Winthrop84