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Adaptive Phase II Study to Evaluate the Safety & Efficacy of NaBen®

Phase 2
Terminated
Conditions
Schizophrenia
Interventions
Drug: Placebo
Registration Number
NCT01908192
Lead Sponsor
SyneuRx International (Taiwan) Corp
Brief Summary

The purpose of this study is to determine if NaBen® is a safe and effective add-on treatment for schizophrenia in adolescents.

Detailed Description

This is a two-part, multi-center, prospective, randomized, placebo-controlled, parallel-group study, in which adolescent subjects with schizophrenia will be enrolled. Overall, eligible subjects will be randomized in a pre-defined 1:1 ratio to NaBen® or placebo.

This study will be conducted in two parts:

In Part 1 (Phase IIb) of the study, 76 subjects (\~ 60% of the total planned subjects) will be randomized in a 1:1 ratio (NaBen® or placebo), of which 38 subjects will be randomized to the NaBen® group and 38 subjects to the placebo group. An interim analysis (IA) will be conducted after the randomization of the 76th subject in Part 1 of the study. The data will be analyzed after all enrolled subjects in Part 1 of the study complete Visit 5 (week 6) or are withdrawn from the study, whichever occurs first. The data from IA will be reviewed by an independent Data Safety and Monitoring Committee (DSMC) that will be responsible for the review of the data from the Part 1 (Phase IIb) of the study for both safety and the effectiveness.

In Part 2 (Phase III) of the study, a total of 50 subjects will be randomized, of which 25 subjects will be randomized to the NaBen® group and 25 subjects to the placebo group. The final subject numbers in the study will depend on the sample size re-estimation after Part 1 of the study.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
60
Inclusion Criteria
  • Male or female subjects who are between 12 and 17 years of age inclusive
  • Physician confirmed DSM-IV or -V diagnosis of schizophrenia based on MINI International Neuropsychiatric Interview for Schizophrenia and Psychotic Disorders Studies for Children and Adolescents, version 6.0 (MINI-KID, Version 6.0)
  • Are clinically stable with residual symptoms, defined as a total score of ≥ 60 of PANSS and a score of ≥ 40 for SANS
  • An unchanged antipsychotic medication regimen for at least eight (8) weeks prior to randomization into the study and expected to remain unchanged during the study (longer for depot or long-acting antipsychotics: ten (10) months for Aripiprazole (Maintena®) and Paliperidone (Xeplion®); six (6) months for Olanzapine pamoate monohydrate (Zypadhera®); and at least 6 times duration of the reported half life or minimum four (4) months for other depot or long-acting antipsychotics)
  • In good general physical health and all physical exam, neurological exam and laboratory assessments (urine/blood routine, biochemical tests and ECG) are clinically unremarkable per the investigator
  • Subject has a negative urine illicit drug screening test
  • Subject understands and is willing to sign the Informed Assent Form (IAF) prior to study entry and agrees to be available for all the study visits
  • The subject's guardian understands and is willing to sign the Informed Consent Form (ICF) prior to study entry and agrees to be available for all the study visits
  • Must not be a danger to self or others and must have family support available to be maintained as outpatients
Exclusion Criteria
  • Meets the DSM-IV or -V criteria at screening for mental retardation, dissociative disorder, bipolar disorder, major depressive disorder, schizoaffective disorder, schizophreniform disorder, autistic disorder, or primary substance induced psychotic disorder. Other comorbid disorders; e.g., attention-deficit hyperactivity disorder (ADHD), are allowed as long as schizophrenia is the primary diagnosis and the comorbid disorder(s) do not require medication.
  • Subjects whose illness was resistant to antipsychotics according to prior trials of two different antipsychotics of adequate dose
  • History of epilepsy, head trauma, or neurological illness other than Tourette's syndrome
  • History of allergic reaction to sodium benzoate
  • Serious medical illnesses such as acute or chronic renal disease, liver failure or heart disease that, in the opinion of the investigator, may interfere with the conduct of the study.
  • Current substance abuse or positive urine illicit drug screening or history of substance dependence (including alcohol, but excluding nicotine and caffeine) in the past three (3) months.
  • Use of depot antipsychotics in the past six (6) months
  • Inability to follow protocol
  • Body Mass Index (BMI) > 35
  • Female subjects who are pregnant (as confirmed by urine pregnancy test performed at screening Visit) or are nursing, or who do not agree to abstinence or birth control during the study
  • Cancer within the last three (3) years except for basal cell carcinoma and squamous cell carcinoma
  • Previous participation in an intervention trial within 30 days of randomization
  • Subjects whose PANSS score has decreased more than 10 percent during the Screening Phase

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboThe control treatment is placebo.
NaBen®NaBen®NaBen® is a white oral tablet (500 mg), which will be taken twice daily at a total dose of 1000 mg/day during this study.
Primary Outcome Measures
NameTimeMethod
Mean change from baseline in Positive and Negative Syndrome Scale (PANSS) total score after 6 weeks of treatmentPositive and Negative Syndrome Scale will be assessed at Visit 1 (Screening), Visit 3, 4, 5, and 6.
Secondary Outcome Measures
NameTimeMethod
Percent change from baseline in Positive and Negative Syndrome Scale (PANSS) total score from baseline after 6 weeks of treatmentPositive and Negative Syndrome Scale will be assessed at Visit 1 (Screening), Visit 3, 4, 5, and 6.
Percentage of subjects with 20% or more reduction in Positive and Negative Syndrome Scale (PANSS) total score from baseline after six (6) weeks of treatmentPositive and Negative Syndrome Scale will be assessed at Visit 1 (Screening), Visit 3, 4, 5, and 6
Percent change in Scale for Assessment of Negative Symptoms (SANS) total scoresScale for Assessment of Negative Symptoms will be assessed at Visit 1 (Screening), Visit 3,4,5, and 6
Percent change in Scale for Assessment of Negative Symptoms (SANS) sub-scale scoresScale for Assessment of Negative Symptoms will be assessed at Visit 1 (Screening), Visit 3,4,5, and 6
Percent change from baseline in the PANSS total score after 6 weeks of treatmentPositive and Negative Syndrome Scale will be assessed at Visit 1 (Screening), Visit 3, 4, 5, and 6
Percent change in Positive and Negative Syndrome Scale (PANSS) sub-scalesPositive and Negative Syndrome Scale will be assessed at Visit 1 (Screening), Visit 3, 4, 5, and 6.

Trial Locations

Locations (22)

John Hopkins University - Hugo W Moser Research Institute at Kennedy Krieger Inc.

🇺🇸

Baltimore, Maryland, United States

University Hospitals Case Medical Center

🇺🇸

Cleveland, Ohio, United States

Premier Clinical Research Institute

🇺🇸

Miami, Florida, United States

University of Minnesota Medical Center - Department of Psychiatry

🇺🇸

Minneapolis, Minnesota, United States

University of Cincinnati - Dept. of Psychiatry and Behavioral Neuroscience

🇺🇸

Cincinnati, Ohio, United States

Focus and Balance LLC

🇺🇸

San Antonio, Texas, United States

Precise Research Centers

🇺🇸

Flowood, Mississippi, United States

Veteran General Hospital Taipei

🇨🇳

Taipei, Taiwan

Children's National Health System

🇺🇸

Washington, District of Columbia, United States

Institute of Living/Hartford Hospital

🇺🇸

Hartford, Connecticut, United States

Michigan Clinical Research Institute

🇺🇸

Ann Arbor, Michigan, United States

Harmonex Neuroscience Research

🇺🇸

Dothan, Alabama, United States

CiTrials

🇺🇸

Riverside, California, United States

Renew Behavioral Health, Inc.

🇺🇸

Long Beach, California, United States

Medical Research Group of Central Florida

🇺🇸

Orange City, Florida, United States

Atlanta Center for Medical Research

🇺🇸

Atlanta, Georgia, United States

University of Massachusetts Medical School - Psychiatry Department

🇺🇸

Worcester, Massachusetts, United States

Finger Lakes Clinical Research

🇺🇸

Rochester, New York, United States

Pacific Institute of Medical Sciences

🇺🇸

Bothell, Washington, United States

Zain Research, LLC

🇺🇸

Richland, Washington, United States

Chang Gung Memorial Hospital (Linkou)

🇨🇳

New Taipei City, Taiwan

Chang Gung Memorial Hospital (Taipei)

🇨🇳

Taipei, Taiwan

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