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Zynerba Pharmaceuticals Presents Data Showing that the Baseline Characteristics of Patients in Phase 2 BRIGHT Trial in Autism Spectrum Disorder Indicate a Moderate-to-Severe Study Population
The poster entitled Phase 2 BRIGHT (An Exploratory Open-Label Tolerability and Efficacy Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Autism Spectrum Disorder) Trial: Baseline Characteristics (poster #27) is being presented at the
Zynerba’s Chief Medical Officer,
“ASD is a complex neurodevelopmental disorder characterized by difficulties with behaviors, communication, and social interaction,” said
The endocannabinoid system - a key modulator of emotion and social behavior - is dysregulated in ASD, and published data suggest that cannabidiol (CBD) may provide therapeutic benefit. However, the efficacy and safety of CBD in patients with ASD have not been well established. Zynerba is undertaking the 14-week BRIGHT Phase 2 exploratory trial in children and adolescents (ages four through 17 years) with ASD as confirmed by Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5) diagnostic criteria to assess the safety and efficacy of Zygel in treating ASD-related behaviors as measured by a variety of efficacy assessments which are shown in Figure 1, below. After completing dosing in the 14-week period, participants may enroll in a six-month extension trial.
Figure 1. Schedule of Screening and Efficacy Assessments is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/a15d5dd4-9ab2-497d-a8ec-7782f612ebca.
The trial protocol included certain inclusion and exclusion criteria to enrich the trial population for disease severity at baseline, as measured by the following assessments:
- A 58-item caregiver-rated scale measuring behaviors across 5 subscales: irritability/agitation (maximum score: 45), lethargy/social withdrawal (maximum score: 48), stereotypic behavior (maximum score: 21), hyperactivity/noncompliance (maximum score: 48), inappropriate speech (maximum score: 12);
- Each behavior is scored from 0 (“not at all a problem”) to 3 (“the problem is severe in degree”);
- Higher scores indicate greater severity of aberrant behavior.
- A diagnostic tool consisting of 5 age - and verbal ability - dependent modules that assess social communication and core behaviors of ASD;
- Each item is scored by a trained test administrator from 0 (“no abnormality of type specified”) to 3 (“moderate to severe abnormality”);
- ADOS total scores are diagnostic; however, standardized comparison scores can be used to measure severity;
- Comparison scores range from 0-10, with scores of <5 indicating mild ASD, scores of 5-7 indicating moderate ASD, and scores of 8-10 indicating severe ASD.
- A 25-item parent-rated scale assessing anxiety in ASD;
- Each item is scored from 0 (“not present”) to 3 (“very frequent and a major problem”);
- Maximum score is 75, with scores >52 indicating possible clinical anxiety.
Baseline Disease Characteristics
As seen in Table 1 below, the majority of patients had moderate or severe ASD at baseline as measured by the ADOS-2 comparison score (94%) and DSM-5 severity levels (92%). In addition, the mean ABC-C Irritability score was 30.0, and 24% of the enrolled patients had PRAS-ASD scores indicative of possible clinical anxiety, further highlighting the severity of symptoms in the enrolled patient population.
Table 1. Baseline Disease Characteristics of Patients Enrolled in BRIGHT
|Disease Characteristics||Patients in BRIGHT
|ABC-C Irritability Subscale score (0-45)|
|Mean (range)||30.0 (18-43)|
|PRAS-ASD score (0-75; >52 suggests possible clinical anxiety)|
|Mean (range)||40.9 (21-68)|
|>52, n (%)||9 (24.3)|
|DSM-5 severity leveli|
|Level 1 (mild), n (%)||3 (8.1)|
|Level 2 (moderate), n (%)||15 (40.5)|
|Level 3 (severe), n (%)||19 (51.4)|
|ADOS®-2 comparison score|
|Mean (range)||7.5 (4-10)|
|<5 (mild ASD), n (%)||2 (5.6)|
|5-7 (moderate ASD), n (%)||19 (52.8)|
|8-10 (severe ASD), n (%)||15 (41.7)|
The authors conclude that the Phase 2 BRIGHT trial has successfully enrolled a broad patient population and was enriched for disease severity to avoid floor effects on outcome measures. The baseline characteristics indicate a patient population with predominantly moderate-to-severe ASD, with a high level of clinically significant anxiety.
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i DSM-5 severity levels are based on degree of social communication impairment and behavioral flexibility. The levels indicate patients “requiring support” (level 1), “requiring substantial support” (level 2), and “requiring very substantial support” (level 3).
Source: Zynerba Pharmaceuticals, Inc.