NT0102 in the Treatment of Children With Attention Deficit Hyperactivity Disorder (ADHD)

The primary efficacy endpoint was derived from the SKAMP-Combined score calculated as the total score of all 13 items of the SKAMP-Combined score. The SKAMP-Combined score was obtained by summing up each item score where each item is rated on a 7-point impairment scale (0=normal to 6=maximal impairment) for a total possible score of 0 to 78. A lower score indicates less symptomatology (i.e., is better). The SKAMP was a rating scale that specifically measures the classroom manifestations of ADHD. The SKAMP ratings were completed for all subjects at baseline (pre-dose) and at 1, 3, 5, 7, 10, 12, and 13 hours post-dose on the classroom testing day (Visit 8). The primary analysis time point for the primary efficacy endpoint was the average of all post-dose SKAMP scores during the 13-hour period.

Time frame: Visit 8 (Day 42)

Population: The full analysis set (FAS) consisted of all participants randomized to treatment who had at least 1 post-dose SKAMP-Combined treatment assessment during the classroom testing session on Visit 8. Participants were analyzed as randomized.

Duration of effect was defined as the last time point at which NT0102 separates from placebo on SKAMP-Combined scores. A separation was defined as a statistically significant difference at the 5% level of active drug over placebo. Data was collected separately for NT0102 and Placebo arms, and is reported as a comparison analysis of the two arms. This assessment was collected on the full classroom day, Visit 8.

Time frame: Visit 8 (Day 42) at 1 hour (h), 3 h, 5 h, 7 h, 10 h, 12 h and 13 h

Population: FAS consisted of all participants randomized to treatment who had at least 1 post-dose SKAMP-Combined treatment assessment during the classroom testing session on Visit 8. Participants were analyzed as randomized.

An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.

Time frame: Visit 9 (Day 43)

Population: The safety population included all participants who took at least 1 dose of study drug.

Onset of effect was defined as the first time point at which NT0102 separates from placebo on SKAMP-Combined scores. A separation was defined as a statistically significant difference at the 5% level of active drug over placebo. Data was collected separately for NT0102 and Placebo arms and is reported as a comparison analysis of the two arms. This assessment was collected on the full classroom day, Visit 8.

Time frame: Visit 8 (Day 42) at 1 hour (h), 3 h, 5 h, 7 h, 10 h, 12 h and 13 h

Population: FAS consisted of all participants randomized to treatment who had at least 1 post-dose SKAMP-Combined treatment assessment during the classroom testing session on Visit 8. Participants were analyzed as randomized.

The PERMP consisted of 400 math problems and was graded as number of problems Attempted (PERMP-A) and number of problems Correct. (PERMP-C). It was an objective measure of performance during the classroom testing day.

Time frame: Visit 8 (Day 42)

Population: FAS consisted of all participants randomized to treatment who had at least 1 post-dose SKAMP-Combined treatment assessment during the classroom testing session on Visit 8. Participants were analyzed as randomized.

The PERMP consisted of 400 math problems and was graded as number of problems Attempted (PERMP-A) and number of problems Correct. (PERMP-C). It was an objective measure of performance during the classroom testing day.

Time frame: Visit 8 (Day 42)

Population: FAS consisted of all participants randomized to treatment who had at least 1 post-dose SKAMP-Combined treatment assessment during the classroom testing session on Visit 8. Participants were analyzed as randomized.

The SKAMP Rating Scale was comprised of 2 behavioral subscales, including the Attention subscale (4 items). The SKAMP-Attention subscore evaluates concentration in the classroom and is obtained by summing up each item score where each item is rated on a 7-point impairment scale (0=normal to 6=maximal impairment) for total possible combined score of 0 to 24. A lower score indicates less symptomatology (i.e. is better). The SKAMP-Attention subscores were derived from 20 minutes of direct observations of participant behavior. Ratings were based on the frequency and quality of behaviors.

Time frame: Visit 8 (Day 42)

Population: The per-protocol set were a subset of the FAS consisting of those participants who satisfied all of the inclusion/exclusion criteria and who correctly received the treatment to which they were randomized.

The SKAMP Rating Scale is comprised of 2 behavioural subscales, including the Deportment subscale (4 items). The SKAMP-Deportment subscore evaluates behaviour in the classroom and is obtained by summing up each item score where each item is rated on a 7-point impairment scale (0=normal to 6=maximal impairment) for total possible combined score of 0 to 24. A lower score indicates less symptomatology (i.e. is better). The SKAMP-Attention subscores were derived from 20 minutes of direct observations of participant behaviour. Ratings were based on the frequency and quality of behaviours.

Time frame: Visit 8 (Day 42)

Population: The Per-Protocol Set were a subset of the FAS consisting of those participants who satisfied all of the inclusion/exclusion criteria and who correctly received the treatment to which they were randomized.