Trial Outcomes & Findings for A Safety/Tolerability and PK Study With Azstarys® in Children With ADHD (NCT NCT05721235)
NCT ID: NCT05721235
Last Updated: 2026-05-08
Results Overview
Summary of Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Total Sleep Disturbance Score to End of Study. The CSHQ is a retrospective, 33-item parent questionnaire to examine sleep behavior in small children. Items are rated on a 3-point scale of "Usually", "Sometimes" and "Rarely" for occurrences in a number of key sleep domains (Bedtime Resistance, sleep onset delay, sleep duration, sleep anxiety, night wakings, parasomnias, sleep disordered breathing, and Daytime Sleepiness). The total Sleep Disturbance Score is the sum of the frequency ratings of the 33 items. The lowest possible score is 33 and the highest possible score is 99. Lower scores indicate improved sleep behavior, and a mean percentage decrease is considered an improvement.
COMPLETED
PHASE4
123 participants
12 Months
2026-05-08
Participant Flow
Across 20 sites in the USA, the first subject was enrolled on 02Jun2023 and the last subject last visit occurred on 31July2025. An appropriate number of new subjects were to be enrolled in addition to roll-over subjects from the KP415.P01 efficacy trial to ensure approximately 100 subjects were enrolled in the study.
Participant milestones
| Measure |
Open Label
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate chloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Overall Study
STARTED
|
123
|
|
Overall Study
COMPLETED
|
63
|
|
Overall Study
NOT COMPLETED
|
60
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Safety/Tolerability and PK Study With Azstarys® in Children With ADHD
Baseline characteristics by cohort
| Measure |
Open Label
n=121 Participants
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Age, Continuous
|
4.5 years
STANDARD_DEVIATION .5 • n=41 Participants
|
|
Sex: Female, Male
Female
|
34 Participants
n=41 Participants
|
|
Sex: Female, Male
Male
|
87 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
16 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
105 Participants
n=41 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
4 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
2 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Black or African American
|
27 Participants
n=41 Participants
|
|
Race (NIH/OMB)
White
|
79 Participants
n=41 Participants
|
|
Race (NIH/OMB)
More than one race
|
9 Participants
n=41 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=41 Participants
|
|
Height
|
110.94 cm
STANDARD_DEVIATION 5.679 • n=41 Participants
|
|
Weight
|
19.87 kg
STANDARD_DEVIATION 2.847 • n=41 Participants
|
PRIMARY outcome
Timeframe: 12 MonthsPopulation: The Safety Analysis Population, as described per protocol, consists of 95 subjects
Summary of Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Total Sleep Disturbance Score to End of Study. The CSHQ is a retrospective, 33-item parent questionnaire to examine sleep behavior in small children. Items are rated on a 3-point scale of "Usually", "Sometimes" and "Rarely" for occurrences in a number of key sleep domains (Bedtime Resistance, sleep onset delay, sleep duration, sleep anxiety, night wakings, parasomnias, sleep disordered breathing, and Daytime Sleepiness). The total Sleep Disturbance Score is the sum of the frequency ratings of the 33 items. The lowest possible score is 33 and the highest possible score is 99. Lower scores indicate improved sleep behavior, and a mean percentage decrease is considered an improvement.
Outcome measures
| Measure |
Open Label
n=95 Participants
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate Hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Changes in Sleep Behavior
|
-6.5 percentage score change
Interval -8.0 to -5.0
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: The Safety Analysis Population, as described per protocol, consists of 95 subjects
Percent Change from Baseline in Body Weight (kg) During Treatment Phase in the Safety Population
Outcome measures
| Measure |
Open Label
n=95 Participants
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate Hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Percent Change in Body Weight
|
6.23 Percent (%) Change of Body Weight
Standard Deviation 8.162
|
PRIMARY outcome
Timeframe: 12 MonthsPopulation: The Safety Analysis Population, as described per protocol, consists of 95 subjects
Percentile Change from Baseline (cm) During Treatment Phase
Outcome measures
| Measure |
Open Label
n=95 Participants
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate Hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Percentile Change in Body Height
|
-10.77 Percentile Change of Body Height
Standard Deviation 12.990
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: The Safety Analysis Population, as described per protocol, consists of 95 subjects
Percentile Change from Baseline in Body Weight (kg) During Treatment Phase in the Safety Population
Outcome measures
| Measure |
Open Label
n=95 Participants
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate Hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Percentile Change in Body Weight
|
-15.68 Percentile Change of Body Weight
Standard Deviation 13.661
|
PRIMARY outcome
Timeframe: 12 MonthsPopulation: The Safety Analysis Population, as described per protocol, consists of 95 subjects
Percent Change from Baseline (cm) During Treatment Phase
Outcome measures
| Measure |
Open Label
n=95 Participants
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate Hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Percent Change in Body Height
|
4.72 Percent Change of Body Height
Standard Deviation 2.227
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: The Efficacy Analysis Population, as described per protocol, consists of 78 subjects
Determine efficacy with respect to the 12-month maintenance of ADHD symptom control through investigator ratings on the ADHD-RS-IV from baseline to 12 months. The ADHD-RS is an 18-item scale based on Diagnostic and Statistical Manual of Mental Disorders criteria of ADHD that rates symptoms on a 4-point scale. Each item is scored using a combination of severity and frequency ratings from a range of 0 (reflecting no symptoms or a frequency of never or rarely) to 3 (reflecting severe symptoms or a frequency of very often), so that the total ADHD-RS scores range from 0 to 54 (low score is better or shows improvement). Scores will be obtained during a clinician-directed interview with the parent/guardian/caregiver at each visit.
Outcome measures
| Measure |
Open Label
n=78 Participants
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate Hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Efficacy of ADHD Symptom Control Through Investigator Ratings on the ADHD-RS-IV
|
-28.1 score on a scale
Interval -30.9 to -25.4
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: The EfficacyAnalysis Population, as described per protocol, consists of 95 subjects
Deterimine the efficacy of the CGI-S from baseline to 12 months. The CGI-S is a clinician-rated scale that evaluates the severity of ADHD symptoms in the study on a scale from 1 (not at all ill) to 7 (among the most severely ill).
Outcome measures
| Measure |
Open Label
n=78 Participants
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate Hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Efficacy of CGI-S
|
-2.3 score on a scale
Standard Deviation 1.15
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 5 daysPopulation: This is the Population PK Dataset using nominal time points, which is a total of 55 subjects. This particular dataset analyzed at 1.5 to 2.5 hours consists of 37 subjects.
To assess the population PK of Azstarys in children 4 and 5 years old with ADHD after at least five days of daily treatment and without any major protocol deviations potentially affecting PK. Here we show model-predicted concentrations (range in ng/mL) for d-MPH and SDX. Predicted concentrations peaked at 1.5 to 2.5 hours (nominal time point) and shown below for the 37 subjects analyzed at this time point.
Outcome measures
| Measure |
Open Label
n=37 Participants
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate Hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Population PK of Azstarys
Dose 39.2 mg/7.8mg - 1.5 to 2.5 hrs
|
33.55 ng/mL
Interval 27.12 to 42.97
|
|
Population PK of Azstarys
Dose 13.1 mg/2.6mg - 1.5 to 2.5 hrs
|
7.37 ng/mL
Interval 6.89 to 10.44
|
|
Population PK of Azstarys
Dose 26.1 mg/5.2 mg - 1.5 to 2.5 hrs
|
18.79 ng/mL
Interval 15.36 to 23.63
|
Adverse Events
Open Label
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Open Label
n=95 participants at risk
13.1 mg/2.6 mg SDX/d-MPH, 26.1/5.2 mg SDX/d-MPH, or 39.2 mg/7.8 mg SDX/d-MPH
Serdexmethylphenidate hydrochloride (SDX) and dexmethylphenidate (d-MPH)
|
|---|---|
|
Infections and infestations
Upper Respiratory Tract Infection
|
7.4%
7/95 • The occurrence of treatment emergent adverse events (>/= 5%), by protocol definition, was assessed following the first dose of study drug in the Treatment Phase and ending five days after the last dose of study drug or early termination visit for the treatment phase population (N=95 subjects). This time period was 12 months. No deaths or SAEs were reported in this study among all enrolled subjects, including those who participated in the dose optimization phase only.
|
|
Infections and infestations
Ear Infection
|
6.3%
6/95 • The occurrence of treatment emergent adverse events (>/= 5%), by protocol definition, was assessed following the first dose of study drug in the Treatment Phase and ending five days after the last dose of study drug or early termination visit for the treatment phase population (N=95 subjects). This time period was 12 months. No deaths or SAEs were reported in this study among all enrolled subjects, including those who participated in the dose optimization phase only.
|
|
Infections and infestations
Pharyngitis Streptococcal
|
6.3%
6/95 • The occurrence of treatment emergent adverse events (>/= 5%), by protocol definition, was assessed following the first dose of study drug in the Treatment Phase and ending five days after the last dose of study drug or early termination visit for the treatment phase population (N=95 subjects). This time period was 12 months. No deaths or SAEs were reported in this study among all enrolled subjects, including those who participated in the dose optimization phase only.
|
|
Infections and infestations
Influenza
|
5.3%
5/95 • The occurrence of treatment emergent adverse events (>/= 5%), by protocol definition, was assessed following the first dose of study drug in the Treatment Phase and ending five days after the last dose of study drug or early termination visit for the treatment phase population (N=95 subjects). This time period was 12 months. No deaths or SAEs were reported in this study among all enrolled subjects, including those who participated in the dose optimization phase only.
|
|
Psychiatric disorders
Insomnia
|
6.3%
6/95 • The occurrence of treatment emergent adverse events (>/= 5%), by protocol definition, was assessed following the first dose of study drug in the Treatment Phase and ending five days after the last dose of study drug or early termination visit for the treatment phase population (N=95 subjects). This time period was 12 months. No deaths or SAEs were reported in this study among all enrolled subjects, including those who participated in the dose optimization phase only.
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
9.5%
9/95 • The occurrence of treatment emergent adverse events (>/= 5%), by protocol definition, was assessed following the first dose of study drug in the Treatment Phase and ending five days after the last dose of study drug or early termination visit for the treatment phase population (N=95 subjects). This time period was 12 months. No deaths or SAEs were reported in this study among all enrolled subjects, including those who participated in the dose optimization phase only.
|
|
Investigations
Weight Decreased
|
5.3%
5/95 • The occurrence of treatment emergent adverse events (>/= 5%), by protocol definition, was assessed following the first dose of study drug in the Treatment Phase and ending five days after the last dose of study drug or early termination visit for the treatment phase population (N=95 subjects). This time period was 12 months. No deaths or SAEs were reported in this study among all enrolled subjects, including those who participated in the dose optimization phase only.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee PI has the right to publish results pertaining to PI's Study activities in accordance with this Agreement for their own publication objectives, provided that it doesn't disclose Confidential Information (CI) as described in this Agreement. At least 90 days prior to submission, PI shall submit to Sponsor review of any Publication. Sponsor will advise PI in writing of any CI that may impair Sponsor's ability to obtain patent protection \& have the right to require PI to remove CI or factual errors
- Publication restrictions are in place
Restriction type: OTHER