Trial Outcomes & Findings for A Pivotal Efficacy Trial to Evaluate HLD200 in Children With ADHD in a Classroom Setting (NCT NCT02493777)

NCT ID: NCT02493777

Last Updated: 2021-07-23

Results Overview

The SKAMP is a validated, 13-item, observer-rated scale designed to assess the level of impairment of classroom-observed behaviors (Wigal and Wigal, 2006). Items 1 through 4 assess subject attention; items 5 through 8 assess deportment; items 9 through 11 assess quality of work; while items 12 and 13 assess subject compliance with teacher/classroom rules. Each individual item is rated on a 7-point scale from 0 (normal, no impairment) to 6 (maximal impairment). When all individual item scores are summed together, they produce a 13-item combined score that ranges from 0 to 78, with higher scores signifying greater impairment. In the present study, the SKAMP rating scale was utilized across 9 sessions occuring at 8:00 am, 9:00 am, 10:00 am, 12:00 pm, 2:00 pm, 4:00 pm, 6:00 pm, 7:00 pm, and 8:00 pm of the laboratory classroom day. Successful training of qualified individuals on the SKAMP scale was required before raters were allowed to perform study assessments.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

125 participants

Primary outcome timeframe

12-hours from 8:00 am to 8:00 pm

Results posted on

2021-07-23

Participant Flow

A total of 125 participants were enrolled in this study, of which 119 were randomized to double-blind, placebo-controlled treatment at 6 sites in the United States from 28 July, 2015 to 04 March, 2015.

The study consisted of a screening period of up-to 4-weeks, followed by a 6-week open-label HLD200 treatment-optimization period that was followed by a 1-week randomized (1:1), placebo-controlled, double-blind period.

Participant milestones

Participant milestones
Measure
HLD200 (Methylphenidate)
Participants received HLD200 capsules (containing beads with methylphenidate in a dual-coated drug-layered core; optimzed at 20, 40, 60, 80 or 100 mg) orally once daily in the evening during a 1-week randomized (1:1), placebo-controlled, double-blind-period that followed a 6-week open-label HLD200 treatment-optimization period.
Placebo
Participants recieved placebo (matched to their dose-optimzed HLD200 capsules, but containing microcrystalline cellulose beads in place of methylphenidate) orally once daily in the evening during a 1-week randomized (1:1), placebo-controlled, double-blind period that followed a 6-week open-label HLD200 treatment-optimization period.
Overall Study
STARTED
65
54
Overall Study
COMPLETED
65
53
Overall Study
NOT COMPLETED
0
1

Reasons for withdrawal

Reasons for withdrawal
Measure
HLD200 (Methylphenidate)
Participants received HLD200 capsules (containing beads with methylphenidate in a dual-coated drug-layered core; optimzed at 20, 40, 60, 80 or 100 mg) orally once daily in the evening during a 1-week randomized (1:1), placebo-controlled, double-blind-period that followed a 6-week open-label HLD200 treatment-optimization period.
Placebo
Participants recieved placebo (matched to their dose-optimzed HLD200 capsules, but containing microcrystalline cellulose beads in place of methylphenidate) orally once daily in the evening during a 1-week randomized (1:1), placebo-controlled, double-blind period that followed a 6-week open-label HLD200 treatment-optimization period.
Overall Study
Lost to Follow-up
0
1

Baseline Characteristics

A Pivotal Efficacy Trial to Evaluate HLD200 in Children With ADHD in a Classroom Setting

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
HLD200 (Methylphenidate)
n=64 Participants
Placebo
n=53 Participants
Total
n=117 Participants
Total of all reporting groups
Age, Continuous
9.6 Years
STANDARD_DEVIATION 1.58 • n=5 Participants
9.3 Years
STANDARD_DEVIATION 1.68 • n=7 Participants
9.4 Years
STANDARD_DEVIATION 1.63 • n=5 Participants
Sex: Female, Male
Female
22 Participants
n=5 Participants
15 Participants
n=7 Participants
37 Participants
n=5 Participants
Sex: Female, Male
Male
42 Participants
n=5 Participants
38 Participants
n=7 Participants
80 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 12-hours from 8:00 am to 8:00 pm

Population: Intent-to-Treat

The SKAMP is a validated, 13-item, observer-rated scale designed to assess the level of impairment of classroom-observed behaviors (Wigal and Wigal, 2006). Items 1 through 4 assess subject attention; items 5 through 8 assess deportment; items 9 through 11 assess quality of work; while items 12 and 13 assess subject compliance with teacher/classroom rules. Each individual item is rated on a 7-point scale from 0 (normal, no impairment) to 6 (maximal impairment). When all individual item scores are summed together, they produce a 13-item combined score that ranges from 0 to 78, with higher scores signifying greater impairment. In the present study, the SKAMP rating scale was utilized across 9 sessions occuring at 8:00 am, 9:00 am, 10:00 am, 12:00 pm, 2:00 pm, 4:00 pm, 6:00 pm, 7:00 pm, and 8:00 pm of the laboratory classroom day. Successful training of qualified individuals on the SKAMP scale was required before raters were allowed to perform study assessments.

Outcome measures

Outcome measures
Measure
HLD200 (Methylphenidate)
n=64 Participants
Placebo
n=53 Participants
Swanson, Kotkin, Agler, M-Flynn and Pelham Combined Score (SKAMP CS) - Model-adjusted Average of All Post-dose Time Points Assessed During a Laboratory Classroom Test Day (Visit 9).
14.8 SKAMP CS (12-hour average)
Standard Error 1.17
20.7 SKAMP CS (12-hour average)
Standard Error 1.22

SECONDARY outcome

Timeframe: PREMB-R AM mean subscale score for the 2-days prior to Visit 9.

Population: Intent-to-Treat

The PREMB-R is an 11-item rating scale designed to assess at-home functional impairments in children with ADHD during both early morning (AM) and late afternoon/evening (PM) time periods. With demonstrated validity and reliablility (Faraone et al., 2015), the AM subscale total score (sum of items 1 to 3) was designated in this study as a key secondary endpoint. Items are scored from 0 (none) to 3 (a lot), with higher scores signifying greater impairment of function. The PREMB-R rating scale was completed by parents during the 2-days prior to study visits at the beginning and end of the open-label period (Visits 2 and 8, respectively), as well as at the end of the randomized, double-blind period (Visit 9). At each visit, these ratings were used only for review by the clinician (MD, PhD, DO, licensed social worker, or any trained mental health professional approved by the sponsor) as part of a structured interview to enable collection of a clinician-rated PREMB-R AM subscale score.

Outcome measures

Outcome measures
Measure
HLD200 (Methylphenidate)
n=64 Participants
Placebo
n=53 Participants
Parent Rating of Evening and Morning Behavior Revised, Morning Subscale (PREMB-R AM).
0.9 PREMB-R AM subscale total score
Standard Error 0.27
2.7 PREMB-R AM subscale total score
Standard Error 0.27

Adverse Events

HLD200 (Methylphenidate)

Serious events: 0 serious events
Other events: 14 other events
Deaths: 0 deaths

Placebo

Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
HLD200 (Methylphenidate)
n=65 participants at risk
Placebo
n=54 participants at risk
Psychiatric disorders
Any Insomnia
7.7%
5/65 • Adverse events were collected across the duration of the study (up to 13-weeks). However, an adverse event is considered treatment-emergent during the randomized, placebo-controlled phase only if it started on or after the first dose of double-blind study medication, but before the last dose of double blind study medication plus 72 hours as reflected in the table below.
Adverse events were recorded by study staff at every post-screening study visit based on spontaneous report from subjects/parents, regardless of causality. However, sleep-related adverse event were directly solicited from subjects/parents by study staff using a structured interview format to assessing sleep disturbances (e.g., issues with sleep onset, quality and quantity), rather than obtained from spontaneous reporting.
9.3%
5/54 • Adverse events were collected across the duration of the study (up to 13-weeks). However, an adverse event is considered treatment-emergent during the randomized, placebo-controlled phase only if it started on or after the first dose of double-blind study medication, but before the last dose of double blind study medication plus 72 hours as reflected in the table below.
Adverse events were recorded by study staff at every post-screening study visit based on spontaneous report from subjects/parents, regardless of causality. However, sleep-related adverse event were directly solicited from subjects/parents by study staff using a structured interview format to assessing sleep disturbances (e.g., issues with sleep onset, quality and quantity), rather than obtained from spontaneous reporting.
Investigations
Blood pressure diastolic increased
13.8%
9/65 • Adverse events were collected across the duration of the study (up to 13-weeks). However, an adverse event is considered treatment-emergent during the randomized, placebo-controlled phase only if it started on or after the first dose of double-blind study medication, but before the last dose of double blind study medication plus 72 hours as reflected in the table below.
Adverse events were recorded by study staff at every post-screening study visit based on spontaneous report from subjects/parents, regardless of causality. However, sleep-related adverse event were directly solicited from subjects/parents by study staff using a structured interview format to assessing sleep disturbances (e.g., issues with sleep onset, quality and quantity), rather than obtained from spontaneous reporting.
13.0%
7/54 • Adverse events were collected across the duration of the study (up to 13-weeks). However, an adverse event is considered treatment-emergent during the randomized, placebo-controlled phase only if it started on or after the first dose of double-blind study medication, but before the last dose of double blind study medication plus 72 hours as reflected in the table below.
Adverse events were recorded by study staff at every post-screening study visit based on spontaneous report from subjects/parents, regardless of causality. However, sleep-related adverse event were directly solicited from subjects/parents by study staff using a structured interview format to assessing sleep disturbances (e.g., issues with sleep onset, quality and quantity), rather than obtained from spontaneous reporting.

Additional Information

Chief Scientific Officer

Ironshore Pharmaceuticals and Development, Inc.

Phone: 13457498174

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place