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INP105 Proof-of-concept Study for the Acute Treatment of Agitation in Adolescents and Young Adults With ASD

Phase 2
Terminated
Conditions
Agitation in Adolescents and Young Adults With ASD
Interventions
Combination Product: INP105
Combination Product: Placebo
Registration Number
NCT05163717
Lead Sponsor
Impel Pharmaceuticals
Brief Summary

This is a Phase 2a, proof-of concept, 2-way, 2-period crossover, double-blind study to evaluate the safety and efficacy of INP105 as an acute treatment versus placebo in adolescents and young adults with autism spectrum disorder (ASD) experiencing agitation. Approximately 32 ASD patients who are currently being treated for agitation/aggression at several inpatient units specializing in behavioral treatment will be enrolled.

INP105 is a novel combination product that sprays a powder formulation of olanzapine to the upper nasal space. An earlier formulation showed a similar extent, but faster rate of absorption compared to the approved intramuscular product. In this study, 5 mg of olanzapine or placebo will be delivered nasally by this combination product to moderately or severely agitated participants.

Participants will undergo several screening assessments, including observation session(s) of episode(s) of agitation resulting from a frustration task (eg, a non-preferred activity). At least one observation session must result in a documented moderate to severe agitation episode prior to the participant being eligible to enroll in the study and be randomized to treatment.

The study will be conducted in 2 phases. A pilot phase will initially enroll at least 6 participants, who will receive both 5 mg INP105 (5 mg olanzapine) and placebo in random order, in the same crossover design as later participants. Participants will be dosed during a documented moderate to severe episode of agitation. Once 6 participants have completed both dosing periods and have at least 48 hours of post-dose safety data collected, a safety and preliminary efficacy analysis will be performed by an independent unblinded statistical group, and a summary report forwarded to a sponsor-led Data and Safety Review Committee (DSRC), who will remain blinded. Enrollment will be paused during the DSRC pilot phase safety and preliminary efficacy results review. Absent any concerning safety signals, the second phase will enroll all remaining participants. The DSRC may suggest revisions to the protocol, and the protocol amended and approved as necessary, prior to further participants being enrolled.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
8
Inclusion Criteria
  • Confirmed autism spectrum disorder diagnosis
  • Admitted as an inpatient to a behavioral unit prior to informed consent
  • Displays episodes of moderate to severe agitation
Exclusion Criteria
  • Hypersensitivity to olanzapine
  • History of severe head trauma, stroke, endocrine disorder, or cardiovascular disease
  • History of hypotension
  • Currently on a chronic dose of olanzapine
  • Currently taking ciprofloxacin, enoxacin, fluvoxamine, or carbamazepine

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
INP105INP105POD-olanzapine (INP105), 5 mg, single dose, to be delivered to each participant
PlaceboPlaceboPOD-placebo, single dose, to be delivered to each participant
Primary Outcome Measures
NameTimeMethod
Incidence of adverse events and serious adverse events in the INP105 and placebo groups up to 48 hours post-doseFrom dosing to 48 hours post dosing

All adverse events, serious or not, will be recorded from time of dosing with either INP105 or placebo up until 48 hours post-dose, or until the next treatment is given, which ever is sooner.

Overall incidence of adverse events and serious adverse events in the INP105 and placebo groupsFrom dosing to end of follow-up (7 days), or to the start of next blinded treatment (48 hours), as applicable

All adverse events will be recorded as treatment emergent from after dosing until the next treatment, or until last study visit, as applicable.

Secondary Outcome Measures
NameTimeMethod
Clinical Global Impressions - Improvement (CGI-I) score at 30 minutes post-dosePre-dose to 30 minutes post-dose

The CGI-I is a 7 point scale that will be used to assess global improvement in the patient's condition on a scale that ranges from 0 "not assessed", 1 "very much improved" to 7 "very much worse".

Change in Agitation-Calmness Evaluation Scale (ACES) score at 30 minutes post-dosePre-dose to 30 minutes post-dose

The ACES is a 9 point scale that measures the degree of agitation versus sedation, ranging from a score of 1 "marked agitation" to 4 "normal" to 9 "unable to be aroused".

Change in Overt Aggression Scale (OAS) score at 30 minutes post-dosePre-dose to 30 minutes post-dose

The OAS measures the degree of aggression, using 4 categories of aggression, defined as "verbal aggression", "aggression against objects", "aggression against self", and "aggression against other people". The higher a category is rated, the more severe the degree of aggression. The total score is the sum of the scores from the 4 categories.

Change in Positive and Negative Syndrome Scale - Excited Component (PEC) score at 30 minutes post-dosePre-dose to 30 minutes post-dose

The PEC describes 5 behaviors related to negative aspects of excitability; excitement, tension, hostility, uncooperativeness, poor impulse control. The rater scores each of these aspects from 1 (not present) to 7 (extreme), for a total score that can range from 5 to 35.

Change in irritability behavior frequency counts at 30 minutes post-dosePre-dose to 30 minutes post-dose

Persons with autism can often display repetitive behaviors. These repetitive behaviors, such as rocking, hitting, kicking, will be counted pre-dose and during intervals post-dose.

Clinical Global Impressions - Efficacy (CGI-E) score at 30 minutes post-dosePre-dose to 30 minutes post-dose

The CGI-E is a scale that measures the efficacy of an intervention balanced by any negative side effects. Scores can range from 1 (vast improvement and no side effects) to 16 (unchanged or worse and side effects that outweigh therapeutic effect).

Change in modified Aberrant Behavior Checklist - Irritability Subscale (ABC-I) score at 60 minutes post-dosePre-dose and at 60 minutes post-dose

The ABC-I lists15 behaviors, including aggression, tantrums, crying, seen with irritability and these are scored from 0 "absent" to 3 "severe". Score can therefore range from 0 to 45 (worst irritability).

Change in Behavioral Activity Rating Scale (BARS) score at 30 minutes post-dosePre-dose to 30 minutes post-dose

The BARS is a 7 point scale measuring the degree of agitated behavior, ranging from 1 "difficult or unable to rouse" to 4 "quiet and awake (normal level of activity)" to 7 "violent, requires restraint.

Time to reach an ACES score of 4 (normal) post-dosingDosing until 120 minutes post-dose

The length of time it will take the participant to move from a score of 2 or 1 on the ACES scale to a score of 4 will be determined.

Frequency of administering pharmaceutical rescue intervention within 120 minutes after dosingDosing until 120 minutes post-dose

The frequency of administering any pharmaceutical intervention other than the study intervention from dosing until 120 minutes after dosing will be recorded and compared for INP105 versus placebo.

Trial Locations

Locations (2)

Maine Behavioral Healthcare

🇺🇸

Portland, Maine, United States

Cincinnati Children's Hospital Medical Center

🇺🇸

Cincinnati, Ohio, United States

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