Measurement of Treatment Effects

Matthew J O'Brien, PhD, BCBA-D

Status and phase: Not yet enrolling; Early Phase 1

Conditions: Autism Spectrum Disorder; Challenging Behavior

Treatments: Behavioral: Behavioral therapy; Drug: Psychotropic medication (at discretion of psychiatrist)

Study type: Interventional

Funder types: Other

Identifiers

NCT07024303

202411526

Details and patient eligibility

About

The overall objective is to evaluate and compare the efficacy of two common treatments for CB in children and adolescents with autism using multiple outcome measures. Our central hypothesis is that both behavioral treatment and medication will produce reductions in CB, but behavioral treatments, including behavior treatment + medication, will produce a greater reduction in CB when measured using direct observation within the contexts most associated with challenging behavior. We have four aims: Aim 1: Evaluate the concordance between indirect and direct measures of challenging behavior. We hypothesize that behavior change (% change) will be discordant between indirect and direct measurement data, regardless of treatment type, with direct measurement resulting in significantly greater change at each measurement timepoint. Aim 2: Compare the efficacy of psychotropic medication, behavior therapy, and combination therapy (medication+behavior therapy) on CB using indirect measures (ABC-I, BPI, CGI, MOSES) and direct observation (antecedent analysis). We hypothesize that all treatment modalities will reduce CB on both outcome measures at each standard timepoint, but behavioral therapy and combination therapy will produce a significantly greater reduction on direct observation of CB in the contexts most associated with challenging behavior. Aim 3: Compare the social validity of psychotropic medication, behavior therapy, and combination therapy using the Treatment Acceptability Rating Form-Revised (Reimers et al., 1992). Aim 4: Compare the social validity of measurement methods (indirect vs. direct).

Full description

Our overall objective of this study is to evaluate and compare the efficacy of two common treatments for CB in children and adolescents with autism using multiple outcome measures. Our central hypothesis is that behavioral treatment, medication, and combination treatment (medication+behavior therapy) will produce reductions in CB, but behavioral treatment and combination treatment will produce a greater reduction in CB when measured using direct observation within the contexts most associated with challenging behavior.

To address this objective, we plan to recruit up to 20 individuals under 13 years of age who are diagnosed with autism and exhibit challenging behavior to participate in this study. Subjects will be recruited from clinics where patients receive consultation on medication for challenging behavior. Subjects will be given the options of initiating/continuing medication, initiating behavior therapy, both medication and behavior therapy, or neither medication nor behavior therapy. The behavioral treatment provided as part of the research study will be functional communication training or FCT. FCT is a scientifically validated behavior therapy that involves teaching the child to use communication (in various forms) instead of challenging behavior to get what they want. It will be delivered at a timing (1 time per week) and dosage (60 minutes per visit) that is common for individuals with challenging behavior. Subjects will participate in a baseline behavioral assessment (baseline), as well as monthly behavioral assessments. Each behavioral assessment will include indirect (rating scales) and direct (antecedent analysis) measures of challenging behavior, as well as measures of social validity (treatment acceptability rating scales). Subjects will be followed for 6 months and comparisons of behavior change across measures will be used to determine treatment effectiveness and concordance between measures.

Enrollment: 20 estimated patients

Sex: All

Ages: 2 to 12 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

The adult subjects will be the caregiver/guardian of the minor subjects. The only inclusion criterion for the adult subjects is that they must be the caregiver/legal guardian of the minor subject and their home is primarily English speaking.

The minor subjects will be individuals who were referred to Child and Adolescent Psychiatry at University of Iowa Healthcare for management of challenging behavior (e.g., physical aggression, self-injury, destruction, elopement, generalized refusal) and meet the following criteria:

1. Between the ages of 2 years, 0 months and 12 years, 0 months
2. Diagnosed with autism spectrum disorder
3. Diagnosed with intellectual disability at the moderate, severe, or profound range (called "profound autism").
4. Display challenging behavior, including one or more of the following: physical aggression, self-injury, destruction, elopement, generalized refusal
5. Live in a home where English is the primary language spoken.
6. The caregiver is considering medication, behavioral therapy, or both to treat the child's challenging behavior.

Subjects would be excluded if:

1. Not diagnosed with autism spectrum disorder
2. Do not have a moderate or severe/profound intellectual disability
3. Do not display challenging behavior
4. Live in a home where English is not the primary language spoken
5. Caregivers are not considering medication, behavior therapy, or both to treat challenging behavior

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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20 participants in 4 patient groups

Psychotropic Medication

Experimental group

Description:

Classes of psychotropic medications used as treatment for challenging behavior include antipsychotics (e.g., aripiprazole, quetiapine), stimulants (e.g., methylphenidate), alpha 2 agonists (e.g., clonidine), and mood-stabilizing anticonvulsants (e.g., valproate). However, other classes of psychotropics may be used to indirectly address challenging behavior (e.g., anxiolytics, antidepressants). Two medications (aripiprazole and risperidone) are FDA approved to treatment irritability for individuals with autism and challenging behavior. Psychotropic medications range in timing and dosage and their effects may be immediate (e.g., stimulants) or take as long as 6 weeks for full therapeutic effects (e.g., risperidone).

Treatment:

Drug: Psychotropic medication (at discretion of psychiatrist)

Behavior Therapy

Experimental group

Description:

In this study, FCT treatment will be provided weekly for approximately 60 min in duration. To facilitate communication during FCT, a word/picture card or microswitch (with recorded voice output) will be paired with any pre-existing child requests. Appropriate requests will be reinforced by the caregiver or therapist, and the occurrence of challenging behavior will be ignored (placed on extinction) or result in the caregiver/therapist providing guided compliance. An individualized FCT treatment plan will be developed based on the results of the functional analysis. For example, if the functional analysis identifies challenging behavior that functioned to escape task demands, then the FCT treatment plan will focus on teaching the child to request breaks appropriately after complying with instructions to complete a task. A typical task demand will be initiated by placing a small amount of developmentally appropriate "work" in front of the child and asking him or her to complete the task

Treatment:

Behavioral: Behavioral therapy

Combined Treatment (Medication+Behavior Therapy)

Experimental group

Description:

Participants in this arm will receive both medication and behavior therapy using the same manipulations as the Psychotropic Medication and Behavior Therapy Arms.

Treatment:

Drug: Psychotropic medication (at discretion of psychiatrist)

Behavioral: Behavioral therapy

No Treatment

No Intervention group

Description:

Participants who chose none of the treatment arms may participate in the assessments.