Prednisone in Adults With an Immune-Mediated Subtype of Autism Spectrum Disorder

ConditionsAutism Spectrum Disorder Autism Spectrum Disorders Autistic Disorder Autism

Trial statusRecruiting

Trial phaseEarly Phase 1

Trial typeInterventional

Biological sexMale

Age18-50

SponsorChristopher John McDougle, M.D.

About this trial

The goal of this clinical trial is to learn how prednisone affects adults with autism spectrum disorder (ASD). It will also learn about the safety of prednisone. The main questions it aims to answer are:

* How does prednisone affect the core features and associated target symptoms of ASD in adults with an immune-mediated subtype of ASD? * Is prednisone safe for autistic adults without causing too many side effects? * Does this study warrant larger trials studying anti-inflammatory drugs in this subject population?

Researchers will compare the drug prednisone to a placebo (a look-alike substance that contains no drug) to see how prednisone affects autistic adult males.

Participants will:

* Visit the clinic 2 times for a screening and baseline visit. * Take prednisone or a placebo every day for 16 weeks. * Visit the clinic 2 times for checkups, tests, questionnaires, and dose changes, and 1 time for a follow-up visit 4 weeks after stopping the study drug. * Provide blood and urine samples for testing up to 4 times. * Complete 8 remote calls every 1-2 weeks for checkups and dose changes. * Keep a diary of the dose and times they take the study drug every day and any symptoms or side effects they experience.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

18 to 50 years of age (inclusive) and assigned male at birth.

Diagnostic Statistical Manual of Mental Disorders (DSM), Fourth Edition, Text Revision (DSM-IV-TR) diagnosed autistic disorder, and DSM, Fifth Edition, Text Revision (DSM-5-TR) diagnosed autism spectrum disorder (ASD), level 2 or 3. A qualified (board-eligible or board-certified) psychiatrist or psychologist, with experience in diagnostic determinations of ASD, will make a final diagnostic determination based on clinical history, clinical observations, medical records, mental status exams, and screening measures.

A Clinical Global Impression-Severity (CGI-S) rating ≥ 4 ("Moderate") at screening (and baseline).

A non-verbal IQ in the range of moderate intellectual disability or higher (≥ 35), as measured by the non-verbal Abbreviated IQ (ABIQ) score of the Stanford-Binet Intelligence Scales, Fifth Edition (SB-5), or mental age of at least 18 months, as measured by the Cognitive and Adaptive Behavior subscales of the Developmental Profile (DP-4) Parent/Caregiver Interview form.

Disqualifiers

DSM-5-TR diagnosed ASD, level 1, or presence of another DSM-IV-TR diagnosed pervasive developmental disorder, such as Asperger's disorder, childhood disintegrative disorder, Rett syndrome, or pervasive developmental disorder not otherwise specified (PDD-NOS). A qualified psychiatrist or psychologist will make a diagnostic determination after reviewing clinical history, clinical observations, medical records, mental status exams, and screening measures.

A CGI-S rating < 4 at screening (or baseline).

A non-verbal IQ in the range of severe or profound intellectual disability (< 35), as measured by the non-verbal ABIQ score of the SB-5, or mental age below 18 months, as measured by the Cognitive and Adaptive Behavior subscales of the DP-4 Parent/Caregiver Interview form. Individuals testing below 18 months may be enrolled after a case review by the PI and study psychologist, especially if testing scores were likely underestimated due to uncooperative behavior.

Previous documentation of a prolonged electroencephalogram (EEG) suggestive of Landau-Kleffner syndrome or continuous spike and wave during sleep (CSWS) syndrome.

Trial design

Design model

Parallel

Treatments tested in this trial

Prednisone
Drug
Starting dose: 5 mg daily. Maximum dose: 60 mg daily. Dosage forms: 5 mg, 10 mg, and 20 mg capsules.
Placebo
Drug
Capsules identical in size and appearance to those containing prednisone. Placebo capsules contain inactive ingredients.

Treatment groups

32 Participants

are divided into 2 treatment groups

Group A: PrednisoneExperimental treatment 1 intervention

Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

Mean difference in Clinical Global Impressions-Improvement (CGI-I) ratings between the prednisone and placebo groups at Visit 9

Mean difference in CGI-I ratings between the prednisone and placebo groups at Visit 9 (efficacy). The CGI-I is a single-item measure of global symptomatic improvement compared to baseline. Improvement is rated on a 7-point scale, ranging from 1 ("Very much improved") to 7 ("Very much worse"). A global domain score of 1 ("Very much improved") or 2 ("Much improved") on the CGI-I scale will constitute a positive treatment response.

Time frame

Baseline to Week 10

Secondary outcomes

Number of randomized study participants

Number of randomized study participants (feasibility).

Time frame

At Baseline

Percent of participants enrolled at Visit 9 (Week 10) and contributing a visit 9 CGI-I rating

Percent of participants enrolled at Visit 9 (Week 10) and contributing a visit 9 CGI-I rating, irrespective of treatment continuation (feasibility).

Time frame

Baseline to Week 10

Percent of participants randomized to prednisone experiencing serious adverse events (SAEs) probably or definitely related to medication

Percent of participants randomized to prednisone experiencing serious adverse events (SAEs) probably or definitely related to medication (safety).

Time frame

Baseline to Week 20

Percent of participants randomized to prednisone who complete a ten-week course of study drug

Percent of participants randomized to prednisone who complete a ten-week course of study drug (tolerability).

Time frame

Baseline to Week 10

Other outcomes

Change in Irritability Subscale Score of the Aberrant Behavior Checklist-Community (ABC-C)

Change in ABC-C Irritability subscale score from Baseline to Week 10. The ABC-C is a 58-item, informant-based scale measuring psychiatric symptoms and behavioral disturbance across five domains (subscales) within the past 28 days. Each item is rated from 0 ("Not a problem") to 3 ("Severe problem"). The Irritability subscale assesses irritability, aggression, self-injury, and negative emotional states. Scores range from 0 to 45, with higher scores indicating more severe irritability symptoms.

Time frame

Baseline to Week 10

Change in Social Withdrawal Subscale Score of the ABC-C

Change in ABC-C Social Withdrawal subscale score from Baseline to Week 10. The ABC-C is a 58-item, informant-based scale measuring psychiatric symptoms and behavioral disturbance across five domains (subscales) within the past 28 days. Each item is rated from 0 ("Not a problem") to 3 ("Severe problem"). The Social Withdrawal subscale assesses social avoidance, reduced emotional responsiveness, and lethargy. Scores range from 0 to 48, with higher scores indicating more severe lethargy and social withdrawal symptoms.

Time frame

Baseline to Week 10

Change in Stereotypic Behavior Subscale Score of the ABC-C

Change in ABC-C Stereotypic Behavior subscale score from Baseline to Week 10. The ABC-C is a 58-item, informant-based scale measuring psychiatric symptoms and behavioral disturbance across five domains (subscales) within the past 28 days. Each item is rated from 0 ("Not a problem") to 3 ("Severe problem"). The Stereotypic Behavior subscale assesses repetitive, stereotyped movements and mannerisms. Scores range from 0 to 21, with higher scores indicating more severe stereotypic behavior symptoms.

Time frame

Baseline to Week 10

Change in Hyperactivity/Noncompliance Subscale Score of the ABC-C

Change in ABC-C Hyperactivity/Noncompliance subscale score from Baseline to Week 10. The ABC-C is a 58-item, informant-based scale measuring psychiatric symptoms and behavioral disturbance across five domains (subscales) within the past 28 days. Each item is rated from 0 ("Not a problem") to 3 ("Severe problem"). The Hyperactivity/Noncompliance subscale assesses hyperactive and non-compliant behaviors. Scores range from 0 to 48, with higher scores indicating more severe symptoms of hyperactivity or noncompliance.

Time frame

Baseline to Week 10

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Christopher John McDougle, M.D.

Lead sponsor

Massachusetts General Hospital

Sponsor institution

Source ClinicalTrials.gov

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