Prednisone for Autism

Overseen By Colleen Buckless

Age: 18 - 65

Sex: Male

Trial Phase: Phase < 1

Sponsor: Christopher John McDougle, M.D.

Must not be taking: Immunosuppressive agents

Disqualifiers: Genetic disorders, Bipolar, Psychotic, others

Compensation: Varies

Number of Visits: 6

Duration: 16 weeks

32 Participants Needed

Trial Summary

Do I need to stop my current medications to join the trial?

The trial requires that any medications or treatments for ASD-related symptoms have been stable for at least 4 weeks before the screening and remain stable throughout the trial. So, you won't need to stop your current medications, but you should not change them during the study.

What data supports the effectiveness of the drug Prednisone for treating autism?

There is no direct evidence from the provided research articles supporting the effectiveness of Prednisone for treating autism. However, Prednisone is known to improve recovery speed in conditions like multiple sclerosis relapses and has been used in asthma treatment, suggesting it may have some impact on inflammation-related conditions. ^{1 2 3 4 5}

How does the drug prednisone differ from other treatments for autism?

Prednisone is a steroid that works by reducing inflammation and suppressing the immune system, which is different from most autism treatments that focus on behavioral therapy or medications targeting neurotransmitters. Its use in autism is novel as it is traditionally used for conditions like asthma and epilepsy, where it helps manage symptoms by reducing inflammation. ^{2 6 7 8 9}

What is the purpose of 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.

Research Team

Christopher J McDougle, M.D.

Principal Investigator

Massachusetts General Hospital

Eligibility Criteria

This trial is for adult males with autism spectrum disorder (ASD) who may have an immune-related subtype. Participants will be required to visit the clinic multiple times, take daily medication, provide blood and urine samples, participate in remote calls, and keep a diary of their medication intake and any symptoms.

Inclusion Criteria

Clinical Global Impression-Severity (CGI-S) rating ≥ 4 at screening

Diagnosed with autistic disorder or autism spectrum disorder (ASD) level 2 or 3 by a qualified psychiatrist or psychologist

Non-verbal IQ in the range of moderate intellectual disability or higher (≥ 35)

Exclusion Criteria

Non-verbal IQ in the range of severe or profound intellectual disability (< 35)

Concomitant medications not stable for at least 4 weeks prior to screening

Diagnosed with ASD level 1 or another pervasive developmental disorder

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

2 visits (in-person)

Treatment

Participants receive prednisone or placebo for 16 weeks, including dose escalation, maintenance, and reduction phases

16 weeks

3 visits (in-person), 8 remote calls

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person)

Treatment Details

Interventions

• Prednisone

Trial Overview The study tests prednisone's effects on ASD core features compared to a placebo over 16 weeks. It aims to determine if prednisone is safe and effective enough to consider larger trials on anti-inflammatory drugs for autistic adults.

Participant Groups

Group I: Prednisone Experimental Treatment 1 Intervention

Eligible participants will receive prednisone for up to 16 weeks. The treatment period consists of 3 structured phases: Dose Escalation (Weeks 1-5), Optimal Dose Maintenance (Weeks 6-10), and Gradual Dose Reduction (Weeks 11-16).

Group II: Placebo Placebo Group 1 Intervention

Eligible participants will receive a matching placebo for up to 16 weeks. The treatment period consists of 3 structured phases: Dose Escalation (Weeks 1-5), Optimal Dose Maintenance (Weeks 6-10), and Gradual Dose Reduction (Weeks 11-16).

Who Is Running the Clinical Trial?

Christopher John McDougle, M.D.

Lead Sponsor

Findings from Research

In a study of 1161 children aged 4 to 17 hospitalized for asthma exacerbations, dexamethasone was compared to prednisone, showing a slightly higher return utilization rate of 3.9% versus 2.2%, respectively.

However, the analysis indicated that the choice of steroid (dexamethasone vs. prednisone) did not significantly impact the likelihood of unplanned readmissions or emergency department visits within 30 days after discharge.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dexamethasone Versus Prednisone in Children Hospitalized With Asthma Exacerbation. Hoefgen, ER., Huang, B., Schuler, CL., et al.[2022]

References

1. Australia pubmed.ncbi.nlm.nih.gov

Lack of efficacy of single-dose prednisolone in moderately severe asthma. [2013]

2. United States pubmed.ncbi.nlm.nih.gov

Prednisone dose limitation of growth hormone treatment of steroid-induced growth failure. [2019]

3. England pubmed.ncbi.nlm.nih.gov

High dose oral steroids commonly used to treat relapses in Canadian MS clinics. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Relationship between alternate-day corticosteroid therapy and behavioral abnormalities. [2016]

5. England pubmed.ncbi.nlm.nih.gov

Prednisolone and salbutamol in the hospital treatment of acute asthma. [2019]

6. England pubmed.ncbi.nlm.nih.gov

A comparison of oral prednisolone given as single or multiple daily doses for active proctocolitis. [2019]

7. United States pubmed.ncbi.nlm.nih.gov

High-dose corticotropin (ACTH) versus prednisone for infantile spasms: a prospective, randomized, blinded study. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

Prednisone therapy in pediatric epilepsy. [2019]

9. United States pubmed.ncbi.nlm.nih.gov

Dexamethasone Versus Prednisone in Children Hospitalized With Asthma Exacerbation. [2022]