Prednisone for Autism

(PREDICT Trial)

Not yet recruiting at 1 trial location

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines how prednisone, a medication often used to reduce inflammation, affects adults with autism spectrum disorder (ASD) who may have an immune-related form of the condition. Researchers aim to determine if prednisone can improve core ASD symptoms and assess its safety and side effects. Participants will receive either prednisone or a placebo for 16 weeks, and their experiences will inform the need for larger studies. This trial may suit male adults diagnosed with moderate or severe autism who show signs of autoimmune issues, such as a family history of autoimmune diseases or related health conditions. As an Early Phase 1 trial, this research focuses on understanding how prednisone works in individuals with ASD and autoimmune issues.

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.

Is there any evidence suggesting that prednisone is likely to be safe for humans?

Research has shown that prednisone, a steroid with anti-inflammatory effects, has been studied for its impact on various conditions, including autism. Some studies suggest that steroids like prednisone might help manage certain symptoms in autistic individuals. For instance, one study found that a similar steroid, prednisolone, improved core symptoms like repetitive behaviors.

While prednisone is usually well-tolerated, some people may experience side effects such as mood changes, increased appetite, and difficulty sleeping. Researchers are still studying the specific effects on autistic adults, which this trial aims to discover.

This trial is in an early phase, meaning researchers are collecting initial safety data. This phase is generally safe and focuses on understanding how the drug works in the body and identifying any side effects. Although prednisone is approved for other uses, its specific effects on adults with autism remain under investigation. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism?

Prednisone is unique in the treatment of autism because it is a corticosteroid that works by reducing inflammation, which is a different approach compared to most current treatments. Typical treatments for autism often focus on behavioral therapies or medications like antipsychotics and antidepressants that manage symptoms such as irritability or anxiety. Researchers are excited about prednisone because it targets potential underlying inflammatory processes that may contribute to autism symptoms, offering a fresh angle that could lead to more comprehensive management of the condition. This approach could potentially complement existing therapies and offer new hope for more effective symptom control.

What evidence suggests that prednisone might be an effective treatment for autism?

Research suggests that prednisone, which participants in this trial may receive, might improve language skills, expression, and social behavior in people with autism. Although no published studies have specifically tested prednisone for autism, research on similar drugs like prednisolone has shown benefits for core autism symptoms, such as repetitive behaviors. Prednisone may help by reducing inflammation, which might play a role in some types of autism. Its ability to lower inflammation could improve certain autism-related behaviors. However, more research is needed to fully understand its effects and safety in adults with autism. ^{1 2 3 5 6}

Who Is on the Research Team?

Christopher J McDougle, M.D.

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9432069/

Effect of prednisolone on language function in children with ... The benefit of prednisolone for language scores was more evident in participants who were younger than five years, with a history of developmental regression.

2. mdpi.com mdpi.com/2218-0532/92/4/61

Corticosteroids and the Pharmacological Management of ... It has also been observed that the use of prednisone over time results in significant improvements in language skills, expression, and social behavior in ...

3. massgeneral.org massgeneral.org/children/autism/lurie-center/prednisone

Lurie Center for Autism: Prednisone Currently there are no published or on-going randomized placebo-controlled trials of prednisone use in autism. This may be due to the many risks involved ...

4. link.springer.com link.springer.com/article/10.1007/s40263-023-00993-x

Immunoregulatory and/or Anti-inflammatory Agents for the ... A beneficial effect of prednisolone, pregnenolone, celecoxib, and/or omega-3 fatty acids on several core symptoms, such as stereotyped behavior, was found.

5. steritas.com steritas.com/insights/steroids-and-autism-what-large-cohorts-and-randomized-trials-do-and-dont-tell-us

Steroids and Autism: What Large Cohorts and Randomized ... Steroids may increase autism risk at term delivery but protect preterm infants. Timing matters more than exposure. New evidence reviewed.

6. ctv.veeva.com ctv.veeva.com/study/prednisone-in-adults-with-an-immune-mediated-subtype-of-autism-spectrum-disorder

Prednisone in Adults With an Immune-Mediated Subtype of ... The goal of this clinical trial is to learn how prednisone affects adults with autism spectrum disorder (ASD). It will also learn about the ...