Propranolol for Autism

Recruiting at 1 trial location

Overseen By Suvekcha Bhattachan, MA

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Jeremy Veenstra-vanderweele

Must not be taking: Antidepressants, Calcium channel blockers, Alcohol, others

Disqualifiers: Asthma, Cardiovascular history, Diabetes, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: 12 weeks

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if propranolol, a type of beta-blocker, can reduce challenging behaviors such as aggression and self-injury in individuals with Autism Spectrum Disorder. Participants will be randomly assigned to one of two groups: one receiving propranolol and the other receiving a placebo (a look-alike pill with no active drug) for 12 weeks. Afterward, all participants will have the opportunity to try propranolol for an additional 12 weeks. This trial may suit individuals diagnosed with autism who experience severe behaviors impacting their daily lives, such as frequent aggression or self-injury. As a Phase 2 trial, the research focuses on assessing propranolol's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

Yes, you may need to stop taking certain medications. The trial excludes participants who are currently using specific medications like calcium channel blockers, ACE inhibitors, and antidepressants, among others. It's important to discuss your current medications with the study team to see if they are allowed.

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

Research has shown that propranolol can be safely administered to individuals with Autism Spectrum Disorder (ASD) to help manage difficult behaviors. Studies have found that high doses of propranolol are usually well-tolerated, with few heart-related side effects, provided there is careful monitoring. The FDA has already approved propranolol for other uses, indicating a certain level of safety. However, each person's experience may vary, so maintaining communication with the healthcare team is important to manage any side effects effectively. ^{1 2 3 4 5}

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

Unlike the standard treatments for autism, which often focus on behavioral therapies or medications like antipsychotics and stimulants, propranolol offers a unique approach by targeting the body's stress response. Propranolol is a beta-blocker that works by damping down the effects of adrenaline, which might help reduce anxiety and improve social functioning in individuals with autism. Researchers are excited about this treatment because it represents a novel mechanism of action, which could potentially address certain symptoms of autism more effectively than current options.

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

Research shows that propranolol, which participants in this trial may receive, might help reduce difficult behaviors in people with Autism Spectrum Disorder (ASD). Earlier studies found that propranolol lowered aggressive symptoms by 50% and challenging behaviors by 37%. Commonly used for high blood pressure, this medication is believed to enhance brain function and provide cognitive and social benefits for those with ASD. Early evidence suggests that high doses can be safe with close monitoring. Overall, propranolol appears promising for managing severe aggression and behaviors associated with autism. ^{6 7 8 9 10}

Who Is on the Research Team?

Eric London, MD

Principal Investigator

New York State Institute for Basic Research (IBR)

Jeremy Veenstra-VanderWeele, MD

Principal Investigator

New York State Psychiatric Institute

Are You a Good Fit for This Trial?

This trial is for adolescents and adults aged 12-40 with Autism Spectrum Disorder who exhibit severe behaviors like aggression or self-injury. They must have a study partner to report on their behavior, be able to swallow capsules, and meet certain health criteria including heart rate and blood pressure. Women of childbearing age must use effective contraception.

Inclusion Criteria

I have been diagnosed with autism based on the Autism Diagnostic Observation Schedule within the last 5 years.

I can swallow pills without any difficulty.

My condition is considered moderately severe or worse.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive propranolol or placebo, titrated over 8 weeks to the highest tolerated dose, followed by 4 weeks at a steady dose

12 weeks

Weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

2 visits (in-person)

Open-label extension

Participants may opt into continuation of propranolol treatment for an additional 12 weeks

12 weeks

Monthly visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Propranolol

Trial Overview The study tests if propranolol can reduce challenging behaviors in those with Autism over 12 weeks compared to a placebo. Afterward, all participants can choose to take propranolol for another 12 weeks.

How Is the Trial Designed?

Group I: Propranolol Experimental Treatment 1 Intervention

Propranolol, oral, starting at 30 mg/day (10 mg TID), titrated weekly based on tolerability to a maximum of 600 mg/day (200 mg TID) by Week 8. Participants will remain on their highest tolerated dose for an additional 4 weeks.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive oral placebo tablets matched in appearance and dosing schedule to Propranolol. The dose will be titrated weekly in parallel with the experimental arm, and participants will remain on the highest tolerated dose for an additional 4 weeks.

Who Is Running the Clinical Trial?

Jeremy Veenstra-vanderweele

Lead Sponsor

United States Department of Defense

Collaborator

Trials

940

Recruited

339,000+

New York State Institute for Basic Research

Collaborator

Trials

7

Recruited

960+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39174017/

High-Dose Propranolol for Severe and Chronic Aggression ... Results: The CGI-I indicated a 50% reduction in symptoms in the propranolol phase, while the ABC-I indicated a 37% reduction in comparison to ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04047355

Propranolol for Challenging Behaviors in Autism There is preliminary evidence that high-dose propranolol can be effective in individuals with ASD who display severe aggression and have not responded to ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/32134849/

The Safety and Effectiveness of High-Dose Propranolol as ... It appears that high-dose propranolol can be given safely with minimal adverse cardiovascular problems, provided that close clinical monitoring is maintained.

4. liebertpub.com liebertpub.com/doi/10.1089/cap.2024.0002?doi=10.1089/cap.2024.0002

Pilot Trial on the Effects of Propranolol on Gastrointestinal ... Pilot Trial on the Effects of Propranolol on Gastrointestinal Symptoms in Autism Spectrum Disorder and Heart Rate Variability as a Treatment Response Biomarker.

5. medicine.missouri.edu medicine.missouri.edu/news/low-cost-blood-pressure-drug-improves-brain-function-individuals-autism

Low-Cost Blood Pressure Drug Improves Brain Function in ... A version of the drug known as propranolol could provide cognitive and social benefits for those living with autism spectrum disorder.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11460740/

High-Dose Propranolol for Severe and Chronic Aggression in ... The safety and effectiveness of high-dose propranolol as a treatment for challenging behaviors in individuals with autism Spectrum disorders.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT07091279

NCT07091279 | Propranolol for Aggression, Self-Injury ... The study will evaluate the efficacy and safety of high dose propranolol in 60 male and female participants between the ages of 12 and 40 years who have a ...

8. journals.lww.com journals.lww.com/psychopharmacology/fulltext/2020/03000/the_safety_and_effectiveness_of_high_dose.4.aspx

The Safety and Effectiveness of High-Dose Propranolol as ... It appears that high-dose propranolol can be given safely with minimal adverse cardiovascular problems, provided that close clinical monitoring is maintained.

9. psychiatryonline.org psychiatryonline.org/doi/10.1176/appi.ajp-rj.2017.120205

Amelioration of Aggression and Echolalia With Propranolol ... We present a case of an adult male with ASD who was hospitalized for aggression and violence and improved with propranolol monotherapy.

10. rwjbh.org rwjbh.org/documents/csh/Interventional-Research-Protocol.pdf

Interventional Research Protocol a) Hypothesis #1- High dose propranolol will show evidence of being effective in treating the symptoms of aggression, self-injurious behaviors and disruptive.