Propranolol for Autism

Overseen By Eric London, M.D.

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Rutgers, The State University of New Jersey

Must be taking: Antipsychotics, Psychotropics

Must not be taking: Clonidine, Guanfacine, Digoxin

Disqualifiers: Asthma, Cardiac diseases, Seizure, Diabetes, others

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 4

8 Participants Needed

Trial Summary

What is the purpose of this trial?

Severe challenging behaviors such as aggression and self-injury can cause significant morbidity and decrease the quality of life for individuals with Autism Spectrum Disorders (ASD). There are only two medications (Risperdal and Abilify) rigorously studied and FDA-approved for the treatment of irritability in individuals with ASD. These medications are not always successful and have many short and long-term side effects. Well-designed studies demonstrating efficacy and safety of alternative medication treatment choices are needed. There is preliminary evidence that high-dose propranolol can be effective in individuals with ASD who display severe aggression and have not responded to antipsychotics or mood stabilizers. Concerns regarding the safety of high dose propranolol have limited its clinical application. Well-designed clinical trials demonstrating the efficacy and safety of high dose propranolol will have significant effects on clinical practice and improve the physical and behavioral quality of life for an underserved subset of individuals with ASD. This study will pilot the safety and efficacy of high dose propranolol. The investigators will randomly assign participants to either propranolol or to placebo later crossing each participant over to the other group. As propranolol can cause changes in blood pressure and heart function, each participant will complete initial comprehensive testing to monitor cardiac safety throughout the study. The investigators will be utilizing telemedicine and computer based telemetry to minimize the burden of office visits on the individual and family.

Research Team

Barbie Zimmerman-Bier, M.D.

Principal Investigator

Rutgers, The State University of New Jersey

Eligibility Criteria

This trial is for New Jersey residents aged 12-30 with Autism Spectrum Disorder who exhibit severe aggression or self-injury and haven't improved enough on two psychotropic drugs, including an antipsychotic. Participants must have a confirmed autism diagnosis, be medically and cardiac cleared, and not have uncontrolled seizures or conditions that propranolol could worsen.

Inclusion Criteria

I engage in self-harm that causes tissue damage.

I engage in self-harm that causes tissue damage.

I have shown physical aggression towards others.

Exclusion Criteria

Pregnancy.

I am not taking medications like clonidine or digoxin that affect blood pressure.

I have had a seizure or changed my seizure medication in the last 6 months.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Baseline

Cognitive and adaptive information is collected before randomization

2 weeks

1 visit (in-person)

Treatment Phase A

Participants receive either Propranolol or Placebo with weekly monitoring

12 weeks

Weekly telemedicine visits

Washout

Participants undergo a washout period before crossover

2 weeks

Treatment Phase B

Participants crossover to receive the alternate treatment (Propranolol or Placebo)

12 weeks

Weekly telemedicine visits

Open-label Extension

Participants continue in an open-label phase with Propranolol

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

2 visits (in-person)

Treatment Details

Interventions

• Propranolol

Trial Overview The study tests high-dose propranolol's safety and effectiveness against challenging behaviors in autism compared to a placebo. Participants will switch between the drug and placebo during the trial. Cardiac safety is monitored through initial testing followed by remote check-ups to reduce office visits.

Participant Groups

Group I: Group A: Propranolol first Experimental Treatment 1 Intervention

Participants randomly assigned to this group will receive Propranolol first. After the washout period, they will receive Placebo. Propranolol will be given in liquid or pill form.

Group II: Group B: Placebo first Placebo Group 1 Intervention

Participants randomly assigned to this group will receive Placebo first. After the washout period, they will receive Propranolol. Placebo will look identical to the study drug Propranolol.

Propranolol is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Inderal for:

• High blood pressure
• Angina pectoris
• Heart rhythm disorders
• Migraine prophylaxis
• Essential tremor
• Performance anxiety

Approved in European Union as Propranolol for:

• Hypertension
• Angina pectoris
• Arrhythmias
• Migraine prophylaxis
• Essential tremor
• Anxiety

Approved in Canada as Propranolol for:

• Hypertension
• Angina pectoris
• Arrhythmias
• Migraine prophylaxis
• Essential tremor
• Anxiety

Who Is Running the Clinical Trial?

Rutgers, The State University of New Jersey

Lead Sponsor

Trials

471

Recruited

81,700+

New Jersey Governor's Council for Medical Research and Treatment of Autism

Collaborator

Trials

2

Recruited

40+

New York State Institute for Basic Research

Collaborator

Trials

7

Recruited

960+