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Autism Spectrum DisorderPhase 3NCT04725383

Amitriptyline for Repetitive Behaviors in Autism Spectrum Disorders

A Phase 3 study testing amitriptyline in people with Autism Spectrum Disorder and Repetitive Compulsive Behavior, run by University of Missouri, Kansas City at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The investigators will recruit 30 children and adolescents (15 per group x 2 groups) aged 6 to 17 years with ASD and significant repetitive behaviors that cause problems to them and to others around them. Read more about this study

Who is it for?
Ages 6 to 17 yearsHealthy volunteers can take part
What is being tested?
amitriptyline
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
1 participants
Where
1 siteUnited States
Study dates
1 Nov 2023to 23 May 2025
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 to 17 years
  • All sexes
  • Healthy volunteers accepted
  • Child

Who may be able to join

  • males and females
  • ages 6-17 years;
  • diagnosis of ASD validated by the Autism Diagnostic Interview- Revised (Lord et al. 1994); CGI-S rating of at least Moderate problem CYBOCS-PDD score of at least 8 or more for compulsive behaviors (sum of items 1A, 2, 3 and 5)
  • Intellectual Disability if present to be no greater than moderate by history (ie IQ>35).

Who cannot join

  • unable to complete an EKG recording, even with low dose risperidone and alprazolam if needed an hour before, and repeated at the time (if needed), of the procedure,
  • QTc on EKG of 440 or more
  • absence of a reliable caregiver
  • amitriptyline allergy
  • previous neuroleptic malignant syndrome
  • seizures in the past 3 months
Show all 11Show fewer
  • bipolar mood disorder
  • current or past psychosis
  • unstable medical illness
  • previous adequate trial of amitriptyline
  • using other psychotropic medications apart from melatonin for sleep or lorazepam 1mg as needed up to once a day for severe outbursts.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • University Health Behavioral Health Canvas Building

    Kansas City, Missouri, 64108

Other trials in United States

About this study

Official title: Amitriptyline for Repetitive Behaviors in Children and Adolescents With Autism Spectrum Disorders

The investigators will recruit 30 children and adolescents (15 per group x 2 groups) aged 6 to 17 years with ASD and significant repetitive behaviors that cause problems to them and to others around them. Subjects will be randomized to either amitriptyline (AMI), dosed flexibly according to response and tolerability with a maximum dose of 100mg per day or 1.5mg/kg/day, in divided doses to minimize side effects, or placebo in look-alike capsules, for 10 weeks. Rating scales will be used to measure outcomes.

Read the detailed description

At the screening visit, investigators will perform a full history-taking, mental status examination, physical and neurological examinations, the ADI-R with a caregiver or parent to confirm autism, an EKG and complete rating scales, as well as a pregnancy test (beta-HCG) in sexually active females of childbearing age. Sexually active females of childbearing age must be on a form of birth control during the study, such as the oral contraceptive pill, intrauterine device or Depo Provera shot. A parent or guardian will sign informed consent, as appropriate, and written assent will be obtained from subjects. Blood tests for CBC and diff, CMP, amitriptyline level will be obtained at at baseline (except for amitriptyline) week 6 and 10.

Subjects will be randomized to amitriptyline (AMI) or placebo. Parents and guardians will be instructed to lock up all medications, warned regarding overdose toxicity and this will be documented in writing. Treatment will be low dose AMI or placebo for 12 weeks. Study visits will occur at weeks 0,1, 2, 3, 4, 5, 6, 8, and 10.

Investigators will complete a Clinical Global Impressions scale (CGI), and have the accompanying adult or parent complete rating scales, at each visit, notably the Child Yale Brown Obsessive Compulsive scale modified for Pervasive Developmental Disorder (CYBOCS-PDD), Aberrant Behavior Checklist-Irritability subscale (ABC-I), Repetitive Behavior Scale-Revised (RBS-R), ADHD-RS, adverse events form, ADI-R item 11 for phrase speech, and a concomitant treatment review form, which will also detail for example any over-the-counter medications, supplements or antibiotics taken. Also a gastrointestinal symptom form based on that used by Valicenti-McDermott et al. 2008.

At all interim visits, the PI will perform a follow-up history taking, mental status examination, vital signs, rating scales as above and study drug dispensing. Any unused capsules must be returned at each visit, to monitor compliance as well as to prevent accidental overdose. Parents and caregivers will agree to lock medication up, and will be reminded of overdose toxicity at each study visit, with written documentation.

Flexible dosing, arranged by calling the pharmacy to randomize the subject and then dispense and mail study capsules, according to the psychiatrist's instructions at each visit, to a maximum dose of 100mg/day or 1.5mg/kg/day maximum by subject weight.

Individuals and their parents or guardians will be questioned about suicidal ideation at each visit, and instructed to call study staff if that should arise. At that time the PI will arrange an urgent visit to closely assess risks of remaining in the study. In our experience suicidal ideation has not occurred. Subjects may drop out at any time however, and in the unlikely case, if warranted, be admitted to hospital for close observation.

The study will be double- blind and placebo-controlled. All investigators except a psychiatry co-investigator will be blinded, as will caregivers, families and subjects.

Being tested
amitriptyline
Keywords
amitriptyline, autism, repetitive behaviors
Medical subject headings
Autism Spectrum Disorder, Autistic Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • amitriptylineDrug

    Also called Elavil

    Amitriptyline is a tricyclic antidepressant being studied off-label here for repetitive behaviors in autism spectrum disorders.

Study groups in NCT04725383
Study groupRoleWhat they receive
amitriptylineActive comparatorSubjects will receive active amitriptyline compounded into look-alike capsules to resemble placebo capsules. Dosing will be as tolerated, up to tid and maximum of 100mg/day or 1.5mg/kg/day, for 10 weeks.
Receives: Drug: amitriptyline
placeboPlacebo comparatorSubjects in this arm will receive placebo compounded into capsules that resemble the compounded amitriptyline capsules, up to 4 capsules a day (1 qam, 1 q4pm and 2 capsules qhs), for 10 weeks.
Receives: Drug: amitriptyline

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Clinical Global Impressions scale-Improvement of Much Improved (2) or Very Much Improved (1)

    1 Very Much Improved to 7 Very Much Worse

    Measured: up to 10 weeks

  2. Child Yale Brown Obsessive Compulsive Scale- Pervasive Developmental Disorders (CYBOCS-PDD)

    0-20; high scores are more severe

    Measured: up to 10 weeks

Secondary outcomes (1)
  1. Repetitive Behavior Scale-revised

    0-129; high scores are more severe

    Measured: up to week 10

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Jan 2021
  2. Study start1 Nov 2023
  3. Primary completion23 May 2025
  4. Study completion23 May 2025

Why it stopped: The PI is retiring.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Randomized double-blind placebo-controlled clinical trial.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
1 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04725383
Sponsor's protocol ID
2015984
Other IDs
17-055
Status last verified
May 2025
First submitted
7 Jan 2021
Last updated
30 May 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Bhatti I, Thome A, Smith PO, Cook-Wiens G, Yeh HW, Gaffney GR, Hellings JA. A retrospective study of amitriptyline in youth with autism spectrum disorders. J Autism Dev Disord. 2013 May;43(5):1017-27. doi: 10.1007/s10803-012-1647-0.

    PubMed 23135317

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing amitriptyline in people with Autism Spectrum Disorder and Repetitive Compulsive Behavior, run by University of Missouri, Kansas City at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 30 May 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: The PI is retiring.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Clinical Global Impressions scale-Improvement of Much Improved (2) or Very Much Improved (1) (time frame: up to 10 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04725383, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 May 2025.

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