Autistic DisorderPhase 1NCT00773812
Placebo-Controlled Pilot Trial of Mecamylamine for Treatment of Autism Spectrum Disorders
A Phase 1 study testing mecamylamine in people with Autistic Disorder and Pervasive Developmental Disorder, run by Ohio State University at 1 site in United States.
Trial summary
The purpose of this study is to examine the safety and efficacy of mecamylamine for the core symptoms of autism. Read more about this study
- Who is it for?
- Ages 4 to 12 years
- Conditions the study is for
- Autistic Disorder, Pervasive Developmental Disorder
- What is being tested?
- mecamylamine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2007to Nov 2012
- Phase
- Phase 1
- 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 4 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Autistic Disorder or Pervasive Developmental Disorder-Not Otherwise Specified
- Age 4-12
- General good health
- IQ of >=36 or mental age of >=18 months
- Parent/caregiver willingness to accompany child to clinic and monitor for side effects
Who cannot join
- Unstable Seizure Disorder
- Psychoactive medication in the process of adjustment
- Antipsychotic medication in previous 3 months before baseline
- Systemic corticoids (inhalers allowed)
- Planned beginning of major behavioral intervention in 3 months of study or the 2 months prior to the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Neuronal Nicotinic Receptor Modulation in the Treatment of Autism: A Pilot Trial of Mecamylamine
The purpose of this study is to examine the safety and efficacy of mecamylamine for the core symptoms of autism.
Read the detailed description
This is a placebo-controlled pilot clinical trial of a nicotinic agent based on evidence that a disturbance occurs in nicotinic cholinergic transmission in the brain of autistic children. This pharmacotherapeutic trial will be conducted as a direct result of findings of a nicotinic receptor abnormality in autopsied brain tissue from individuals who suffered from autism. A pharmacological agent, mecamylamine, with specificity for the nicotinic receptors implicated in autism, will be tested in children with autism spectrum disorder. Twenty children who meet inclusion/exclusion criteria will be randomized in a 2:3 ratio to 14 weeks of either matched placebo (n=8) or mecamylamine (n=12). Children randomized to placebo who do not respond will be given the opportunity to enroll in a ten week open label extension.
The trial has been completed and published in J. of Child \& Adolescent Psychopharmacology.
- Being tested
- mecamylamine, placebo
- Keywords
- Autistic Disorder, Autism, Mecamylamine, Pervasive Developmental Disorder-Not Otherwise Specified
- Medical subject headings
- Autistic Disorder, Child Development Disorders, Pervasive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mecamylamineDrug
Also called Inversine
The dosage is titrated at 6 weeks of .5mg, 2 weeks of 2.5 mg, and 6 weeks of 5.0 mg. One pill is taken once daily.
placeboDrug
One pill is taken once daily.
| Study group | Role | What they receive |
|---|---|---|
| Active Mecamylamine | Experimental | There will be 12 children in this arm. These children will receive the active medication (mecamylamine). Receives: Drug: mecamylamine |
| Placebo | Placebo comparator | There will be 8 children in this arm. These children will receive placebo instead of the active medication. Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary measure will be a global composite derived by calculating the z scores on each measure other than CGI from published norms (or from the baseline descriptives, then averaging the z scores of the various outcome measures for each individual.
Measured: Baseline, weeks 2, 4, 6, 8, 10,12, and 14.
Secondary outcomes (8)
OSU Autism Rating Scale-DSM-IV
Measured: Baseline, week 6, 8, and 14.
Repetitive Behavior Questionnaire
Measured: Baseline, week 2, 4, 6, 8, 10, 12, and 14
Aberrant Behavior Checklist
Measured: Baseline, week 2, 4, 6, 8, 10, 12, and 14.
Ohio Autism Clinical Impressions Scale
Measured: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.
Social Responsiveness Scale
Measured: Baseline, weeks 2, 4, 6, 8, 10, 12, and 14.
Expressive Vocabulary test-Second Edition
Measured: Baseline and week 14
Adverse Experience checklist and AE log
Measured: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.
Cognitive Battery
Measured: baseline, weeks 6, 8, and 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2008 (estimated)
- Study startJul 2007
- Primary completionNov 2012
- Study completionNov 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Autistic DisorderStudies of the same condition, open and under way.
- More trials run by Ohio State UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ohio State University
- Sponsor type
- University, hospital or other
- Collaborators
- Autism Speaks
- Responsible party
- Principal investigator, L. Eugene Arnold, Ohio State University
- Investigators
- L E Arnold, M.D., Principal investigator, Ohio State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00773812
- Sponsor's protocol ID
- 2007H0096
- Status last verified
- Jan 2017
- First submitted
- 15 Oct 2008
- Last updated
- 13 Jan 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Arnold LE, Aman MG, Hollway J, Hurt E, Bates B, Li X, Farmer C, Anand R, Thompson S, Ramadan Y, Williams C. Placebo-controlled pilot trial of mecamylamine for treatment of autism spectrum disorders. J Child Adolesc Psychopharmacol. 2012 Jun;22(3):198-205. doi: 10.1089/cap.2011.0056. Epub 2012 Apr 26.
PubMed 22537359Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2.
PubMed 37811711
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Neuronal Nicotinic Receptor Modulation in the Treatment of Autism: A Pilot Trial of Mecamylamine
- ClinicalTrials.gov ID
- NCT00773812
- Acronym / sponsor ID
- 2007H0096
- Keywords
- Autistic Disorder, Autism, Mecamylamine, Pervasive Developmental Disorder-Not Otherwise Specified
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Ohio State UniversityUniversity, hospital or other
- Collaborators
- Autism Speaks
- Study start
- Jul 2007
- Primary completion
- Nov 2012 (actual)
- Study completion
- Nov 2012 (actual)
- First posted
- 16 Oct 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to examine the safety and efficacy of mecamylamine for the core symptoms of autism.
Detailed description
This is a placebo-controlled pilot clinical trial of a nicotinic agent based on evidence that a disturbance occurs in nicotinic cholinergic transmission in the brain of autistic children. This pharmacotherapeutic trial will be conducted as a direct result of findings of a nicotinic receptor abnormality in autopsied brain tissue from individuals who suffered from autism. A pharmacological agent, mecamylamine, with specificity for the nicotinic receptors implicated in autism, will be tested in children with autism spectrum disorder. Twenty children who meet inclusion/exclusion criteria will be randomized in a 2:3 ratio to 14 weeks of either matched placebo (n=8) or mecamylamine (n=12). Children randomized to placebo who do not respond will be given the opportunity to enroll in a ten week open label extension.
The trial has been completed and published in J. of Child \& Adolescent Psychopharmacology.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active Mecamylamine | Experimental | Drug: mecamylamineThere will be 12 children in this arm. These children will receive the active medication (mecamylamine). |
| Placebo | Placebo comparator | Drug: placeboThere will be 8 children in this arm. These children will receive placebo instead of the active medication. |
Who may qualify
- Ages
- 4 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Autistic Disorder or Pervasive Developmental Disorder-Not Otherwise Specified
- Age 4-12
- General good health
- IQ of >=36 or mental age of >=18 months
- Parent/caregiver willingness to accompany child to clinic and monitor for side effects
Key exclusion criteria (5 listed)
- Unstable Seizure Disorder
- Psychoactive medication in the process of adjustment
- Antipsychotic medication in previous 3 months before baseline
- Systemic corticoids (inhalers allowed)
- Planned beginning of major behavioral intervention in 3 months of study or the 2 months prior to the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mecamylamineAlso: InversineThe dosage is titrated at 6 weeks of .5mg, 2 weeks of 2.5 mg, and 6 weeks of 5.0 mg. One pill is taken once daily. | Drug | Active Mecamylamine |
| placeboOne pill is taken once daily. | Drug | Placebo |
Primary outcome
The primary measure will be a global composite derived by calculating the z scores on each measure other than CGI from published norms (or from the baseline descriptives, then averaging the z scores of the various outcome measures for each individual.
Time frame: Baseline, weeks 2, 4, 6, 8, 10,12, and 14.
8 secondary outcomes
OSU Autism Rating Scale-DSM-IV
Time frame: Baseline, week 6, 8, and 14.
Repetitive Behavior Questionnaire
Time frame: Baseline, week 2, 4, 6, 8, 10, 12, and 14
Aberrant Behavior Checklist
Time frame: Baseline, week 2, 4, 6, 8, 10, 12, and 14.
Ohio Autism Clinical Impressions Scale
Time frame: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.
Social Responsiveness Scale
Time frame: Baseline, weeks 2, 4, 6, 8, 10, 12, and 14.
Expressive Vocabulary test-Second Edition
Time frame: Baseline and week 14
Adverse Experience checklist and AE log
Time frame: Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.
Cognitive Battery
Time frame: baseline, weeks 6, 8, and 14
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2017.
- Results publication: PubMed 22537359Arnold LE, Aman MG, Hollway J, Hurt E, Bates B, Li X, Farmer C, Anand R, Thompson S, Ramadan Y, Williams C. Placebo-controlled pilot trial of mecamylamine for treatment of autism spectrum disorders. J Child Adolesc Psychopharmacol. 2012 Jun;22(3):198-205. doi: 10.1089/cap.2011.0056. Epub 2012 Apr 26.
- Derived from this study: PubMed 37811711Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
L E Arnold, M.D. principal investigator
Ohio State University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing mecamylamine in people with Autistic Disorder and Pervasive Developmental Disorder, run by Ohio State University 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 5 inclusion and 5 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: finished, as of the sponsor's last update on 13 Jan 2017. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The primary measure will be a global composite derived by calculating the z scores on each measure other than CGI from published norms (or from the baseline descriptives, then averaging the z scores of the various outcome measures for each individual. (time frame: Baseline, weeks 2, 4, 6, 8, 10,12, and 14.). 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 NCT00773812, which also lists 2 linked publications.
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 13 Jan 2017.