Autism Spectrum DisordersEarly Phase 1NCT02081027
Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorders
A Early Phase 1 study testing Riluzole in people with Autism Spectrum Disorders, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
Determine if riluzole shows evidence of efficacy, safety, and tolerability targeting drug-refractory irritability (DRI) in persons with autism spectrum disorders (ASD). Read more about this study
- Who is it for?
- Ages 12 to 25 years
- Conditions the study is for
- Autism Spectrum Disorders
- What is being tested?
- Riluzole
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Sep 2013to May 2015
- Phase
- Early 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 12 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age ≥ 12 and < 26 years.
- Weight greater than 50 kg.
- Diagnosis of ASD
- Drug-refractory irritability, as defined as screening ABC Irritability subscale (ABC-I) score of ≥18 AND 1) failure of clinically adequate treatment trials of both aripiprazole and risperidone or 2) failure of at least three previous clinically adequate drug trials targeting irritability (one trial must include aripiprazole or risperidone), as confirmed by caregiver reports and medical record review when available.
- Stable dosing of all concomitant psychotropic medications (including those targeting irritability) for four weeks prior to screening visit and during the study.
- Presence of parent/guardian willing to serve as informant for behavioral outcome measures and shipping control sample for Extracellular Signal-Related Kinase biomarker (ERK) assay.
Who cannot join
- Current use of more than two concomitant psychotropic drugs targeting irritability.
- Current use of valproic acid.
- Current use of drugs with known interaction with riluzole
- Current use of drugs with concomitant glutamatergic or glutamatergic- modulating action medications.
- For female subjects of child bearing potential, a positive serum pregnancy test.
- History of pancreatitis.
Show all 10Show fewer
- Hemoglobin less than or equal to 8.0 gm/dL.
- Neutropenia with absolute neutrophil count less than or equal to 1.0 K/mcL.
- Problems with kidney functioning, as assessed by lab work
- Any major chronic medical or chronic respiratory illness considered to be uncontrolled by the Principal Investigator.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Cross-Over Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorders
Determine if riluzole shows evidence of efficacy, safety, and tolerability targeting drug-refractory irritability (DRI) in persons with autism spectrum disorders (ASD).
Read the detailed description
A randomized, double blind, placebo-controlled, 12-week cross-over study (5-week treatment periods with 2-week washout) of adjunctive riluzole in 12 persons with ASD and DRI between the ages of 12 and 25 years.
- Conditions
- Autism Spectrum Disorders
- Being tested
- Riluzole, placebo
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RiluzoleDrug
Also called Other name: Rilutek
placeboOther
| Study group | Role | What they receive |
|---|---|---|
| Riluzole | Experimental | The maximum dose of riluzole to be used in this study is 200 mg per day divided BID Receives: Drug: Riluzole |
| Placebo | Placebo comparator | Placebo will be administered in the same manner as the riluzole group, in order to maintain subject assignment throughout the study. Receives: Other: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Global Impression-Improvement
A clinician-rated global assessment of symptom change rated on a scale from 1 to 7
Measured: Change from baseline to end of week 12
Aberrant Behavior Checklist- Irritability
A parent questionnaire measuring five behavioral domains
Measured: Change between baseline and end of each phase
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2014 (estimated)
- Study start19 Sep 2013
- Primary completionApr 2015
- Study completionMay 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autism Spectrum DisordersStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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
- Early Phase 1
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Logan Wink, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02081027
- Sponsor's protocol ID
- CIN001- Riluzole in Autism
- Status last verified
- Apr 2017
- First submitted
- 5 Aug 2013
- Last updated
- 14 Jan 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Wink LK, Adams R, Horn PS, Tessier CR, Bantel AP, Hong M, Shaffer RC, Pedapati EV, Erickson CA. A Randomized Placebo-Controlled Cross-Over Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorder. J Autism Dev Disord. 2018 Sep;48(9):3051-3060. doi: 10.1007/s10803-018-3562-5.
PubMed 29644582
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Cross-Over Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorders
- ClinicalTrials.gov ID
- NCT02081027
- Acronym / sponsor ID
- CIN001- Riluzole in Autism
- Conditions
- Autism Spectrum Disorders
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 19 Sep 2013 (actual)
- Primary completion
- Apr 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 7 Mar 2014 (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.
Determine if riluzole shows evidence of efficacy, safety, and tolerability targeting drug-refractory irritability (DRI) in persons with autism spectrum disorders (ASD).
Detailed description
A randomized, double blind, placebo-controlled, 12-week cross-over study (5-week treatment periods with 2-week washout) of adjunctive riluzole in 12 persons with ASD and DRI between the ages of 12 and 25 years.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Riluzole | Experimental | Drug: RiluzoleThe maximum dose of riluzole to be used in this study is 200 mg per day divided BID |
| Placebo | Placebo comparator | Other: placeboPlacebo will be administered in the same manner as the riluzole group, in order to maintain subject assignment throughout the study. |
Who may qualify
- Ages
- 12 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Age ≥ 12 and < 26 years.
- Weight greater than 50 kg.
- Diagnosis of ASD
- Drug-refractory irritability, as defined as screening ABC Irritability subscale (ABC-I) score of ≥18 AND 1) failure of clinically adequate treatment trials of both aripiprazole and risperidone or 2) failure of at least three previous clinically adequate drug trials targeting irritability (one trial must include aripiprazole or risperidone), as confirmed by caregiver reports and medical record review when available.
- Stable dosing of all concomitant psychotropic medications (including those targeting irritability) for four weeks prior to screening visit and during the study.
Key exclusion criteria (10 listed)
- Current use of more than two concomitant psychotropic drugs targeting irritability.
- Current use of valproic acid.
- Current use of drugs with known interaction with riluzole
- Current use of drugs with concomitant glutamatergic or glutamatergic- modulating action medications.
- For female subjects of child bearing potential, a positive serum pregnancy test.
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 |
|---|---|---|
| RiluzoleAlso: Other name: Rilutek | Drug | Riluzole |
| placebo | Other | Placebo |
Primary outcome
Clinical Global Impression-Improvement
Time frame: Change from baseline to end of week 12
A clinician-rated global assessment of symptom change rated on a scale from 1 to 7
Aberrant Behavior Checklist- Irritability
Time frame: Change between baseline and end of each phase
A parent questionnaire measuring five behavioral domains
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 14 Jan 2021.
- Derived from this study: PubMed 29644582Wink LK, Adams R, Horn PS, Tessier CR, Bantel AP, Hong M, Shaffer RC, Pedapati EV, Erickson CA. A Randomized Placebo-Controlled Cross-Over Pilot Study of Riluzole for Drug-Refractory Irritability in Autism Spectrum Disorder. J Autism Dev Disord. 2018 Sep;48(9):3051-3060. doi: 10.1007/s10803-018-3562-5.
- Journal of Autism and Developmental Disorders (2018) 48:3051-3060Link provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Logan Wink, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Riluzole in people with Autism Spectrum Disorders, run by Children's Hospital Medical Center, Cincinnati 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 6 inclusion and 10 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 14 Jan 2021. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Clinical Global Impression-Improvement (time frame: Change from baseline to end of week 12). 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 NCT02081027, 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 14 Jan 2021; AzTrials last refreshed its copy on 7 Nov 2026.