ASDPhase 2ACEP4NCT03243552
Proof of Mechanism Study for the Treatment of Social Anhedonia in ASD
A Phase 2 study testing Social Skills Training in people with ASD, run by University of California, Los Angeles at 1 site in United States.
Trial summary
This project will use the experimental medicine approach of a Phase IIa Proof of Mechanism 16-week, randomized, double-blind, controlled trial of L-DOPA versus placebo administration in combination with a 16 week social skills training group in order to: 1) identify differences in social reward processes in adolescent… Read more about this study
- Who is it for?
- Ages 13 to 30 yearsHealthy volunteers can take part
- Conditions the study is for
- ASD
- What is being tested?
- Social Skills Training
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 56 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jun 2017to Dec 2023
- Phase
- Phase 2
- 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 13 to 30 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- ages 13 - 30 years inclusive;
- meets diagnostic criteria for ASD by clinical evaluation and ADOS;
- estimated FS IQ >70; 4) English reading ability of 6th grade;
- ability to participate and complete protocol expectations (fMRI scan, testing) in the examining clinician's judgment; and
- planned enrollment and acceptance for the UCLA PEERS® adolescent or young adult social skills training program.
Who cannot join
- significant perceptual deficits;
- need for continuation or anticipated of use of prohibited dopamine-modifying medications (stimulants, antipsychotics);
- presence of serious behavioral comorbidity such as aggression, major depressive disorder requiring additional intervention, or self-injurious behavior, or current of past history of suspected psychotic disorder;
- history of tic disorder;
- presence of significant medical illness which may impact CNS function.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, Los Angeles
Los Angeles, California, 90095
About this study
Official title: Proof of Mecahism Study for the Treatment of Social Anhedonia in ASD
This project will use the experimental medicine approach of a Phase IIa Proof of Mechanism 16-week, randomized, double-blind, controlled trial of L-DOPA versus placebo administration in combination with a 16 week social skills training group in order to: 1) identify differences in social reward processes in adolescent and young adult ASD participants versus healthy controls as measured by fMRI activation in reward circuitry; 2) provide evidence of dopaminergic moderating effects on social reward components in ASD with greater pre- to post-treatment changes expected in the subjects randomized to L-DOPA versus placebo; 3) examine the hypothesis that baseline readouts of putative dopamine signaling (wanting activation responses) will predict the extent of fMRI reward-related activation changes pre- to post-treatment; and, 4) examine the proposed relationship between pre- to post- L-DOPA fMRI reward changes and changes in individual self-report ratings of social wanting and ratings of videotaped positive affect in a structured interaction with an examiner. The study will enroll 56 participants with DSM-5 ASD between the ages of 13-30 years of age and 18 healthy control participants without histories of psychopathology for baseline comparisons.
Read the detailed description
Participants will comprise two groups: 1) 56 (male and female) adolescents and young adults likely to meet inclusion/exclusion confirmed by study assessment who will be outpatients, ages 13 to 30 years, inclusive; and 2) 18 (male and female) healthy adolescents and young adults without any history of significant psychiatric disorders or treatment, who will be ambulatory, and will reflect the racial, ethnic, and socio-economic composition of Los Angeles, and will be recruited without regard to gender, race, or ethnic background.
Healthy control adolescents (and parents) and young adults who meet all inclusion and exclusion criteria, will undergo a one-day 4-5 hour research evaluation of current functioning and behavior, cognitive function, including intellectual testing, urine drug screen, completion of questionnaires about possible psychological symptoms, perform brief tests to measure reward response, and complete a research Magnetic Resonance Imaging scan. Healthy control will not receive any interventions or medication.
All eligible ASD participants will be enrolled concurrently in a 16-week, structured, group-based, social skills training program (PEERS®). After screening evaluations, eligible subjects will undergo baseline MRI scanning and behavioral assessments (self- and parent-report measures, videotaped interaction) and then randomized in a blocked schedule to yield 1:1 L-DOPA: placebo assignment, before beginning weekly social skills training. Medication administration will begin simultaneously with social skills training, and subjects will be seen weekly for the 1st 8 weeks, then biweekly for the final 4 weeks for safety assessments, dose titration, and compliance checks. During the 16-week study period, primary behavioral assessments will be repeated at Week 8 and at end of study on Week 16. A second MRI scan will be obtained within + 4 days of last social skills session. A follow-up safety phone call to assess post-study condition will be performed 30 days after completion of double-blind. The rich set of behavioral observations obtained across the 16 weeks will allow the possible identification of early clinical efficacy changes associated with L-DOPA administration, which could add further support for investigating this mechanism in future studies. Results from the project should inform reseacher's understanding of determinants of social dysfunction in ASD and may support further investigation of treatments modulating central nervous system dopamine function as a therapeutic strategy to enhance social functioning in ASD.
- Conditions
- ASD
- Being tested
- L-DOPA versus Placebo, Social Skills Training
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
L-DOPA versus PlaceboDrug
Participants will be randomized in a blocked schedule to yield 1:1 L-DOPA: placebo assignment.
Social Skills TrainingBehavioural
Also called PEERS
ASD participants will be enrolled concurrently in a 16-week manualized, structured, group-based, social skills training program (PEERS).
| Study group | Role | What they receive |
|---|---|---|
| L-DOPA versus Placebo | Active comparator | L-DOPA or placebo (1:1 randomization). Dosing will begin at 25mg carbidopa/100mg L-DOPA in 3 divided doses, with a fixed-flexible titration schedule, allowing dose increases once per week of 100mg L-DOPA. Maximum dose is 600mg/d. Receives: Drug: L-DOPA versus Placebo; Behavioral: Social Skills Training |
| Social Skills | Experimental | All participants will receive 16-week manualized social skills training. Receives: Drug: L-DOPA versus Placebo; Behavioral: Social Skills Training |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in fMRI: Social Reward Task from Baseline to week 16 (4 months)
BOLD Activation: VS, ACC, OFC, Amygdala, Hippocampus (same units of measure)
Measured: Baseline, week 16
Secondary outcomes (8)
Change on Anticipatory and Consummatory Interpersonal Pleasure Scale Adolescent (ACIPS-A)
Total Score (change in score by timepoint; before treatment, midpoint, end)
Measured: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
Change on Anticipatory and Consummatory Interpersonal Pleasure Scale (ACIPS)
Total Score (change in score by timepoint; before treatment, midpoint, end)
Measured: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
Change on Child Behavior Checklist (CBCL)
T Scores of Syndrome Scales for ages 18 or under
Measured: Baseline, week 16 (4 month/end of study)
Change on Youth Self Report (YSR)
T Scores of Syndrome Scales for ages 18 or under
Measured: Baseline, week 16 (4 month/end of study)
Change on Adult Behavior Checklist (ABCL)
T Scores of Syndrome Scales for ages 18 or over
Measured: Baseline, week 16 (4 month/end of study)
Change on SRS Self Report
Social Anhedonia
Measured: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
Change on SRS Parent Report
Social Anhedonia
Measured: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
change on Social Communication Interaction Test (SCIT)
6 subscales and total score (0-30)
Measured: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2017
- Study start1 Jun 2017
- Primary completionDec 2023 (estimated)
- Study completionDec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ASDStudies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Phase IIa Proof of Mechanism 16-week, randomized, double-blind, controlled trial of L-DOPA versus placebo.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Responsible party
- Principal investigator, James McCracken, University of California, Los Angeles
- Investigators
- James T McCracken, MD, Principal investigator, University of California, Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03243552
- Sponsor's protocol ID
- ACEPIV
- Acronym
- ACEP4
- Status last verified
- Jun 2023
- First submitted
- 27 Jul 2017
- Last updated
- 28 Jun 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Proof of Mecahism Study for the Treatment of Social Anhedonia in ASD
- ClinicalTrials.gov ID
- NCT03243552
- Acronym / sponsor ID
- ACEP4 / ACEPIV
- Conditions
- ASD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Study start
- 1 Jun 2017 (actual)
- Primary completion
- Dec 2023 (estimated)
- Study completion
- Dec 2023 (estimated)
- First posted
- 9 Aug 2017 (actual)
- 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.
This project will use the experimental medicine approach of a Phase IIa Proof of Mechanism 16-week, randomized, double-blind, controlled trial of L-DOPA versus placebo administration in combination with a 16 week social skills training group in order to: 1) identify differences in social reward processes in adolescent and young adult ASD participants versus healthy controls as measured by fMRI activation in reward circuitry; 2) provide evidence of dopaminergic moderating effects on social reward components in ASD with greater pre- to post-treatment changes expected in the subjects randomized to L-DOPA versus placebo; 3) examine the hypothesis that baseline readouts of putative dopamine signaling (wanting activation responses) will predict the extent of fMRI reward-related activation changes pre- to post-treatment; and, 4) examine the proposed relationship between pre- to post- L-DOPA fMRI reward changes and changes in individual self-report ratings of social wanting and ratings of videotaped positive affect in a structured interaction with an examiner. The study will enroll 56 participants with DSM-5 ASD between the ages of 13-30 years of age and 18 healthy control participants without histories of psychopathology for baseline comparisons.
Detailed description
Participants will comprise two groups: 1) 56 (male and female) adolescents and young adults likely to meet inclusion/exclusion confirmed by study assessment who will be outpatients, ages 13 to 30 years, inclusive; and 2) 18 (male and female) healthy adolescents and young adults without any history of significant psychiatric disorders or treatment, who will be ambulatory, and will reflect the racial, ethnic, and socio-economic composition of Los Angeles, and will be recruited without regard to gender, race, or ethnic background.
Healthy control adolescents (and parents) and young adults who meet all inclusion and exclusion criteria, will undergo a one-day 4-5 hour research evaluation of current functioning and behavior, cognitive function, including intellectual testing, urine drug screen, completion of questionnaires about possible psychological symptoms, perform brief tests to measure reward response, and complete a research Magnetic Resonance Imaging scan. Healthy control will not receive any interventions or medication.
All eligible ASD participants will be enrolled concurrently in a 16-week, structured, group-based, social skills training program (PEERS®). After screening evaluations, eligible subjects will undergo baseline MRI scanning and behavioral assessments (self- and parent-report measures, videotaped interaction) and then randomized in a blocked schedule to yield 1:1 L-DOPA: placebo assignment, before beginning weekly social skills training. Medication administration will begin simultaneously with social skills training, and subjects will be seen weekly for the 1st 8 weeks, then biweekly for the final 4 weeks for safety assessments, dose titration, and compliance checks. During the 16-week study period, primary behavioral assessments will be repeated at Week 8 and at end of study on Week 16. A second MRI scan will be obtained within + 4 days of last social skills session. A follow-up safety phone call to assess post-study condition will be performed 30 days after completion of double-blind. The rich set of behavioral observations obtained across the 16 weeks will allow the possible identification of early clinical efficacy changes associated with L-DOPA administration, which could add further support for investigating this mechanism in future studies. Results from the project should inform reseacher's understanding of determinants of social dysfunction in ASD and may support further investigation of treatments modulating central nervous system dopamine function as a therapeutic strategy to enhance social functioning in ASD.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Phase IIa Proof of Mechanism 16-week, randomized, double-blind, controlled trial of L-DOPA versus placebo.
| Arm | Type | Receives |
|---|---|---|
| L-DOPA versus Placebo | Active comparator | Drug: L-DOPA versus Placebo, Behavioral: Social Skills TrainingL-DOPA or placebo (1:1 randomization). Dosing will begin at 25mg carbidopa/100mg L-DOPA in 3 divided doses, with a fixed-flexible titration schedule, allowing dose increases once per week of 100mg L-DOPA. Maximum dose is 600mg/d. |
| Social Skills | Experimental | Drug: L-DOPA versus Placebo, Behavioral: Social Skills TrainingAll participants will receive 16-week manualized social skills training. |
Who may qualify
- Ages
- 13 to 30 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- ages 13 - 30 years inclusive;
- meets diagnostic criteria for ASD by clinical evaluation and ADOS;
- estimated FS IQ >70; 4) English reading ability of 6th grade;
- ability to participate and complete protocol expectations (fMRI scan, testing) in the examining clinician's judgment; and
- planned enrollment and acceptance for the UCLA PEERS® adolescent or young adult social skills training program.
Key exclusion criteria (5 listed)
- significant perceptual deficits;
- need for continuation or anticipated of use of prohibited dopamine-modifying medications (stimulants, antipsychotics);
- presence of serious behavioral comorbidity such as aggression, major depressive disorder requiring additional intervention, or self-injurious behavior, or current of past history of suspected psychotic disorder;
- history of tic disorder;
- presence of significant medical illness which may impact CNS function.
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 |
|---|---|---|
| L-DOPA versus PlaceboParticipants will be randomized in a blocked schedule to yield 1:1 L-DOPA: placebo assignment. | Drug | L-DOPA versus Placebo, Social Skills |
| Social Skills TrainingAlso: PEERSASD participants will be enrolled concurrently in a 16-week manualized, structured, group-based, social skills training program (PEERS). | Behavioural | L-DOPA versus Placebo, Social Skills |
Primary outcome
Change in fMRI: Social Reward Task from Baseline to week 16 (4 months)
Time frame: Baseline, week 16
BOLD Activation: VS, ACC, OFC, Amygdala, Hippocampus (same units of measure)
8 secondary outcomes
Change on Anticipatory and Consummatory Interpersonal Pleasure Scale Adolescent (ACIPS-A)
Time frame: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
Change on Anticipatory and Consummatory Interpersonal Pleasure Scale (ACIPS)
Time frame: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
Change on Child Behavior Checklist (CBCL)
Time frame: Baseline, week 16 (4 month/end of study)
Change on Youth Self Report (YSR)
Time frame: Baseline, week 16 (4 month/end of study)
Change on Adult Behavior Checklist (ABCL)
Time frame: Baseline, week 16 (4 month/end of study)
Change on SRS Self Report
Time frame: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
Change on SRS Parent Report
Time frame: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
change on Social Communication Interaction Test (SCIT)
Time frame: Baseline, week 8 (midpoint), week 16 (4 month/end of study)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jun 2023.
- 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.
James T McCracken, MD principal investigator
University of California, Los Angeles
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Social Skills Training in people with ASD, run by University of California, Los Angeles 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: status not confirmed, as of the sponsor's last update on 28 Jun 2023. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
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?
Change in fMRI: Social Reward Task from Baseline to week 16 (4 months) (time frame: Baseline, week 16). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Jun 2023; AzTrials last refreshed its copy on 8 Nov 2026.