- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02947048
Safety of L1-79 in Autism
A Phase 2 Safety Study of L1-79 for the Treatment of Autism
Study Overview
Detailed Description
Protocol Number: HT 02-121
Protocol Title: Phase 2 Safety Study of L1-79 for the Treatment of Autism Study Phase: 2
The first cohort of 20 patients to be enrolled will all receive L1-79 100 mg t.i.d., and will be comprised of 3 groups of patients. The first group of patients to receive 100 mg will differ from the others in that they will get blood samples drawn for PK analysis and EKGs will be taken. The safety and PK data from this group will be submitted for FDA review and acceptance before the 200 mg t.i.d. cohort will be enrolled. The remaining 15 patients in this cohort will be randomized to receive either L1-79 100 mg t.i.d. or placebo on a 2:1 basis (2 L1-79 patients for each placebo patient). While the FDA is reviewing the data from the first 5 patients all 100 mg t.i.d. patients will continue to be treated.
The second cohort is identical to the first. The initial 5 patients to be enrolled will differ from the others in that they will get blood samples drawn for PK analysis and EKGs will be taken. The remaining 15 patients in this cohort will be randomized to receive either L1-79 200 mg t.i.d. or placebo on a 2:1 active:placebo.
Sample Size: N=40
- Group 1 (n=5) open100mg L1-79 (1x100mg capsule+1 placebo capsule)
- Group 2 (n=10) blind100mg L1-79 (1x100mg capsule+1 placebo capsule)
- Group 3 (n=5) open200 mg L1-79 (2x100 mg capsules)
- Group 4 (n=10) blind200 mg L1-79 (2x100 mg capsules)
- Group 5 (n=10) Placebo (2 placebo capsules) All Groups will receive the assigned study drug three-times daily
Study Population: Male subjects with autism between the ages of 13 and 21 years of age who meet the entry criteria and who are able to complete standardized measures allowing them to participate in this study.
Evaluation Schedule: Subjects will be evaluated within one week prior to study accession, and weekly throughout the dosing period, and again 4 weeks after the cessation of treatment. The Assigned Dosage Groups (Groups 1 and 3) will have PK blood draws and EKG the randomized group will not have.
Safety Measures: All Groups will have regularly scheduled complete history and physical examination that includes orthostatic blood pressure measurements, vital signs, CBC, differential, platelet counts, urine analysis, and serum analytes including: total protein, albumin, glucose, BUN, creatinine, direct and total bilirubin, alkaline phosphatase, phosphorous, calcium, AST, ALT, sodium, potassium, chloride, bicarbonate, T4, TSH, and adverse events assessments. The Assigned Groups (1 and 3) will also have electrocardiograms taken at the study screening visit and weekly throughout the treatment interval.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
New Jersey
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Livingston, New Jersey, United States, 07039
- Eric Bartky MD, Bartky Health Care Center
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Sea Girt, New Jersey, United States, 07850
- F. Peter Halas MD, Sea Girt Pediatrics
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Males who are not sexually active
- 13 and 21 years of age
- Signed informed consent
- Normal clinical laboratory values
- DSM-5 compliant diagnosis of autism spectrum disorder, confirmed by the Autistic Diagnosis Interview Review (ADIR), and by the Autism Diagnosis Observation Schedule (ADOS) score consistent with a diagnosis of autism
- No more than one concomitant medication for the treatment of autism, on a stable for at least 2 weeks prior to enrollment and no planned changes in psychosocial interventions during the trial
- No medications for any other pathology
Exclusion Criteria:
- Any co-morbidities, including Fragile-X syndrome, epilepsy, Retts syndrome, ADHD, or other disease or syndrome aside from autism that requires treatment
- Any other psychiatric disorder, or out of range lab values
- DSM-5 diagnosis of schizophrenia, schizoaffective disorder, alcohol use disorder
- Active medical problems: unstable seizures (>2 in past month)
- Concomitant physical illness
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 100 mg open
open-label lead-in 100 mg L1-79 t.i.d.
|
|
Experimental: 100 mg blinded
blinded and randomized 100 mg L1-79 t.i.d.
|
|
Experimental: 200 mg open
open-label lead-in 200 mg L1-79 t.i.d.
|
|
Experimental: 200 mg blinded
blinded and randomized 200 mg L1-79 t.i.d.
|
|
Placebo Comparator: Placebo
placebo t.i.d.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Adverse event frequency
Time Frame: 56 days
|
Adverse events will be solicited over 56 days from the start of treatment
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56 days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change from baseline in CGI
Time Frame: Week 0 and weekly for first 28 days, and at day 56.
|
The attending physicians assessment as quantified by the Clinical Global Impressions Scale completed at baseline and weekly through 56 days of treatment and follow-up.
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Week 0 and weekly for first 28 days, and at day 56.
|
Change from Baseline in Vineland Adaptive Behavior Scales - 2nd Edition
Time Frame: Day 0 and at days 28 and 56.
|
Changes from baseline in Communication and Socialization domain of Vineland Adaptive Behavior Scale 2nd edition (VABS II)
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Day 0 and at days 28 and 56.
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Change from Baseline in the Autism Diagnostic Observation Schedule (ADOS)
Time Frame: Day 0 and at days 28 and 56.
|
Changes from baseline in the Autism Diagnostic Observation Schedule (ADOS) total and domain scores.
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Day 0 and at days 28 and 56.
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Change from baseline in Aberrant Behavior Checklist - Community
Time Frame: Week 0 and weekly for first 28 days, and at day 56.
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Weekly Changes from baseline in the Aberrant Behavior Checklist-Community (ABC-C) domains of irritability, social withdrawal and lethargy, hyperactivity, inappropriate speech and stereotypical behavior.
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Week 0 and weekly for first 28 days, and at day 56.
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Change from baseline in the Social Responsiveness Scale - 2nd edition (SRS-2)
Time Frame: Week 0 and weekly for first 28 days, and at day 56.
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Weekly changes from baseline in overall and subdomains of the Social Responsiveness Scale (SRS).
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Week 0 and weekly for first 28 days, and at day 56.
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Changes from baseline in the Repetitive Behavior Scale - Revised (RBS-R).
Time Frame: Week 0 and weekly for first 28 days, and at day 56.
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Weekly changes from baseline in the overall and subdomains of the Repetitive Behavior Scale - Revised (RBS-R).
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Week 0 and weekly for first 28 days, and at day 56.
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Plasma concentrations of L1-79
Time Frame: Week 0 and weekly for 28 days; random sampling times will be employed
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Blood will be collected from assigned patients in each dose group at baseline visit one hour after dose and again at each clinic visit at random times after dose for determination of L1-79 concentrations
|
Week 0 and weekly for 28 days; random sampling times will be employed
|
Collaborators and Investigators
Sponsor
Investigators
- Study Director: John Rothman, PhD, Yamo Pharmaceuticals
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- HT 02-121
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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