Phase 2
N=42
Safety of L1-79 in Adolescent and Adult Males With Autism
Autism
Bottom Line
View on ClinicalTrials.gov: NCT02947048 ↗Enrolled (actual)
42
Serious AEs
0.0%
Results posted
Aug 2024
Primary outcome: Primary: Adverse Events — 2; 1; 2; 6 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- L1-79 (Drug); Placebo (Drug)
- Age
- Pediatric, Adult · 12+ yrs
- Sex
- Male
- Sponsor
- Yamo Pharmaceuticals LLC
- Primary completion
- Dec 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Adverse Events |
2; 1; 2; 6; 5; 5 | — |
| SECONDARY Change From Baseline in Clinical Global Impression Scale (CGI) |
-0.5; -0.2; -0.8; -0.3; 3.0; 2.8 | 0.61 |
| SECONDARY Change From Baseline in Vineland Adaptive Behavior Scale - 2nd Edition Socialization Standard Score |
-4.2; 0.0; 7.6; 1.5 | — |
| SECONDARY Change From Baseline in Vineland Adaptive Behavior Scale - 2nd Edition Communication Standard Score |
10.1; 7.7; 2.7; 3.0 | — |
| SECONDARY Change From Baseline in the Autism Diagnostic Observation Schedule 2nd Edition (ADOS-2) Overall Total Score |
-0.6; 0.3; -1.0; -0.3 | — |
| SECONDARY Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score |
-0.4; -3.8; -8.6; -9.3; 1.0; -0.8 | — |
| SECONDARY Change From Baseline in Aberrant Behavior Checklist - Community (ABC-C) |
-0.9; -5.3; -3.1; -4.5; -1.6; -3.5 | — |
| SECONDARY Change From Baseline in the Repetitive Behavior Scale - Revised (RBS-R) Total Score |
-3.2; -9.3; -17.6; -16.5; -0.6; -2.3 | — |
Summary
This is a five-arm designed to assess the safety of L1-79 that incorporates 15 prospectively randomized, placebo controlled patients and 5 open label patients at either 100 tid (three times daily) or 200 tid dosing for 28 days. The open label patients will be assessed for the purpose of understanding PK/PD and to determine if there are any EKG changes associated with the administration of L1-79. Additional safety information will be provided by the 30 patients randomized 2:1 active:placebo.
Eligibility Criteria
Inclusion Criteria
- Males who are not sexually active
- 12 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
Data sourced from ClinicalTrials.gov (NCT02947048). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.