Table 2.
Double-blind treatment phase study timeline.
Measurement | Screen (Visit 1) | Baselineb (Visit 2) | Month 1 (Visit 3) | Month 2e (Visit 4) | Month 3 (Visit 5) | Month 4e (Visit 6) | Month 5e (Visit 7) | Month 6 (Visit 8) | Follow-up | PRN |
---|---|---|---|---|---|---|---|---|---|---|
Visit Windows | Within 42d of Screen | ±14d | ±14d | ±14d | ±14d | ±14d | ±14d |
28d after last visit ±14d |
||
Medical History | X | X | X | X | X | X | X | X | X | |
Clinical Interview | X | X | X | X | X | X | X | X | ||
Inclusion/Exclusion Criteria | X | X | ||||||||
Cgi Scales: Severity & Improvement | Xc | X | X | X | X | X | X | |||
Vital Signs | X | X | X | X | X | |||||
Physical/Neurological Exam | X | X | X | |||||||
Dermoscopy | X | X | X | |||||||
Microbiome/Mycobiome Sample Collection | X | X | X | |||||||
Tanner Staging | X | X | ||||||||
Side Effects (CTCAE V5.0 + DOTES) | X | X | X | X | X | X | X | X | X | |
Laboratory Testsa | X | X | X | X | X | |||||
Everolimus Level (Only Done After M1 If Needed) | X | X | ||||||||
PTEN-Associated Proteins | Xd | Xd | X | X | ||||||
Concomitant Treatment Log | X | X | X | X | X | X | X | X | X | |
Autism Diagnostic Interview- Revised | X | |||||||||
Primary Efficacy Outcome Neurocognitive Composite | X | X | X | X | ||||||
Columbia-Suicide Severity Rating Scale | X | X | X | X | ||||||
Cpt-3/K-Cpt-2) | X | X | X | X | ||||||
SB-5/Mullen Scales Of Early Learning (MSEL) | X | X | ||||||||
SB-5 Working Memory Subscale Only | X | X | ||||||||
Purdue Pegboard (Pp) | X | X | X | X | ||||||
Wechsler Processing Speed Index | X | X | X | |||||||
Wide Range Assessment of Memory and Learning-2 (WRAML-2) | X | X | X | |||||||
Peabody Picture Vocabulary Test – Fourth Edition (PPVT-4) | X | X | X | |||||||
Expressive Vocabulary Test – Second Edition (EVT-2) | X | X | X | |||||||
Autism Diagnostic Observation Schedule (ADOS-2) | X | X | ||||||||
Social Responsiveness Scale – Second Edition (SRS-2) | X | X | X | |||||||
Repetitive Behavior Scale – Revised (RBS-R) | X | X | X | |||||||
Developmental Coordination Disorder Questionnaire (DCDQ) | X | X | X | X | ||||||
Behavior Rating Inventory of Executive Function (BRIEF) | X | X | X | |||||||
Adult/Child Behavior Checklist | X | X | X | X | ||||||
Sensory Profile Questionnaire - Short Form (SPQ) | X | x | x | |||||||
Vineland Adaptive Behavior Scales (VABS-III) – caregiver report | X | X | X | |||||||
Eye Tracking (Optional) | X | X | X | |||||||
Resting State EEG/Auditory Evoked Potentials (Optional) | X | X | X | |||||||
Participant Unblinding | X | X |
**: primary outcome mesaure only.
Coagulation testing will only be performed at screening, month 3, and month 6. Pregnancy testing, serum is done at screening and month 6 and urine may be used for onsite visits (if deemed clinically necessary) for women of childbearing potential.
Monthly visit windows are calculated based on date of baseline visit.
At the baseline visit, only the CGI: Severity scale will be completed.
PTEN-associated protein blood collections will be collected at either the screening or baseline visit (as well as at the 3-month and 6-month visits). CGI: Clinical Global Impressions Scale; CPT: Conners' Continuous Performance Test; IE: Independent Evaluator; CTCAE: Common Terminology Criteria for Adverse Events; DOTES: Dosage Record and Treatment Emergent Symptom Scale; SB-5: Stanford-Binet Intelligence Scales, Fifth Edition. PRN: Pro Re Nata (visits as needed).
May be performed by phone or in person.