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. 2021 Feb 6;21:100733. doi: 10.1016/j.conctc.2021.100733

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.

a

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.

b

Monthly visit windows are calculated based on date of baseline visit.

c

At the baseline visit, only the CGI: Severity scale will be completed.

d

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).

e

May be performed by phone or in person.