Clinical trial NCT02586935 (phase 2, status: completed)
Conditions: autism spectrum disorders
ICD codes: Z16.12
Drugs: tideglusib
SMILES: O=C1SN(C(=O)N1CC1=CC=CC=C1)C1=C2C=CC=CC2=CC=C1
Eligibility criteria:

        Inclusion Criteria:

          1. Outpatients 12-17 years of age inclusive with a mental age equivalent ≥ 18 months at
             Screening.

          2. Weigh a minimum of 30 kg (the 3rd percentile for 12 years of age)

          3. Meet Diagnostic and Statistical Manual of Mental Disorders. Diagnostic and Statistical
             Manual (DSM-5) criteria will be established by a clinician with expertise with
             individuals with ASD.

          4. Have a Clinician's Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at
             Screening.

          5. If already receiving stable concomitant medications affecting behaviour, have stable
             regimens with no changes during the preceding 1 month prior to Screening (with the
             exception of fluoxetine, where a period of 6 weeks is needed), and will not electively
             initiate new or modify ongoing medications for the duration of the study

          6. If already receiving stable non-pharmacological educational and behavioural
             interventions, have continuous participation during the preceding 3 months prior to
             Screening, and not electively initiate new or modify ongoing interventions for the
             duration of the study

          7. Have normal physical examination and laboratory test results at Screening. If
             abnormal, the finding(s) must be deemed clinically insignificant by the Investigator.

          8. Ability to obtain written informed consent from the participant, if developmentally
             appropriate. If a participant does not have the capacity to consent, ability to obtain
             assent (if developmentally appropriate), as well as written informed consent from
             their parent(s)/legal guardian.

        Exclusion Criteria:

          1. Patients with a primary psychiatric diagnosis other than ASD

          2. Pregnant female patients; sexually active female patients on inadequate birth control.

          3. Patients with known phosphatase and tensin homolog (PTEN) mutations as they are
             unlikely to respond to this medication

          4. Patients with a serious medical condition that, based on Investigator judgment, might
             interfere with the conduct of the study, confound interpretation of the study results,
             or endanger their own well-being. Patients with evidence of any significant
             hematological, endocrine, cardiovascular (including uncorrected symptomatic congenital
             heart disease), respiratory, renal, hepatic, or gastrointestinal disease, not
             including mild common pediatric diseases in these areas that are stable (e.g. mild
             asthma, constipation, etc.).

          5. Patients with unstable epilepsy (i.e. seizures occurring within the last 6 months), or
             patients with epilepsy who are not on stable doses of antiepileptic medications (i.e.
             dose changes within the last 3 months).

          6. Patients with hypersensitivity to tideglusib or any components of its formulation.

          7. Patients unable to tolerate venipuncture procedures for blood sampling.

          8. Patients actively enrolled in another intervention study.

          9. Patients who have elevated liver enzymes ≥ 3 times the normal amount before the study
             begins.

         10. Patients who have serum creatinine of >150 μmol/L and creatinine clearance ≤60ml/m
             (according to Cockcroft-Gault formula) at Screening.

         11. Patients taking strong CYP3A4 inhibitors (e.g. clarithromycin, telithromycin,
             ketoconazole, itraconazole, posaconazole, nefazodone, indinavir, ritonavir)

         12. Inability to speak and understand English sufficiently enough to allow for the
             completion of all study assessments (parent; patient, if verbal).