Safety, Tolerability and Pharmacokinetics Study of STP1 in a Subgroup of Patients With Autism Spectrum Disorder (ASD)

After obtaining written informed consent, those patients who are deemed eligible for the study, will be randomized on Day 1, in a double-blinded manner, in a 3:1ratio to receive either oral STP1 (twice daily) or placebo (twice daily). The total study duration is 6 weeks, including a screening phase of up to 2 weeks, a treatment phase of 2 weeks and a post-treatment follow-up phase of 2 weeks.

Key Inclusion Criteria:

• Male or female individuals, between 18 and 40 years, diagnosed of ASD.
• Patients will be assessed for specific developmental anthropometric & anatomical criteria as well as personal and family medical history as assessed by the ASD-Phen1 semi structured interview form.
• Patients must have a parent or reliable caregiver who can provide information about the pre-natal period and early developmental period, as required by the protocol.
• Patient and/or parent or legal guardian willing and consenting to participate.
• Patients with ASD and comorbid seizure disorder should be seizure-free for at least 6 months prior to screening.
• Before enrolling in the study, subjects must agree to use double-barrier birth control methods if they engage in intercourse.

Key Exclusion Criteria:

• Patients with an identified genetic cause of ASD in their medical record will be excluded from the study.
• History of traumatic head injury, cerebrovascular disorder, congestive heart failure, hepatic or renal disease.
• Thrombocytopenia.
• Type 1 Diabetes Mellitus or uncontrolled type 2 Diabetes Mellitus, or latent autoimmune diabetes of the adult.
• A significant risk for suicidal behavior.
• Initiation of, or a major change in psychological / behavioral intervention within 4 weeks prior to randomization.
• Patient with any active infection.
• Systolic blood pressure (SBP) <80 mmHg or diastolic blood pressure (DBP) <40 mmHg or a drop in SBP of ≥20 mm Hg, or in DBP of ≥10 mm Hg, during the orthostatic recordings.
• Clinically relevant electrocardiogram (ECG) abnormalities.
• Clinically significant abnormal laboratory test.
• Active clinically significant disease.
• History of malignancy.
• Pregnant (confirmed by laboratory testing) or lactating female patient.