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

Study Overview
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Brief Summary
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The main purpose of this study is to evaluate safety and tolerability, Pharmacokinetics and Pharmacodynamics, as well as exploratory efficacy of STP1, in a subgroup of patients with Autism Spectrum Disorder (ASD).

Detailed Description
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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.

Official Title
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A Phase 1b, Double-Blind, Placebo-Controlled, First-in-Human Study to Evaluate Safety, Tolerability and Pharmacokinetics of a Two-Week Oral Treatment With STP1 in a Subgroup of Patients With Autism Spectrum Disorder

Conditions
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Autism Spectrum Disorder

Intervention / Treatment
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* Drug: STP1
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 40 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: STP1 Low Dose<br>1 capsule and 1 tablet per intake | Drug: STP1<br>* STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor<br>|
| Experimental: STP1 High Dose<br>1 capsule and 1 tablet per intake | Drug: STP1<br>* STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor<br>|
| Placebo Comparator: Placebo<br>1 placebo capsule and 1 placebo tablet per intake | Drug: Placebo<br>* Placebo medication (capsule and tablet) identical in appearance to active medication<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and Tolerability | Incidence, nature and severity of adverse events, serious adverse events and adverse events of special interest | 28 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Plasma concentration of STP1 (PK) | Standard non-compartmental analysis | Day 1, Day 7, Day 14, Day 15 (and optionally: Day 16, Day 17 and Day 18) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ASD, Autism Spectrum Disorder