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

Primary Purpose

Autism Spectrum Disorder

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

STP1

Placebo

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring ASD, Autism Spectrum Disorder

Eligibility Criteria

18 Years - 40 Years (Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Cincinnati Children's Hospital Medical Center

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Arm Label

STP1 Low Dose

STP1 High Dose

Placebo

Arm Description

1 capsule and 1 tablet per intake

1 placebo capsule and 1 placebo tablet per intake

Outcomes

Primary Outcome Measures

Safety and Tolerability

Incidence, nature and severity of adverse events, serious adverse events and adverse events of special interest

Secondary Outcome Measures

Plasma concentration of STP1 (PK)

Standard non-compartmental analysis

Full Information

NCT ID

NCT04644003

First Posted

November 16, 2020

Last Updated

February 1, 2022

Sponsor

Stalicla SA

1. Study Identification

Unique Protocol Identification Number

NCT04644003

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2022

Overall Recruitment Status

Completed

Study Start Date

December 7, 2020 (Actual)

Primary Completion Date

January 28, 2022 (Actual)

Study Completion Date

January 28, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Stalicla SA

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Autism Spectrum Disorder

Keywords

ASD, Autism Spectrum Disorder

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

STP1 Low Dose

Arm Type

Experimental

Arm Description

1 capsule and 1 tablet per intake

Arm Title

STP1 High Dose

Arm Type

Experimental

Arm Description

1 capsule and 1 tablet per intake

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

1 placebo capsule and 1 placebo tablet per intake

Intervention Type

Drug

Intervention Name(s)

STP1

Intervention Description

STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo medication (capsule and tablet) identical in appearance to active medication

Primary Outcome Measure Information:

Title

Safety and Tolerability

Description

Incidence, nature and severity of adverse events, serious adverse events and adverse events of special interest

Time Frame

28 days

Secondary Outcome Measure Information:

Title

Plasma concentration of STP1 (PK)

Description

Standard non-compartmental analysis

Time Frame

Day 1, Day 7, Day 14, Day 15 (and optionally: Day 16, Day 17 and Day 18)

Other Pre-specified Outcome Measures:

Title

ABC-C: Aberrant Behavior Checklist-Community

Description

Assess maladaptive behaviors across 5 original subscales: Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/non-compliance, Inappropriate Speech

Time Frame

28 days

Title

OACIS: Ohio Autism Clinical Impression Scale

Description

The OACIS is composed of two scales: the OACIS-Severity scale (OACIS-S), which measures global severity of illness at a given point in time as well as scores for 9 anchors: social interactions; aberrant/abnormal behaviors; repetitive/ritualistic behaviors; verbal communication; non-verbal communication; hyperactivity/inattention; anxiety/fears; sensory sensitivities; restricted and narrow interests and the OACIS improvement scale (OACIS-C), which permits a global evaluation by the clinician of the subject's improvement over time. The OACIS-S is a 7-point scale ranging from 1 (no symptoms) to 7 (very severe). The OACIS-C is a seven-point scale, ranging from 1 (very much improved) to 7 (very much worse).

Time Frame

28 days

Title

CGI-S: Clinical Global Impressions-Severity reflected by the Clinical Global Impressions-Improvement (CGI-I) scale

Description

Illness severity rating is made on a scale of 1 to 7, with 1 being "normal not at all mentally ill" and 7 being "among the most extremely ill patients". Subsequently, the patient's condition on the study drug (or placebo) is compared to the patient's condition before the initiation of the study drug (or placebo) (baseline) via additional CGI-S ratings or the CGI-I item. The CGI is a clinician-rated scale utilizing history from the caregiver and incorporating it into a clinical rating. The CGI-I will be used to judge the change in clinical impression as 1=very much improved since the initiation of treatment; 2=much improved; 3=minimally improved; 4=no change from baseline (the initiation of treatment); 5=minimally worse; 6= much worse; 7=very much worse since the initiation of treatment

Time Frame

28 days

Title

NIH-TCB: NIH Toolbox Cognitive Battery

Description

Computer-based test, that assesses cognition

Time Frame

28 days

Title

KiTAP test battery: The Test of Attentional Performance

Description

The KiTAP test is a computer-based Continuous Performance Tasks (CPT) and Executive Function (EF) battery test

Time Frame

28 days

Title

Social Responsiveness Scale, 2nd Edition (SRS-2)

Description

The SRS-2 is a 65-item parent/caregiver rating scale used to assess the severity of social impairment within patients with ASD.

Time Frame

28 days

Title

CSHQ: Children Sleep Habit Questionnaire

Description

The CSHQ is a retrospective, 45-item parent questionnaire, which includes items relating to a number of key sleep domains that encompass the major presenting clinical sleep complaints in this age group: bedtime behavior and sleep onset; sleep duration; anxiety around sleep; behavior occurring during sleep and night wakings; sleep-disordered breathing; parasomnias; and morning waking/daytime sleepiness.

Time Frame

28 days

Title

EEG: Electroencephalogram

Description

Auditory Event Related Potentials (ERP) will be measured.

Time Frame

28 days

Title

Eye-tracking

Description

Change from baseline in eye gaze to eye regions during viewing of static faces and change in eye gaze to social scene viewing during viewing of dynamic video

Time Frame

28 days

Title

Lactate/Pyruvate Ratio - L:P

Description

Change from baseline, measured in blood

Time Frame

28 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

40 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Craig Erickson, MD

Organizational Affiliation

Children's Hospital Medical Center, Cincinnati

Official's Role

Principal Investigator

Facility Information:

Facility Name

Cincinnati Children's Hospital Medical Center

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

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