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

Autism Spectrum Disorder Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04644003
• Other study ID #: STP1-C004
• Status: Completed
• Phase: Phase 1
• Start date: December 7, 2020
• Est. completion date: January 28, 2022

Study information

• Verified date: February 2022
• Source: Stalicla SA
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: January 28, 2022
• Est. primary completion date: January 28, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 40 Years

Eligibility

Key Inclusion Criteria:

1. Male or female individuals, between 18 and 40 years, diagnosed of ASD.

2. 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.

3. 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.

4. Patient and/or parent or legal guardian willing and consenting to participate.

5. Patients with ASD and comorbid seizure disorder should be seizure-free for at least 6 months prior to screening.

6. Before enrolling in the study, subjects must agree to use double-barrier birth control methods if they engage in intercourse.

Key Exclusion Criteria:

1. Patients with an identified genetic cause of ASD in their medical record will be excluded from the study.

2. History of traumatic head injury, cerebrovascular disorder, congestive heart failure, hepatic or renal disease.

3. Thrombocytopenia.

4. Type 1 Diabetes Mellitus or uncontrolled type 2 Diabetes Mellitus, or latent autoimmune diabetes of the adult.

5. A significant risk for suicidal behavior.

6. Initiation of, or a major change in psychological / behavioral intervention within 4 weeks prior to randomization.

7. Patient with any active infection.

8. 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.

9. Clinically relevant electrocardiogram (ECG) abnormalities.

10. Clinically significant abnormal laboratory test.

11. Active clinically significant disease.

12. History of malignancy.

13. Pregnant (confirmed by laboratory testing) or lactating female patient.

Related Conditions & MeSH terms

• Autism Spectrum Disorder
• Autistic Disorder
• Child Development Disorders, Pervasive
• Disease

Intervention

Drug:
• STP1
STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor
• Placebo
Placebo medication (capsule and tablet) identical in appearance to active medication

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Stalicla SA

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	ABC-C: Aberrant Behavior Checklist-Community	Assess maladaptive behaviors across 5 original subscales: Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/non-compliance, Inappropriate Speech	28 days
Other	OACIS: Ohio Autism Clinical Impression Scale	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).	28 days
Other	CGI-S: Clinical Global Impressions-Severity reflected by the Clinical Global Impressions-Improvement (CGI-I) scale	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	28 days
Other	NIH-TCB: NIH Toolbox Cognitive Battery	Computer-based test, that assesses cognition	28 days
Other	KiTAP test battery: The Test of Attentional Performance	The KiTAP test is a computer-based Continuous Performance Tasks (CPT) and Executive Function (EF) battery test	28 days
Other	Social Responsiveness Scale, 2nd Edition (SRS-2)	The SRS-2 is a 65-item parent/caregiver rating scale used to assess the severity of social impairment within patients with ASD.	28 days
Other	CSHQ: Children Sleep Habit Questionnaire	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.	28 days
Other	EEG: Electroencephalogram	Auditory Event Related Potentials (ERP) will be measured.	28 days
Other	Eye-tracking	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	28 days
Other	Lactate/Pyruvate Ratio - L:P	Change from baseline, measured in blood	28 days
Primary	Safety and Tolerability	Incidence, nature and severity of adverse events, serious adverse events and adverse events of special interest	28 days
Secondary	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)