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

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ClinicalTrials.gov Identifier: NCT04644003
Recruitment Status : Recruiting
First Posted : November 25, 2020
Last Update Posted : June 18, 2021
Sponsor:
Information provided by (Responsible Party):
Stalicla SA

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).

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: STP1 Drug: Placebo Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : December 7, 2020
Estimated Primary Completion Date : November 30, 2021
Estimated Study Completion Date : December 31, 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: STP1 Low Dose
1 capsule and 1 tablet per intake
Drug: STP1
STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor

Experimental: STP1 High Dose
1 capsule and 1 tablet per intake
Drug: STP1
STP1 is a combination of two drugs, a PDE inhibitor and an NKCC1 inhibitor

Placebo Comparator: Placebo
1 placebo capsule and 1 placebo tablet per intake
Drug: Placebo
Placebo medication (capsule and tablet) identical in appearance to active medication




Primary Outcome Measures :
  1. Safety and Tolerability [ Time Frame: 28 days ]
    Incidence, nature and severity of adverse events, serious adverse events and adverse events of special interest


Secondary Outcome Measures :
  1. Plasma concentration of STP1 (PK) [ Time Frame: Day 1, Day 7, Day 14, Day 15 (and optionally: Day 16, Day 17 and Day 18) ]

Other Outcome Measures:
  1. ABC-C: Aberrant Behavior Checklist-Community [ Time Frame: 28 days ]
    Assess maladaptive behaviors across 5 original subscales: Irritability, Social Withdrawal, Stereotypic Behavior, Hyperactive/non-compliance, Inappropriate Speech

  2. OACIS: Ohio Autism Clinical Impression Scale [ Time Frame: 28 days ]
    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).

  3. CGI-S: Clinical Global Impressions-Severity reflected by the Clinical Global Impressions-Improvement (CGI-I) scale [ Time Frame: 28 days ]
    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

  4. NIH-TCB: NIH Toolbox Cognitive Battery [ Time Frame: 28 days ]
    Computer-based test, that assesses cognition

  5. KiTAP test battery: The Test of Attentional Performance [ Time Frame: 28 days ]
    The KiTAP test is a computer-based Continuous Performance Tasks (CPT) and Executive Function (EF) battery test

  6. Social Responsiveness Scale, 2nd Edition (SRS-2) [ Time Frame: 28 days ]
    The SRS-2 is a 65-item parent/caregiver rating scale used to assess the severity of social impairment within patients with ASD.

  7. CSHQ: Children Sleep Habit Questionnaire [ Time Frame: 28 days ]
    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.

  8. EEG: Electroencephalogram [ Time Frame: 28 days ]
    Auditory Event Related Potentials (ERP) will be measured.

  9. Eye-tracking [ Time Frame: 28 days ]
    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

  10. Lactate/Pyruvate Ratio - L:P [ Time Frame: 28 days ]
    Change from baseline, measured in blood



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 40 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04644003


Contacts
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Contact: Hannah Meinen 513-636-2592 Hannah.Meinen@cchmc.org

Locations
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United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Craig A Erickson, MD    513-803-8653    autism@cchmc.org   
Sponsors and Collaborators
Stalicla SA
Investigators
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Principal Investigator: Craig Erickson, MD Children's Hospital Medical Center, Cincinnati
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Responsible Party: Stalicla SA
ClinicalTrials.gov Identifier: NCT04644003    
Other Study ID Numbers: STP1-C004
First Posted: November 25, 2020    Key Record Dates
Last Update Posted: June 18, 2021
Last Verified: June 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Stalicla SA:
ASD
Autism Spectrum Disorder
Additional relevant MeSH terms:
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Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders