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

NCT04644003 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: STP1-C004
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The main purpose of this study is to evaluate safety and tolerability in a subgroup of patients with Autism Spectrum Disorder (ASD). In addition, Pharmacokinetics and Pharmacodynamics, as well as efficacy of STP1 are being explored.

Conditions

Autism Spectrum Disorder

Keywords

ASD; Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability

  Number of Participants with Adverse Events (nature and frequency of non-serious adverse events, serious adverse events and adverse events of special interest).

  14 days

Study Arms (3)

STP1 Low Dose

EXPERIMENTAL

1 capsule and 1 tablet per intake

Drug: STP1

STP1 High Dose

EXPERIMENTAL

1 capsule and 1 tablet per intake

Drug: STP1

Placebo

PLACEBO COMPARATOR

1 placebo capsule and 1 placebo tablet per intake

Drug: Placebo

Interventions

STP1 DRUG

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

STP1 High Dose; STP1 Low Dose

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Stalicla SA: lead

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (1)

• Erickson CA, Perez-Cano L, Pedapati EV, Painbeni E, Bonfils G, Schmitt LM, Sachs H, Nelson M, De Stefano L, Westerkamp G, de Souza ALS, Pohl O, Laufer O, Issachar G, Blaettler T, Hyvelin JM, Durham LA. Safety, Tolerability, and EEG-Based Target Engagement of STP1 (PDE3,4 Inhibitor and NKCC1 Antagonist) in a Randomized Clinical Trial in a Subgroup of Patients with ASD. Biomedicines. 2024 Jun 27;12(7):1430. doi: 10.3390/biomedicines12071430.

  PMID: 39062003 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Eric Painbeni, PhD - Head of Clinical Operations
• Organization: STALICLA SA

clinical@stalicla.com

+41 (0)79 918 33 16

Study Officials

• Craig Erickson, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2020
• First Posted: November 25, 2020
• Study Start: December 7, 2020
• Primary Completion: January 28, 2022
• Study Completion: January 28, 2022
• Last Updated: November 8, 2024
• Results First Posted: November 8, 2024

Record last verified: 2024-08

Locations

US (1)