28-Day Daily-dose Crossover Study of the Safety and Tolerability of SB-121 (Lactobacillus Reuteri With Sephadex® and Maltose) in Subjects, Ages 15 to 45 Years, Diagnosed With Autistic Disorder

NCT04944901 | Completed Phase 1 Results FDA Drug

• 1 other identifier: SBI-SB121-20-01
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

SB-121 is being developed for use in the treatment of autistic disorder (AD). This study is a multiple-dose, randomized, double-blind, placebo-controlled, cross-over single-site Phase I study. The primary objective is to evaluate the safety and tolerability of multiple doses of SB-121 in subjects ages 15 to 45 years with AD. Additionally, multiple measures of AD, as well as mechanistic biomarkers, will be assessed in order to inform later stage trials.

Conditions

Autistic Disorder

Keywords

Lactobacillus reuteri; autistic disorder; microbiome

Outcome Measures

Primary Outcomes (3)

• Adverse Event of Special Interest (AESIs) and Adverse Events (AEs) Leading to Discontinuation

  Adverse event of special interest (AESIs) and adverse events (AEs) leading to discontinuation from the study are presented. Treatment Period 1: 2 participants reported 4 events in the SB-121 group and 3 participant reported 6 events in the placebo group. Treatment Period 2: 1 participant reported 3 events in the SB-121 group and 1 participant reported 4 events in the placebo group.

  Approximately 98 days

• Sephadex Microspheres in the Stool

  The presence of Sephadex microspheres in the stool was assessed. The number of participants with data available at each stage are presented.

  Period 1: Days 1 (pre-dose), 28 and 35; Period 2: Days 28 and 42 (period = 28 days and 14 days wash-out)

• Symptomatic Bacteremia With Positive L. Reuteri Identification

  The presence of symptomatic bacteremia with positive L. reuteri identification was assessed and none of the participants in either group showed any clinical features of suspected bacteremia in this study.

  Approximately 98 days

Secondary Outcomes (6)

• Mean Percent Change From Baseline in Biomarkers: Tumor Necrosis Factor-α

  Day -28 to Day 0, Period 1: Day 28; Period 2: Days 1 (pre-dose) and 28 (period = 28 days and 14 days wash-out)

• Mean Percent Change From Baseline in Biomarkers: Serum High-sensitivity C-reactive Protein (Hs-CRP)

  Day -28 to Day 0, Period 1: Day 28; Period 2: Days 1 (pre-dose) and 28 (period = 28 days and 14 days wash-out)

• Mean Percent Change From Baseline in Biomarkers: Stool Biomarkers, Fecal Calprotectin

  Period 1: Days 1 (pre-dose), 28 and 35; Period 2: Days 28 and 35 (period = 28 days and 14 days wash-out)

• Mean Percent Change From Baseline in Biomarkers: Stool Biomarkers, Fecal Lactoferrin

  Period 1: Days 1 (pre-dose), 28 and 35; Period 2: Days 28 and 35 (period = 28 days and 14 days wash-out)

• Mean Percent Change From Baseline in Biomarkers: Plasma Oxytocin

  Day -28 to Day 0, Period 1: Day 28; Period 2: Days 1 and 28 (period = 28 days and 14 days wash-out)

Study Arms (2)

SB-121

EXPERIMENTAL

One dose of SB-121 daily for 28 days according to the treatment group to which they are allocated. Administration: Oral

Drug: SB-121

Placebo

PLACEBO COMPARATOR

One dose of placebo daily for 28 days according to the treatment group to which they are allocated. Administration: Oral

Drug: Placebo

Interventions

SB-121 DRUG

SB-121 is a formulation of L. reuteri

SB-121

Placebo DRUG

Placebo oral formulation

Placebo

Eligibility Criteria

• Age: 15 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subject/parent (or authorized designee) has provided written informed consent for the study.
• Subject is ≥15 and ≤45 years of age at the time of enrollment.
• Diagnosis of autistic disorder (AD) as confirmed by the gold standard clinical interview using Diagnostic and Statistical Manual of Mental Disorders (DSM)-5 criteria and administration of the Autism Diagnostic Observation Schedule-2.
• Subject, if female and of childbearing potential, is not lactating or pregnant.
• Subject, if female, is either not of childbearing potential or is practicing an acceptable effective method of birth control.
• Subject is willing to comply with all study requirements (including the requirements for stool sampling and biobanking) and to return to the study facility for the follow-up evaluations, as required.

You may not qualify if:

• Subject has known allergy or significant adverse reaction to L reuteri, Sephadex®, maltose, or related compounds.
• Subject has previously had GI surgery, intestinal obstruction, Clostridium difficile infection or diverticulitis.
• Subject has travelled outside of the USA in the 30 days prior to screening.
• Subject has had a diarrheal illness in 30 days prior to screening.
• Subject currently has a fever or active/uncontrolled gastrointestinal (GI) symptoms (e.g., nausea, vomiting, diarrhea, constipation, abdominal distention, abdominal pain/cramps, flatulence) or has had these within 14 days prior to screening. If the GI symptoms are stable, in the opinion of the investigator, the subject can be enrolled.
• Subject has any immunological/autoimmune disorder including, but not limited to, systemic lupus erythematosis, rheumatoid arthritis, Sjögren's syndrome, inflammatory bowel disease, or immunoglobulin-deficiency disorder, that would increase the risk to the subject or interfere with the evaluation of SB-121.
• Subject has a documented history of human immunodeficiency virus (HIV), hepatitis B and/or hepatitis C
• Subject has implanted prosthetic devices including prosthetic heart valves.
• Subject has taken, or is taking, any of the following prohibited medications:
• A proton pump inhibitor within 2 weeks prior to screening
• Use of supplemental probiotics within 2 weeks prior to screening except for yogurt
• Current use of immunosuppressive medications, including corticosteroids
• Treatment with monoclonal antibodies within 4 weeks prior to screening
• Systemic antibiotics within 2 weeks prior to screening
• Subject has diabetes mellitus or is prediabetic.

Sponsors & Collaborators

• Scioto Biosciences, Inc.: lead

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (1)

• Schmitt LM, Smith EG, Pedapati EV, Horn PS, Will M, Lamy M, Barber L, Trebley J, Meyer K, Heiman M, West KHJ, Hughes P, Ahuja S, Erickson CA. Results of a phase Ib study of SB-121, an investigational probiotic formulation, a randomized controlled trial in participants with autism spectrum disorder. Sci Rep. 2023 Mar 30;13(1):5192. doi: 10.1038/s41598-023-30909-0.

  PMID: 36997569 DERIVED

MeSH Terms

Conditions

Autistic Disorder

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Phoevos Hughes, VP Clinical Operations
• Organization: Scioto Biosciences

phoevos@sciotobiosciences.com

866-672-4686

Study Officials

• Craig Erickson, MD

  University of Cincinnati

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2021
• First Posted: June 30, 2021
• Study Start: August 2, 2021
• Primary Completion: March 3, 2022
• Study Completion: March 3, 2022
• Last Updated: March 25, 2024
• Results First Posted: March 25, 2024

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)