Clinical trial NCT05003804: Allergic Disease Onset Prevention Study
Official title: A Phase 1b/2, Randomized, Double-blind, Placebo-controlled, Multi-center Study of STMC-103H in Neonates and Infants at Risk for Developing Allergic Disease
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Atopic Dermatitis
Type 1 Hypersensitivity
Lead sponsor: Siolta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2021-08-12
Enrollment: 264
Sex: ALL
Minimum age (years): 0
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Participants will be enrolled in a three-part sequential approach (Parts A1, A2, and B). Part A will enroll at risk participants of 1 year to less than 6 years of age; after safety review, Part A2 will enroll at risk participants of 1 month to less than 12 months of age; and after safety review, Part B will enroll at -risk participants between 0 and 14 days of life.
Primary purpose: PREVENTION

Brief summary: This is a Phase 1b/2, randomized, double-blind, multi-center study to evaluate the safety, tolerability, and preliminary clinical efficacy of STMC-103H in neonates and infants at risk for developing allergic disease (Type 1 hypersensitivity). Subjects will be enrolled in a three-part sequential approach. Participants in the safety-run portion of the study (Part A1: 1 year to \<6 years of age and A2: 1 month to \<12 months of age) will receive 28 days of treatment with STMC-103H or placebo, followed by 28 days of follow-up. A Data and Safety Monitoring Committee (DSMC) will review safety data after all patients in each part complete 28 days of therapy prior to enrolling the next part. After A2, Part B will enroll 224 patients for 336 days of treatment with STMC-103H or placebo, followed by 336 days of follow-up. Stool, blood, and optional samples will be collected in Parts A2 and part B. Primary safety endpoints are frequency, type and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs), as well as findings on physical exams, vitals, and safety laboratories. The primary efficacy endpoint is incidence of physician-diagnosed atopic dermatitis at day 336.


Eligibility criteria: Inclusion Criteria:

* All Parts (A1, A2, B)

  1. Subject's parent(s)/legal representative(s) providing consent must be 18 years or older
  2. Biological mother and/or biological father and/or full sibling(s), have a history of asthma, atopic dermatitis, food allergy, or allergic rhinitis as determined by the screening questionnaire
  3. Subject's parent(s)/legal representative(s) (if appropriate according to local laws) is/are willing and able to give informed consent for participation in the study
  4. Subject's parent(s)/legal representative(s) (if appropriate according to local laws) is/are willing and able, in the PI's opinion, to comply with all study requirements

Part A1 Only

Inclusion criteria 1-4 for all parts plus:

5 (A1). Subject is between 1 year and \< 6 years old at the time of enrollment

Part A2 Only

Inclusion criteria 1-4 for all parts plus:

5 (A2). Subject is between 28 days and \< 12 months of life at the time of enrollment 6 (A2). Subject's parent(s)/legal representative(s) do not plan to give probiotics (including infant formula that contain probiotics) to the subject during the trial

Part B Only

Inclusion criteria 1-4 for all parts plus:

5 (B). Subject is ≤ 14 days of life at the time of enrollment. Sites should make every effort to enroll newborns as soon as possible after birth.

6 (B). Subject has a birthweight ≥ 2.5 kg and ≤ 4.5 kg 7 (B). Subject's parent(s)/legal representative(s) do not plan to give probiotics (including infant formula that contain probiotics) to the subject from the time of birth to the end of the trial.

Exclusion Criteria:

* All Parts (A1, A2, B)

  1. Subject's twin (or higher order multiple) is enrolled in STMC-103H-102
  2. Subject has any congenital abnormalities or condition, significant disease, illness, physical exam finding, or disorder that, in the opinion of the PI, may put the subject at safety risk or is likely to hinder feeding or affect metabolism that may influence the results of the study. (Neonatal hyperbilirubinemia (jaundice), including jaundice that requires phototherapy, should not be considered exclusionary).
  3. Subject is acutely ill or on systemic antibiotics at the time of enrollment
  4. Subject is participating in another interventional clinical study involving investigational medication, formula, probiotic, or prebiotic use within 30 days (or five half-lives, whichever is longer) of this study
  5. Subject has evidence of immune deficiency/immune compromise in the judgment of the investigator

Part B Only

Exclusion Criteria 1-5 for all parts plus:

6 (B). Subject was born at \< 35 weeks' gestation 7 (B). Biological maternal medical condition during the pregnancy that, in the opinion of the PI, may put the subject at risk because of participation in the study. (Maternal antibiotics during the time of delivery should not be considered exclusionary.)

Primary outcomes:
measure: Part A1 and A2: Assess safety and tolerability of STMC-103H in children and infants at risk for development of allergic disease by assessing adverse events (AE), serious adverse events (SAE), and AEs of special interest
timeFrame: Through 56 days of study
measure: Part B: Assess the safety, tolerability of STMC-103H in neonate and infants subjects at risk for development of atopic disease by monitoring AEs, SAEs, AESI, physical exam findings, and clinical safety laboratories.
timeFrame: Through 672 days of study
measure: Part B: Primary Efficacy Endpoint: Incidence of physician-diagnosed atopic dermatitis at 336 days
timeFrame: Day 336
Secondary outcomes:
measure: Part B Secondary Efficacy Endpoint - physician-diagnosed atopic dermatitis
timeFrame: At days 168 and 672
measure: Part B - Secondary Efficacy Endpoint - atopic disease assessments
timeFrame: At days 168, 336 and 672
measure: Part B Secondary Efficacy Endpoint - incidence of sensitization to food and aeroallergen
timeFrame: At days 168, 336, and 672
measure: Part B Secondary Efficacy Endpoint - incidence of food allergy, allergic rhinitis/conjunctivitis, urticaria, and wheezing illness/asthma
timeFrame: At days 168, 336, and 672
measure: Part B Secondary Efficacy Endpoint - Time to atopic dermatitis diagnosis
timeFrame: Through 672 days of study
measure: Part B Secondary Efficacy Endpoint - Time to first wheezing episode
timeFrame: Through 672 days of study
measure: Part B Secondary Efficacy Endpoint - severity of atopic dermatitis by Investigator Global Assessment x Body Surface Area (IGAxBSA) assessment
timeFrame: At days 168, 336 and 672
measure: Part B Secondary Efficacy Endpoint - severity of atopic dermatitis by Severity Scoring Of Atopic Dermatitis (SCORAD) assessment
timeFrame: At days 168, 336 and 672
measure: Part B Secondary Efficacy Endpoint - Severity of Wheezing Illness/Asthma
timeFrame: At days 68, 336, and 672 days
measure: Part B Secondary Efficacy Endpoint - use of concomitant medications for allergic symptoms or diagnosis
timeFrame: Through 672 days of study
measure: Part B Secondary Efficacy Endpoint - Total Serum IgE
timeFrame: At days 168, 336, and 672
measure: Part B Secondary Efficacy Endpoint - Peripheral Eosinophil Counts
timeFrame: At day 336
Other outcomes:
measure: Part B Key Exploratory Endpoint - Mean fecal concentration of 12,13-diHOME
timeFrame: At day 336
Locations:
city: Tucson
country: United States
facility: University of Arizona Health Sciences
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Research Institute
state: Arkansas
city: Los Angeles
country: United States
facility: UCLA Health
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital - San Diego
state: California
city: San Francisco
country: United States
facility: UCSF Benioff Children's Hospital
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Lurie Children's Hospital
state: Illinois
city: Chicago
country: United States
facility: University of Chicago Medicine
state: Illinois
city: Indianapolis
country: United States
facility: Riley Children's Health at University of Indiana
state: Indiana
city: Baltimore
country: United States
facility: Johns Hopkins University School of Medicine
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Health
state: Michigan
city: Great Neck
country: United States
facility: Northwell Healthcare
state: New York
city: New York
country: United States
facility: NYU Langone Fink Children's
state: New York
city: New York
country: United States
facility: Mt. Sinai Jaffe Allergy Institute
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Greenville
country: United States
facility: Tribe Clinical Research
state: South Carolina
city: Summerville
country: United States
facility: Coastal Pediatrics Research
state: South Carolina
city: Austin
country: United States
facility: Dell Medical School at UT Austin
state: Texas
city: Dallas
country: United States
facility: UT Southwestern/Children's Health
state: Texas
city: Seattle
country: United States
facility: Seattle Allergy and Asthma Research Institute
state: Washington
city: Madison
country: United States
facility: Univ. of Wisconsin-Madison/Jackson Research Group
state: Wisconsin
city: Westmead
country: Australia
facility: The Children's Hospital at Westmead
state: New South Wales
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
state: Queensland
city: Adelaide
country: Australia
facility: The Women's and Children's Hospital
state: South Australia
city: Clayton
country: Australia
facility: Monash Children's Hospital
state: Victoria
city: Parkville
country: Australia
facility: Murdoch Children's Research Institute
state: Victoria
city: Murdoch
country: Australia
facility: Fiona Stanley Hospital
state: Western Australia
city: Caguas
country: Puerto Rico
facility: Centro de Neumologia Pediatrica
Study officials:
References: