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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
QUADRUPLE
Enrollment
283
University of Arizona Health Sciences
Tucson, Arizona, United States
Arkansas Children's Research Institute
Little Rock, Arkansas, United States
UCLA Health
Los Angeles, California, United States
Rady Children's Hospital - San Diego
San Diego, California, United States
UCSF Benioff Children's Hospital
San Francisco, California, United States
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
Frequency, type, and severity of AEs and SAEs, including AEs of special interest (AESI) as in Appendix 9 (Adverse Events of Special Interest) and Appendix 10 (Adverse Event Grading Scale)
Time frame: Through 56 days of study
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.
Frequency, type and severity of AEs, SAEs, and AESIs as in Appendix 9 (Adverse Events of Special Interest) and Appendix 10 (Adverse Event Grading Scale), as well as clinically significant findings on physical examinations including growth (length, weight, height and head circumference) and vital signs (RR, HR, and temperature); clinical safety laboratories including complete blood count with manual differential and blood chemistry
Time frame: Through 672 days of study
Part B: Primary Efficacy Endpoint: Incidence of physician-diagnosed atopic dermatitis at 336 days
Incidence of physician-diagnosed atopic dermatitis at 336 days in STMC-103H-treated subjects compared to placebo
Time frame: Day 336
Part B Secondary Efficacy Endpoint - physician-diagnosed atopic dermatitis
Incidence of physician-diagnosed atopic dermatitis
Time frame: At days 168 and 672
Part B - Secondary Efficacy Endpoint - atopic disease assessments
Proportion of subjects who develop any atopic disease (atopic dermatitis, food allergy, allergic rhinitis/conjunctivitis, asthma)
Time frame: At days 168, 336 and 672
Part B Secondary Efficacy Endpoint - incidence of sensitization to food and aeroallergen
Incidence of sensitization to food and aeroallergen as measured by specific serum IgE levels
Time frame: At days 168, 336, and 672
Part B Secondary Efficacy Endpoint - incidence of food allergy, allergic rhinitis/conjunctivitis, urticaria, and wheezing illness/asthma
Incidence of physician-diagnosed food allergy, allergic rhinitis/conjunctivitis, urticaria and wheezing illnesses/asthma using physician assessment, Allergic Disease Assessment and Diagnosis questionnaire, and Allergic Disease Diagnostic Criteria \& Severity Evaluation
Time frame: At days 168, 336, and 672
Part B Secondary Efficacy Endpoint - Time to atopic dermatitis diagnosis
Time to atopic dermatitis diagnosis by physician assessment
Time frame: Through 672 days of study
Part B Secondary Efficacy Endpoint - Time to first wheezing episode
Time to first wheezing episode by physician assessment
Time frame: Through 672 days of study
Part B Secondary Efficacy Endpoint - severity of atopic dermatitis by Investigator Global Assessment x Body Surface Area (IGAxBSA) assessment
Severity of atopic dermatitis by IGAxBSA assessment
Time frame: At days 168, 336 and 672
Part B Secondary Efficacy Endpoint - severity of atopic dermatitis by Severity Scoring Of Atopic Dermatitis (SCORAD) assessment
Severity of atopic dermatitis by SCORAD assessment
Time frame: At days 168, 336 and 672
Part B Secondary Efficacy Endpoint - Severity of Wheezing Illness/Asthma
Severity of wheezing illness/asthma by Wheezing Severity Assessment
Time frame: At days 68, 336, and 672 days
Part B Secondary Efficacy Endpoint - use of concomitant medications for allergic symptoms or diagnosis
Concomitant medications prescribed/used for allergic symptoms or diagnosis and use of rescue medications for atopic dermatitis and wheezing/asthma
Time frame: Through 672 days of study
Part B Secondary Efficacy Endpoint - Total Serum IgE
Total serum IgE levels
Time frame: At days 168, 336, and 672
Part B Secondary Efficacy Endpoint - Peripheral Eosinophil Counts
Peripheral eosinophil counts by automated differential
Time frame: At day 336
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