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
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 283
- 试验地点
- 31
- 主要终点
- 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
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Double-blind, placebo-controlled
入排标准
- 年龄范围
- 0 Days 至 14 Days（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •All Parts (A1, A2, B)
- •Subject's parent(s)/legal representative(s) providing consent must be 18 years or older
- •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
- •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
- •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.
排除标准
- •All Parts (A1, A2, B)
- •Subject's twin (or higher order multiple) is enrolled in STMC-103H-102
- •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).
- •Subject is acutely ill or on systemic antibiotics at the time of enrollment
- •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
- •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.)
研究组 & 干预措施
STMC-103H Part A1
Once daily dosing with one capsule of STMC-103H mixed with milk, formula, or a milk product for 28 days
干预措施: STMC-103H (Biological)
Placebo Part A1
Once daily dosing with one capsule of placebo mixed with milk, formula, or a milk product for 28 days
干预措施: Placebo (Biological)
STMC-102H Part A2
Once daily dosing with one capsule of STMC-103H mixed with milk, formula, or a milk product for 28 days
干预措施: STMC-103H (Biological)
Placebo Part A2
Once daily dosing with one capsule of placebo mixed with milk, formula or a milk product for 28 days
干预措施: Placebo (Biological)
STMC-103H Part B
Once daily dosing with one capsule of STMC-103H mixed with breastmilk, formula or a milk product for 336 days
干预措施: STMC-103H (Biological)
Placebo Part B
Once daily dosing with one capsule of placebo mixed with breastmilk, formula or a milk product for 336 days
干预措施: Placebo (Biological)
结局指标
主要结局
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
时间窗: Through 56 days of study
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)
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.
时间窗: Through 672 days of study
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
Part B: Primary Efficacy Endpoint: Incidence of physician-diagnosed atopic dermatitis at 336 days
时间窗: Day 336
Incidence of physician-diagnosed atopic dermatitis at 336 days in STMC-103H-treated subjects compared to placebo
次要结局
- Part B Secondary Efficacy Endpoint - Time to first wheezing episode(Through 672 days of study)
- Part B Secondary Efficacy Endpoint - physician-diagnosed atopic dermatitis(At days 168 and 672)
- Part B Secondary Efficacy Endpoint - incidence of food allergy, allergic rhinitis/conjunctivitis, urticaria, and wheezing illness/asthma(At days 168, 336, and 672)
- Part B Secondary Efficacy Endpoint - severity of atopic dermatitis by Severity Scoring Of Atopic Dermatitis (SCORAD) assessment(At days 168, 336 and 672)
- Part B Secondary Efficacy Endpoint - incidence of sensitization to food and aeroallergen(At days 168, 336, and 672)
- Part B Secondary Efficacy Endpoint - Time to atopic dermatitis diagnosis(Through 672 days of study)
- Part B Secondary Efficacy Endpoint - severity of atopic dermatitis by Investigator Global Assessment x Body Surface Area (IGAxBSA) assessment(At days 168, 336 and 672)
- Part B Secondary Efficacy Endpoint - Severity of Wheezing Illness/Asthma(At days 68, 336, and 672 days)
- Part B Secondary Efficacy Endpoint - Total Serum IgE(At days 168, 336, and 672)
- Part B - Secondary Efficacy Endpoint - atopic disease assessments(At days 168, 336 and 672)
- Part B Secondary Efficacy Endpoint - use of concomitant medications for allergic symptoms or diagnosis(Through 672 days of study)
- Part B Secondary Efficacy Endpoint - Peripheral Eosinophil Counts(At day 336)