NCT03588013

Brief Summary

Environmental Enteropathy (EE) is an acquired sub-clinical inflammatory gut condition in which alterations in intestinal structure, function, and local and systemic immune activation lead to impaired vaccine responses, decreased cognitive potential and undernutrition in low-middle income countries. Approximately half of all global deaths in children aged less than five years are attributable to undernutrition making the study of EE an area of critical priority. However, given the operational limitations and ethical considerations for safely obtaining intestinal biopsies from young children in low resource settings, there have been few detailed investigations of human intestinal tissue in this vulnerable patient group for whom reversal of EE would provide the greatest benefit. EE biomarkers have been studied in different settings but these have not been correlated with the gold standard histopathology confirmation. The Study of Environment Enteropathy and Malnutrition in Pakistan (SEEM Pakistan) is designed to better understand the pathophysiology, predictors, biomarkers, and potential management strategies of EE to inform strategies to eradicate this debilitating pathology.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
416

participants targeted

Target at P75+ for not_applicable

Timeline
Completed

Started Mar 2016

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2016

Completed
2.3 years until next milestone

First Submitted

Initial submission to the registry

June 19, 2018

Completed
28 days until next milestone

First Posted

Study publicly available on registry

July 17, 2018

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 31, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 31, 2019

Completed
Last Updated

September 8, 2021

Status Verified

September 1, 2021

Enrollment Period

3.4 years

First QC Date

June 19, 2018

Last Update Submit

September 6, 2021

Conditions

Outcome Measures

Primary Outcomes (6)

  • Nutritional status in children assessed by measuring parameters of growth at 3-6 months of age

    Our birth cohort will be assessed for nutritional status by measuring parameters of growth, length, weight and height. These measurements will be calculated as Z scores according to WHO tables and children will be divided into the appropriate categories: Stunting as Length for Age Z-score \< -2 SD (standard deviation), underweight as Weight-for-Age Z-score \< -2 SD and wasting as Weight-for-Length Z-score \< -2 SD, or normal-for-age.

    Approximately 3-6 months after entry into study

  • Nutritional status in children assessed by measuring parameters of growth at 9 months of age

    Our birth cohort will be assessed for nutritional status by measuring parameters of growth, length, weight and height. These measurements will be calculated as Z scores according to WHO tables and children will be divided into the appropriate categories: Stunting as Length for Age Z-score \< -2 SD (standard deviation), underweight as Weight-for-Age Z-score \< -2 SD and wasting as Weight-for-Length Z-score \< -2 SD, or normal-for-age.

    At approximately 9 months after entry into study

  • Association of biomarkers with Environmental Enteropathy at 3-6 months of age

    Serum, stool, and urine biomarkers of EE will be collected and tested in malnourished and well-nourished children

    Approximately 3-6 months after entry into study

  • Association of biomarkers with Environmental Enteropathy at 9 months of age

    Serum, stool, and urine biomarkers of EE will be collected and tested in malnourished and well-nourished children

    At approximately 9 months after entry into study

  • Association of biomarkers with Environmental Enteropathy at the time of endoscopy and biopsy

    Serum, stool, and urine biomarkers of EE will be collected and tested in those children who are eligible to undergo upper GI endoscopy and biopsy

    At approximately 15 months after entry into study

  • The use of UGI biopsy tissue for histopathological and multiomic workup for validation of biomarkers associated with EE

    Biopsy specimens from UGI endoscopy obtained from eligible children will be used for detailed histopathologic and multiomic work up to study the pathophysiology of EE, validate current biomarkers, and generate novel biomarker candidates

    At approximately 15 months after entry into study

Study Arms (5)

Moderate/severe malnourishment

EXPERIMENTAL

Pakistani children from age 0 to 6 months with weight for height Z score (WHZ) \< -2 at the time of enrollment. Parents/caregivers of all participants will undergo a series of rehabilitative interventions to improve the child's nutrition and growth. Those participants who remain WHZ \< -2 despite interventions are eligible for medical evaluation for more advanced workup of malnutrition, including UGI endoscopy and biopsy.

Other: Nutritional educationDietary Supplement: Ready to Use Therapeutic FoodProcedure: Upper gastrointestinal endoscopy with biopsy

Well nourished children

ACTIVE COMPARATOR

Pakistani children from age 0 to 6 months who would be growing normally, with WHZ \> 0, to serve as controls. Parents/caregivers of all participants will undergo a series of rehabilitative interventions to improve the child's nutrition and growth.

Other: Nutritional education

US children with celiac disease

NO INTERVENTION

Comparative group for the Pakistani WHZ \<-2 children who undergo UGI endoscopy and biopsy. Environmental Enteropathy and celiac disease have some shared features therefore we plan to enroll children under the age of 6 years with newly diagnosed celiac disease per endoscopy at CCHMC to assess the extent to which gene signatures and associated biologic pathways for children with celiac disease or environmental enteropathy overlap or differ.

US children with Crohn's disease

NO INTERVENTION

Comparative group for the Pakistani WHZ \<-2 children who undergo UGI endoscopy and biopsy. As some differentially expressed ileal gene signatures for Crohn's disease bear remarkable similarities to individual gene expression patterns previously reported for EE, children under the age of 10 years with newly diagnosed Crohn's disease per endoscopy at CCHMC will be enrolled to study these similarities and any differences

Healthy age-matched US children

NO INTERVENTION

Comparative group for the Pakistani WHZ \<-2 children who undergo UGI endoscopy and biopsy. Number of upper gastrointestinal endoscopies performed in children less than 2 years old are limited in Pakistan, therefore US age-matched controls will be used; healthy children \< 3 years old will be enrolled, who will undergo endoscopy at CCHMC as part of a diagnostic workup for digestive symptoms, but whose biopsies and diagnoses are not supportive of eosinophilic esophagitis, celiac disease, or inflammatory bowel disease, and who were not treated with antibiotics ≤ 4 weeks prior to endoscopy.

Interventions

4 week home delivered educational program that will focus on breast feeding and complimentary feeding. If child remains WHZ \< -2 at by 9 months of age families will be shown 10 minute educational video detailing best practices for complimentary feeding.

Also known as: Education
Moderate/severe malnourishmentWell nourished children

If child remains WHZ \< -2 despite nutritional education then will be managed according to Pakistan's Community Management of Acute Malnutrition protocol. This will include provision of ready to use therapeutic food (RUTF) to the child at home with close follow up. Utilization of the RUTF will be monitored closely by bi-weekly home visits.

Also known as: RUTF
Moderate/severe malnourishment

If child remains at WHZ \< -2 despite educational and dietary interventions, then will undergo medical evaluation for assessment of need for more advanced workup of malnutrition, including UGI endoscopy at AKUH. Results of the advanced work up, including the UGI endoscopy, will guide future management of these children.

Also known as: UGI endoscopy, Biopsy, Esophagoduodenoscopy
Moderate/severe malnourishment

Eligibility Criteria

Age0 Days - 10 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17)

You may qualify if:

  • Children aged 0-6 months from Matiari, Pakistan with weight for height Z score (WHZ) \< -2 at the time of enrollment
  • Children aged 0 to 6 months from Matiari, Pakistan who would be growing normally, with WHZ \> 0, to serve as controls
  • Healthy control children under the age of 3 years, who will undergo endoscopy at CCHMC
  • Children under the age of 6 years with newly diagnosed celiac disease per endoscopy at CCHMC
  • Children under the age of 10 years with newly diagnosed Crohn's disease per endoscopy at CCHMC

You may not qualify if:

  • For healthy control children \< 3 years old undergoing endoscopy at CCHMC, biopsies and diagnoses should not supportive of eosinophilic esophagitis
  • For healthy control children \< 3 years old undergoing endoscopy at CCHMC, biopsies and diagnoses should not supportive of celiac disease
  • For healthy control children \< 3 years old undergoing endoscopy at CCHMC, biopsies and diagnoses should not supportive of inflammatory bowel disease
  • For healthy control children \< 3 years old undergoing endoscopy at CCHMC, children should not have been treated with antibiotics ≤ 4 weeks prior to endoscopy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Aga Khan University

Karachi, Sindh, 74800, Pakistan

Location

Related Publications (4)

  • Ahmed S, Iqbal J, Sadiq K, Umrani F, Rizvi A, Kabir F, Jamil Z, Syed S, Ehsan L, Zulqarnain F, Sajid M, Hotwani A, Rahman N, Ma JZ, McNeal M, Ann Costa Clemens S, Talat Iqbal N, Moore SR, Ali A. Association of Anti-Rotavirus IgA Seroconversion with Growth, Environmental Enteric Dysfunction and Enteropathogens in Rural Pakistani Infants. Vaccine. 2022 May 31;40(25):3444-3451. doi: 10.1016/j.vaccine.2022.04.032. Epub 2022 May 6.

  • Zhao X, Setchell KDR, Huang R, Mallawaarachchi I, Ehsan L, Dobrzykowski Iii E, Zhao J, Syed S, Ma JZ, Iqbal NT, Iqbal J, Sadiq K, Ahmed S, Haberman Y, Denson LA, Ali SA, Moore SR. Bile Acid Profiling Reveals Distinct Signatures in Undernourished Children with Environmental Enteric Dysfunction. J Nutr. 2021 Dec 3;151(12):3689-3700. doi: 10.1093/jn/nxab321.

  • Haberman Y, Iqbal NT, Ghandikota S, Mallawaarachchi I, Tzipi Braun, Dexheimer PJ, Rahman N, Hadar R, Sadiq K, Ahmad Z, Idress R, Iqbal J, Ahmed S, Hotwani A, Umrani F, Ehsan L, Medlock G, Syed S, Moskaluk C, Ma JZ, Jegga AG, Moore SR, Ali SA, Denson LA. Mucosal Genomics Implicate Lymphocyte Activation and Lipid Metabolism in Refractory Environmental Enteric Dysfunction. Gastroenterology. 2021 May;160(6):2055-2071.e0. doi: 10.1053/j.gastro.2021.01.221. Epub 2021 Jan 29.

  • Iqbal NT, Syed S, Sadiq K, Khan MN, Iqbal J, Ma JZ, Umrani F, Ahmed S, Maier EA, Denson LA, Haberman Y, McNeal MM, Setchell KDR, Zhao X, Qureshi S, Shen L, Moskaluk CA, Liu TC, Yilmaz O, Brown DE, Barratt MJ, Kung VL, Gordon JI, Moore SR, Ali SA. Study of Environmental Enteropathy and Malnutrition (SEEM) in Pakistan: protocols for biopsy based biomarker discovery and validation. BMC Pediatr. 2019 Jul 22;19(1):247. doi: 10.1186/s12887-019-1564-x.

MeSH Terms

Conditions

Child Nutrition DisordersIntestinal DiseasesGrowth DisordersGastrointestinal Diseases

Interventions

Educational StatusEndoscopy, Digestive SystemBiopsy

Condition Hierarchy (Ancestors)

Nutrition DisordersNutritional and Metabolic DiseasesDigestive System DiseasesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Socioeconomic FactorsPopulation CharacteristicsDiagnostic Techniques, Digestive SystemDiagnostic Techniques and ProceduresDiagnosisEndoscopyDiagnostic Techniques, SurgicalDigestive System Surgical ProceduresSurgical Procedures, OperativeMinimally Invasive Surgical ProceduresCytodiagnosisCytological TechniquesClinical Laboratory TechniquesSpecimen HandlingInvestigative Techniques

Study Officials

  • Syed Asad Ali, MD, MPH

    Aga Khan University

    PRINCIPAL INVESTIGATOR
  • Sean R Moore, MD

    University of Virginia

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
PREVENTION
Intervention Model
PARALLEL
Model Details: 1. Moderately or severely malnourished (weight for height Z score (WHZ) \< -2) children 2. Well-nourished (WHZ \> 0) children 3. Control group of children in the US with celiac disease 4. Control group of children in the US with Crohn's disease 5. Control group of healthy age-matched children in the US
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Professor, Department of Pediatrics and Child Health

Study Record Dates

First Submitted

June 19, 2018

First Posted

July 17, 2018

Study Start

March 1, 2016

Primary Completion

July 31, 2019

Study Completion

July 31, 2019

Last Updated

September 8, 2021

Record last verified: 2021-09

Locations