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| Name | Class |
|---|---|
| Patient-Centered Outcomes Research Institute | OTHER |
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The purpose of this interventional study is to test and compare the effectiveness of two elimination diets-the 1-food elimination diet (1FED, milk only) and the 4-food elimination diet (4FED, milk, egg, wheat, and soy) for eosinophilic esophagitis (EoE). The study will also test the effectiveness of swallowed glucocorticoid therapy in some of the study participants for whom diet therapy was not effective.
This study will consist of two phases, plus a screening period. During the screening period, subject eligibility for the study will be determined. During Phase 1, qualified participants will be randomly assigned to one of two elimination diet therapies--the 1FED or the 4FED. Participants will remain on the assigned dietary therapy for 12 weeks. At the end of 12 weeks of therapy, esophageal biopsies from participant's standard of care (i.e. normal, routine care) endoscopy will be evaluated to determine disease status. Participants whose EoE is in remission (i.e. <15 eos/hpf) will be done with the study.
Participants whose EoE is still active (i.e. ≥15 eos/hpf) will continue into Phase 2 of the study. During Phase 2, participants who were on 1FED in Phase 1 will receive 4FED therapy for 12 weeks, and participants who were on 4FED during Phase 1 will receive swallowed glucocorticoid therapy for 12 weeks. At the end of 12 weeks of therapy, esophageal biopsies from participant's standard of care (i.e. normal, routine care) endoscopy will be evaluated to determine disease status.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1FED | Active Comparator | 1-food elimination diet: Participants eliminate milk from the diet in Phase 1 |
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| 4FED | Active Comparator | 4-food elimination diet: Participants eliminate milk, egg, wheat, soy from the diet in Phase 1 |
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| 1FED Non-Responders (4FED) | Other | Participants that fail to respond to 1FED in Phase 1 eliminate milk, egg, wheat, soy from the diet in Phase 2 |
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| 4FED Non-Responders (SGC) | Other | Participants that fail to respond to 4FED in Phase 1 administer swallowed glucocorticosteroids (Flovent HFA) 800 mcg twice daily in Phase 2 |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 1 Food Elimination Diet | Other | Participants eliminate milk from the diet for 12 weeks |
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| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Pediatric EoE Symptom Score Version 2.0 (PEESS V2.0) at 12 Weeks | The PEESS V2.0 questionnaire captures EoE-specific symptoms (dysphagia, gastro-esophageal reflux disease (GERD), nausea/vomiting, and pain) as reported by children with EoE (8-18 years of age) and their parents (for children 2-18 years of age). The range for PEESS v2.0 scores is 0 to 100, with a higher score being indicative of more frequent and/or severe symptoms. Scores were obtained at baseline and 12 weeks. Change in score is defined as total score at 12 weeks minus total score at baseline. The parent-proxy PEESS total score change from pre-treatment to post-treatment is the primary efficacy endpoint. 1FED vs 4FED changes are compared. A reduction in score (negative change) is indicative of a reduction in symptoms. | Baseline and 12 weeks |
| Within-group Comparisons (Baseline v. Week 12) of PEESS V2.0 Scores | The PEESS V2.0 questionnaire captures EoE-specific symptoms. The range for PEESS v2.0 scores is 0 to 100, with a higher score being indicative of more frequent and/or severe symptoms. Baseline vs Week 12 scores are compared within each treatment group (1FED and 4FED). | Baseline and 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Percent of Participants in Histologic Remission (<15 Eosinophils Per High Power Field) at 12 Weeks | Percent of participants in remission in 1FED and 4FED groups. Remission is defined as clinical esophageal peak eosinophil count < 15 eosinophils per high power field (eos/hpf). Complete remission is defined as ≤ 1 peak eos/hpf and partial remission as 2 - 14 peak eos/hpf. | 12 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Marc E Rothenberg, MD, PhD | Children's Hospital Medical Center, Cincinnati | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
The raw anonymized data tables, data dictionaries, and study documentation will be provided to outside investigators upon request, with the proviso that it may not be linked with external datasets so as to minimize the risk of re-identification. When the resources and the associated research findings have been published, data will be made available for research purposes to qualified individuals within the scientific community. Consortium participants will retain rights to any subject inventions generated.
After the associated research findings have been published, data will be made available for research purposes
Data will be provided in a form suitable for a variety of databases. Data will be provided to qualified individuals within the scientific community, according to federal guidelines.
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67 participants screened, 4 excluded (2 did not meet inclusion criteria, 2 met exclusion criteria), and 63 randomized
Participants were recruited from eosinophilic esophagitis (EoE) specialty clinics (10 sites) in the USA between November 2015 and February 2018.
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| ID | Title | Description |
|---|---|---|
| FG000 | 1FED | 1-food elimination diet: participants eliminate milk from the diet for 12 weeks |
| FG001 | 4FED | 4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Phase 1 |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 28, 2016 | Jun 5, 2018 |
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| 4 Food Elimination Diet | Other | Participants eliminate milk, egg, wheat, soy from the diet for 12 weeks |
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| 4 Food Elimination Diet (post 1FED failure) | Other | Participants that fail to respond to 1FED in phase 1 eliminate milk, egg, wheat, soy from the diet for 12 weeks in Phase 2 |
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| Fluticasone Propionate, 800 mcg twice daily (post 4FED failure) | Drug | Participants that fail to respond to 4FED in phase 1 administer swallowed glucocorticosteroids (Flovent HFA) 800 mcg twice daily for 12 weeks in Phase 2 |
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| Percent of Participants on Swallowed Glucocorticoids (SGC) in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2 | Percent of 4FED non-responders on SGC in Phase 2 in histologic remission. Remission is defined as esophageal peak eosinophil count < 15 eosinophils per high power field (eos/hpf). Complete remission is defined as ≤ 1 peak eos/hpf and partial remission as 2 - 14 peak eos/hpf. | 12 weeks |
| Percent of 1FED Non-responders on 4FED in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2 | Percent of 1FED non-responders on 4FED in histologic remission in phase 2. Remission is defined as esophageal peak eosinophil count < 15 eosinophils per high powered field. Complete remission is defined as ≤ 1 peak eos/hpf and partial remission as 2 - 14 peak eos/hpf. | 12 weeks |
| Change From Baseline in Pediatric Quality of Life Inventory Version 3.0 EoE Module (PedsQL 3.0 EoE) at 12 Weeks | The PedsQL 3.0 EoE measures symptoms and problems related to treatment, worry, communication, food/eating, and feelings. The range for PedsQL 3.0 EoE scores is 0 to 100, with a higher score indicating better quality of life. Scores were obtained at baseline and 12 weeks. Change in score is defined as the PedsQL 3.0 EoE total score at 12 weeks minus total score at baseline. 1FED vs 4FED changes are compared. An increase in score (positive change) is indicative of improved quality of life. | Baseline and 12 weeks |
| Change From Baseline in Pediatric Quality of Life Inventory Version 4.0 (PedsQL 4.0) Generic Core Scales at 12 Weeks | The PedsQL 4.0 measures physical and psychosocial function. The range for PedsQL 4.0 scores is 0 to 100, with a higher score indicating better quality of life. Scores were obtained at baseline and 12 weeks. Change in score is defined as the PedsQL 4.0 total score at 12 weeks minus total score at baseline. 1FED vs 4FED changes are compared. An increase in score (positive change) is indicative of improved quality of life. | Baseline and 12 weeks |
| Change From Baseline in Endoscopic Reference Score at 12 Weeks | The EoE Endoscopic Reference Score (EREFS) measures features of EoE including esophageal edema, rings, exudate, furrows, and strictures. The instrument grades edema and furrows as absent (0) or present (1); rings as absent (0), mild (1, subtle circumferential ridges), moderate (2, distinct rings) and severe (3, rings that impair passage of a standard adult diagnostic endoscope); exudates as absent (0), mild (1, less than 10% of the esophageal surface area) or severe (2, greater or equal to 10% of the esophageal surface area); and strictures as absent (0) or present (1) with an estimation of the minimal luminal diameter. Higher scores indicate more severe disease (range 0 - 9). Scores were obtained at baseline and 12 weeks. Change in score is defined as the EREFS total score at 12 weeks minus total score at baseline. 1FED vs 4FED changes are compared. A reduction in score (negative change) is indicative of a reduction in esophageal abnormalities. | Baseline and 12 weeks |
| Percent of Participants With Positive and Negative Milk Skin Prick Tests Responding to 1FED | Reactions to skin prick test (SPT) to milk is positive if the wheal size of the milk test is at least 3 mm larger than the wheal size of the negative control. Treatment response is defined as clinical histologic remission (<15 eos/hpf). | Baseline and 12 weeks |
| FG002 | 1FED Non-responders (4FED) | Participants that fail to respond to 1FED in phase 1 eliminate milk, egg, wheat, soy from the diet for 12 weeks in phase 2 |
| FG003 | 4FED Non-responders (SGC) | Participants that fail to respond to 4FED in phase 1 administer swallowed glucocorticosteroids (Flovent HFA) 800 mcg twice daily for 12 weeks in phase 2 |
| COMPLETED |
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| NOT COMPLETED |
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| Phase 2 |
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| ID | Title | Description |
|---|---|---|
| BG000 | 1FED | 1-food elimination diet: participants eliminate milk from the diet for 12 weeks |
| BG001 | 4FED | 4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Atopy | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in Pediatric EoE Symptom Score Version 2.0 (PEESS V2.0) at 12 Weeks | The PEESS V2.0 questionnaire captures EoE-specific symptoms (dysphagia, gastro-esophageal reflux disease (GERD), nausea/vomiting, and pain) as reported by children with EoE (8-18 years of age) and their parents (for children 2-18 years of age). The range for PEESS v2.0 scores is 0 to 100, with a higher score being indicative of more frequent and/or severe symptoms. Scores were obtained at baseline and 12 weeks. Change in score is defined as total score at 12 weeks minus total score at baseline. The parent-proxy PEESS total score change from pre-treatment to post-treatment is the primary efficacy endpoint. 1FED vs 4FED changes are compared. A reduction in score (negative change) is indicative of a reduction in symptoms. | We performed intent to treat analysis including participants who had at least one clinical observation post randomization. | Posted | Mean | Standard Deviation | units on a scale | Baseline and 12 weeks |
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| Primary | Within-group Comparisons (Baseline v. Week 12) of PEESS V2.0 Scores | The PEESS V2.0 questionnaire captures EoE-specific symptoms. The range for PEESS v2.0 scores is 0 to 100, with a higher score being indicative of more frequent and/or severe symptoms. Baseline vs Week 12 scores are compared within each treatment group (1FED and 4FED). | We performed intent to treat analysis including participants who had at least one clinical observation post randomization | Posted | Mean | Standard Deviation | units on a scale | Baseline and 12 weeks |
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| Secondary | Percent of Participants in Histologic Remission (<15 Eosinophils Per High Power Field) at 12 Weeks | Percent of participants in remission in 1FED and 4FED groups. Remission is defined as clinical esophageal peak eosinophil count < 15 eosinophils per high power field (eos/hpf). Complete remission is defined as ≤ 1 peak eos/hpf and partial remission as 2 - 14 peak eos/hpf. | We performed intent to treat analysis including participants who had at least one clinical observation post randomization. | Posted | Number | percentage of participants | 12 weeks |
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| Secondary | Percent of Participants on Swallowed Glucocorticoids (SGC) in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2 | Percent of 4FED non-responders on SGC in Phase 2 in histologic remission. Remission is defined as esophageal peak eosinophil count < 15 eosinophils per high power field (eos/hpf). Complete remission is defined as ≤ 1 peak eos/hpf and partial remission as 2 - 14 peak eos/hpf. | We performed intent to treat analysis including participants who had at least one clinical observation post randomization | Posted | Number | percentage of participants | 12 weeks |
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| Secondary | Percent of 1FED Non-responders on 4FED in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2 | Percent of 1FED non-responders on 4FED in histologic remission in phase 2. Remission is defined as esophageal peak eosinophil count < 15 eosinophils per high powered field. Complete remission is defined as ≤ 1 peak eos/hpf and partial remission as 2 - 14 peak eos/hpf. | We performed Intent to treat analysis including participants who had at least one clinical observation post randomization. | Posted | Number | percentage of participants | 12 weeks |
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| Secondary | Change From Baseline in Pediatric Quality of Life Inventory Version 3.0 EoE Module (PedsQL 3.0 EoE) at 12 Weeks | The PedsQL 3.0 EoE measures symptoms and problems related to treatment, worry, communication, food/eating, and feelings. The range for PedsQL 3.0 EoE scores is 0 to 100, with a higher score indicating better quality of life. Scores were obtained at baseline and 12 weeks. Change in score is defined as the PedsQL 3.0 EoE total score at 12 weeks minus total score at baseline. 1FED vs 4FED changes are compared. An increase in score (positive change) is indicative of improved quality of life. | We performed intent to treat analysis including participants who had at least one clinical observation post randomization | Posted | Mean | Standard Deviation | units on a scale | Baseline and 12 weeks |
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| Secondary | Change From Baseline in Pediatric Quality of Life Inventory Version 4.0 (PedsQL 4.0) Generic Core Scales at 12 Weeks | The PedsQL 4.0 measures physical and psychosocial function. The range for PedsQL 4.0 scores is 0 to 100, with a higher score indicating better quality of life. Scores were obtained at baseline and 12 weeks. Change in score is defined as the PedsQL 4.0 total score at 12 weeks minus total score at baseline. 1FED vs 4FED changes are compared. An increase in score (positive change) is indicative of improved quality of life. | We performed intent to treat analysis including participants who had at least one clinical observation post randomization | Posted | Mean | Standard Deviation | units on a scale | Baseline and 12 weeks |
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| Secondary | Change From Baseline in Endoscopic Reference Score at 12 Weeks | The EoE Endoscopic Reference Score (EREFS) measures features of EoE including esophageal edema, rings, exudate, furrows, and strictures. The instrument grades edema and furrows as absent (0) or present (1); rings as absent (0), mild (1, subtle circumferential ridges), moderate (2, distinct rings) and severe (3, rings that impair passage of a standard adult diagnostic endoscope); exudates as absent (0), mild (1, less than 10% of the esophageal surface area) or severe (2, greater or equal to 10% of the esophageal surface area); and strictures as absent (0) or present (1) with an estimation of the minimal luminal diameter. Higher scores indicate more severe disease (range 0 - 9). Scores were obtained at baseline and 12 weeks. Change in score is defined as the EREFS total score at 12 weeks minus total score at baseline. 1FED vs 4FED changes are compared. A reduction in score (negative change) is indicative of a reduction in esophageal abnormalities. | We performed intent to treat analysis including participants who had at least one clinical observation post randomization | Posted | Mean | Standard Deviation | units on a scale | Baseline and 12 weeks |
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| Secondary | Percent of Participants With Positive and Negative Milk Skin Prick Tests Responding to 1FED | Reactions to skin prick test (SPT) to milk is positive if the wheal size of the milk test is at least 3 mm larger than the wheal size of the negative control. Treatment response is defined as clinical histologic remission (<15 eos/hpf). | We performed intent to treat analysis including participants who had at least one clinical observation post randomization | Posted | Number | percentage of participants | Baseline and 12 weeks |
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Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 1FED | 1-food elimination diet (Phase 1) | 0 | 38 | 1 | 38 | 5 | 38 |
| EG001 | 4FED | 4-food elimination diet (Phase 1) | 0 | 25 | 1 | 25 | 8 | 25 |
| EG002 | 1FED Non-responders (4FED) | 4-food elimination diet (Phase 2) | 0 | 19 | 0 | 19 | 6 | 19 |
| EG003 | 4FED Non-responders (SGC) | Swallowed glucocorticosteroids (Flovent HFA) (Phase 2) | 0 | 10 | 0 | 10 | 6 | 10 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal Pain | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ear Pain | Ear and labyrinth disorders | CTCAE (4.0) | Systematic Assessment |
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| Abdominal Pain | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Otitis Media | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
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| Sinusitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
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| Headache | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Sinus Disorder | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Marc E. Rothenberg, MD, PhD | Cincinnati Childrens Hospital Medical Center | 513-636-7177 | Marc.Rothenberg@cchmc.org |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 5, 2015 | Jun 5, 2018 | SAP_001.pdf |
| ID | Term |
|---|---|
| D057765 | Eosinophilic Esophagitis |
| ID | Term |
|---|---|
| D004941 | Esophagitis |
| D004935 | Esophageal Diseases |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
| D005759 | Gastroenteritis |
| D004802 | Eosinophilia |
| D007960 | Leukocyte Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D000068298 | Fluticasone |
| ID | Term |
|---|---|
| D000730 | Androstadienes |
| D000736 | Androstenes |
| D000731 | Androstanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
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| Lost to Follow-up |
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| Physician Decision |
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| Pregnancy |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Black or African American |
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| Other |
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| Not Atopic |
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