Trial Outcomes & Findings for Eosinophilic Esophagitis Intervention Trial-1 Food vs. 4 Food Elimination Diet Followed by Swallowed Glucocorticoids (NCT NCT02610816)

NCT ID: NCT02610816

Last Updated: 2018-08-27

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

67 participants

Primary outcome timeframe

Baseline and 12 weeks

Results posted on

2018-08-27

Participant Flow

Participants were recruited from eosinophilic esophagitis (EoE) specialty clinics (10 sites) in the USA between November 2015 and February 2018.

67 participants screened, 4 excluded (2 did not meet inclusion criteria, 2 met exclusion criteria), and 63 randomized

Participant milestones

Participant milestones
Measure
1FED
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
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
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
Phase 1
STARTED
38
25
0
0
Phase 1
COMPLETED
34
17
0
0
Phase 1
NOT COMPLETED
4
8
0
0
Phase 2
STARTED
0
0
19
10
Phase 2
COMPLETED
0
0
8
4
Phase 2
NOT COMPLETED
0
0
11
6

Reasons for withdrawal

Reasons for withdrawal
Measure
1FED
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
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
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
Phase 1
Withdrawal by Subject
2
6
0
0
Phase 1
Lost to Follow-up
2
1
0
0
Phase 1
Physician Decision
0
1
0
0
Phase 2
Withdrawal by Subject
0
0
9
3
Phase 2
Lost to Follow-up
0
0
2
0
Phase 2
Physician Decision
0
0
0
2
Phase 2
Pregnancy
0
0
0
1

Baseline Characteristics

Eosinophilic Esophagitis Intervention Trial-1 Food vs. 4 Food Elimination Diet Followed by Swallowed Glucocorticoids

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
1FED
n=38 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
n=25 Participants
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Total
n=63 Participants
Total of all reporting groups
Age, Continuous
12.3 Years
STANDARD_DEVIATION 3.1 • n=5 Participants
12.1 Years
STANDARD_DEVIATION 3.7 • n=7 Participants
12.2 Years
STANDARD_DEVIATION 3.3 • n=5 Participants
Sex: Female, Male
Female
12 Participants
n=5 Participants
9 Participants
n=7 Participants
21 Participants
n=5 Participants
Sex: Female, Male
Male
26 Participants
n=5 Participants
16 Participants
n=7 Participants
42 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
4 Participants
n=5 Participants
3 Participants
n=7 Participants
7 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
33 Participants
n=5 Participants
21 Participants
n=7 Participants
54 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
1 Participants
n=7 Participants
2 Participants
n=5 Participants
Race/Ethnicity, Customized
White
33 Participants
n=5 Participants
22 Participants
n=7 Participants
55 Participants
n=5 Participants
Race/Ethnicity, Customized
Black or African American
4 Participants
n=5 Participants
1 Participants
n=7 Participants
5 Participants
n=5 Participants
Race/Ethnicity, Customized
Other
1 Participants
n=5 Participants
2 Participants
n=7 Participants
3 Participants
n=5 Participants
Atopy
Atopic
34 Participants
n=5 Participants
19 Participants
n=7 Participants
53 Participants
n=5 Participants
Atopy
Not Atopic
4 Participants
n=5 Participants
6 Participants
n=7 Participants
10 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 12 weeks

Population: We performed intent to treat analysis including participants who had at least one clinical observation post randomization.

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.

Outcome measures

Outcome measures
Measure
1FED
n=33 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
n=16 Participants
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Change From Baseline in Pediatric EoE Symptom Score Version 2.0 (PEESS V2.0) at 12 Weeks
-14.3 units on a scale
Standard Deviation 17.1
-21.6 units on a scale
Standard Deviation 17.3

PRIMARY outcome

Timeframe: Baseline and 12 weeks

Population: We performed intent to treat analysis including participants who had at least one clinical observation post randomization

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).

Outcome measures

Outcome measures
Measure
1FED
n=38 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
n=24 Participants
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Within-group Comparisons (Baseline v. Week 12) of PEESS V2.0 Scores
Week 12 - Total Score
23.5 units on a scale
Standard Deviation 18.3
16.0 units on a scale
Standard Deviation 13.0
Within-group Comparisons (Baseline v. Week 12) of PEESS V2.0 Scores
Baseline - Total Score
38.1 units on a scale
Standard Deviation 14.2
42.9 units on a scale
Standard Deviation 16.0

SECONDARY outcome

Timeframe: 12 weeks

Population: We performed intent to treat analysis including participants who had at least one clinical observation post randomization.

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.

Outcome measures

Outcome measures
Measure
1FED
n=34 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
n=17 Participants
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Percent of Participants in Histologic Remission (<15 Eosinophils Per High Power Field) at 12 Weeks
Partial remission (2 - 14 eos/hpf)
23.5 percentage of participants
23.5 percentage of participants
Percent of Participants in Histologic Remission (<15 Eosinophils Per High Power Field) at 12 Weeks
Remission (< 15 eos/hpf)
44.1 percentage of participants
41.2 percentage of participants
Percent of Participants in Histologic Remission (<15 Eosinophils Per High Power Field) at 12 Weeks
Complete remission (≤ 1 eos/hpf)
20.6 percentage of participants
17.6 percentage of participants

SECONDARY outcome

Timeframe: 12 weeks

Population: We performed intent to treat analysis including participants who had at least one clinical observation post randomization

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.

Outcome measures

Outcome measures
Measure
1FED
n=4 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Percent of Participants on Swallowed Glucocorticoids (SGC) in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2
Remission (< 15 eos/hpf)
50.0 percentage of participants
Percent of Participants on Swallowed Glucocorticoids (SGC) in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2
Complete remission (≤ 1 eos/hpf)
25.0 percentage of participants
Percent of Participants on Swallowed Glucocorticoids (SGC) in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2
Partial remission (2 - 14 eos/hpf)
25.0 percentage of participants

SECONDARY outcome

Timeframe: 12 weeks

Population: We performed Intent to treat analysis including participants who had at least one clinical observation post randomization.

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.

Outcome measures

Outcome measures
Measure
1FED
n=8 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Percent of 1FED Non-responders on 4FED in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2
Complete remission (≤ 1 eos/hpf)
0 percentage of participants
Percent of 1FED Non-responders on 4FED in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2
Partial remission (2 - 14 eos/hpf)
12.5 percentage of participants
Percent of 1FED Non-responders on 4FED in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2
Remission (< 15 eos/hpf)
12.5 percentage of participants

SECONDARY outcome

Timeframe: Baseline and 12 weeks

Population: We performed intent to treat analysis including participants who had at least one clinical observation post randomization

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.

Outcome measures

Outcome measures
Measure
1FED
n=38 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
n=25 Participants
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Change From Baseline in Pediatric Quality of Life Inventory Version 3.0 EoE Module (PedsQL 3.0 EoE) at 12 Weeks
Baseline - Total Score
71.3 units on a scale
Standard Deviation 13.0
67.5 units on a scale
Standard Deviation 13.6
Change From Baseline in Pediatric Quality of Life Inventory Version 3.0 EoE Module (PedsQL 3.0 EoE) at 12 Weeks
Change from Baseline to Week 12
9.7 units on a scale
Standard Deviation 11.3
9.8 units on a scale
Standard Deviation 14.1

SECONDARY outcome

Timeframe: Baseline and 12 weeks

Population: We performed intent to treat analysis including participants who had at least one clinical observation post randomization

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.

Outcome measures

Outcome measures
Measure
1FED
n=38 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
n=24 Participants
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Change From Baseline in Pediatric Quality of Life Inventory Version 4.0 (PedsQL 4.0) Generic Core Scales at 12 Weeks
Baseline - Total Score
80.3 units on a scale
Standard Deviation 10.8
79.9 units on a scale
Standard Deviation 14.5
Change From Baseline in Pediatric Quality of Life Inventory Version 4.0 (PedsQL 4.0) Generic Core Scales at 12 Weeks
Change from Baseline to Week 12
3.7 units on a scale
Standard Deviation 9.9
5.3 units on a scale
Standard Deviation 16.4

SECONDARY outcome

Timeframe: Baseline and 12 weeks

Population: We performed intent to treat analysis including participants who had at least one clinical observation post randomization

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.

Outcome measures

Outcome measures
Measure
1FED
n=22 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
n=12 Participants
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Change From Baseline in Endoscopic Reference Score at 12 Weeks
Baseline - Total Score
2.5 units on a scale
Standard Deviation 1.3
3.3 units on a scale
Standard Deviation 1.8
Change From Baseline in Endoscopic Reference Score at 12 Weeks
Change from Baseline to Week 12
-0.7 units on a scale
Standard Deviation 2.2
-1.3 units on a scale
Standard Deviation 2.2

SECONDARY outcome

Timeframe: Baseline and 12 weeks

Population: We performed intent to treat analysis including participants who had at least one clinical observation post randomization

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).

Outcome measures

Outcome measures
Measure
1FED
n=28 Participants
1-food elimination diet: participants eliminate milk from the diet for 12 weeks
4FED
4-food elimination diet: participants eliminate milk, egg, wheat, soy from the diet for 12 weeks
Percent of Participants With Positive and Negative Milk Skin Prick Tests Responding to 1FED
Percent of total with positive milk SPT
11 percentage of participants
Percent of Participants With Positive and Negative Milk Skin Prick Tests Responding to 1FED
Percent of total with negative milk SPT
89 percentage of participants
Percent of Participants With Positive and Negative Milk Skin Prick Tests Responding to 1FED
Percent with positive SPT responding to 1FED
33 percentage of participants
Percent of Participants With Positive and Negative Milk Skin Prick Tests Responding to 1FED
Percent with negative SPT responding to 1FED
48 percentage of participants

Adverse Events

1FED

Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths

4FED

Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths

1FED Non-responders (4FED)

Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths

4FED Non-responders (SGC)

Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
1FED
n=38 participants at risk
1-food elimination diet (Phase 1)
4FED
n=25 participants at risk
4-food elimination diet (Phase 1)
1FED Non-responders (4FED)
n=19 participants at risk
4-food elimination diet (Phase 2)
4FED Non-responders (SGC)
n=10 participants at risk
Swallowed glucocorticosteroids (Flovent HFA) (Phase 2)
Gastrointestinal disorders
Abdominal Pain
2.6%
1/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
4.0%
1/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).

Other adverse events

Other adverse events
Measure
1FED
n=38 participants at risk
1-food elimination diet (Phase 1)
4FED
n=25 participants at risk
4-food elimination diet (Phase 1)
1FED Non-responders (4FED)
n=19 participants at risk
4-food elimination diet (Phase 2)
4FED Non-responders (SGC)
n=10 participants at risk
Swallowed glucocorticosteroids (Flovent HFA) (Phase 2)
Ear and labyrinth disorders
Ear Pain
0.00%
0/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
5.3%
1/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Gastrointestinal disorders
Abdominal Pain
5.3%
2/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
8.0%
2/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Gastrointestinal disorders
Constipation
0.00%
0/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
5.3%
1/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Gastrointestinal disorders
Vomiting
2.6%
1/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
8.0%
2/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
5.3%
1/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
20.0%
2/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Infections and infestations
Otitis Media
0.00%
0/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
5.3%
1/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Infections and infestations
Sinusitis
0.00%
0/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
10.0%
1/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Nervous system disorders
Headache
0.00%
0/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
5.3%
1/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Respiratory, thoracic and mediastinal disorders
Cough
2.6%
1/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
8.0%
2/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
10.0%
1/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
2.6%
1/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
10.0%
1/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Respiratory, thoracic and mediastinal disorders
Sinus Disorder
0.00%
0/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
5.3%
1/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
10.0%
1/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
Skin and subcutaneous tissue disorders
Urticaria
0.00%
0/38 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
8.0%
2/25 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/19 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).
0.00%
0/10 • Adverse events were collected from informed consent/assent to 30 days after the cessation of the study agent (2 years, 2 months).

Additional Information

Marc E. Rothenberg, MD, PhD

Cincinnati Childrens Hospital Medical Center

Phone: 513-636-7177

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place