OBS in Adolescent and Adults With EOE: A Phase II, Randomized, Double-Blind, Placebo Controlled, Study With an Open Label Extension

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Single-blind Placebo	Eligible participants entered a 4-week, single-blind, placebo baseline period. Participants ingested the first dose of placebo in the clinic in the presence of study center personnel, and the remaining doses were ingested at home. Participants took placebo twice daily (bid); once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Double-blind Placebo	Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Double-blind Oral Budesonide Suspension (OBS) 2 mg	Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Placebo to Open-label Oral Budesonide Suspension (OBS) 2 mg	During the first 12 weeks of open-label extension treatment, participants took 2 mg OBS (formulation MB-9) once daily (qd) at bedtime. The volume per dose was 10 mL of oral liquid (10 mL qd; 0.2 mg/mL). Thereafter, an optional dose increase to 1.5 mg twice daily (bid) (7.5 mL bid; 0.2 mg/mL) and then to 2 mg bid (10 mL bid; 0.2 mg/mL) was allowed for subjects whose response to the 2 mg once daily dose was inadequate, as determined by the Investigator; a dose decrease to 2 mg once daily was allowed at any time.
Oral Budesonide Suspension (OBS) 2 mg to Open-label OBS 2 mg	During the first 12 weeks of open-label extension treatment, participants took 2 mg OBS (formulation MB-9) once daily (qd) at bedtime. The volume per dose was 10 mL of oral liquid (10 mL qd; 0.2 mg/mL). Thereafter, an optional dose increase to 1.5 mg twice daily (bid) (7.5 mL bid; 0.2 mg/mL) and then to 2 mg bid (10 mL bid; 0.2 mg/mL) was allowed for subjects whose response to the 2 mg once daily dose was inadequate, as determined by the Investigator; a dose decrease to 2 mg once daily was allowed at any time.

Participant Flow for 3 periods

Period 1:   Single-blind Baseline

	Single-blind Placebo	Double-blind Placebo	Double-blind Oral Budesonide Suspension (OBS) 2 mg	Placebo to Open-label Oral Budesonide Suspension (OBS) 2 mg	Oral Budesonide Suspension (OBS) 2 mg to Open-label OBS 2 mg
STARTED	119	0	0	0	0
COMPLETED	93	0	0	0	0
NOT COMPLETED	26	0	0	0	0
Did Not Meet Eligibility Criteria	26	0	0	0	0

Period 2:   Double-blind Treatment

	Single-blind Placebo	Double-blind Placebo	Double-blind Oral Budesonide Suspension (OBS) 2 mg	Placebo to Open-label Oral Budesonide Suspension (OBS) 2 mg	Oral Budesonide Suspension (OBS) 2 mg to Open-label OBS 2 mg
STARTED	0	42	51	0	0
COMPLETED	0	39	49	0	0
NOT COMPLETED	0	3	2	0	0
Not Specified	0	2	1	0	0
Non-compliance	0	1	0	0	0
Adverse Event	0	0	1	0	0

Period 3:   Open-label Extension

	Single-blind Placebo	Double-blind Placebo	Double-blind Oral Budesonide Suspension (OBS) 2 mg	Placebo to Open-label Oral Budesonide Suspension (OBS) 2 mg	Oral Budesonide Suspension (OBS) 2 mg to Open-label OBS 2 mg
STARTED	0	0	0	37 [1]	45 [2]
COMPLETED	0	0	0	27	37
NOT COMPLETED	0	0	0	10	8
Adverse Event	0	0	0	3	0
Sponsor Decision	0	0	0	7	4
Consent Withdrawn	0	0	0	0	2
Lost to Follow-up	0	0	0	0	1
Not Specified	0	0	0	0	1

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The Safety Analysis Set, defined as all randomized participants who received at least 1 dose of double-blind study drug (placebo or OBS); subjects were analyzed based on the actual treatment received.

Reporting Groups

	Description
Double-blind Placebo	Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Double-blind Oral Budesonide Suspension (OBS) 2 mg	Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Total	Total of all reporting groups

Baseline Measures

	Double-blind Placebo	Double-blind Oral Budesonide Suspension (OBS) 2 mg	Total
Overall Participants Analyzed  [Units: Participants]	42	51	93
Age  [Units: Years] Mean (Standard Deviation)	20.8  (7.50)	22.3  (7.92)	21.6  (7.73)
Age, Customized  [Units: Participants]
<18 Years Of Age	17	18	35
>/= 18 Years Of Age	25	33	58
Gender  [Units: Participants]
Female	13	16	29
Male	29	35	64

1.  Primary:

Percent of Participants Who Were Histologic Responders   [ Time Frame: Week 16 ]

2.  Primary:

Change From Baseline in The Dysphagia Symptom Questionnaire (DSQ) Score at The Final Treatment Period Evaluation   [ Time Frame: Baseline, Week 16 ]

3.  Secondary:

Change From Baseline in The DSQ Score Over Time   [ Time Frame: Baseline, Weeks 8 and 12 ]

4.  Secondary:

Change From Baseline in The DSQ Score For The 50th Percentile of Participants at The Final Treatment Period Evaluation   [ Time Frame: Baseline, Week 16 ]

5.  Secondary:

Percent of Participants With a Peak Eosinophil Count </= 15/High Power Field (Light Microscopy) (HPF) And </= 1/HPF at The Final Treatment Period Evaluation   [ Time Frame: Week 16 ]

6.  Secondary:

Percent of Participants With a >/= 30% And >/= 50% Reduction In The DSQ Score From Baseline to The Final Treatment Period Evaluation   [ Time Frame: Baseline, Week 16 ]

7.  Secondary:

Percent of Participants Who Were Overall Responders at The Final Treatment Period Evaluation   [ Time Frame: Week 16 ]

8.  Secondary:

Change From Baseline in The Histopathologic Epithelial Features Combined Total Score at The Final Treatment Period Evaluation   [ Time Frame: Baseline, Week 16 ]

9.  Secondary:

Change From Baseline in The Total Endoscopy Score at The Final Treatment Period Evaluation   [ Time Frame: Baseline, Week 16 ]

10.  Secondary:

Change From Baseline in The Peak Eosinophil Count at Each Available Esophageal Level at The Final Treatment Period Evaluation   [ Time Frame: Baseline, Week 16 ]

11.  Secondary:

Change From Baseline in The Physician’s Global Assessment (PGA) of Disease Activity at The Final Treatment Period Evaluation   [ Time Frame: Baseline, Week 16 ]

12.  Secondary:

Distribution of Responses For The Patient Global Impression of Change (PGIC) Survey at The Final Treatment Evaluation   [ Time Frame: Week 16 ]

13.  Secondary:

Percent of Participants With Improved Symptoms on The Eosinophilic Esophagitis (EoE) Symptom Survey at The Final Treatment Period Evaluation   [ Time Frame: Week 16 ]

14.  Secondary:

Percent of Participants With No Change in Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation   [ Time Frame: Week 16 ]

15.  Secondary:

Percent of Participants With Worsened Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation   [ Time Frame: Week 16 ]

16.  Secondary:

Percent of Participants Without Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation   [ Time Frame: Week 16 ]

17.  Secondary:

Percent of Participants With New Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation   [ Time Frame: Week 16 ]

18.  Secondary:

Percent of Participants Who Were Symptom Responders on The DSQ+Pain Scale at The Final Treatment Period Evaluation   [ Time Frame: Week 16 ]

19.  Secondary:

Percent of Days That Participants Reported That They Avoided Solid Food During The Baseline And Treatment Periods   [ Time Frame: From 14 days prior to the baseline visit to the final treatment period evaluation ]

20.  Secondary:

Change From Baseline in The Scores of DSQ Question 1 During The Treatment Period   [ Time Frame: Baseline, Weeks 8, 12, and 16 ]

21.  Secondary:

Change From Baseline in The Scores of DSQ Question 4 During The Treatment Period   [ Time Frame: Baseline, Weeks 8, 12, and 16 ]

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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