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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01642212
First received: July 13, 2012
Last updated: November 3, 2015
Last verified: March 2014
Results First Received: September 30, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Eosinophilic Esophagitis
Interventions: Drug: Oral Budesonide Suspension (MB-9)
Drug: Placebo

  Participant Flow
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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 
[1] Two participants completed double-blind treatment but did not enter the open-label extension.
[2] Four participants completed double-blind treatment but did not enter the open-label extension.



  Baseline Characteristics
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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 


  Outcome Measures
  Show All Outcome Measures

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 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Physician
Organization: Shire Development LLC
phone: +1 866 842 5335



Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01642212     History of Changes
Other Study ID Numbers: MPI-101-06
Study First Received: July 13, 2012
Results First Received: September 30, 2015
Last Updated: November 3, 2015
Health Authority: United States: Food and Drug Administration