Clinical trial NCT05587309: A Study to Evaluate the Efficacy and Safety of BLI5100 in Patients with Erosive Esophagitis
Official title: A Phase 3, Double-Blind, Randomized, Two-Phase, Active-Controlled Study to Evaluate the Efficacy and Safety of BLI5100 in Patients with Erosive Esophagitis
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Erosive Esophagitis
Lead sponsor: Braintree Laboratories
Sponsor class: INDUSTRY
First posted: 2022-10-20
Enrollment: 1250
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The objective of the Healing Phase of the study is to evaluate the safety and efficacy of up to 8 weeks of once daily oral administration of BLI5100 versus a PPI control in healing EE. The objective of the Maintenance Phase of the study is to evaluate the safety and efficacy of 24 weeks of once daily oral administration of BLI5100 (low or high dose) versus a PPI control in the maintenance of healed EE.


Eligibility criteria: Inclusion Criteria:

1. Aged ≥18 years at the time of signing informed consent;
2. Have experienced both heartburn and regurgitation within 7 days prior to the Screening Visit;
3. Current evidence of EE of LA grades A to D based on an upper GI endoscopy;
4. Able to understand and comply with the protocol requirements;
5. Willing and able to provide written informed consent at Screening;
6. A female of reproductive potential defined as a non-post-menopausal female who has not had a bilateral oophorectomy or medically documented ovarian failure; or If a female of childbearing potential, agrees to use an acceptable form of birth control and avoid egg donation from the Screening Visit until 6 months after the last dose of study drug.
7. If a male, agrees to use an acceptable form of birth control from the Screening Visit until 3 months after the last dose of study drug.
8. If a male, agrees to abstain from sperm donation through 3 months after administration of the last dose of study drug.

Exclusion Criteria:

1. Unable to undergo an upper GI endoscopy;
2. Presence of esophageal stricture, gastroesophageal varix (including post sclerotherapy or ligation), untreated Barrett's esophagus, gastric bleeding, infection, tumor, or gastric or duodenal ulcer on the upper GI endoscopy;

   o Note: Patients with diagnosis of Schatzki's ring (mucosal tissue ring around lower esophageal sphincter) are eligible to participate.
3. Alarm symptoms such as odynophagia, severe dysphagia, upper GI bleeding, weight loss, anemia, or hematochezia within 4 weeks prior to Screening, unless the presumed malignancy is ruled out;
4. History of eosinophilic esophagitis, achalasia, or other primary esophageal motility disorder; functional heartburn; physiochemical trauma (including radiation, mucosal resection, or cryotherapy); or documented history of delayed gastric emptying;
5. History of a connective tissue disorder associated with GI symptoms (eg, scleroderma or systemic lupus erythematous) or inflammatory bowel disease;
6. History of acid-suppressive, esophageal, or gastric surgery;

   o Note: This is not applicable to appendectomy, cholecystectomy, or endoscopic excision of benign tumor.
7. History of malignancy within the past 5 years (with the exception of resected basal cell or squamous cell carcinoma of the skin);

   o Note: This is not applicable to patients who had complete response or pathological complete response and whose tumor had not recurred for at least 5 years from the date of last treatment, or patients whose tumor had been removed by endoscopic resection without any findings indicating recurrence of the tumor within 3 years.
8. History of an allergic disease, or hypersensitivity or intolerance to the active ingredient or excipients of the study drug;
9. History of alcoholism, chronic opiate use, or substance addiction in the 12 months before Screening or a positive urine drug screen for opiates or substances of abuse;

   * Note: Patients on prescribed opioids are eligible to participate if they have been on a stable dose for \>3 months prior to Screening.
   * Note: Retesting of a positive urine drug screen requires Medical Monitor approval. Positive urine drug screen for all drugs will require verification of prescription for the positive tested substance.
10. Presence of manic-depression, anxiety disorder, panic disorder, somatoform disorder, personality disorder, or other psychological disorder;

    o Note: A diagnosis of manic-depression, anxiety disorder, panic disorder, somatoform disorder, personality disorder, or other psychological disorder is not exclusionary, as long as the patient is asymptomatic and well-controlled on stable treatment (ie, stable dose for \>6 months prior to Screening), including non-medical therapy.
11. Current use of antipsychotics, antidepressants, anxiolytics, or prescription sleeping medications, with the exception of a stable dose for \>6 months prior to Screening;
12. Use of any gastric acid-suppressive agents, including PPIs, within 2 weeks prior to the upper GI endoscopy at Screening;
13. Use of 2 or more commercial doses of ref lux esophagitis-related medications (including H2 blockers, prostaglandins, mucosal protective agents, and prokinetics) within 1 week prior to the upper GI endoscopy at Screening;
14. Requirement of persistent use of non-steroidal anti-inflammatory drugs (NSAIDs) during the course of the study;

    o Note: Low-dose (≤100 mg/day) aspirin is allowed provided that it has been used for prophylaxis prior to study participation.
15. If a female, is pregnant, breastfeeding, or planning to become pregnant during the study or within 6 months after the last dose of study drug;
16. Positive test result for human immunodeficiency virus, hepatitis B surface antigen, or hepatitis C virus at Screening;
17. Positive test result for H pylori at Screening or diagnosis and treatment of H pylori within 6 weeks prior to randomization;

    o Note: Patients who test positive for H pylori at Screening will be offered treatment according to local standard of care and may be re-screened for eligibility following completion of treatment.
18. Abnormal laboratory results with clinical relevance at Screening as follows:

    * Aspartate aminotransferase (AST), ALT, or alkaline phosphatase level of ≥2 × upper limit of normal (ULN);
    * Total bilirubin level of ≥2 × ULN, unless Gilbert's syndrome is confirmed when direct bilirubin is ≤0.3 mg/dL;
    * Estimated glomerular filtration rate \<30 mL/min; or
    * Serum magnesium \< lower limit of normal.
19. Abnormal ECG of clinical significance (eg, major arrhythmia, multifocal premature ventricular contractions, or 2° atrioventricular block anomaly);
20. Presence of any gastric acid hypersecretory conditions, such as Zollinger-Ellison syndrome;
21. Involvement in another clinical study within 4 weeks of initiation of study drug;
22. Any other clinically relevant condition that would confound study endpoints or adversely affect patient compliance with the study procedures in the medical judgment of the Investigator or Medical Monitor based on previous medical history or findings on Screening assessments.

Primary outcomes:
measure: Healing Phase: Percentage of patients with complete healing by Week 8.
timeFrame: 8 Weeks
measure: Maintenance Phase: Percentage of patients who maintain complete healing through Week 24.
timeFrame: 24 Weeks
Secondary outcomes:
measure: Healing Phase: Percentage of 24-hour heartburn-free days through Week 8.
timeFrame: 8 Weeks
measure: Maintenance Phase: Percentage of 24-hour heartburn-free days through Week 24.
timeFrame: 24 Weeks
Other outcomes:
Locations:
city: Homewood
country: United States
facility: Research Site 145
state: Alabama
city: Huntsville
country: United States
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state: Alabama
city: Peoria
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state: Arizona
city: Peoria
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facility: Research Site 150
state: Arizona
city: Phoenix
country: United States
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state: Arizona
city: Tucson
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state: Arizona
city: Little Rock
country: United States
facility: Research Site 87
state: Arkansas
city: North Little Rock
country: United States
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state: Arkansas
city: Arcadia
country: United States
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state: California
city: Bell Gardens
country: United States
facility: Research Site 66
state: California
city: Camarillo
country: United States
facility: Research Site 156
state: California
city: Canoga Park
country: United States
facility: Research Site 19
state: California
city: Chula Vista
country: United States
facility: Research Site 94
state: California
city: Huntington Park
country: United States
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state: California
city: La Mesa
country: United States
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state: California
city: Los Angeles
country: United States
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state: California
city: San Diego
country: United States
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state: California
city: Santa Ana
country: United States
facility: Research Site 85
state: California
city: Santa Ana
country: United States
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state: California
city: Santa Maria
country: United States
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state: California
city: Westlake
country: United States
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state: California
city: Littleton
country: United States
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state: Colorado
city: Bristol
country: United States
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state: Connecticut
city: Boca Raton
country: United States
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state: Florida
city: Cooper City
country: United States
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city: Doral
country: United States
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city: Hialeah
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city: Homestead
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city: Miami Lakes
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city: Miami
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state: Florida
city: Miami
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state: Florida
city: Miami
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city: Atlanta
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city: Marietta
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city: Sandy Springs
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state: Georgia
city: Boise
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state: Idaho
city: Idaho Falls
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state: Illinois
city: Gurnee
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state: Illinois
city: Gurnee
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state: Illinois
city: Oaklawn
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state: Illinois
city: South Bend
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state: Indiana
city: Covington
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city: Houma
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city: Marrero
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city: New Orleans
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state: Minnesota
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state: Missouri
city: Columbia
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city: Kansas City
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city: Henderson
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state: Nevada
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city: Reno
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state: New Jersey
city: Freehold
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state: New Jersey
city: Jackson
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state: New Jersey
city: Brooklyn
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state: New York
city: Great Neck
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state: New York
city: Rochester
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state: North Carolina
city: High Point
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city: Salisbury
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state: North Carolina
city: Columbus
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state: Ohio
city: Columbus
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city: Columbus
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city: Mentor
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city: Springboro
country: United States
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state: Ohio
city: Westlake
country: United States
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state: Ohio
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state: South Carolina
city: North Charleston
country: United States
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state: South Carolina
city: Hermitage
country: United States
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state: Tennessee
city: Kingsport
country: United States
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state: Tennessee
city: Nashville
country: United States
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state: Tennessee
city: Nashville
country: United States
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state: Tennessee
city: Shelbyville
country: United States
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state: Tennessee
city: Bellaire
country: United States
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state: Texas
city: Dallas
country: United States
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state: Texas
city: Forney
country: United States
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state: Texas
city: Houston
country: United States
facility: Research Site 154
state: Texas
city: Houston
country: United States
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state: Texas
city: Katy
country: United States
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state: Texas
city: Lewisville
country: United States
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state: Texas
city: McAllen
country: United States
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state: Texas
city: Pearland
country: United States
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state: Texas
city: Red Oak
country: United States
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state: Texas
city: San Antonio
country: United States
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state: Texas
city: San Antonio
country: United States
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state: Texas
city: San Antonio
country: United States
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state: Texas
city: Tomball
country: United States
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state: Texas
city: Waco
country: United States
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state: Texas
city: Ogden
country: United States
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state: Utah
city: Salt Lake City
country: United States
facility: Research Site 57
state: Utah
city: Sandy
country: United States
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state: Utah
city: South Ogden
country: United States
facility: Research Site 16
state: Utah
city: Chesapeake
country: United States
facility: Research Site 77
state: Virginia
city: Richmond
country: United States
facility: Research Site 136
state: Virginia
city: Morgantown
country: United States
facility: Research Site 97
state: West Virginia
Study officials:
affiliation: Braintree Laboratories / Sebela Pharmaceuticals
name: Leah Hollins
role: STUDY_DIRECTOR
References: