Prospective randomized controlled trial of an injectable esophageal prosthesis versus a sham procedure for endoscopic treatment of gastroesophageal reflux disease.
BACKGROUND
This study aimed to assess whether endoscopic implantation of an injectable esophageal prosthesis, the Gatekeeper Reflux Repair System (GK), is a safe and effective therapy for controlling gastroesophageal reflux disease (GERD).


METHODS
A prospective, randomized, sham-controlled, single-blinded, international multicenter study planned final enrollment of 204 patients in three groups: up to 60 lead-in, 96 GK, and 48 sham patients. The sham patients were allowed to cross over to the GK treatment arm or exit the study at 6 months. The primary end points were (1) reduction in serious device- and procedure-related adverse device effects compared with a surgical composite complication rate and (2) reduction in heartburn symptoms 6 months after the GK procedure compared with the sham procedure. The secondary end point was improved esophageal pH (total time pH was <4) 6 months after the GK procedure compared with baseline.


RESULTS
A planned interim analysis was performed after 143 patients were enrolled (25 lead-in, 75 GK, and 43 sham patients), and the GK study was terminated early due to lack of compelling efficacy data. Four reported serious adverse events had occurred (2 perforations, 1 pulmonary infiltrate related to a perforation, and 1 severe chest pain) at termination of the study with no mortality or long-term sequelae. Heartburn symptoms had improved significantly at 6 months compared with baseline in the GK group (p < 0.0001) and the sham group (p < 0.0001), but no significant between-group difference in improvement was observed (p = 0.146). Esophageal acid exposure had improved significantly at 6 months compared with baseline in the GK group (p = 0.021) and the sham group (p = 0.003), but no significant between-group difference in improvement was observed (p = 0.27).


CONCLUSIONS
The GK procedure was associated with some serious but infrequent complications. No statistically significant difference in outcomes was observed between the treatment and control groups at 6 months compared with baseline.
Fockens
Paul
P
Academic Medical Center, University of Amsterdam, PO Box 22700, 1100 DE Amsterdam, the Netherlands.
Cohen
Lawrence
L
Edmundowicz
Steven A
SA
Binmoeller
Kenneth
K
Rothstein
Richard I
RI
Smith
Daniel
D
Lin
Edward
E
Nickl
Nicholas
N
Overholt
Bergein
B
Kahrilas
Peter J
PJ
Vakil
Nimish
N
Abdel Aziz Hassan
Ayman M
AM
Lehman
Glen A
GA
D000328:Adult; D000368:Aged; D016099:Endoscopy, Gastrointestinal / Q000379:methods*; D050782:Esophageal pH Monitoring; D004947:Esophagus; D005260:Female; D005500:Follow-Up Studies; D005764:Gastroesophageal Reflux / Q000175:diagnosis / Q000601:surgery*; D006801:Humans; D020136:Hydrogel, Polyethylene Glycol Dimethacrylate / Q000008:administration & dosage*; D007267:Injections; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D019736:Prostheses and Implants; D011788:Quality of Life; D016037:Single-Blind Method; D011795:Surveys and Questionnaires; D016896:Treatment Outcome; D055815:Young Adult
D020136:Hydrogel, Polyethylene Glycol Dimethacrylate