The purpose of this study is to evaluate the combined remission of complex perianal fistulas, defined as the clinical assessment at Week 24 of closure of all treated external openings that were draining at baseline despite gentle finger compression, and absence of collections greater than (\>) 2 centimeter (cm) (in at least 2 dimensions) confirmed by blinded central magnetic resonance imaging (MRI) assessment at Week 24.
This study is to assess the efficacy and safety of Cx601, eASC, for the treatment of complex perianal fistulas in participants with Crohn's disease. The study will randomize approximately 554 participants. * Cx601 eASCs intralesional injection * Placebo - Cx601 placebo-matching eASCs intralesional injection Study treatments will be allocated, on a 1:1 ratio, by central randomization through interactive web response system (IWRS). The study will follow an add-on design, participants receiving any ongoing concomitant medical treatment, at stable doses at the time of screening, for the CD will be allowed to continue it throughout the study. The primary efficacy analysis, will be conducted at Week 24 timepoint. The double blind design will be maintained up to Week 52 (both participant and investigator) by a specific blinding for study treatment administration and for evaluating its efficacy. This multicenter trial will be conducted globally across 150 centers. The overall time to participate in this study is approximately 5 years.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
568
Scottsdale Mayo Clinic
Scottsdale, Arizona, United States
UC San Diego Health Systems
La Jolla, California, United States
University of Southern California (USC) Norris Comprehensive Cancer Center
Los Angeles, California, United States
UC Irvine Medical Center - Chao Family Comprehensive Cancer
Orange, California, United States
Inland Empire Liver Foundation
Rialto, California, United States
Percentage of Participants With Combined Remission at Week 24
Combined remission was defined as the closure of all treated external openings that were draining at baseline despite gentle finger compression and absence of collection(s) \>2 cm (in at least 2 dimensions) of the treated perianal fistula(s) confirmed by blinded central magnetic resonance imaging (MRI) assessment. Percentages are rounded off to whole number at the nearest decimal.
Time frame: Week 24
Percentage of Participants With Clinical Remission at Week 24
Clinical remission was defined as closure of all treated external openings that were draining at baseline despite gentle finger compression. Percentages are rounded off to whole number at the nearest decimal.
Time frame: Week 24
Time to Clinical Remission at Week 24
Time to clinical remission was defined as the time from treatment start to first visit with closure of all treated external openings that were draining at baseline despite gentle finger compression, as clinically assessed.
Time frame: Week 24
Percentage of Participants With Combined Remission at Week 52
Combined remission was defined as the closure of all treated external openings that were draining at baseline despite gentle finger compression, and absence of collections \>2 cm (in at least 2 dimensions) confirmed by blinded central MRI assessment. Percentages are rounded off to whole number at the nearest decimal.
Time frame: Week 52
Percentage of Participants With Clinical Remission at Week 52
Clinical remission was defined as closure of all treated external openings that were draining at baseline despite gentle finger compression. Percentages are rounded off to whole number at the nearest decimal.
Time frame: Week 52
Percentage of Participants With Clinical Response at Week 24
Clinical response was defined as closure of at least 50 percent (%) of all treated external openings that were draining at baseline despite gentle finger compression. Percentages are rounded off to whole number at the nearest decimal.
Time frame: Week 24
Percentage of Participants With Clinical Response at Week 52
Clinical response was defined as closure of at least 50% of all treated external openings that were draining at baseline, despite gentle finger compression. Percentages are rounded off to whole number at the nearest decimal.
Time frame: Week 52
Time to Clinical Remission at Week 52
Time to clinical remission was defined as the time from treatment start to first visit with closure of all treated external openings that were draining at baseline despite gentle finger compression, as clinically assessed.
Time frame: Week 52
Time to Clinical Response at Week 24
Time to clinical response was defined as the time from treatment start to first visit with closure of at least 50% of all treated external openings that were draining at baseline, despite gentle finger compression, as clinically assessed.
Time frame: Week 24
Time to Clinical Response at Week 52
Time to clinical response was defined as time from treatment start to first visit with closure of at least 50% of all treated external openings that were draining at baseline despite gentle finger compression, as clinically assessed.
Time frame: Week 52
Percentage of Participants With Relapse by Week 52 After Achieving Combined Remission at Week 24
Relapse was defined as reopening of any of the treated fistulas external openings with active drainage as clinically assessed, or the development of a perianal fluid collection \>2 cm of the treated perianal fistula confirmed by centrally read MRI assessment in participants who were in combined remission at week 24. Percentages are rounded off to whole number at the nearest decimal.
Time frame: From Week 24 to Week 52
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Treatment-emergent Adverse Events of Special Interest (TEAESIs)
An adverse event(AE)=any untoward medical occurrence in a clinical investigation participant receiving a medicinal product; it did not necessarily have to have a causal relationship with this treatment. Serious adverse event(SAE)=any untoward medical occurrence that at any dose resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital abnormality/birth defect, or was a medically significant event or required intervention to prevent at least one of the outcomes listed above, or was a suspected transmission of an infectious agent. AESIs included tumorigenicity, ectopic tissue formation, hypersensitivity reactions, transmission of infectious agents, immunogenicity/alloimmune reactions, and medication errors, as reported by the investigator. TEAE=AE whose onset occurred, severity worsened, or intensity increased after receiving the study treatment.
Time frame: From first dose of study drug to end of follow up period (up to Week 52)
Number of Participants With Clinically Significant Changes in Vital Sign Parameters
Vital signs included measurement of pulse rate, systolic and diastolic blood pressure, respiratory rate, and body temperature. Clinically significant vital signs assessment was based on investigator interpretation. Number of participants with clinically significant changes in vital signs were reported.
Time frame: From first dose of study drug to end of follow up period (up to Week 52)
Number of Participants With Clinically Significant Changes in Laboratory Parameters
Laboratory parameters included blood chemistry and hematology. Clinically significant laboratory parameters assessment was based on investigator interpretation. Number of participants with clinically significant changes in laboratory parameters (hematology and blood chemistry) were reported.
Time frame: From first dose of study drug to end of follow up period (up to Week 52)
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Kaiser Permamente
San Francisco, California, United States
University of California San Francisco
San Francisco, California, United States
Vallejo Hospital and Medical Offices
Vallejo, California, United States
Cedar-Sinai Medical Center
West Hollywood, California, United States
University of Colorado Hospital
Aurora, Colorado, United States
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