Cryptoglandular Perianal Fistula
Conditions
Brief summary
The purpose of this study is to determine if experimental therapy (E-CEL UVEC Cells), a very minimally invasive procedure, can treat patients with perianal fistulas.
Interventions
BIOLOGICALE-CEL UVEC Cells
IP treatment involves percutaneous injections along the entire fistula tract and submucosal injections into the internal tract opening using anoscopic visualization
Sponsors
Angiocrine Bioscience
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This study is designed as an adequate and well-controlled study per 21 CFR 314.126. Specifically, this study utilizes one of the recognized controls: no treatment concurrent control. Primary endpoint consists of objective measurements of effectiveness. Placebo effect for this indication and target population is regarded as negligible. Moreover, a placebo concurrent control is regarded as infeasible for the treatment regimen to treat perianal fistula.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age 18 or older 2. Willing and able to provide informed consent and medically eligible 3. Diagnosed with perianal fistula per the following criteria: 1. Diagnosis documented by physical/clinical examination and imaging specifically performed to assess the number and course of the perianal fistula tract---i.e., magnetic resonance imaging (MRI) and/or ultrasound 2. Single tract without residual abscess cavity 3. Low transsphincteric and intersphincteric fistulas that cross less than 30% of the external sphincter i. Fistula internal opening width ≤ 3 mm. d. Maximum tract length of 10 cm e. Consistent with cryptoglandular origin f. Fistula-related pain of NRS \< 3 (NRS 0-10) g. Absence of history of Crohn's disease, ulcerative colitis or other inflammatory bowel diseases 4. For female patients of childbearing potential: 1. A negative serum or urine pregnancy test at screening is required prior to enrollment 2. Patient must be willing to use a highly effective method of contraception from the start of the screening period until at least 3 months after completion of last treatment 5. For male patients who can father a child and are having intercourse with females of childbearing potential who are not using adequate contraception: 1. Patient must be willing to use a recommended method of contraception and refrain from sperm donation from the start of screening period until for at least 3 months after completion of the last treatment
Exclusion criteria
1. Concomitant rectovaginal fistulas 2. Patients with an active abscess or suspicion of an active, untreated microabscess 3. Presence of signs and symptoms suggestive or suspicious of an acute active infection or flare-up. 4. Fistula-related pain measured as NRS ≥ 3 (NRS Scale 0-10) in the last 7 days. • If the subject is eligible except for this
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Clinical response (CR) rate at Week 12, comparing Arm A to Arm B in parallel. | Week 12 | CR is defined as achieving all the following four criteria: 1. Closure of internal opening, plus 2. Closure of external tract, plus 3. No discharge upon digital pressure, plus 4. No clinically meaningful pain |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in the anal fistula quality of life (AF-QoL) scale | Weeks 12, 24, and 36 after first treatment | To assess changes in the quality of life * Pre-treatment (just prior to 1st treatment) versus 12 weeks after the 1st treatment * Pre-treatment (just prior to 1st treatment) versus 24 weeks after the 1st treatment * Pre-treatment (just prior to 1st treatment) versus 36 weeks after the 1st treatment |
Countries
United States
Contacts
CONTACTJohn R Jaskowiak
Outcome results
None listed