A Phase 1 interventional study of Upadacitinib in Crohn's Diseases, Active Anal Fistula and Upadacitinib, sponsored by Sixth Affiliated Hospital, Sun Yat-sen University. Recruiting at 1 site in China. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2025-03-30.
Sponsored by Sixth Affiliated Hospital, Sun Yat-sen University · Phase 1, Interventional, and Treatment
Among 17 studies in Rectal Fistula with a site in China — 6 open to participants now, 6 under way, none of them phase 3. Rectal Fistula · China.
Among the 17 studies in Rectal Fistula with a site in China, this one is the 12th largest by planned enrollment of the 15 interventional ones, is one of 3 that started in 2024, is one of 3 in phase 1, and was the 11th registered.
169 studies on the registry are indexed under Rectal Fistula; 45 are open to participants now.
This study's planned enrollment of 27 is below the median of 66 across 115 interventional studies indexed under Rectal Fistula.
Browse Rectal Fistula studies →6 of the 17 studies in Rectal Fistula with a site in China are open now, in 1 country. Each count lists the studies.
Of the 26 Rectal Fistula studies recruiting now, 18 (69%) set no upper age limit and 7 (27%) stop at 75 or younger; 2 take children under 18; 1 take healthy volunteers.
This study: 18 Years to 70 Years · all sexes · no healthy volunteers.
The full table on the hub →17 studies; the count open now beside each. This study's row is marked.
First in 2006; this study’s year is marked. 3 started in 2024.
Median enrollment 92 across the 15 interventional studies, 663 across the 2 observational. Of the 6 that completed or were terminated, 0 (0%) have results posted.
1 of 17 have an industry lead sponsor. The 10 most frequent:
Sixth Affiliated Hospital, Sun Yat-sen University is the lead sponsor of 217 studies on the registry; 111 are open to participants now.
Sixth Affiliated Hospital, Sun Yat-sen University has 3 studies filed under Rectal Fistula — fewer than ten, so there is no comparison to draw.
None of the studies under way reaches primary completion within a year.
Among the 17 studies in Rectal Fistula with a site in China, from their registry updates:
Nothing recorded among them since 2026-09-25.
Anal fistula is the most common perianal lesion of Crohn's disease (CD), and the incidence of anal fistula in eastern CD population is significantly higher than that in Western population. The treatment of CD active anal fistula is difficult, which seriously affects the quality of life of patients and consumes a lot of medical resources. Injection of biological agents is the most commonly used method for the treatment of CD anal fistula, small molecule drugs can be taken orally, and the curative effect is more lasting. Upadacitinib was the first small molecule drug approved for CD treatment in China on June 30, 2023. At present, there is only one post-subgroup analysis of a global Phase 3 clinical study on Upatinib in the treatment of CD anal fistula, and the number of active anal fistula cases included is small, and the study objects are mostly western populations. This study intends to include CD patients with active anal fistula, and adopts the method of single-center single-arm study to explore the efficacy of Upatinib in the treatment of CD anal fistula, so as to provide more evidence-based medical evidence for the drug selection of CD anal fistula in China.
Crohn's disease (CD) is a chronic non-specific inflammatory disease of the intestine, and anal fistula is the most common perianal lesion in CD. Active anal fistula refers to the infection of the fistula, which causes perianal pain, discharge, redness, swelling, and even fever in patients. Biologics are currently the most widely used drugs for treating CD anal fistula. Studies with fistula healing as the main endpoint of observation show that the clinical remission rate of fistula is about 50% at most. CD anal fistula is difficult to treat, has a high recurrence rate, seriously affects the quality of life of patients, and consumes a large amount of medical resources.
CD anal fistula is a special subtype of CD, and exploring CD anal fistula has very important clinical significance. The incidence of anal fistula in Asian CD patients is significantly higher than that in Western CD patients; the prognosis of CD patients with anal fistula is worse, and the risk of intestinal stenosis or perforation is 3 to 4 times higher than that of patients without anal fistula; approximately 10% of patients present with anal fistula as the first manifestation of CD, and the symptoms of anal fistula most affect the quality of life of patients during the course of the disease; the susceptibility genes of CD anal fistula patients are different from those of other CD patients, and the susceptibility genes of the Asian CD population are different from those of the Western population. Our previous study included Han CD patients from southern China for analysis and found that polymorphisms in the IRGM, AOX1, and NKX2-3 genes are associated with the development of anal fistula.Compared with the widely used biologics for treating CD, small molecule drugs have great prospects. Biologics are complex proteins that require injection for treatment, have immunogenicity, are prone to secondary failure, and are relatively expensive. In contrast, small molecule drugs have a relatively small molecular weight, are easier to pass through cell membranes; have a short half-life and can be taken orally; have no antigenicity or immunogenicity, and have better sustained efficacy; and have lower production costs.
Upadacitinib was approved for the treatment of CD on June 30, 2023, and it is the first small molecule drug approved for CD treatment in China. The New England Journal of Medicine recently published the results of a phase 3 clinical study of upadacitinib for CD [8]: In the induction period study, the clinical remission rate of the upadacitinib 45 mg treatment group was higher than that of the placebo group (U-EXCEL study, 49.5% vs. 29.1%; U-EXCEED study, 38.9% vs. 21.1%), and the endoscopic response rate was also higher than that of the placebo group (U-EXCEL study, 45.5% vs. 13.1%; U-EXCEED study, 34.6% vs. 3.5%); in the maintenance period study (U-ENDURE), the clinical remission rates (37.3% and 47.6%) and endoscopic response rates (27.6% and 40.1%) of the upadacitinib 15 mg and 30 mg treatment groups at week 52 were both higher than those of the placebo group (15.1% and 7.3%). The above study results show that upadacitinib is effective in treating CD.However, as a new small molecule drug, the efficacy of upadacitinib for the special subtype of CD anal fistula is not clear.
JAK/STAT is involved in innate and adaptive immunity. After activation, the signal is rapidly transmitted from the membrane to the nucleus, and then cytokines are activated, white blood cell transport is promoted, and cell proliferation is initiated, triggering intestinal inflammation and playing an important role in the pathogenesis of CD. The JAK/STAT signaling pathway may also be an important pathogenic mechanism of CD anal fistula. Manreet et al. found that CD anal fistula patients have gene variations in the JAK/STAT signaling pathway [9]. Upadacitinib targets and inhibits JAK1. Mechanistically, it may be effective for CD anal fistula. At present, only post hoc subgroup analysis data from the aforementioned three clinical studies on CD (not yet published) are available. In these studies, the complete remission rates of patients with active fistulas treated with different doses of upadacitinib at week 52 were 25% (3/12) and 10% (1/10), respectively, which were superior to the placebo group (0%, 0/23 and 0/7). However, the number of active fistula cases included in this post hoc subgroup analysis was relatively small, and the study subjects were mostly Western populations. Therefore, more efficacy data on this new small molecule drug for CD fistulas in the Chinese population are needed.
Therefore, this study intends to include CD patients with active fistulas and use a single-arm clinical study approach to explore the efficacy of upadacitinib in treating Chinese CD fistula patients. This will prepare for subsequent randomized controlled trials (RCTs) to further investigate the efficacy of different doses of upadacitinib in treating fistulas and compare the efficacy of upadacitinib with other biological agents in treating fistulas, and also provide more evidence-based medical evidence for the drug selection of CD fistulas.
Our hospital is one of the largest inflammatory bowel disease centers in the country. Additionally, due to the reputation of the anorectal surgery department, it has gathered a large number of fistula patients, providing a guarantee for the number of cases in this study.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Combined response refers to clinical response plus imaging response. Clinical response was defined as 100% closure of the external orifice of the fistula and no fluid seepage from the patient's complaint or physician's acupressure. Imaging remission was defined as the absence of \> 2cm pyo-filling fistula on perianal MRI and the absence of edema and active inflammation. Clinical response for the primary endpoint was judged by two experienced senior physicians and, if inconsistent, by a third clinician. The imaging was judged by two senior doctors in the independent third party diagnostic team, and if the judgment was inconsistent, the third team physician made the judgment
Drug: Upadacitinib
Enrolled patients received oral upatinib treatment with a conventional induction dose of 45mg/d for 12 weeks, followed by a maintenance dose of 15m/d or 30mg/d
Clinical response at 12 weeks of treatment
Clinical response was defined as 100% closure of the external orifice of the fistula and no fluid seepage from the patient's complaint or physician's acupressure
Time frame: 12 weeks
Imaging remission at 12 weeks of treatment
Imaging remission was defined as the absence of \> 2 cm pyo-filling fistula on perianal MRI and the absence of edema and active inflammation
Time frame: 12 weeks
The clinical response rate of anal fistula at week 12/48 of treatment.
Clinical response: At least 50% of the external openings with exudate upon digital pressure at baseline are closed
Time frame: 12 weeks、48 weeks
The clinical remission rate of anal fistula at week 12/48 of treatment.
Clinical remission: The patient has no symptoms of perianal fistula exudate, and 100% of the external openings show no exudate upon digital pressure by the doctor.
Time frame: 12 weeks、48 weeks
The imaging remission rate of anal fistula at week 12/48 of treatment
Imaging remission: No abscess fistula larger than 2 cm is found on perianal MRI, and there is no edema or active inflammation (judged blindly by one radiologist).
Time frame: 12 weeks、48 weeks
The Perianal Disease Activity Index score at week 12/48 of treatment
Perianal Disease Activity Index(PDAI) is the core tool for evaluating perianal lesions in Crohn's disease, with a total score ranging from 0 to 20. A higher score indicates more severe perianal disease activity and a poorer prognosis
Time frame: 12 weeks、48 weeks
The van Assche Magnetic Resonance Imaging (MRI) Score of anal fistula at week 12/48 of treatment.
van Assche Magnetic Resonance Imaging (MRI) Score is an imaging assessment tool for Crohn's disease intestinal lesions, with a total score of 0-30 points. High scores reflect more severe intestinal damage and poorer clinical outcomes.
Time frame: 12 weeks、48 weeks
The CAF-QoL score of anal fistula at week 12/48 of treatment.
Complex Anal Fistula Quality of Life Questionnaire of anal fistula is a specific assessment tool used to evaluate the quality of life in patients with anal fistula, with scores typically ranging from 0 to 60 points. Higher scores indicate greater impairment in quality of life and poorer clinical outcomes
Time frame: 12 weeks、48 weeks
The CDAI of CD at week 12/48 of treatment.
Crohn's Disease Activity Index(CDAI) is a tool for assessing the activity level of Crohn's disease, with a score range of 0 to 600. A higher score indicates a worse disease state.
Time frame: 12 weeks、48 weeks
The mucosal healing rate (SES-CD) at week 12/48 of treatment.
Simple Endoscopic Score for Crohn's Disease(SES-CD) is the core assessment tool for evaluating the severity of endoscopic lesions in Crohn's disease, with a total score ranging from 0 to 60 points. Higher scores directly reflect more severe mucosal damage and poorer clinical outcomes.
Time frame: 12 weeks、48 weeks
Safety evaluation within 56 weeks of treatment.
Safety was evaluated at each follow-up, with adverse events recorded. The safety assessments included the name, frequency, and severity of adverse events, vital signs, physical examination findings, and laboratory test results.
Time frame: 56 weeks
Started Jan 2024 (actual). Primary completion is estimated for Dec 2025, 23 months after the start.
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Studies of the drug tested here: Upadacitinib
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