Crohn DiseasePhase 2NCT03752970
A Study Testing How BI 655130 Works in Patients With Fistulizing Crohn's Disease
A Phase 2 study testing Spesolimab in people with Crohn Disease, run by Boehringer Ingelheim at 10 sites in 8 countries.
Trial summary
This is a study in adults with Crohn's Disease who also have fistulas near the anus. The study has 2 parts. The first part is to find out more about what causes the fistulas. In this part of the study, tissue samples are taken from patients. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Crohn Disease
- What is being tested?
- Spesolimab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 10 sitesin 8 countries
- Study dates
- 5 Feb 2019to 4 Jul 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18-75 years at date of signing informed consent
- Male or female patients. Women of childbearing potential (WOCBP)1 must be ready and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. Restrictions regarding women of childbearing potential. Restrictions regarding contraception for female patients are not applicable for Screening Cohort
- Diagnosis of clinical Crohn´s Disease ≥ 3 months prior to screening by clinical and endoscopic evidence and corroborated by a histopathology report
- Has ≥ 1 perianal active* fistula(s) with clinical indication for seton drainage (≥ 4 weeks duration before enrolment, as a complication of CD) **
- Criteria for Active Fistula: As per clinical evaluation: Presence of spontaneous drainage or drainage after gentle finger compression at the external openings \& as confirmed by radiological (MRI) exploration
- Patients who are screened with a seton drainage in place are eligible provided the drainage has not been in place for > 3 months and the patient meets the rest of the eligibility criteria
Show all 49Show fewer
- Absent, mild or moderate clinical activity with CDAI < 250. CDAI is not applicable for Screening Cohort
- Demonstrated in the past inadequate fistula response or loss of response or have had unacceptable side effects with approved doses of at least one of the following compounds: immunosuppressive agents (e.g. thiopurines, methotrexate), TNFɑ antagonists (e.g. infliximab, adalimumab, certolizumab pegol; or respective biosimilars), vedolizumab, ustekinumab, azathioprine and / or antibiotics
- Patients with family history of colorectal cancer or personal history of increased colorectal cancer risk must have had a negative ileocolorectal cancer screening within <1 year prior to screening per local guidance
- Signed and dated written informed consent in accordance with ICH-GCP and local legislation prior to admission to the trial
- Further inclusion criteria apply
- Gastrointestinal Exclusion Criteria (Study cohort only)
- Complications of Crohn's Disease such as symptomatic strictures, functional stenosis distal from fistula(s), short gut syndrome, or any other manifestation that might require surgery, could preclude the use of the PDAI and CDAI to assess response to therapy, or would possibly confound the evaluation of benefit from treatment with BI 655130
- Rectovaginal fistulas
- Anticipated to require surgical intervention for CD including any fistula surgical procedures (except seton drainage)
- Has an abscess that the investigator feels requires drainage beyond fistula drainage with a seton (based on either clinical assessment or MRI)
- Any kind of bowel resection or diversion within 6 months or any other intraabdominal surgery within 3 months prior to screening.
- Ileostomy, colostomy or known fixed symptomatic stenosis of the intestine at screening
- Positive stool examinations for C. difficile or other intestinal pathogens < 30 days prior to screening
- -- Evidence of colonic mucosal dysplasia or colonic adenomas, unless properly removed (properly according to the investigator's assessment)
- Faecal Microbiota transplant (FMT) within 6 months prior to randomization
- Treatment with:
- any non-biologic medication (incl. cyclosporine, JAK inhibitors such as tofacitinib, tacrolimus, sirolimus, mycophenolate mofetile, S1P modulators, SMAD7 antisense inhibitors such as mongersen), other than those allowed per chapter 4.2.1 within 30 days prior to randomisation unless these patients show an undetectable plasma concentration
- any biologic treatment approved for CD other than anti-TNFα inhibitors within 8 weeks prior to randomization unless these patients show an undetectable plasma concentration
- any investigational or non-approved biologic for CD (including but not limited to IL-23 inhibitors) within 12 weeks prior to randomisation or etrolizumab within 8 weeks prior to randomization unless these patients show an undetectable plasma concentration
- rectal 5-ASA, rectal Tacrolimus, parenteral or rectal corticosteroids (incl. budesonide) within 2 weeks prior to randomisation
- any antibiotics within 1 week prior to randomisation
- any prior autologous or allogeneic, haematopoietic (HSC) or mesenchymal stem cell (MSC) therapy
- any prior exposure to BI 655130
- any chronic use of NSAID within 2 weeks prior to randomisation (occasional use of NSAIDs and acetaminophen for headache, arthritis, myalgias, menstrual cramps, etc., and daily use of baby or low dose (81-162.5mg) aspirin for cardiovascular prophylaxis are permitted)
- any life-attenuated vaccines within 6 weeks prior to randomization
- Infectious Disease Exclusion Criteria (Study cohort only)
- Increased risk of infectious complications (e.g. due to past organ or stem cell transplantation)
- Live or attenuated vaccination within 6 weeks prior to randomization
- Patients with a positive QuantiFERON TB test during screening are excluded, unless:
- Patient had previous diagnosis of active or latent TB and has completed appropriate treatment per local practice/guidelines within the last 3 years and at least 6 months before first administration of trial medication under this protocol (patients may be re-screened once to meet this criterion)
- Patients with suspected false positive or indeterminate QuantiFERON TB result may be re-tested once
- If Quantiferon not available or providing indeterminate results after repeat testing : A tuberculin skin test reaction ≥10mm (≥5mm if receiving ≥15mg/d prednisone or its equivalent)
- Relevant chronic or acute infections including active tuberculosis, human immunodeficiency virus (HIV) infection or viral hepatitis. A patient can be re-screened if the patient was treated and is cured from the acute infection.
- General Exclusion Criteria (Study cohort only)
- Any documented active or suspected malignancy or history of malignancy within 5 years prior to screening, except appropriately treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ carcinoma of uterine cervix.
- Major surgery (major according to the investigator's assessment) performed within 12 weeks prior to randomization or planned during study conduct, e.g. hip replacement.
- Pathological safety lab parameters: haemoglobin < 8 g/dL, total white blood count (WBC) < 3000 cells/μl, neutrophils < 1000 cells/μl, thrombocytes < 100.000/μl, creatinine ≥ 2 mg/dL, total bilirubin > 2 x ULN with ratio of direct/indirect >1 (patients with Gilbert's syndrome are not excluded), Alkaline Phosphatase >3 x ULN.
- Patients who must or wish to continue the intake of restricted medications or any drug considered likely to interfere with the safe conduct of the trial
- Previous enrolment in this trial (exception: patients of screening cohort may be enrolled in study cohort)
- Currently enrolled in another investigational device or drug trial
- Women who are pregnant, nursing, or who plan to become pregnant in the trial
- Evidence of a current or previous disease, medical condition (including chronic alcohol or drug abuse) other than Crohn´s disease, surgical procedure, medical examination finding (including vital signs and electrocardiogram (ECG)), or laboratory value at the screening visit outside the reference range that in the opinion of the investigator is clinically significant and would make the study participant unreliable to adhere to the protocol or to complete the trial, compromise the safety of the patient, or compromise the quality of the data
- History of allergy/hypersensitivity to the systemically administered trial medication agent or its excipients or to contrast media
Who cannot join
None listed.
Trial locations and contacts
10 sites in 8 countries.
10 sites in 8 countries
Belgium2 sites
UZ Leuven
Leuven, 3000
Centre Hospitalier Universitaire de Liège
Liège, 4000
Germany2 sites
Universitätsklinikum Erlangen
Erlangen, 91054
Universitätsklinikum Ulm
Ulm, 89081
About this study
Official title: Mechanism of Action and Clinical Effect of BI 655130 in Patients With Fistulizing Crohn's Disease
This is a study in adults with Crohn's Disease who also have fistulas near the anus. The study has 2 parts. The first part is to find out more about what causes the fistulas. In this part of the study, tissue samples are taken from patients. The second part of the study tests whether a medicine called spesolimab (BI 655130) helps patients with Crohn's Disease.
Participants get study medication for 24 weeks. The participants are put into 2 groups. It is decided by chance who gets into which group. One group gets an intravenous drip that contains spesolimab every 4 weeks. The other group gets a placebo drip every 4 weeks. The placebo drip looks like the spesolimab drip, but contains no medicine.
The doctors regularly examine fistulas of the participants. The results of the fistula examinations are compared between the groups. The doctors also check the general health of the patients.
- Conditions
- Crohn Disease
- Being tested
- Spesolimab, Placebo
- Medical subject headings
- Crohn Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SpesolimabDrug
Spesolimab 1200 milligram intravenously every 4 weeks (week 0, 4, 8, 12, 16 and 20). At week 12 Placebo patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20).
PlaceboDrug
Placebo intravenously every 4 weeks (week 0, 4, 8). At week 12 achievement of combined perianal fistula remission was determined, patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). Patients with combined perianal fistula remission remained on Placebo and were treated with placebo intravenously every 4 weeks (week 12, 16 and 20).
| Study group | Role | What they receive |
|---|---|---|
| Screening Cohort | No intervention | Patients enrolled in the Screening Cohort did not receive study treatment. This was a feasibility phase for fistula preparation and seton placement to determine the tissue samples that were suitable for analysis of the primary endpoint in the Study Cohort. |
| Study Cohort - Placebo | Placebo comparator | Patients with perianal fistulising Crohn's disease received Placebo intravenously every 4 weeks (week 0, 4, 8). At week 12 achievement of combined perianal fistula remission was determined, patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). Patients with combined perianal fistula remission remained on Placebo and were treated with placebo intravenously every 4 weeks (week 12, 16 and 20). Receives: Drug: Spesolimab; Drug: Placebo |
| Study Cohort - Spesolimab | Experimental | Patients with perianal fistulising Crohn's disease received Spesolimab 1200 milligram intravenously every 4 weeks (week 0, 4, 8, 12, 16 and 20). At week 12 Placebo patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). Receives: Drug: Spesolimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Total Number of Deregulated Genes at Week 4
The total number of deregulated genes based on biopsies from the inner fistula orifice at Week 4 comparing changes in gene expression from baseline between the two treatment groups. For each gene, a repeated measures linear regression model was utilized with treatment (BI 655130 or Placebo), visit (baseline, week 4), treatment by visit interaction as fixed effect and patient as a blocking factor.
Changes will be quantified by log2 fold changes (FC) and associated False Discovery Rate (FDR) adjusted p-values.
Genes will be considered deregulated if they fulfil the following criteria:
- FDR adjusted p-value ≤ 0.05
- |fold change| ≥ 1.5 (|log2 fold change| ≥ 0.58)
Measured: Biopsies taken at screening (Week -3) and at week 4 of treatment.
Secondary outcomes (3)
Number of Patients With Perianal Fistula Response at Week 12
Number of patients with perianal fistula response at Week 12 defined as closure of at least 50% of external openings, no drainage/discharge despite gentle finger compression of fistulas that were draining at baseline and without new emerging fistulas.
The no response imputation (NRI) approach is applied: missing visits where imputed whereby all subsequent visits after a patient took rescue medication for the disease under study or died due to any cause were considered to be missing.
Measured: At baseline (day 1) and week 12 (day 85) of treatment.
Number of Patients With Perianal Fistula Remission at Week 12
Proportion of patients with perianal fistula remission at Week 12 defined as closure of all external openings, no drainage/discharge despite gentle finger compression of fistulas that were draining at baseline and without new emerging fistulas.
The no response imputation (NRI) approach is applied: missing visits where imputed whereby all subsequent visits after a patient took rescue medication for the disease under study or died due to any cause were considered to be missing.
Measured: At baseline (day 1) and week 12 (day 85) of treatment.
Number of Patients With Combined Perianal Fistula Remission at Week 12
Number of patients with combined perianal fistula remission at Week 12 defined as closure of all external openings, no drainage/discharge despite gentle finger compression of fistulas that were draining at baseline and without new emerging fistulas, AND absence collections of >2 centimeter, confirmed by magnetic resonance imaging (MRI) in at least 2 of 3 dimensions - blinded and centrally read.
The no response imputation (NRI) approach is applied: missing visits where imputed whereby all subsequent visits after a patient took rescue medication for the disease under study or died due to any cause were considered to be missing.
Measured: At baseline (day 1) and week 12 (day 85) of treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2018
- Study start5 Feb 2019
- Primary completion1 Dec 2021
- Study completion4 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Crohn DiseaseStudies of the same condition, open and under way.
- More trials run by Boehringer IngelheimThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boehringer Ingelheim
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03752970
- Sponsor's protocol ID
- 1368-0008
- Other IDs
- 2017-003090-34, 2017
- Status last verified
- Oct 2025
- First submitted
- 22 Nov 2018
- Last updated
- 16 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization). For more details refer to: https://www.mystudywindow.com/msw/datatransparency
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mechanism of Action and Clinical Effect of BI 655130 in Patients With Fistulizing Crohn's Disease
- ClinicalTrials.gov ID
- NCT03752970
- Acronym / sponsor ID
- 1368-0008
- Conditions
- Crohn Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Boehringer IngelheimIndustry (pharma, biotech, devices)
- Study start
- 5 Feb 2019 (actual)
- Primary completion
- 1 Dec 2021 (actual)
- Study completion
- 4 Jul 2022 (actual)
- First posted
- 26 Nov 2018 (actual)
- Results
- Posted 16 Feb 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a study in adults with Crohn's Disease who also have fistulas near the anus. The study has 2 parts. The first part is to find out more about what causes the fistulas. In this part of the study, tissue samples are taken from patients. The second part of the study tests whether a medicine called spesolimab (BI 655130) helps patients with Crohn's Disease.
Participants get study medication for 24 weeks. The participants are put into 2 groups. It is decided by chance who gets into which group. One group gets an intravenous drip that contains spesolimab every 4 weeks. The other group gets a placebo drip every 4 weeks. The placebo drip looks like the spesolimab drip, but contains no medicine.
The doctors regularly examine fistulas of the participants. The results of the fistula examinations are compared between the groups. The doctors also check the general health of the patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Screening Cohort | No intervention | –Patients enrolled in the Screening Cohort did not receive study treatment. This was a feasibility phase for fistula preparation and seton placement to determine the tissue samples that were suitable for analysis of the primary endpoint in the Study Cohort. |
| Study Cohort - Placebo | Placebo comparator | Drug: Spesolimab, Drug: PlaceboPatients with perianal fistulising Crohn's disease received Placebo intravenously every 4 weeks (week 0, 4, 8). At week 12 achievement of combined perianal fistula remission was determined, patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). Patients with combined perianal fistula remission remained on Placebo and were treated with placebo intravenously every 4 weeks (week 12, 16 and 20). |
| Study Cohort - Spesolimab | Experimental | Drug: SpesolimabPatients with perianal fistulising Crohn's disease received Spesolimab 1200 milligram intravenously every 4 weeks (week 0, 4, 8, 12, 16 and 20). At week 12 Placebo patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (49 listed)
- 18-75 years at date of signing informed consent
- Male or female patients. Women of childbearing potential (WOCBP)1 must be ready and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. Restrictions regarding women of childbearing potential. Restrictions regarding contraception for female patients are not applicable for Screening Cohort
- Diagnosis of clinical Crohn´s Disease ≥ 3 months prior to screening by clinical and endoscopic evidence and corroborated by a histopathology report
- Has ≥ 1 perianal active* fistula(s) with clinical indication for seton drainage (≥ 4 weeks duration before enrolment, as a complication of CD) **
- Patients who are screened with a seton drainage in place are eligible provided the drainage has not been in place for > 3 months and the patient meets the rest of the eligibility criteria
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SpesolimabSpesolimab 1200 milligram intravenously every 4 weeks (week 0, 4, 8, 12, 16 and 20). At week 12 Placebo patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). | Drug | Study Cohort - Placebo, Study Cohort - Spesolimab |
| PlaceboPlacebo intravenously every 4 weeks (week 0, 4, 8). At week 12 achievement of combined perianal fistula remission was determined, patients without combined perianal fistula remission were switched to spesolimab and were treated with spesolimab 1200 milligram intravenously every 4 weeks (week 12, 16 and 20). Patients with combined perianal fistula remission remained on Placebo and were treated with placebo intravenously every 4 weeks (week 12, 16 and 20). | Drug | Study Cohort - Placebo |
Primary outcome
The Total Number of Deregulated Genes at Week 4
Time frame: Biopsies taken at screening (Week -3) and at week 4 of treatment.
The total number of deregulated genes based on biopsies from the inner fistula orifice at Week 4 comparing changes in gene expression from baseline between the two treatment groups. For each gene, a repeated measures linear regression model was utilized with treatment (BI 655130 or Placebo), visit (baseline, week 4), treatment by visit interaction as fixed effect and patient as a blocking factor. Changes will be quantified by log2 fold changes (FC) and associated False Discovery Rate (FDR) adjusted p-values. Genes will be considered deregulated if they fulfil the following criteria: * FDR adjusted p-value ≤ 0.05 * |fold change| ≥ 1.5 (|log2 fold change| ≥ 0.58)
3 secondary outcomes
Number of Patients With Perianal Fistula Response at Week 12
Time frame: At baseline (day 1) and week 12 (day 85) of treatment.
Number of Patients With Perianal Fistula Remission at Week 12
Time frame: At baseline (day 1) and week 12 (day 85) of treatment.
Number of Patients With Combined Perianal Fistula Remission at Week 12
Time frame: At baseline (day 1) and week 12 (day 85) of treatment.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No. Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization). For more details refer to: https://www.mystudywindow.com/msw/datatransparency
Related trials for Crohn Disease
Active, not recruitingPhase 4
Glucagon-like Peptide-1 Receptor Agonist (GLP-1 RA) and Diet in Inflammatory Bowel Disease (IBD) Patients
Crohn Disease
Enrolling by invitationPhase 4
INSIGHT-IBD Pragmatic Study
Ulcerative Colitis (UC), Crohn Disease (CD)
RecruitingObservational study
Longitudinal Immunophenotyping of Patients With Inflammatory Bowel Disease
Inflammatory Bowel Disease, Crohn's Disease
RecruitingPhase 2
NOV-ERA - A Clinical Trial to Assess the Efficacy and Safety of Ontunisertib Compared to Placebo in Patients With Fibrostenosing Crohn's Disease
Fibrostenotic Crohn's Disease
Recruiting
A Randomized Crossover Trial of Bright Light Therapy in Crohn's Disease on Intestinal Barrier Homeostasis
Inflammatory Bowel Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Spesolimab in people with Crohn Disease, run by Boehringer Ingelheim at 10 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 49 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Belgium, Germany, Austria, Denmark, Hungary and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Total Number of Deregulated Genes at Week 4 (time frame: Biopsies taken at screening (Week -3) and at week 4 of treatment.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2025.