Atorvastatin for Ulcerative Colitis

Not currently recruiting at 4 trial locations

Overseen By Guang-Yu Yang, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Northwestern University

Must be taking: Mesalamine, Thiopurines, Biologics

Must not be taking: Corticosteroids, Statins, Cyclosporine, others

Disqualifiers: Ulcerative proctitis, HIV, Pregnancy, others

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 6 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines how atorvastatin, a drug typically used to lower cholesterol, might benefit people with ulcerative colitis who have a specific genetic mutation that raises their risk of colon cancer. Researchers aim to determine if atorvastatin can help prevent cancer in these patients, who already face an increased cancer risk due to their ulcerative colitis. The trial includes two groups: one taking atorvastatin and the other a placebo (a non-active pill) for a year. People with ulcerative colitis in remission for over 8 years, who are stable on their current treatment, and have a history of certain colon issues might be suitable candidates for the trial. As a Phase 2 trial, this research focuses on assessing atorvastatin's effectiveness in an initial, smaller group, offering a chance to contribute to significant findings.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but you must not have used statins in the last 12 months or corticosteroids in the past 3 months. You also cannot use certain medications like cyclosporine, fibrates, or strong CYP3A4 inhibitors or inducers.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that atorvastatin is generally safe for patients. One study found that people with ulcerative colitis who took atorvastatin experienced improvements in their condition without more side effects than those who didn't take it. Another study discovered that long-term use of atorvastatin reduced the need for surgery to remove part or all of the colon in these patients. These findings suggest that atorvastatin is safe for people with ulcerative colitis. It is already approved for other uses, such as lowering cholesterol and preventing heart problems. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ulcerative colitis?

Atorvastatin is unique because it offers a fresh approach to treating ulcerative colitis by utilizing a cholesterol-lowering medication, commonly used for heart conditions, to potentially reduce inflammation in the colon. Unlike standard treatments for ulcerative colitis, which include anti-inflammatory drugs like mesalamine or immunosuppressants, atorvastatin may work by modulating the immune response differently. Researchers are excited about its potential to not only manage symptoms but also address underlying inflammation through a novel mechanism that could complement existing therapies.

What evidence suggests that atorvastatin could be an effective treatment for ulcerative colitis?

Research has shown that atorvastatin, which participants in this trial may receive, might help with ulcerative colitis (UC) by improving gut health and aiding in healing. One study found that atorvastatin reduced inflammation and eased symptoms in people with UC. Another study discovered that when used alongside other UC treatments, atorvastatin improved patients' quality of life and further reduced inflammation. These findings suggest that atorvastatin could be a promising option for managing UC. Participants in this trial will receive either atorvastatin or a placebo to evaluate its effectiveness. ^{1 2 4 6 7}

Who Is on the Research Team?

Stephen B Hanauer, MD

Principal Investigator

Northwestern University

Are You a Good Fit for This Trial?

Adults aged 18-70 with longstanding ulcerative colitis and a specific P53 mutation, in clinical remission for at least 8 years. They must not have used statins or had chemotherapy within the last year or two respectively, no recent corticosteroid use, and cannot be pregnant. Participants should have stable cholesterol levels and agree to contraception during the study.

Inclusion Criteria

Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (SGPT) =< 1.5 X institutional upper limit of normal (ULN)

I have had ulcerative colitis for over 8 years and am in remission, confirmed by yearly endoscopy.

My ulcerative colitis is in remission, and any precancerous growths were fully removed.

Exclusion Criteria

Pregnant or lactating participants are excluded

I have used corticosteroids in the last 3 months due to a high risk of my disease returning.

I have not had chemotherapy in the last 2 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive atorvastatin or placebo orally once daily for 12 months, with colonoscopy and blood collection

12 months

Monthly visits for monitoring and sample collection

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

2 weeks

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Atorvastatin Calcium
• Placebo Administration

Trial Overview The trial is testing if Atorvastatin can lower colon cancer risk in patients with ulcerative colitis who are at increased risk of large intestinal cancer. It involves taking Atorvastatin versus a placebo while monitoring health through biopsies, blood tests, questionnaires, and biospecimen collection.

How Is the Trial Designed?

Group I: Arm I (atorvastatin, biospecimen collection) Experimental Treatment 4 Interventions

Patients receive atorvastatin PO QD for 12 months. Patients also undergo colonoscopy with biopsy, and collection of blood on the trial.

Group II: Arm II (placebo, biospecimen collection) Placebo Group 4 Interventions

Patients receive placebo PO QD for 12 months. Patients also undergo colonoscopy with biopsy, and collection of blood on the trial.

Atorvastatin Calcium is already approved in European Union, United States, Canada, Japan, China, Switzerland for the following indications:

Approved in European Union as Lipitor for:

• Hypercholesterolemia
• Mixed dyslipidemia
• Prevention of cardiovascular disease

Approved in United States as Lipitor for:

• Hypercholesterolemia
• Mixed dyslipidemia
• Prevention of cardiovascular disease
• Reduction of risk of myocardial infarction, stroke, angina, and revascularization procedures

Approved in Canada as Lipitor for:

• Hypercholesterolemia
• Mixed dyslipidemia
• Prevention of cardiovascular disease

Approved in Japan as Lipitor for:

• Hypercholesterolemia
• Mixed dyslipidemia
• Prevention of cardiovascular disease

Approved in China as Lipitor for:

• Hypercholesterolemia
• Mixed dyslipidemia
• Prevention of cardiovascular disease

Approved in Switzerland as Lipitor for:

• Hypercholesterolemia
• Mixed dyslipidemia
• Prevention of cardiovascular disease

Who Is Running the Clinical Trial?

Northwestern University

Lead Sponsor

Trials

1,674

Recruited

989,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Published Research Related to This Trial

In a study of 497 ulcerative colitis patients, those with dyslipidaemia exhibited more severe disease activity, particularly with low levels of high-density lipoprotein (HDL) cholesterol, which was linked to increased risks of severe disease (p < 0.05).

Patients with persistent dyslipidaemia faced significantly higher risks of needing surgery (HR: 3.27) and developing tumors (HR: 7.92), along with shorter surgery- and tumor-free survival, highlighting the importance of monitoring lipid levels for better management of ulcerative colitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dyslipidaemia Is Associated with Severe Disease Activity and Poor Prognosis in Ulcerative Colitis: A Retrospective Cohort Study in China. Liu, Z., Tang, H., Liang, H., et al.[2023]

In a study on rats with TNBS-induced ulcerative colitis, both simvastatin and rosuvastatin significantly reduced colonic mucosal damage and inflammation, as indicated by lower disease activity index scores and reduced neutrophil accumulation.

The beneficial effects of simvastatin and rosuvastatin were attributed to their anti-inflammatory and antioxidant properties, suggesting they may be effective treatments for ulcerative colitis beyond their cholesterol-lowering effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Protective effect of simvastatin and rosuvastatin on trinitrobenzene sulfonic acid-induced colitis in rats. Maheshwari, RA., Balaraman, R., Sailor, GU., et al.[2018]

A 41-year-old man developed severe gastric ulceration after 3 months of taking atorvastatin for high cholesterol, marking the first reported case of this side effect.

Switching from atorvastatin to simvastatin and starting proton pump inhibitors led to complete resolution of the patient's gastrointestinal symptoms, highlighting the need for awareness of potential gastric issues in patients on atorvastatin.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Atorvastatin-induced severe gastric ulceration: a case report. El-Hajj, II., Mourad, FH., Shabb, NS., et al.[2019]

In a long-term study involving 1124 patients with ulcerative colitis treated with tofacitinib, lipid levels remained stable over time, indicating that the medication does not significantly alter cholesterol or triglyceride levels.

The incidence of major adverse cardiovascular events was very low at 0.26 per 100 patient-years, suggesting that tofacitinib is safe in terms of cardiovascular risk for patients with ulcerative colitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lipid Profiles in Patients With Ulcerative Colitis Receiving Tofacitinib-Implications for Cardiovascular Risk and Patient Management. Sands, BE., Colombel, JF., Ha, C., et al.[2022]

The combination of mesalazine and atorvastatin significantly improved symptoms of ulcerative colitis in rats, reducing diarrhea, rectal bleeding, and weight loss while enhancing colon health compared to single drug treatments.

This combination therapy worked by decreasing inflammatory markers like IL-6 and TNF-α and increasing protective factors such as IL-10 and ZO-1, suggesting a mechanism that restores balance in the colon's inflammatory response.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Targeting IL-10, ZO-1 gene expression and IL-6/STAT-3 trans-signaling by a combination of atorvastatin and mesalazine to enhance anti-inflammatory effects and attenuates progression of oxazolone-induced colitis. El-Mahdy, NA., El-Sayad, ME., El-Kadem, AH., et al.[2021]

In a study involving 30 female mice with colitis induced by DSS, both simvastatin and atorvastatin significantly reduced disease activity, with reductions of 64% and 76% respectively, compared to a 41% reduction in the control group.

The effectiveness of statins in modifying disease activity in this colitis model was found to be comparable to that of an anti-TNFalpha treatment, suggesting a potential therapeutic role for statins in inflammatory bowel disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Disease modifying effect of statins in dextran sulfate sodium model of mouse colitis. Kanagarajan, N., Nam, JH., Noah, ZA., et al.[2018]

Statins and omega-3 polyunsaturated fatty acids were found to positively influence lipid levels and acute phase proteins in patients with ulcerative colitis, suggesting potential benefits for managing this condition.

The study highlights the importance of these interventions in addressing subclinical atherosclerosis, indicating a dual role in both inflammatory bowel disease and cardiovascular health.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Treatment optimization of subclinical atherosclerosis in patients with ulcerative colitis]. Chopeĭ, IV., Ternushchak, TM., Chubirko, KI., et al.[2015]

In a study of 64 patients with mild to moderately severe acute exacerbation of ulcerative colitis (UC), atorvastatin did not show significant clinical improvement compared to placebo, with only 25% of patients in the atorvastatin group meeting the primary outcome criteria for improvement.

Interestingly, atorvastatin was associated with a paradoxical increase in disease activity in some patients, highlighting the need for caution and further research in larger studies to confirm these findings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized clinical trial: atorvastatin versus placebo in patients with acute exacerbation of mild to moderate ulcerative colitis. Dhamija, P., Hota, D., Kochhar, R., et al.[2021]

Citations

1. sciencedirect.com sciencedirect.com/science/article/pii/S0024320524003801

Atorvastatin improved ulcerative colitis in association with ... Atorvastatin could alleviate UC by regulating intestinal flora disorders, promoting microbial tryptophan metabolism, and repairing the intestinal barrier.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8510297/

Services Unavailable for 24+ Hours Starting 9 PM EDT Atorvastatin decreases Seo Index in patients with short duration of disease in ulcerative colitis: a randomized placebo-controlled clinical trial.

3. frontiersin.org frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1690513/full

Atorvastatin as an immunomodulatory adjunct in ulcerative ... Conclusion: Adjunctive atorvastatin with 5-ASA significantly improved clinical disease activity, quality of life, and inflammatory biomarkers ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8247597/

Association Between Statin Use and Inflammatory Bowel ... Statin use was associated with a lower risk of CD, but not of UC. The association with CD risk appeared strongest for current statin use.

5. journals.lww.com journals.lww.com/ajg/fulltext/2025/10002/s1622_comparative_effectiveness_of_statins_in.1622.aspx

S1622 Comparative Effectiveness of Statins in Inflammatory... Among high-intensity statins, rosuvastatin was associated with significantly lower all-cause mortality compared to atorvastatin in IBD (HR 0.73, P < 0.001), CD ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05567068

NCT05567068 | Atorvastatin Efficacy and Safety in Patients ... Study Details | NCT05567068 | Atorvastatin Efficacy and Safety in Patients With Ulcerative Colitis | ClinicalTrials.gov.

7. healio.com healio.com/news/gastroenterology/20210916/lipitor-reduces-colectomy-rate-in-ulcerative-colitis

Lipitor reduces colectomy rate in ulcerative colitis “Long-term atorvastatin (Lipitor) use is associated with reduced rate of colectomy in patients with UC,” Purvesh Khatri, PhD, from the Institute ...