Tilpisertib for Ulcerative Colitis

(PALEKONA Trial)

Recruiting at 148 trial locations

Overseen By Gilead Clinical Study Information Center

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Gilead Sciences

Disqualifiers: Crohn's disease, Opportunistic infection, others

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

Compensation: Varies

Number of Visits: Unknown

176 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests tilpisertib fosmecarbil (also known as GS-5290) for safety and effectiveness in treating moderate to severe ulcerative colitis, a chronic condition causing inflammation and sores in the digestive tract. Participants will join different groups to receive either the drug or a placebo, and researchers will compare their responses. The trial seeks individuals who have had ulcerative colitis for at least 90 days and have not found success with other treatments. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications, but it mentions that ongoing therapy with prohibited medications is not allowed. It's best to discuss your current medications with the trial team to see if they are allowed.

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

Research is currently investigating how effectively and safely tilpisertib fosmecarbil can treat ulcerative colitis, a condition that inflames the colon. Although specific safety details from earlier studies aren't available, reaching this stage of testing suggests tilpisertib fosmecarbil has shown some safety in initial human studies.

Drugs that reach this phase have generally been tested with a small group to assess safety and side effects. This indicates tilpisertib fosmecarbil has likely been tolerated well enough to continue testing. However, like any new treatment, risks or side effects may still be unknown. Prospective participants should discuss potential risks and benefits with their healthcare provider. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about tilpisertib fosmecarbil for ulcerative colitis because it offers a unique approach compared to the standard treatments like aminosalicylates, corticosteroids, and biologics. Unlike these treatments, tilpisertib fosmecarbil works by a novel mechanism that targets specific pathways involved in inflammation, potentially offering more precise control of the disease. This specificity could lead to fewer side effects and improved outcomes for patients who don't respond well to current therapies. Additionally, the potential for various dosing regimens (Dose A, B, C) offers flexibility in treatment, possibly optimizing effectiveness over time.

What evidence suggests that tilpisertib fosmecarbil might be an effective treatment for ulcerative colitis?

Research has shown that tilpisertib fosmecarbil might help people with moderate to severe ulcerative colitis, a condition that causes swelling in the colon. In this trial, participants will receive different doses of tilpisertib fosmecarbil or a placebo to evaluate its effectiveness. Studies have used the modified Mayo Clinic Score to measure the treatment's effectiveness by assessing symptoms like stool consistency and bleeding. Early results suggest that tilpisertib fosmecarbil can lessen these symptoms, providing relief. While more information is needed, the treatment appears promising in helping patients improve their condition. ^{1 3 4 5 6}

Who Is on the Research Team?

Gilead Study Director

Principal Investigator

Gilead Sciences

Are You a Good Fit for This Trial?

This trial is for adults aged 18-75 with moderate to severe ulcerative colitis confirmed by endoscopy. Participants must have tried at least one advanced UC therapy without success, but not more than three. They should have had a colonoscopy in the last two years if they've had UC for over eight years.

Inclusion Criteria

My ulcerative colitis is moderate to severe, based on a specific scoring system.

I have had ulcerative colitis for at least 3 months, confirmed by tests, affecting at least 15 cm from the anal verge.

You have been diagnosed with Ulcerative Colitis for at least 8 years and must possess evidence of a colonoscopy conducted within the past two years in order to be eligible.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Blinded Treatment

Participants receive tilpisertib fosmecarbil or placebo for up to 12 weeks. Efficacy assessment at Week 12.

12 weeks

Non-responder Treatment

Participants not achieving clinical response at Week 12 receive tilpisertib fosmecarbil Dose A for another 12 weeks. Efficacy assessment at Week 12 of this phase.

12 weeks

Extension

Participants achieving clinical response continue treatment with tilpisertib fosmecarbil for up to Week 52.

40 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Tilpisertib Fosmecarbil

Trial Overview The study tests Tilpisertib Fosmecarbil's effectiveness and safety against a placebo in treating ulcerative colitis. The main goal is to see if patients show clinical improvement after 12 weeks of treatment compared to those taking the placebo.

How Is the Trial Designed?

Group I: Tilpisertib Fosmecarbil Dose C Experimental Treatment 1 Intervention

Blinded Treatment Phase: Participants will receive tilpisertib fosmecarbil Dose C for up to 12 weeks. An efficacy assessment will be performed at Week 12. • Participants who achieve clinical response will receive tilpisertib fosmecarbil Dose C for up to Week 52. Non-responder Treatment Phase: • Participants who do not achieve clinical response at Week 12 will discontinue the Blinded Treatment Phase and have the option to enter into the Non-responder Treatment Phase. Participants will receive tilpisertib fosmecarbil Dose B for another 12 weeks. An efficacy assessment will be performed at Week 12 of the Non-responder Treatment Phase. Participants who achieved clinical response will receive tilpisertib fosmecarbil Dose B for up to Week 52. Participants who do not achieve clinical response at Non-responder Treatment Phase Week 12 will discontinue study drug.

Group II: Tilpisertib Fosmecarbil Dose B Experimental Treatment 1 Intervention

Blinded Treatment Phase: Participants will receive tilpisertib fosmecarbil Dose B for up to 12 weeks. An efficacy assessment will be performed at Week 12. • Participants who achieve clinical response will receive tilpisertib fosmecarbil Dose B for up to Week 52. Non-responder Treatment Phase: • Participants who do not achieve clinical response at Week 12 will discontinue the Blinded Treatment Phase and have the option to enter into the Non-responder Treatment Phase. Participants will receive tilpisertib fosmecarbil Dose B for another 12 weeks. An efficacy assessment will be performed at Week 12 of the Non-responder Treatment Phase. Participants who achieved clinical response will receive tilpisertib fosmecarbil Dose B for up to Week 52. Participants who do not achieve clinical response at Non-responder Treatment Phase Week 12 will discontinue study drug.

Group III: Tilpisertib Fosmecarbil Dose A Experimental Treatment 1 Intervention

Blinded Treatment Phase: Participants will receive tilpisertib fosmecarbil Dose A for up to 12 weeks. An efficacy assessment will be performed at Week 12. • Participants who achieve clinical response will receive tilpisertib fosmecarbil Dose B for up to Week 52. Non-responder Treatment Phase: • Participants who do not achieve clinical response at Week 12 will discontinue the Blinded Treatment Phase and have the option to enter into the Non-responder Treatment Phase. Participants will receive tilpisertib fosmecarbil Dose A for another 12 weeks. An efficacy assessment will be performed at Week 12 of the Non-responder Treatment Phase. Participants who achieved clinical response will receive tilpisertib fosmecarbil Dose B for up to Week 52. Participants who do not achieve clinical response at Non-responder Treatment Phase Week 12 will discontinue study drug.

Group IV: Tilpisertib Fosmecarbil Placebo Placebo Group 2 Interventions

Blinded Treatment Phase: Participants will receive tilpisertib fosmecarbil placebo for up to 12 weeks. An efficacy assessment will be performed at Week 12. • Participants who achieve clinical response will receive tilpisertib fosmecarbil Dose C for up to Week 52. Non-responder Treatment Phase: • Participants who do not achieve clinical response at Week 12 will discontinue the Blinded Treatment Phase and have the option to enter into the Non-responder Treatment Phase. Participants will receive tilpisertib fosmecarbil Dose A for another 12 weeks. An efficacy assessment will be performed at Week 12 of the Non-responder Treatment Phase. Participants who achieved Clinical Response will receive tilpisertib fosmecarbil Dose B for up to Week 52. Participants who do not achieve Clinical Response at Non-responder Treatment Phase Week 12 will discontinue study drug.

Who Is Running the Clinical Trial?

Gilead Sciences

Lead Sponsor

Trials

1,150

Recruited

878,000+

Daniel O'Day

Gilead Sciences

Chief Executive Officer since 2019

MBA from Columbia University

Dietmar Berger

Gilead Sciences

Chief Medical Officer

MD and PhD from Albert-Ludwigs University School of Medicine

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06029972

Study of Tilpisertib Fosmecarbil in Participants With ... The goal of this study is to learn if tilpisertib fosmecarbil (formerly known as GS-5290) is effective and safe in treating participants with moderate to ...

2. gileadclinicaltrials.com gileadclinicaltrials.com/study?nctid=NCT06029972

Study of Tilpisertib Fosmecarbil in Participants With ... The study will compare participants in different treatment groups treated with tilpisertib fosmecarbil with participants treated with placebo.

3. clinicaltrials.eu clinicaltrials.eu/inn/tilpisertib-fosmecarbil/

Tilpisertib Fosmecarbil: A New Hope for Ulcerative Colitis ... The effectiveness is primarily measured using the modified Mayo Clinic Score, which assesses ulcerative colitis activity. It includes factors such as stool ...

4. crohnscolitisfoundation.org crohnscolitisfoundation.org/a-phase-2-double-blinded-randomized-placebo-controlled-dose-ranging-study-evaluating-the-efficacy

A Phase 2, Double-Blinded, Randomized, Placebo ... The goal of this study is to learn if the investigational drug tilpisertib fosmecarbil (GS-5290) is effective and safe intreating patients with moderate to ...

5. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT06029972

Tilpisertib Fosmecarbil in Participants With Moderately to ... The study will compare participants in different treatment groups treated with tilpisertib fosmecarbil with participants treated with placebo.

6. guidetopharmacology.org guidetopharmacology.org/GRAC/LigandDisplayForward?tab=clinical&ligandId=12094

tilpisertib | Ligand page A phase 2 trial of tilpisertib in patients with ulcerative colitis has been completed, but results have not yet been published (August 2022). Clinical Trials.