Mirikizumab for Pediatric Crohn's Disease

(AMAY Trial)

Recruiting at 154 trial locations

Overseen By Physicians interested in becoming principal investigators please contact

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Eli Lilly and Company

Disqualifiers: Symptomatic strictures, Stenosis, Short gut, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 19

90 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called mirikizumab (also known as Omvoh) to evaluate its effectiveness and safety for children with Crohn's disease. Depending on the child's weight, mirikizumab will be administered either through a vein (IV) or as an injection under the skin (subcutaneously). The trial will last about 74 weeks and include around 19 visits. Children with moderate to severe Crohn's disease who have not improved with other treatments might be suitable candidates for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering early access to a potentially effective treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, since participants must have a history of inadequate response or intolerance to certain medications, it's possible that some adjustments might be needed. Please consult with the trial coordinators for specific guidance.

Is there any evidence suggesting that mirikizumab is likely to be safe for pediatric patients with Crohn's disease?

Studies have shown that mirikizumab is generally safe for people with Crohn's disease. The safety profile of mirikizumab, which refers to its effects on people, is considered favorable, meaning patients usually tolerate it well. Previous research confirmed the known safety results of the treatment. This is encouraging for those considering a clinical trial for mirikizumab, as it suggests that any side effects might be manageable. However, individual reactions can vary, so discussing any concerns with healthcare providers is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Crohn's disease?

Researchers are excited about Mirikizumab for pediatric Crohn's disease because it offers a novel approach by targeting the interleukin (IL)-23 pathway. Unlike the standard treatments like corticosteroids, immunomodulators, or TNF inhibitors, Mirikizumab specifically blocks IL-23, a protein involved in inflammation, potentially leading to fewer side effects and more targeted efficacy. With its flexible administration methods—either intravenously or subcutaneously—and weight-based dosing, it promises a more personalized treatment experience for young patients. This innovative mechanism and adaptable delivery could make Mirikizumab a promising option for managing Crohn's disease in children.

What evidence suggests that this trial's treatments could be effective for pediatric Crohn's disease?

Research has shown that mirikizumab holds promise for treating Crohn's disease. In studies, over 90% of patients without active symptoms after one year remained symptom-free after two years of ongoing treatment. Additionally, 78% of patients stopped using steroid medications to control their symptoms. The treatment matched existing safety data. This trial will evaluate different doses of mirikizumab based on weight categories to determine its effectiveness and safety in children with Crohn's disease. These results strongly support mirikizumab as an effective option for managing Crohn's disease, offering hope for children with the condition. ^{1 5 6 7 8}

Who Is on the Research Team?

Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 8 AM - 8 PM Eastern time (UTC/GMT - 5 hours, EST)

Principal Investigator

Eli Lilly and Company

Are You a Good Fit for This Trial?

This trial is for pediatric patients with moderately to severely active Crohn's Disease, confirmed by specific medical criteria. They should have tried at least one treatment without success or could not tolerate it. Kids who've had recent bowel surgery or have complications like strictures that might need surgery can't join.

Inclusion Criteria

I have Crohn's disease and treatments I've tried haven't worked well for me.

My recent endoscopy shows active Crohn's disease.

I have been diagnosed with Crohn's disease or fistulizing Crohn's disease affecting my colon, ileum, or both.

Exclusion Criteria

I do not have severe Crohn's disease complications that might need surgery.

I do not have an abscess.

I haven't had bowel surgery in the last 6 months or any abdominal surgery in the last 3 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Induction

Participants receive mirikizumab or another intervention to evaluate efficacy, safety, and tolerability

12 weeks

Up to 6 visits (in-person)

Maintenance

Participants continue treatment to assess long-term efficacy and safety

40 weeks

Up to 10 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

16 weeks

3 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Mirikizumab

Trial Overview The study tests Mirikizumab's effectiveness and safety in children with Crohn's Disease over approximately 74 weeks, including a 12-week induction phase and up to Week 52 maintenance period, followed by a safety follow-up. Participants are randomly assigned to receive either Mirikizumab or another intervention.

How Is the Trial Designed?

Group I: Mirikizumab Dose 3 Experimental Treatment 1 Intervention

Mirikizumab administered IV or SC in participants that weigh greater than or equal to (≥) 9 kg to less than or equal to ≤20 kg. Dosing is based on assessments of the participant's weight and appropriate weight class.

Group II: Mirikizumab Dose 2 Experimental Treatment 1 Intervention

Mirikizumab administered IV or SC in participants that weigh \>20 kg to less than or equal to (≤) 40 kg. Dosing is based on assessments of the participant's weight and appropriate weight class.

Group III: Mirikizumab Dose 1 Experimental Treatment 1 Intervention

Mirikizumab administered intravenously (IV) or subcutaneously (SC) in participants that weigh greater than (\>) 40 kilograms (kg).

Mirikizumab is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Omvoh for:

• Moderately to severely active ulcerative colitis

Approved in United States as Omvoh for:

• Moderately to severely active ulcerative colitis

Approved in Canada as Omvoh for:

• Moderately to severely active ulcerative colitis

Approved in Japan as Omvoh for:

• Moderately to severely active ulcerative colitis

Who Is Running the Clinical Trial?

Eli Lilly and Company

Lead Sponsor

Trials

2,708

Recruited

3,720,000+

Dr. Daniel Skovronsky

Eli Lilly and Company

Chief Medical Officer since 2018

MD from Harvard Medical School

David A. Ricks

Eli Lilly and Company

Chief Executive Officer since 2017

BSc from Purdue University, MBA from Indiana University

Published Research Related to This Trial

Mirikizumab, an anti-interleukin-23 treatment, significantly improved histological and endoscopic outcomes in patients with moderately-to-severely active ulcerative colitis, showing better results compared to placebo in a study involving 1162 patients at Week 12 and 544 patients at Week 40.

Achieving early endoscopic and histological improvements with mirikizumab was linked to better long-term outcomes, including clinical remission and reduced symptoms, highlighting the importance of early intervention in managing ulcerative colitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Resolving Histological Inflammation in Ulcerative Colitis With Mirikizumab in the LUCENT Induction and Maintenance Trial Programmes. Magro, F., Pai, RK., Kobayashi, T., et al.[2023]

In a study involving patients with moderate to severe ulcerative colitis who initially did not respond to mirikizumab after 12 weeks, 50% of those receiving an additional 12 weeks of 600 mg mirikizumab achieved a clinical response, indicating that extended treatment can be effective for nonresponders.

Among those who responded to the extended treatment, 65.8% maintained their clinical response for up to 52 weeks, demonstrating the long-term efficacy of mirikizumab without new safety concerns.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Continued Treatment With Mirikizumab in a Phase 2 Trial of Patients With Ulcerative Colitis. Sandborn, WJ., Ferrante, M., Bhandari, BR., et al.[2022]

In a study of 191 patients with moderate-to-severe Crohn's disease, mirikizumab significantly improved endoscopic response at Week 12 compared to placebo, with the highest response rate observed in the 1000 mg group (43.8%).

Mirikizumab demonstrated durable efficacy, maintaining a high endoscopic response rate of around 58.5% at Week 52, while the safety profile was comparable to placebo, indicating it is a promising treatment option for Crohn's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Mirikizumab in a Randomized Phase 2 Study of Patients With Crohn's Disease. Sands, BE., Peyrin-Biroulet, L., Kierkus, J., et al.[2022]

In a study of 100 patients with moderate-to-severe Crohn's disease who had previously failed multiple treatments, risankizumab achieved a steroid-free clinical remission rate of 45.8% and a clinical response rate of 78.5% by week 12, indicating its effectiveness in a real-world setting.

The treatment was generally well-tolerated, with 20 adverse events reported, including 7 serious cases, but the majority of patients experienced significant improvements in their condition, highlighting risankizumab's potential as a viable option for refractory Crohn's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness and safety of risankizumab induction therapy for 100 patients with Crohn's disease: A GETAID multicentre cohort study. Fumery, M., Defrance, A., Roblin, X., et al.[2023]

Mirikizumab, an anti-interleukin-23 monoclonal antibody, demonstrated pharmacokinetic properties typical of monoclonal antibodies, with a half-life of approximately 9.5 days and a subcutaneous bioavailability of 48%, based on data from 1362 patients with ulcerative colitis.

The study found that while body weight and serum albumin levels influenced the drug's clearance and distribution, these effects were relatively small compared to individual variability, suggesting that no dose adjustments are necessary based on patient characteristics.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mirikizumab Pharmacokinetics in Patients with Moderately to Severely Active Ulcerative Colitis: Results from Phase III LUCENT Studies. Chua, L., Friedrich, S., Zhang, XC.[2023]

In a study of 170 participants with Crohn's disease, mirikizumab treatment led to significantly higher rates of histologic response (66% vs 27%) and remission (26% vs 6%) at 12 weeks compared to placebo, indicating its efficacy in promoting mucosal healing.

The study found that early histologic and endoscopic responses were associated with better long-term outcomes, as participants who showed combined responses at week 12 had a higher rate of endoscopic remission at one year (45% vs 4% for those with no response).

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of Mirikizumab on Histologic Resolution of Crohn's Disease in a Randomized Controlled Phase 2 Trial. Magro, F., Protic, M., De Hertogh, G., et al.[2023]

Mirikizumab, an anti-IL-23p19 monoclonal antibody, was approved in March 2023 in Japan for treating moderate to severe ulcerative colitis, marking it as the first IL-23p19 inhibitor approved for this condition.

In the EU, mirikizumab received a positive opinion for adult patients with active ulcerative colitis who did not respond to conventional or biologic therapies, highlighting its potential as a new treatment option for difficult-to-treat cases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mirikizumab: First Approval. Keam, SJ.[2023]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04844606

Study Details | NCT04844606 | A Master Protocol (AMAZ) ... The main purpose of this study is to evaluate the long-term efficacy of mirikizumab in pediatric participants with ulcerative colitis (UC) or Crohn's ...

2. investor.lilly.com investor.lilly.com/news-releases/news-release-details/most-patients-lillys-omvohr-mirikizumab-mrkz-crohns-disease

Most patients on Lilly's Omvoh® (mirikizumab-mrkz) for ... More than 90% of Omvoh-treated patients who were in clinical remission at one year sustained it with two years of continuous treatment.

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39581202/

Efficacy and safety of mirikizumab in patients with ... The safety of mirikizumab in Crohn's disease was consistent with its known favourable profile. Interpretation: Mirikizumab was safe and ...

4. prnewswire.com prnewswire.com/news-releases/lillys-omvoh-mirikizumab-mrkz-is-the-first-and-only-il-23p19-antagonist-to-show-four-years-of-sustained-corticosteroid-free-comprehensive-patient-outcomes-in-ulcerative-colitis-302576255.html

Lilly's Omvoh (mirikizumab-mrkz) is the first and only IL ... 78% achieved corticosteroid-free clinical remission · 78% sustained long-term clinical remission · 81% sustained endoscopic remission, defined as ...

5. ddw.digitellinc.com ddw.digitellinc.com/p/s/pk-efficacy-and-safety-of-mirikizumab-as-induction-therapy-in-pediatric-patients-with-moderately-to-severely-active-ulcerative-colitis-results-from-the-phase-2-shine-1-study-3117

PK, EFFICACY AND SAFETY OF MIRIKIZUMAB AS ... Mirikizumab was superior to placebo in inducing clinical remission as well as other symptomatic, clinical, and endoscopic endpoints and had an acceptable safety ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03926130

NCT03926130 | A Study of Mirikizumab (LY3074828) in ... The reason for this study is to see if the study drug mirikizumab is safe and effective in participants with moderately to severely active Crohn's disease.

7. investor.lilly.com investor.lilly.com/node/51431/pdf

Lilly reports one-year histologic outcomes in Phase 3 study ... The overall safety profile of mirikizumab in patients with moderately to severely active Crohn's disease was consistent with the known safety ...

8. lilly.gcs-web.com lilly.gcs-web.com/news-releases/news-release-details/lillys-omvoh-mirikizumab-mrkz-demonstrated-early-and-sustained

Lilly's Omvoh (mirikizumab-mrkz) demonstrated early and ... Lilly's Omvoh (mirikizumab-mrkz) demonstrated early and sustained improvement in bowel urgency outcomes for patients with ulcerative colitis.