Mirikizumab for Pediatric Crohn's Disease

(AMAY Trial)

Recruiting at 129 trial locations

Overseen By There may be multiple sites in this clinical trial. 1-877-CTLILLY (1-877-285-4559) or

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

Trial Summary

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.

What data supports the effectiveness of the drug Mirikizumab for treating Crohn's disease?

Mirikizumab has shown effectiveness in treating Crohn's disease and ulcerative colitis, which are both inflammatory bowel diseases. It works by targeting a specific protein involved in inflammation, and has been approved for use in ulcerative colitis in Japan and received positive opinions in the EU. ^{1 2 3 4 5}

Is Mirikizumab safe for humans?

Mirikizumab has been tested in people with conditions like ulcerative colitis and Crohn's disease, and it was generally well tolerated in these studies. It is a type of medicine called a monoclonal antibody, which targets specific proteins in the body to help reduce inflammation. ^{1 2 3 6 7}

How is the drug Mirikizumab different from other treatments for Crohn's disease?

Mirikizumab is unique because it targets interleukin 23p19, a specific part of the immune system involved in inflammation, and is the first drug of its kind approved for ulcerative colitis, which is similar to Crohn's disease. This makes it different from other treatments that may target different pathways or use different mechanisms to reduce inflammation. ^{1 2 3 4 7}

What is the purpose of this trial?

Study participants will be screened during the platform study and randomly assigned to receive mirikizumab or another intervention. The purpose of the mirikizumab study is to evaluate efficacy, safety, tolerability, and how well mirikizumab absorbs into the body of pediatric participants with Crohn's disease. Study periods for the intervention-specific appendix (ISA) will be as follows: * A 12-week induction period * A maintenance period from Week 12 to Week 52, and * A safety follow-up period up to 16 weeks. The study will last about 74 weeks and may include up to 19 visits.

Research Team

Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST)

Principal Investigator

Eli Lilly and Company

Eligibility Criteria

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

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)

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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-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 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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Mirikizumab in a Randomized Phase 2 Study of Patients With Crohn's Disease. [2022]

2. New Zealand pubmed.ncbi.nlm.nih.gov

Mirikizumab: First Approval. [2023]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Mirikizumab Pharmacokinetics in Patients with Moderately to Severely Active Ulcerative Colitis: Results from Phase III LUCENT Studies. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Effects of Mirikizumab on Histologic Resolution of Crohn's Disease in a Randomized Controlled Phase 2 Trial. [2023]

5. England pubmed.ncbi.nlm.nih.gov

Resolving Histological Inflammation in Ulcerative Colitis With Mirikizumab in the LUCENT Induction and Maintenance Trial Programmes. [2023]

6. England pubmed.ncbi.nlm.nih.gov

Effectiveness and safety of risankizumab induction therapy for 100 patients with Crohn's disease: A GETAID multicentre cohort study. [2023]

7. United States pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Continued Treatment With Mirikizumab in a Phase 2 Trial of Patients With Ulcerative Colitis. [2022]