Tadekinig Alfa for Genetic Autoinflammatory Diseases

Recruiting at 11 trial locations

Overseen By Eduardo Schiffrin, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: AB2 Bio Ltd.

Must not be taking: Other immunosuppression

Disqualifiers: Malignancy, Infection, Tuberculosis, 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

Compensation: Varies

Number of Visits: Unknown

Duration: 26 weeks

11 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing Tadekinig alfa, a drug that blocks a protein called IL-18 to reduce inflammation. It targets children with specific genetic conditions causing severe inflammation. The goal is to manage their symptoms until they can receive a stem cell transplant.

Will I have to stop taking my current medications?

The trial allows the use of certain medications like NSAIDs, glucocorticoids, cyclosporine, tacrolimus, and IL-1 inhibitors. However, other immunosuppressive medications are not allowed, so you may need to stop those.

What data supports the effectiveness of the drug Tadekinig alfa for genetic autoinflammatory diseases?

The research highlights that novel IL-1-targeted drugs, like Tadekinig alfa, are particularly effective for many autoinflammatory diseases, offering life-changing effects for patients. ^{1 2 3 4 5}

How is the drug Tadekinig alfa different from other treatments for genetic autoinflammatory diseases?

Tadekinig alfa is unique because it targets specific pathways involved in inflammation, potentially offering a new approach for patients who do not respond well to existing treatments like IL-1 inhibitors. This drug may provide an alternative for those with genetic mutations that affect typical inflammatory responses. ^{4 5 6 7 8}

Research Team

Eduard Behrens, MD

Principal Investigator

Children Hospital of Philadelphia

Eligibility Criteria

This trial is for patients with specific genetic disorders (NLRC4 mutation or XIAP deficiency) who were in a previous Phase III study and either completed it or needed rescue immunosuppression. They must not have withdrawn voluntarily, be pregnant, or have severe organ issues, life-threatening infections or bleeding, recent live vaccinations, tuberculosis history, or use of certain immunosuppressants.

Inclusion Criteria

Patients must have participated in AB2 Bio ltd. Phase III clinical trial NLRC4/XIAP.2016.001 (IND N° 127953) by meeting one of the following mechanisms: a) Completed the first 18-week RCT phase but were not eligible for the RW phase due to flare symptoms. b) Completed the first 18-week RCT phase and RW phase. c) Exited RCT or RW phase due to treatment failure requiring rescue immunosuppression, waiting a minimum of 4 weeks after treatment discontinuation before enrolling in this OLE. Women of childbearing potential with negative urine pregnancy test (UPT) at all visits

Exclusion Criteria

I have not received a live vaccine in the last 3 months.

I have had tuberculosis in the past.

I have not had severe bleeding in the last 2 weeks.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Active drug treatment with Tadekinig alfa for patients with NLRC4 Mutation and XIAP Deficiency

26 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Long-term safety and tolerability of Tadekinig alfa are evaluated in patients with IL-18 driven monogenic autoinflammatory conditions

Long-term

Treatment Details

Interventions

• Tadekinig alfa

Trial Overview The trial tests the long-term safety and effectiveness of Tadekinig alfa in children with monogenic autoinflammatory diseases due to NLRC4 mutations/XIAP deficiency. It's an extension for those who've been part of a prior related trial.

Participant Groups

Group I: Tadekinig alfa Experimental Treatment 1 Intervention

Active drug treatment during 26 weeks

Who Is Running the Clinical Trial?

AB2 Bio Ltd.

Lead Sponsor

Trials

3

Recruited

50+

References

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

Therapy of autoinflammatory syndromes. [2022]

2. India pubmed.ncbi.nlm.nih.gov

Growth Parameters of Turkish Children With an Autoinflammatory Disease Before and After Canakinumab Treatment. [2021]

3. England pubmed.ncbi.nlm.nih.gov

Colchicine - an effective treatment for children with a clinical diagnosis of autoinflammatory diseases without pathogenic gene variants. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Clinical characteristics and genetic analyses of 187 patients with undefined autoinflammatory diseases. [2023]

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

Treatment of autoinflammatory diseases: results from the Eurofever Registry and a literature review. [2022]

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

Role of interleukin-1β in NLRP12-associated autoinflammatory disorders and resistance to anti-interleukin-1 therapy. [2011]

7. Netherlands pubmed.ncbi.nlm.nih.gov

Molecular and genetic characteristics of hereditary autoinflammatory diseases. [2019]

8. England pubmed.ncbi.nlm.nih.gov

Development of the autoinflammatory disease damage index (ADDI). [2022]