Study for Children and Teens With IL-18 Driven Monogenic Autoinflammatory Conditions

Why are we doing this research?

Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to evaluate the safety and effectiveness of the study drug Tadekinig alfa when added to the standard of care compared with placebo (an inactive substance that contains no medicine) in children and teens with IL-18 (interleukin-18) driven monogenic autoinflammatory conditions due to NLRC4 mutation and XIAP deficiency.

Who can participate?

Children and teens up to 17 years old who have an IL-18 driven autoinflammatory condition due to a genetic diagnosis of NLRC4-MAS mutation or XIAP deficiency may be eligible to participate.

Conditions

  • Rheumatology
  • Immune Deficiency and Histiocytosis
  • Gastroenterology, Hepatology and Nutrition

What will happen in the study?

This study includes 16 study visits to Cincinnati Children’s over 26 weeks or about 7 months. There are two phases in this study.

For the first study phase, your child will receive the study drug Tadekinig alfa for 18 weeks and will be treated with standard of care (SOC), which is a treatment plan that the majority of the medical community would accept as appropriate, to control flares of the disease. For this study, your child may receive Dexamethasone (a common anti-inflammatory drug) in combination with Tadekinig alfa for the first 28 days of the study. If your child responds to treatment with the combination of SOC and Tadekinig alfa, he/she will then receive injections of only Tadekinig alfa for 14 more weeks.

If your child completes study phase 1 without an ongoing flare at the end of the 18 weeks, he or she may continue into study phase 2, which is an additional 8 weeks. In the second study phase, your child will be assigned at random (like flipping a coin) to one of 2 study groups to either receive Tadekinig alfa or placebo. Participants in each study group will be given injections of Tadekinig alfa or placebo every 2 days for a maximum of 8 weeks.

In addition, the study visits will be similar to your child’s regular checkup visits with his/her doctor and will include physical exams, measurements, and blood samples. We will also ask you and your child questions about his/her health, disease, medications, etc.

If you want to learn more about this study, the research team will give you a consent form that thoroughly explains all of the details of the study. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.

What are the good things that can happen from this research?

It is possible that your child may benefit from the study drug Tadekinig alfa, but it is unknown at this time whether it will improve your child’s health. We do think that the information from this study will help researchers to learn more about this study drug as a potential treatment for IL-18 driven autoinflammatory conditions.

What are the bad things that can happen from this research?

Your child may have side effects while in this study. With any drug, unusual, unexpected or previously not reported reactions and side affects may occur. Possible risks and discomforts will be discussed with parents or guardians interested in learning more about the study.

Will you/your child be paid to be in this research study?

Families will not be compensated for participation in this study. However, the study sponsor AB2 Bio will pay for all procedures and tests completed specifically for this research study.

Depending on how far you live from the hospital, you may be reimbursed for reasonable travel costs incurred for each visit to cover the cost of transportation, parking and possible accommodations and meals for each completed visit.

Contact

Contact Us.Kelly McIntosh
513-803-0460
kelly.mcintosh@cchmc.org
Cincinnati Children’s Hospital Medical Center
3333 Burnet Ave.
Cincinnati, OH 45229-3039

Study Doctor

Contact Us.Rebecca Marsh, MD
Bone Marrow Transplantation & Immune Deficiency
Cincinnati Children’s Hospital Medical Center