Summary

for people ages up to 17 years (full criteria)
at La Jolla, California and other locations
study started
estimated completion:

Description

Summary

This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with monogenic, interleukin-18 (IL 18) driven autoinflammation due to Nucleotide-binding oligomerization domain, leucine-rich repeat and caspase recruiting domain (CARD domain) containing 4 (NLRC4) - Macrophage activation syndrome (MAS) mutation (NLRC4-MAS mutation) or X-linked inhibitor of apoptosis (XIAP) deficiency. Because of the likelihood for pathogenic IL-18 in certain monogenic diseases, patients known to harbor deleterious mutations in NLRC4-MAS or XIAP and who have a history of ongoing inflammation will be enrolled if they have ferritin ≥ 500 ng/mL or persistent C reactive protein (CRP) elevation > 2 times the upper limit of normal (ULN) and the patients should have a Modified Autoinflammatory Disease Activity Index (mAIDAI) ≥ 4.

Official Title

Multicenter, Randomized, Double-blind, Placebo-controlled Trial With Tadekinig Alfa (r-hIL-18BP) in Patients With IL-18 Driven Monogenic Auto Inflammatory Conditions: NLRC4 Mutation and XIAP Deficiency

Details

The study is designed with a Randomized Double-Blind Placebo-Controlled (RDBPC) 18-week treatment phase, followed by an 8-week Randomized Withdrawal (RW) period for further efficacy and safety evaluation, with no interruption between the two phases of treatment.

Keywords

NLRC4-MAS XIAP Deficiency Lymphoproliferative Disorders Genetic Diseases, X-Linked Tadekinig alfa

Eligibility

You can join if…

Open to people ages up to 17 years

  1. Patients ≤ 17 years of age
  2. Patients with genetic diagnosis of NLRC4-MAS mutation or XIAP deficiency (caused by BIRC4 gene mutation)
  3. Ferritin ≥ 500 ng/mL or persistent elevation of CRP ≥ 2x ULN and mAIDAI ≥4
  4. Patients receiving stable doses of corticosteroids, non-steroidal anti-inflammatory drugs (NSAIDs) or disease modifying anti rheumatic drugs (DMARDs), and/or IL-1 blockade for at least 2 weeks prior to enrollment are allowed into the study. Patients not receiving any of these treatments before start of therapy are also allowed
  5. Women of childbearing potential with negative urine pregnancy test (UPT) at all visits

You CAN'T join if...

  1. Patients > 17 years of age
  2. Positive test for or prior history of HIV, Hepatitis B or Hepatitis C (serology)
  3. Presence of active infections or a history of pulmonary TB infection with or without documented adequate therapy
  4. Presence of life threatening infections
  5. Oncologic causes of symptoms; current or previous history of malignancy
  6. Presence of CNS manifestations
  7. Patients suffering from familial hemophagocytic lymphohistiocytosis (f HLH)
  8. Patients who are pregnant or nursing, women of childbearing potential who are unwilling to use highly effective birth control methods
  9. Concomitant use of immunosuppression therapies excluded by the protocol.
  10. . Patients and/or parents (or legal representative, if applicable) not willing to sign assent/informed consent

Locations

  • UCSD _ Department of Pediatrics / Rady Children's Hospital accepting new patients
    La Jolla California 92056 United States
  • Texas Children's Hospital _ Baylor College of Medicine accepting new patients
    Houston Texas 77030 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
AB2 Bio Ltd.
Links
Sponsor details
ID
NCT03113760
Phase
Phase 3
Study Type
Interventional
Last Updated