Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency As Open Label Extension
NCT ID: NCT03512314
Last Updated: 2025-03-17
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
11 participants
INTERVENTIONAL
2018-01-24
2024-05-08
Brief Summary
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Detailed Description
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Patients with X-linked inhibitor of apoptosis (XIAP) deficiency and suffering from Hemophagocytic-Lymphohistiocytosis (HLH), a MAS-like syndrome, also show high levels of serum IL-18 and may benefit from IL-18 blockade treatment until a curative hematopoietic stem cell transplantation can be performed The safety of IL-18 blockade during long-term periods is of major interest for the treatment of these patients
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Tadekinig alfa
Active drug treatment during 26 weeks
Tadekinig alfa
Open label, 26 weeks on Tadekinig alfa treatment.
Interventions
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Tadekinig alfa
Open label, 26 weeks on Tadekinig alfa treatment.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Women of childbearing potential with negative urine pregnancy test (UPT) at all visits
Exclusion Criteria
2. Evidence or history of malignancy
3. Evidence of invasive or life-threatening infection
4. History of tuberculosis
5. Life-threatening bleeding within 2 weeks of screening
6. Vaccination with a live vaccine within the previous 3 months
7. Evidence of severe organ compromise including but not limited to: (see details in the protocol)
8. Pregnant or breastfeeding females
9. Inability to follow highly effective birth control recommendations during the study and until 1 month after the end of the treatment.
10. Inability to provide informed consent, and also assent if applicable
11. Life expectancy less than 4 weeks
12. Concomitant use of other immunosuppression except NSAIDs, glucocorticoids, cyclosporine, tacrolimus, IL-1 inhibitors (Anakinra, Canakinumab, or Rilonacept)
ALL
No
Sponsors
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AB2 Bio Ltd.
INDUSTRY
Responsible Party
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Principal Investigators
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Eduard Behrens, MD
Role: PRINCIPAL_INVESTIGATOR
Children Hospital of Philadelphia
Locations
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UCSD _ Department of Pediatrics / Rady Children's Hospital
La Jolla, California, United States
Shands Children's Hospital
Gainsville, Florida, United States
Children's Healthcare of Atlanta at Egleston
Atlanta, Georgia, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Texas Children's Hospital _ Baylor College of Medicine
Houston, Texas, United States
The Hospital for Sick Children
Toronto, Ontario, Canada
CHU Sainte-Justine
Montreal, , Canada
Universitätsklinikum Freiburg, Centrum für Chronische Immundefizienz (CCI) - Paediatric Unit
Freiburg im Breisgau, Baden-Wurttemberg, Germany
Countries
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Other Identifiers
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OLE-NLRC4/XIAP.2016.001
Identifier Type: -
Identifier Source: org_study_id
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