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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 25 January 2021
Main ID:  NCT03113760
Date of registration: 28/02/2017
Prospective Registration: Yes
Primary sponsor: AB2 Bio Ltd.
Public title: Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
Scientific title: Multicenter, Double-blind, Placebo-controlled, Randomized Withdrawal Trial With Tadekinig Alfa (r-hIL-18BP) in Patients With IL-18 Driven Monogenic Autoinflammatory Conditions: NLRC4 Mutation and XIAP Deficiency
Date of first enrolment: July 21, 2017
Target sample size: 10
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT03113760
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).  
Phase:  Phase 3
Countries of recruitment
Canada Germany United States
Contacts
Name:     Eduardo Schiffrin, MD
Address: 
Telephone: +41 21 694 00 43
Email: eduardo.schiffrin@ab2bio.com
Affiliation: 
Name:     Ed M Behrens, MD
Address: 
Telephone:
Email:
Affiliation:  Children Hospital of Philadelphia
Key inclusion & exclusion criteria

INCLUSION CRITERIA

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

EXCLUSION CRITERIA

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



Age minimum: N/A
Age maximum: 17 Years
Gender: All
Health Condition(s) or Problem(s) studied
NLRC4-MAS
XIAP Deficiency
Intervention(s)
Drug: Tadekinig alfa
Other: 0.9% sodium chloride
Primary Outcome(s)
Prevention of flares [Time Frame: 8 weeks]
Secondary Outcome(s)
Adverse events will be reported [Time Frame: 26 weeks (SAOL + RW phases)]
Change in mAIDAI score [Time Frame: 8 weeks]
Change in Physician Global Assessment (PGA) [Time Frame: 8 weeks]
Flare rate [Time Frame: 8 weeks]
Hospital length of stay [Time Frame: 8 weeks]
Immunogenicity evaluation [Time Frame: 26 weeks (SAOL + RW phases)]
Improvement in serum albumin and liver transaminases, anemia and/or platelet count [Time Frame: 8 weeks]
Improvement of fevers, improvement of hepato/splenomegaly [Time Frame: 8 weeks]
Improvement of stool output for hospitalized patients if intestinal dysfunction was present at Baseline [Time Frame: 8 weeks]
Intensity of flares [Time Frame: 8 weeks]
Laboratory assessments [Time Frame: 26 weeks (SAOL + RW phases)]
Local tolerability at the injection site [Time Frame: 26 weeks (SAOL + RW phases)]
Physical examination findings and vital signs [Time Frame: 26 weeks (SAOL + RW phases)]
Presence of skin rash - evolution if present at Baseline or appearance during the study [Time Frame: 8 weeks]
Response to therapy/treatment failures [Time Frame: 8 weeks]
Serum CRP, Serum Ferritin [Time Frame: 8 weeks]
Tolerance to oral/enteral nutrition for hospitalized patients if intestinal dysfunction was present at Baseline [Time Frame: 8 weeks]
Secondary ID(s)
NLRC4/XIAP.2016.001
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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