Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label Extension

Description

This is an open-label extension study for patients previously enrolled in the AB2 Bio Ltd. ongoing Phase III clinical trial NLRC4/XIAP.2016.001 (IND N° 127953). This OLE study will evaluate the long-term safety and tolerability of Tadekinig alfa in patients suffering from pediatric monogenic autoinflammatory diseases harboring deleterious mutations of NLRC4 and XIAP.

Study Start Date

January, 24 2018

Estimated Completion Date

July 2020

Interventions

  • Drug: Tadekinig alfa

Study ID

AB2 Bio Ltd. -- OLE-NLRC4/XIAP.2016.001

Status

Recruiting

Trial ID

NCT03512314

Study Type

Interventional

Trial Phase

Phase 3

Enrollment Quota

10

Sponsor

AB2 Bio Ltd.

Inclusion Criteria

    (both criteria must be met) 1. Patients have participated in AB2 Bio ltd. Phase III clinical trial NLRC4/XIAP.2016.001 (IND N° 127953) by one of the following mechanisms : a) Patients that have completed the first 18-week RCT phase of the preceding clinical trial but were not eligible for the RW phase due to flare symptoms. Or b) Patients that completed the first 18-week RCT phase and completed the RW phase of the preceding clinical trial. Or c) Patients who have exited either the RCT or RW phase of the preceding clinical trial due to treatment failure requiring rescue immunosuppression. Such patients must wait a minimum of 4 weeks after treatment discontinuation from the preceding clinical trial before enrolling in this OLE. If patients do not consent to enroll in the OLE after their early termination in the main study, they will be asked to continue with the planned visits of the main study 2. Women of childbearing potential with negative urine pregnancy test (UPT) at all visits

Exclusion Criteria

    1. Patients may not enter the OLE if they voluntarily withdrew from RCT or RW study or if the time period between participation exceeds 3 months 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)

Gender

All

Ages

N/A

Accepts Healthy Volunteers

No

Study Locations and Contact Information (7)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Fatma Dedeoglu Dr 617-355-6117 None
Children Hospital of Philadelphia - Philadelphia, Pennsylvania 270.8 miles Ed Behrens Dr None None
Childrens Hospital of Pittsburgh of UPMC - Pittsburgh, Pennsylvania 477.6 miles Scott Canna Dr 412-692-9934 None
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Rebecca Marsh Dr 513-803-9063 None
Childrens of Alabama CoA - Birmingham, Alabama 1,051.7 miles Randy Cron Dr 205-996-7584 None
Texas Childrens Hospital Baylor College of Medicine - Houston, Texas 1,609.9 miles Lisa Forbes Dr 832-824-1319 None
UCSD Department of Pediatrics Rady Childrens Hospital - La Jolla, California 2,573.3 miles Harold Hoffman Dr None None

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