Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and X... | NCT03512314

Status Module

Record Verification Date

Jan 2025

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 24, 2018Actual

Primary Completion Date

Apr 8, 2024Actual

Completion Date

May 8, 2024Actual

First Submitted Date

Apr 9, 2018

First Submission Date that Met QC Criteria

Apr 19, 2018

First Posted Date

Apr 30, 2018Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 13, 2025

Last Update Posted Date

Mar 17, 2025Actual

Description Module

Brief Summary

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.

Detailed Description

Pediatric auto-inflammatory conditions related to spontaneous activating mutations of the NLRC4 and with recurrent MAS-like flares with constitutive IL-18 hypersecretion, may require long-term blockade of the IL-18 pathway.

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

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

11Actual

Label	Type	Description	Intervention Names
Tadekinig alfa	Experimental	Active drug treatment during 26 weeks	Drug: Tadekinig alfa

Name	Type	Description	Arm Group Labels	Other Names
Tadekinig alfa	Drug	Open label, 26 weeks on Tadekinig alfa treatment.	Tadekinig alfa

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Reports of adverse events	The incidence, nature and severity of AEs will be reported	26 weeks
Reports of abnormal physical examination	Measurements will be done using the modified Auto-inflammatory Disease Activity Index (mAIDAI) including multiple measurements aggregated as 1 / 0.	26 weeks
Reports of abnormal laboratory results	Report of clinically significant abnormal laboratory results (i.eSerum CRP (ug/mL), Serum Ferritin (ng/mL). and any other abnormal lab results	26 weeks
Immunogenicity evaluation	Generation of anti-recombinant human Interleukin-18 Binding Protein (anti-rhIL-18BP) antibodies	26 weeks
Evaluation of the local tolerability at the injection site	Evaluation will be done based on the Local Tolerability Index where the patients will be asked to assess the degree of pain, redness, swelling, bruising, tenderness and itching, they are experiencing from each injection.	26 weeks

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria: (both criteria must be met)

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
Women of childbearing potential with negative urine pregnancy test (UPT) at all visits

Exclusion Criteria:

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
Evidence or history of malignancy
Evidence of invasive or life-threatening infection
History of tuberculosis
Life-threatening bleeding within 2 weeks of screening
Vaccination with a live vaccine within the previous 3 months
Evidence of severe organ compromise including but not limited to: (see details in the protocol)
Pregnant or breastfeeding females
Inability to follow highly effective birth control recommendations during the study and until 1 month after the end of the treatment.
Inability to provide informed consent, and also assent if applicable
Life expectancy less than 4 weeks
Concomitant use of other immunosuppression except NSAIDs, glucocorticoids, cyclosporine, tacrolimus, IL-1 inhibitors (Anakinra, Canakinumab, or Rilonacept)

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Release Date	Unrelease Date	Unrelease Date Unknown	Reset Date	MCP Release Number
Apr 29, 2025			May 13, 2025	11
May 20, 2025			Jun 5, 2025	12