NLRC4-MASPhase 3NCT03113760
Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
A Phase 3 study testing Tadekinig alfa and 0.9% sodium chloride in people with NLRC4-MAS and XIAP Deficiency, run by AB2 Bio Ltd. at 9 sites in 3 countries.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- NLRC4-MAS, XIAP Deficiency
- What is being tested?
- Tadekinig alfa and 0.9% sodium chloride
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 9 sitesin 3 countries
- Study dates
- 21 Jul 2017to 2 Nov 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with genetic diagnosis of NLRC4-MAS mutation or XIAP deficiency (caused by BIRC4 gene mutation) as confirmed by analysis performed at the central genetics laboratory. If possible, flow cytometry assay will be performed in parallel to confirm diagnosis of XIAP deficiency. (Note: Previous flow cytometry assay results will be permitted for confirmation of XIAP deficiency diagnosis.)
- Patients with XIAP deficiency and a previous bone marrow transplantation are allowed, if they show evidence of primary or secondary graft failure, or failure to achieve phenotypic correction with evidence of XIAP-related disease recurrence or clinically significant mixed chimerism.
- Ferritin ≥ 500 ng/mL or persistent elevation of CRP ≥ 2x ULN and mAIDAI ≥4
- Patients receiving corticosteroids, non-steroidal anti-inflammatory drugs (NSAIDs) or disease modifying anti-rheumatic drugs (DMARDs), and/or IL-1 blockade with insufficient response to treatment upon enrollment are allowed into the study. Patients not receiving any of these treatments before start of therapy are also allowed.
- Women of childbearing potential with negative urine pregnancy test (UPT) at all visits (if UPT is positive, a blood test for human chorionic gonadotropin (hCG) to be performed) and who agree to follow highly effective birth control recommendations during the study and until 1 month after the end of the treatment. Birth control methods considered highly effective are: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner or sexual abstinence. In each case of delayed menstrual period (over one month between menstruations, confirmation of absence of pregnancy is strongly recommended. This recommendation also applies to women of childbearing potential with infrequent or irregular menstrual cycles. A post-study contraception duration of 4 weeks is recommended taking into account the median half-life of Tadekinig alfa of almost 40h and 5 half-lives representing a duration of 200 hours.
Who cannot join
- Patients with life-threatening co-morbidities not associated with the underlying NLRC4-mutation or XIAP deficiency
- Positive test for or prior history of HIV, Hepatitis B or Hepatitis C (serology)
- Presence of active infections or a history of pulmonary TB infection with or without documented adequate therapy
- Presence of life threatening infections
- Oncologic causes of symptoms; current or previous history of malignancy
- Presence of CNS manifestations (i.e. seizures, altered mental status, signs of increased intracranial pressure, chronic papilledema, loss of vision, other sensorineural deficiencies, etc.)
Show all 11Show fewer
- Patients suffering from biallelic mutations in any of the following genes: PRF1/Perforin, UNC13D/Munc 13-4, STX11/Syntaxin11, STXB2/Munc 18-2, RAB27A/Rab27a (Griscelli syndrome type 2), LYST (Chediak-Higashi syndrome), AP3B1, ADTB3A, HPS2 mutations (Hermansky-Pudlak syndrome 2) and X-linked lymphoproliferative syndrome (XLP)-1 with SH2D1A mutation
- Patients who are pregnant or nursing, women of childbearing potential who are unwilling to use highly effective birth control methods (see definition in Inclusion Criteria above) through 4 weeks after the end of their participation in the study
- Concomitant use of immunosuppression therapies excluded by the protocol. Note: NSAIDs, glucocorticoids, cyclosporine, tacrolimus, and IL-1 inhibitors (anakinra, canakinumab, or rilonacept, or others) are allowed
- Patients and/or parents (or legal representative, if applicable) not willing to sign assent/informed consent
- Hypersensitivity to the active substance or one of the excipients of the investigational product
Trial locations and contacts
9 sites in 3 countries.
9 sites in 3 countries
United States6 sites
Children's Healthcare of Atlanta at Egleston
Atlanta, Georgia, 30322
Boston Children's Hospital
Boston, Massachusetts, 02115
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Texas Children's Hospital _ Baylor College of Medicine
Houston, Texas, 77030
UCSD _ Department of Pediatrics / Rady Children's Hospital
La Jolla, California, 92056
Children Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Canada2 sites
CHU Sainte-Justine
Montreal, Providence, QC H3T 1C5
The Hospital for Sick Children
Toronto, Ontario, ON M5G 1X8
Germany1 site
Universitätsklinikum Freiburg, Centrum für Chronische Immundefizienz (CCI) - Paediatric Unit
Freiburg im Breisgau, Baden-Wurttemberg, 79106
About this study
Official 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
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.
Read the detailed description
The study is designed with single-arm, open-label phase (SAOL) of Tadekinig alfa treatment duration for 18-week followed by an up to 16-week Randomized Withdrawal (RW) period for efficacy and safety evaluation, with no interruption between the two phases of treatment. The screening period will occur before the SAOL phase and before the first dose of Investigational Medicinal Product (IMP)
- Conditions
- NLRC4-MAS, XIAP Deficiency
- Being tested
- Tadekinig alfa, 0.9% sodium chloride
- Medical subject headings
- Lymphoproliferative Syndrome, X-Linked, 2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tadekinig alfaDrug
Also called IL-18BP, r-hIL-18BP
Tadekinig alfa is a soluble glycoprotein of 164 amino acids produced from Chinese Hamster Ovary cell line. Tadekinig alfa is supplied as a colorless to slightly yellow, sterile solution for injection in glass vials containing sodium chloride, and 0.02M sodium phosphate buffer as excipients. It is available in a concentration of 20mg/0.5mL.
0.9% sodium chlorideOther
To ensure that the treatment remains blinded for the entire study period, the placebo solutions will be supplied in identical vials and similar labelling as the active drug and will be indistinguishable in terms of their texture, color, and smell.
| Study group | Role | What they receive |
|---|---|---|
| Tadekinig alfa | Experimental | Patients that showed response to treatment in the SAOL phase will receive Tadekinig alfa for up to 16 weeks. Receives: Drug: Tadekinig alfa |
| 0.9% sodium chloride | Placebo comparator | Patients that showed response to treatment in the SAOL phase will receive placebo comparator for up to 16 weeks. Receives: Other: 0.9% sodium chloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevention of Flares
The primary outcome measure is the time to first occurrence of disease reactivation during the 16-week RW phase.
Method of assessment: Biomarkers (CRP / Ferritin) and clinical manifestations of systemic inflammation and end-organ damage.
Measured: 16 weeks
Secondary outcomes (21)
Best Response
Best response to therapy during the SAOL phase including either complete response, partial response or disease improvement.
Method of assessment: Biomarkers (CRP / Ferritin) and clinical manifestations of systemic inflammation and end-organ damage.
Measured: 18 weeks
Duration of Response During the SAOL Phase (Until End of Phase or Disease Reactivation)
Duration of response to therapy during the SAOL phase is assessed for patients having achieved a complete or partial response to therapy during the SAOL phase. It is defined as the time from first evaluation of partial or complete response until the time of subsequent disease reactivation or end of SAOL phase, whichever occurs first.
Of the 11 patients achieving partial or complete response during the SAOL phase, 8 maintained treatment response until the end of the SAOL phase.
2 of the 11 patients had a temporary disease reactivation during the protocol mandated steroid weaning in the SAOL phase; 1 of the 11 patients was withdrawn from blinded treatment following a disease reactivation.
Measured: The actual mean treatment duration in the SAOL phase was 17.6 weeks (minimum / maximum 5.9 / 22.1 weeks).
Change From Baseline in mAIDAI Total Score in the SAOL Phase
The mAIDAI (modified autoinflammatory disease activitiy index) is an assessment of global disease activity that measures 14 different components for disease as either absent (0 points) or present (2 points for Uveitis 3+/4+ and 1 point for all other symptoms) at each visit. The mAIDAI total score is the sum of the points assigned across all components and ranges from 0 to 15, with a higher score indicating more severe disease activity.
Measured: 18 weeks
Change From Baseline in Serum Ferritin
Laboratory measure
Measured: 34 weeks
Change From Baseline in Serum CRP
Laboratory measure
Measured: 34 weeks
Resolution of Fevers, Hepato/Splenomegaly and Skin Rash
Clinical assessments if present at Baseline; number displays percentage of patients with resolution of individual disease component at Week 18 or early termination visit based on the number of patients with a baseline assessment for the component of interest.
Measured: 18 weeks
Improvement in Laboratory Markers - AST (SGOT)
Change from Baseline mean value to Week 18 mean value
Measured: 18 weeks
Improvement in Laboratory Markers - ALT (SGPT)
Change from Baseline mean value to Week 18 mean value
Measured: 18 weeks
Improvement in Laboratory Markers - Albumin
Change from Baseline mean value to Week 18 mean value
Measured: 18 weeks
Improvement in Laboratory Markers - Hemoglobin
Change from Baseline mean value to Week 18 mean value
Measured: 18 weeks
Improvement in Laboratory Markers - Platelets
Change from Baseline mean value to Week 18 mean value
Measured: 18 weeks
Improvement in Laboratory Markers - Erythrocyte Sedimentation Rate
Change from Baseline mean value to Week 18 mean value
Measured: 18 weeks
Improvement in Laboratory Markers - Fibrinogen
Change from Baseline mean value to Week 18 mean value
Measured: 18 weeks
Improvement in Laboratory Markers - D-Dimer
Change from Baseline mean value to Week 18 mean value
Measured: 18 weeks
Hospital Length of Stay
Length of hospitalisation; emergency room attendance and unscheduled visits for treatment of disease reactivations not included
Measured: 34 weeks
Change in Physician Global Assessment (PGA)
The PGA is a direct surrogate of how a patient functions and assesses the severity of disease-related (auto-inflammatory) symptoms by selecting an integer score from 0 (no impact on subject; no symptoms) to 10 (no normal activities possible; highest severity of symptoms possible).
The outcome lists the change from baseline of treatment phase to end of treatment phase/study in the PGA symptom severity score.
Measured: 34 weeks
Change in Patient/Caregiver Qualitative Evaluation of Health Status in Randomized Withdrawal Phase
The individual disease-related symptoms score is the sum of (x) individual symptom scores within domain each ranging from 0 (None) to 5 (Very severe) for: general wellbeing (5), gastrointestinal (7), musculoskeletal (5), skin (2), eye (2), central nervous system (3), and lymphatic (2). Highest values indicate more severe disease-related symptoms within total score range from 0-130.
Measured: 16 weeks
Immunogenicity Evaluation
Generation of anti-recombinant human IL-18BP (anti-rhIL-18BP) antibodies
Measured: 34 weeks (SAOL + RW phases)
Local Tolerability at the Injection Site (as Defined by Number of Participants With Adverse Events of Special Interest)
Adverse events of special interest (AESIs) are defined for this protocol as injection site reactions (including pruritus, erythema, swelling).
Measured: 34 weeks (SAOL + RW phases)
Disease Reactivation Rate
Disease reactivation rate for individual subjects (number of disease reactivations experienced per week while each subject is on the study treatment during the SAOL phase)
Measured: 18 weeks
Treatment Failures
Treatment failures (i.e. patients who experience at least one disease reactivation) during the SAOL phase
Measured: 18 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2017
- Study start21 Jul 2017
- Primary completion31 Oct 2023
- Study completion2 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for NLRC4-MASStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Initial phase of 18 weeks is a single arm, open-label (SAOL) period where Tadekinig alfa is administered in addition to the standard of care treatment used for the control of flares. The SAOL period will be followed by an up to 16-week randomized withdrawal phase (RW). All patients who showed response to treatment at the end of the SAOL phase will be enrolled in the RW phase. In the RW phase patients will be randomized to either Tadekinig alfa or placebo.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- AB2 Bio Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ed M Behrens, MD, Principal investigator, Children Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03113760
- Sponsor's protocol ID
- NLRC4/XIAP.2016.001
- Status last verified
- Jun 2025
- First submitted
- 28 Feb 2017
- Last updated
- 2 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: This information will be provided soon
Publications
Krei JM, Moller HJ, Larsen JB. The role of interleukin-18 in the diagnosis and monitoring of hemophagocytic lymphohistiocytosis/macrophage activation syndrome - a systematic review. Clin Exp Immunol. 2021 Feb;203(2):174-182. doi: 10.1111/cei.13543. Epub 2020 Nov 23.
PubMed 33128796
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT03113760
- Acronym / sponsor ID
- NLRC4/XIAP.2016.001
- Conditions
- NLRC4-MAS, XIAP Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- AB2 Bio Ltd.Industry (pharma, biotech, devices)
- Study start
- 21 Jul 2017 (actual)
- Primary completion
- 31 Oct 2023 (actual)
- Study completion
- 2 Nov 2023 (actual)
- First posted
- 14 Apr 2017 (actual)
- Results
- Posted 2 Jul 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The study is designed with single-arm, open-label phase (SAOL) of Tadekinig alfa treatment duration for 18-week followed by an up to 16-week Randomized Withdrawal (RW) period for efficacy and safety evaluation, with no interruption between the two phases of treatment. The screening period will occur before the SAOL phase and before the first dose of Investigational Medicinal Product (IMP)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Initial phase of 18 weeks is a single arm, open-label (SAOL) period where Tadekinig alfa is administered in addition to the standard of care treatment used for the control of flares. The SAOL period will be followed by an up to 16-week randomized withdrawal phase (RW). All patients who showed response to treatment at the end of the SAOL phase will be enrolled in the RW phase. In the RW phase patients will be randomized to either Tadekinig alfa or placebo.
| Arm | Type | Receives |
|---|---|---|
| Tadekinig alfa | Experimental | Drug: Tadekinig alfaPatients that showed response to treatment in the SAOL phase will receive Tadekinig alfa for up to 16 weeks. |
| 0.9% sodium chloride | Placebo comparator | Other: 0.9% sodium chloridePatients that showed response to treatment in the SAOL phase will receive placebo comparator for up to 16 weeks. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patients with genetic diagnosis of NLRC4-MAS mutation or XIAP deficiency (caused by BIRC4 gene mutation) as confirmed by analysis performed at the central genetics laboratory. If possible, flow cytometry assay will be performed in parallel to confirm diagnosis of XIAP deficiency. (Note: Previous flow cytometry assay results will be permitted for confirmation of XIAP deficiency diagnosis.)
- Patients with XIAP deficiency and a previous bone marrow transplantation are allowed, if they show evidence of primary or secondary graft failure, or failure to achieve phenotypic correction with evidence of XIAP-related disease recurrence or clinically significant mixed chimerism.
- Ferritin ≥ 500 ng/mL or persistent elevation of CRP ≥ 2x ULN and mAIDAI ≥4
- Patients receiving corticosteroids, non-steroidal anti-inflammatory drugs (NSAIDs) or disease modifying anti-rheumatic drugs (DMARDs), and/or IL-1 blockade with insufficient response to treatment upon enrollment are allowed into the study. Patients not receiving any of these treatments before start of therapy are also allowed.
- Women of childbearing potential with negative urine pregnancy test (UPT) at all visits (if UPT is positive, a blood test for human chorionic gonadotropin (hCG) to be performed) and who agree to follow highly effective birth control recommendations during the study and until 1 month after the end of the treatment. Birth control methods considered highly effective are: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner or sexual abstinence. In each case of delayed menstrual period (over one month between menstruations, confirmation of absence of pregnancy is strongly recommended. This recommendation also applies to women of childbearing potential with infrequent or irregular menstrual cycles. A post-study contraception duration of 4 weeks is recommended taking into account the median half-life of Tadekinig alfa of almost 40h and 5 half-lives representing a duration of 200 hours.
Key exclusion criteria (11 listed)
- Patients with life-threatening co-morbidities not associated with the underlying NLRC4-mutation or XIAP deficiency
- Positive test for or prior history of HIV, Hepatitis B or Hepatitis C (serology)
- Presence of active infections or a history of pulmonary TB infection with or without documented adequate therapy
- Presence of life threatening infections
- Oncologic causes of symptoms; current or previous history of malignancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tadekinig alfaAlso: IL-18BP, r-hIL-18BPTadekinig alfa is a soluble glycoprotein of 164 amino acids produced from Chinese Hamster Ovary cell line. Tadekinig alfa is supplied as a colorless to slightly yellow, sterile solution for injection in glass vials containing sodium chloride, and 0.02M sodium phosphate buffer as excipients. It is available in a concentration of 20mg/0.5mL. | Drug | Tadekinig alfa |
| 0.9% sodium chlorideTo ensure that the treatment remains blinded for the entire study period, the placebo solutions will be supplied in identical vials and similar labelling as the active drug and will be indistinguishable in terms of their texture, color, and smell. | Other | 0.9% sodium chloride |
Primary outcome
Prevention of Flares
Time frame: 16 weeks
The primary outcome measure is the time to first occurrence of disease reactivation during the 16-week RW phase. Method of assessment: Biomarkers (CRP / Ferritin) and clinical manifestations of systemic inflammation and end-organ damage.
21 secondary outcomes
Best Response
Time frame: 18 weeks
Duration of Response During the SAOL Phase (Until End of Phase or Disease Reactivation)
Time frame: The actual mean treatment duration in the SAOL phase was 17.6 weeks (minimum / maximum 5.9 / 22.1 weeks).
Change From Baseline in mAIDAI Total Score in the SAOL Phase
Time frame: 18 weeks
Change From Baseline in Serum Ferritin
Time frame: 34 weeks
Change From Baseline in Serum CRP
Time frame: 34 weeks
Resolution of Fevers, Hepato/Splenomegaly and Skin Rash
Time frame: 18 weeks
Improvement in Laboratory Markers - AST (SGOT)
Time frame: 18 weeks
Improvement in Laboratory Markers - ALT (SGPT)
Time frame: 18 weeks
Improvement in Laboratory Markers - Albumin
Time frame: 18 weeks
Improvement in Laboratory Markers - Hemoglobin
Time frame: 18 weeks
Improvement in Laboratory Markers - Platelets
Time frame: 18 weeks
Improvement in Laboratory Markers - Erythrocyte Sedimentation Rate
Time frame: 18 weeks
Improvement in Laboratory Markers - Fibrinogen
Time frame: 18 weeks
Improvement in Laboratory Markers - D-Dimer
Time frame: 18 weeks
Hospital Length of Stay
Time frame: 34 weeks
Change in Physician Global Assessment (PGA)
Time frame: 34 weeks
Change in Patient/Caregiver Qualitative Evaluation of Health Status in Randomized Withdrawal Phase
Time frame: 16 weeks
Immunogenicity Evaluation
Time frame: 34 weeks (SAOL + RW phases)
Local Tolerability at the Injection Site (as Defined by Number of Participants With Adverse Events of Special Interest)
Time frame: 34 weeks (SAOL + RW phases)
Disease Reactivation Rate
Time frame: 18 weeks
Treatment Failures
Time frame: 18 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33128796Krei JM, Moller HJ, Larsen JB. The role of interleukin-18 in the diagnosis and monitoring of hemophagocytic lymphohistiocytosis/macrophage activation syndrome - a systematic review. Clin Exp Immunol. 2021 Feb;203(2):174-182. doi: 10.1111/cei.13543. Epub 2020 Nov 23.
- Sponsor detailsLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. This information will be provided soon
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RecruitingObservational study
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ed M Behrens, MD principal investigator
Children Hospital of Philadelphia
9 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Tadekinig alfa and 0.9% sodium chloride in people with NLRC4-MAS and XIAP Deficiency, run by AB2 Bio Ltd. at 9 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Canada, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Prevention of Flares (time frame: 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03113760, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jul 2025.