BridgeMD
Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label Extension
EU CTIS ID: 2023-504534-21-00
What this study is testing
This is an open-label extension study for patients previously enrolled in the preceding clinical trial NLRC4/XIAP.2016.001 to evaluate the long-term safety and tolerability of TA in patients suffering from pediatric monogenic autoinflammatory diseases harboring deleterious mutations of NLRC4 and XIAP.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Patients have participated in the preceding clinical trial NLRC4/XIAP.2016.001 (IND N° 127953) by one of the following mechanisms: a. Patients that have completed the first 18-week SAOL phase of the preceding clinical trial, but were not eligible for the RW phase due to flare symptoms. b. Patients that completed the first 18-week SAOL phase and completed the RW phase of the preceding clinical trial. c. Patients who have exited either the SAOL 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 of the main study they will be asked to continue with the planned visits of the main study. The time period between participation in the SAOL or RW and the OLE study should not exceed 3 months. After this period, patients are no longer eligible for enrollment into the OLE study.
- 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.
You likely can't join if
- Patients may not enter the OLE if they voluntarily withdrew from SAOL or RW study or if the time period between participation exceeds 3 months
- 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)
- Hypersensitivity to the active substance or one of the excipients of the investigational product.
- Evidence or history of malignancy
See the full eligibility criteria
Who can join
- Patients have participated in the preceding clinical trial NLRC4/XIAP.2016.001 (IND N° 127953) by one of the following mechanisms: a. Patients that have completed the first 18-week SAOL phase of the preceding clinical trial, but were not eligible for the RW phase due to flare symptoms. b. Patients that completed the first 18-week SAOL phase and completed the RW phase of the preceding clinical trial. c. Patients who have exited either the SAOL 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 of the main study they will be asked to continue with the planned visits of the main study. The time period between participation in the SAOL or RW and the OLE study should not exceed 3 months. After this period, patients are no longer eligible for enrollment into the OLE study.
- 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.
What rules you out
- Patients may not enter the OLE if they voluntarily withdrew from SAOL or RW study or if the time period between participation exceeds 3 months
- 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)
- Hypersensitivity to the active substance or one of the excipients of the investigational product.
- 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: ▪ Intractable encephalopathy, psychosis, or seizures ▪ Creatinine of > 2X ULN (patients receiving dialysis are also excluded) ▪ Albumin < 1.5 g/dL, ALT>10x ULN, or evidence of acute liver failure ▪ Mechanical ventilation (including invasive and non-invasive forms) ▪ Heart failure requiring medical support including medications ▪ Hypotension from any cause requiring the use of vasopressors
- Evidence of severe organ compromise including but not limited to: ▪ Intractable encephalopathy, psychosis, or seizures ▪ Creatinine of > 2X ULN (patients receiving dialysis are also excluded) ▪ Albumin < 1.5 g/dL, ALT>10x ULN, or evidence of acute liver failure ▪ Mechanical ventilation (including invasive and non-invasive forms) ▪ Heart failure requiring medical support including medications ▪ Hypotension from any cause requiring the use of vasopressors
- Inability to follow highly effective birth control recommendations during the study and until 1 month after the end of the treatment.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.