BridgeMD
A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb≥10 g/dL in response to anti-C5 antibody and switch to iptacopan
EU CTIS ID: 2022-502148-10-00
What this study is testing
The main question this trial is designed to answer: is iptacopan treatment equally effective to standard of care when switching treatment to iptacopan?
- 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
- Signed informed consent must be obtained prior to participation in the study
- Male and female participants ≥ 18 years of age, at the time of ICF signatures and with a diagnosis of PNH confirmed by PNH clone size WBCs ≥ 10% based on historical data.
- Stable regimen (dose and intervals) of anti-C5 antibody treatment (either eculizumab or i.v. ravulizumab) for at least 6 months prior to screening
- Mean hemoglobin level ≥10 g/dL, documented by the mean of all available Hb assessments (minimum 2 measurements) over a period of 6 months before screening visit, collected at any laboratory. In addition to fulfill the Hb eligibility criterion, participants must have two different samples collected during the screening period and tested by the central laboratory with the mean >10 g/dL, prior to starting iptacopan.
- Vaccination against Neisseria meningitidis and S. pneumoniae infection are required prior to the start of iptacopan treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine are to be given according to local regulations, at least 2 weeks prior to first dosing. However, administration of these vaccines less than 2 weeks prior to start of iptacopan treatment is at the discretion of the investigator. If iptacopan treatment is started less than 2 weeks post-vaccination, participant must be given prophylactic antibiotic at the start of iptacopan and for at least 2 weeks after vaccination.
- If not received previously, vaccination against Haemophilus influenzae infections is recommended, if available and according to local regulations. The vaccines should be given at least 2 weeks prior to initiation of iptacopan treatment. However, administration of these vaccines less than 2 weeks prior to start of iptacopan treatment, is at the discretion of the investigator. If iptacopan treatment is started less than 2 weeks post-vaccination, participant must be given prophylactic antibiotic at the start of iptacopan and for at least 2 weeks after vaccination.
You likely can't join if
- Participation in any other investigational drug trial or use of other investigational drugs at the time of enrollment
- Ongoing drug or alcohol abuse that could interfere with patient's participation in the trial.
- Any medical condition deemed likely to interfere with the patient's participation in the study
- Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
- Patients requiring red blood cell transfusion in the 6 months prior to screening or during screening
- History of stem cell transplantation or any solid organ transplantation
See the full eligibility criteria
Who can join
- Signed informed consent must be obtained prior to participation in the study
- Male and female participants ≥ 18 years of age, at the time of ICF signatures and with a diagnosis of PNH confirmed by PNH clone size WBCs ≥ 10% based on historical data.
- Stable regimen (dose and intervals) of anti-C5 antibody treatment (either eculizumab or i.v. ravulizumab) for at least 6 months prior to screening
- Mean hemoglobin level ≥10 g/dL, documented by the mean of all available Hb assessments (minimum 2 measurements) over a period of 6 months before screening visit, collected at any laboratory. In addition to fulfill the Hb eligibility criterion, participants must have two different samples collected during the screening period and tested by the central laboratory with the mean >10 g/dL, prior to starting iptacopan.
- Vaccination against Neisseria meningitidis and S. pneumoniae infection are required prior to the start of iptacopan treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine are to be given according to local regulations, at least 2 weeks prior to first dosing. However, administration of these vaccines less than 2 weeks prior to start of iptacopan treatment is at the discretion of the investigator. If iptacopan treatment is started less than 2 weeks post-vaccination, participant must be given prophylactic antibiotic at the start of iptacopan and for at least 2 weeks after vaccination.
- If not received previously, vaccination against Haemophilus influenzae infections is recommended, if available and according to local regulations. The vaccines should be given at least 2 weeks prior to initiation of iptacopan treatment. However, administration of these vaccines less than 2 weeks prior to start of iptacopan treatment, is at the discretion of the investigator. If iptacopan treatment is started less than 2 weeks post-vaccination, participant must be given prophylactic antibiotic at the start of iptacopan and for at least 2 weeks after vaccination.
- Able to communicate well with the investigator, to understand and comply with the requirements of the study
What rules you out
- Participation in any other investigational drug trial or use of other investigational drugs at the time of enrollment
- Ongoing drug or alcohol abuse that could interfere with patient's participation in the trial.
- Any medical condition deemed likely to interfere with the patient's participation in the study
- Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
- Patients requiring red blood cell transfusion in the 6 months prior to screening or during screening
- History of stem cell transplantation or any solid organ transplantation
- Active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to study drug administration
- Presence of fever ≥ 38.0 °C (100.4 °F) within 7 days prior to study drug administration
- Human immunodeficiency virus (HIV) infection (known history of HIV or test positive for HIV antibody at Screening)
- A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus
- Unstable medical condition including, but not limited to, myocardial ischemia, active gastrointestinal bleeding, coexisting chronic anemia unrelated to PNH, or unstable thrombotic event not amenable to active treatment as judged by the investigator at Screening.
- History of cancer of any part of the body within the past 5 years
The study team makes the final eligibility decision.
Where it's taking place
- Turkey
- United Kingdom
- Korea, Republic of
- United States
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, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Turkey; United Kingdom; Korea, Republic of; United States. 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.