A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb10 g/dL in response to anti-C5 antibody and switch to iptacopan
ESTADO CLÍNICO
Reclutando
FASE/S DEL ENSAYO
Fase III
PATOLOGÍA
Insuficiencias medulares y aplasia medular
FECHA DE APERTURA
Septiembre, 2023
FECHA DE CIERRE
,
CRITERIOS DE INCLUSIÓN
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
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
CRITERIOS DE EXCLUSIÓN
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
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
DESCRIPCIÓN
The main question this trial is designed to answer: is iptacopan treatment equally effective to standard of care when switching treatment to iptacopan?
PALABRAS CLAVE
iptacopan, paroxysmal nocturnal hemoglobinuria, pnh
