A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of iptacopan in patients with generalized Myasthenia Gravis, followed by an open label extension phase
ESTADO CLÍNICO
Reclutando
FASE/S DEL ENSAYO
Fase III
PATOLOGÍA
Insuficiencias medulares y aplasia medular
CENTRO INVESTIGADOR
Hospital Ruber Juan Bravo
INVESTIGADOR PRINCIPAL
Rafael Arroyo Gonzalez
FECHA DE APERTURA
Junio, 2024
FECHA DE CIERRE
,
CRITERIOS DE INCLUSIÓN
Adult patients with gMG (age 18-85 years) at screening
Positive serology testing for AChR+ antibody at screening
Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG at screening and likely not in need of a respirator for the duration of the study, as judged by the Investigator
The confirmation of the diagnosis of gMG should be documented and supported by 1 of the following 3 tests: History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation. History of positive test with short-acting acetylcholinesterase inhibitors (e.g. neostigmine or edrophonium chloride) Patient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician.
Baseline MG-ADL score 6, with 50% of the total score due to non-ocular symptoms
Participants receiving at least one of the following treatments for gMG for 6 months prior to baseline One or more NSISTs; or plasmapheresis, plasma exchange, or intravenous immunoglobulin (at least quarterly) to control symptoms despite treatment with steroids and NSISTs; or an approved FcRN antagonist; or rituximab; or other approved gMG disease modifying therapies excluding complement inhibitors
Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster was required, the vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated at the start of the study treatment and continued until at least 2 weeks after vaccination or booster was completed.. Note: for US sites participating in Study CLNP023Q12301, the completion of the meningococcal vaccination or booster is required for patients with gMG prior to initiating study treatment, irrespective of prophylactic antibiotic use.
If not previously vaccinated, vaccination against Haemophilus influenzae infections should be given, if available and according to local guidelines, at least 2 weeks prior to first study drug administration.
Positive serology testing for AChR+ antibody at screening
Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG at screening and likely not in need of a respirator for the duration of the study, as judged by the Investigator
The confirmation of the diagnosis of gMG should be documented and supported by 1 of the following 3 tests: History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation. History of positive test with short-acting acetylcholinesterase inhibitors (e.g. neostigmine or edrophonium chloride) Patient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician.
Baseline MG-ADL score 6, with 50% of the total score due to non-ocular symptoms
Participants receiving at least one of the following treatments for gMG for 6 months prior to baseline One or more NSISTs; or plasmapheresis, plasma exchange, or intravenous immunoglobulin (at least quarterly) to control symptoms despite treatment with steroids and NSISTs; or an approved FcRN antagonist; or rituximab; or other approved gMG disease modifying therapies excluding complement inhibitors
Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster was required, the vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated at the start of the study treatment and continued until at least 2 weeks after vaccination or booster was completed.. Note: for US sites participating in Study CLNP023Q12301, the completion of the meningococcal vaccination or booster is required for patients with gMG prior to initiating study treatment, irrespective of prophylactic antibiotic use.
If not previously vaccinated, vaccination against Haemophilus influenzae infections should be given, if available and according to local guidelines, at least 2 weeks prior to first study drug administration.
CRITERIOS DE EXCLUSIÓN
Have been treated with intravenous immunoglobulin (IVIG)/plasma exchange (PLEX) in the past month, with rituximab in the past 6 months, eculizumab in the past 2 months, ravulizumab or other complement inhibitors in the past 3 months, efgartigimod or other anti-FcRn therapies in the past 3 months, or had a thymectomy in the past 6 months or a planned thymectomy during the trial period.
Participants with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with: Active Hepatitis B Virus (HBV); Active Hepatitis C Virus (HCV); Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count 200 cells/mm3
Female participants who are pregnant or lactating, or are intending to become pregnant
Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment and an additional one week following cessation of study treatment.
Active systemic bacterial, viral (including COVID-19) or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy within 14 days prior to study drug administration
History of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae
Presence of fever 38 °C (100.4 °F) within 7 days prior to study drug administration
Participants with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with: Active Hepatitis B Virus (HBV); Active Hepatitis C Virus (HCV); Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count 200 cells/mm3
Female participants who are pregnant or lactating, or are intending to become pregnant
Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment and an additional one week following cessation of study treatment.
Active systemic bacterial, viral (including COVID-19) or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy within 14 days prior to study drug administration
History of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae
Presence of fever 38 °C (100.4 °F) within 7 days prior to study drug administration
DESCRIPCIÓN
To demonstrate efficacy of iptacopan compared to placebo in patients with gMG on stable SOC in reducing the total score of the Myasthenia Gravis Activity of Daily Living (MG-ADL) scale at 6 months (Day 180) of treatment
PALABRAS CLAVE
anemia, idiopathic thrombocytopenic purpura, idiopathic thrombocytopenic purpura (itp), immune thrombocytopenia, iptacopan, myelodysplastic syndrome, myelofibrosis, purpura, púrpura trombocitopénica idiopática (pti), trombocitopenia
