Clinical trial • Phase III • Immunology|Rare Disease|Haematology
DANICOPAN for Paroxysmal nocturnal haemoglobinuria
Phase III trial of DANICOPAN for Paroxysmal nocturnal haemoglobinuria. open-label, none/not specified-controlled. 55 participants.
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease|Haematology
- Trial Disease
- Paroxysmal nocturnal haemoglobinuria
- Trial Stage
- Phase III
- Drug Modality
- Small molecule|Monoclonal antibody
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 07-05-2024
- First CTIS Authorization Date
- 04-07-2024
Trial design
open-label, none/not specified-controlled Phase III trial across 19 sites in France, Spain, Greece and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 55
- Trial Duration For Participant
- 152
Eligibility
Recruits 55 Vulnerable population selected (isVulnerablePopulationSelected = true). Subject information and informed consent forms are listed in the application documents (multiple ICF documents available per country). Specific assent/consent handling details are not provided in the JSON..
- Pregnancy Exclusion
- Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.
- Vulnerable Population
- Vulnerable population selected (isVulnerablePopulationSelected = true). Subject information and informed consent forms are listed in the application documents (multiple ICF documents available per country). Specific assent/consent handling details are not provided in the JSON.
Inclusion criteria
- {"criterion_text":"- All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment"}
- {"criterion_text":"- Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors."}
Exclusion criteria
- {"criterion_text":"- Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant."}
- {"criterion_text":"- Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Change in hemoglobin (Hgb) values over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Proportion of patients with Hgb increase of ≥ 2g/dL in the absence of transfusion over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in absolute reticulocyte count over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in lactate dehydrogenase (LDH) over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Proportion of patients with LDH ≤ 1.5 × upper limit of normal (ULN) over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Proportion of patients with transfusion avoidance (TA), defined as patients who remain transfusion free and do not require transfusion as per protocol specified guidelines","definition_or_measurement_approach":"Defined as patients who remain transfusion free and do not require transfusion as per protocol specified guidelines"}
- {"endpoint_text":"- Change in FACIT Fatigue scores over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in EORTC-QLQ-C30 scores over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- Change in safety laboratory parameters over time","definition_or_measurement_approach":""}
- {"endpoint_text":"- TEAEs leading to discontinuation","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 55
- Recruitment Window Months
- 43
- Consent Approach
- Informed consent obtained using subject information and informed consent forms. ICF documents are provided in multiple country-specific versions (examples in Spanish, Greek, Czech, Polish, Italian, English). Specific age-based assent documents or detailed consent process steps are not available in the JSON.
Geography
- Total Number Of Sites
- 19
- Total Number Of Participants
- 25
France
- Latest Decision Or Authorization Date
- 04-07-2024
- Number Of Sites
- 4
- Number Of Participants
- 5
Sites
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Service Maladies du Sang
- Principal Investigator Name
- Louis TERRIOU
- Principal Investigator Email
- louis.terriou@chru-lille.fr
- Contact Person Name
- Louis TERRIOU
- Contact Person Email
- louis.terriou@chru-lille.fr
- Site Name
- Centre Hospitalier Universitaire De Bordeaux
- Department Name
- Service d'Hematologie et de therapie cellulaire
- Principal Investigator Name
- Edouard FORCADE
- Principal Investigator Email
- edouard.forcade@chu-bordeaux.fr
- Contact Person Name
- Edouard FORCADE
- Contact Person Email
- edouard.forcade@chu-bordeaux.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Service d'Hematologie-Greffe
- Principal Investigator Name
- Flore SICRE de FONTBRUNE
- Principal Investigator Email
- flore.sicre-de-fontbrune@aphp.fr
- Contact Person Name
- Flore SICRE de FONTBRUNE
- Contact Person Email
- flore.sicre-de-fontbrune@aphp.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Service d'Hematologie
- Principal Investigator Name
- Fiorenza BARRACO
- Principal Investigator Email
- florenza.barraco@chu-lyon.fr
- Contact Person Name
- Fiorenza BARRACO
- Contact Person Email
- florenza.barraco@chu-lyon.fr
Spain
- Latest Decision Or Authorization Date
- 20-08-2025
- Number Of Sites
- 4
- Number Of Participants
- 7
Sites
- Site Name
- Hospital Germans Trias I Pujol
- Department Name
- Haematology Department
- Principal Investigator Name
- Ines Hernandez Rodriguez
- Principal Investigator Email
- ihernandez@iconcologia.net
- Contact Person Name
- Ines Hernandez Rodriguez
- Contact Person Email
- ihernandez@iconcologia.net
- Site Name
- Hospital Universitario Puerta De Hierro De Majadahonda
- Department Name
- Haematology Department
- Principal Investigator Name
- Rafael Fores
- Principal Investigator Email
- rafael.fores@salud.madrid.org
- Contact Person Name
- Rafael Fores
- Contact Person Email
- rafael.fores@salud.madrid.org
- Site Name
- Hospital Clinic De Barcelona
- Department Name
- Haematology Department
- Principal Investigator Name
- Anna Gaya Valls
- Principal Investigator Email
- AGAYAV@clinic.cat
- Contact Person Name
- Anna Gaya Valls
- Contact Person Email
- AGAYAV@clinic.cat
- Site Name
- University Hospital Virgen Del Rocio S.L.
- Department Name
- Haematology Department
- Principal Investigator Name
- Ramiro Nuñez Vazquez
- Principal Investigator Email
- ramirojosenv@gmail.com
- Contact Person Name
- Ramiro Nuñez Vazquez
- Contact Person Email
- ramirojosenv@gmail.com
Greece
- Latest Decision Or Authorization Date
- 04-03-2026
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Geniko Nosokomeio Thessalonikis George Papanikolaou
- Department Name
- Hematology Clinic
- Principal Investigator Name
- Chrysavgi Elissavet Lalagianni
- Principal Investigator Email
- bmt.gpapanikolaou@n3.syzefxis.gov.gr
- Contact Person Name
- Chrysavgi Elissavet Lalagianni
- Contact Person Email
- bmt.gpapanikolaou@n3.syzefxis.gov.gr
- Site Name
- Hippokration Hospital
- Department Name
- B' Internal Medicine Clinic
- Principal Investigator Name
- Stavroula Giannouli
- Principal Investigator Email
- sgiannoul@med.uoa.gr
- Contact Person Name
- Stavroula Giannouli
- Contact Person Email
- sgiannoul@med.uoa.gr
Poland
- Latest Decision Or Authorization Date
- 25-08-2025
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Uniwersyteckie Centrum Kliniczne
- Department Name
- Klinika Hematologii i Transplantologii
- Principal Investigator Name
- Agnieszka Piekarska
- Principal Investigator Email
- babajaga@gumed.edu.pl
- Contact Person Name
- Agnieszka Piekarska
- Contact Person Email
- babajaga@gumed.edu.pl
Czechia
- Latest Decision Or Authorization Date
- 29-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- Interní hematologická a onkologická klinika (IHOK)
- Principal Investigator Name
- Libor Červinek
- Principal Investigator Email
- cervinek.libor@fnbrno.cz
- Contact Person Name
- Libor Červinek
- Contact Person Email
- cervinek.libor@fnbrno.cz
Italy
- Latest Decision Or Authorization Date
- 08-05-2026
- Number Of Sites
- 7
- Number Of Participants
- 9
Sites
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- S.C. Ematologia
- Principal Investigator Name
- Bruno Fattizzo
- Principal Investigator Email
- bruno.fattizzo@policlinico.mi.it
- Contact Person Name
- Bruno Fattizzo
- Contact Person Email
- bruno.fattizzo@policlinico.mi.it
- Site Name
- Azienda Ospedaliero-Universitaria Policlinico Umberto I
- Department Name
- UO Ematologia
- Principal Investigator Name
- Maurizio Martelli
- Principal Investigator Email
- martelli@bce.uniroma1.it
- Contact Person Name
- Maurizio Martelli
- Contact Person Email
- martelli@bce.uniroma1.it
- Site Name
- Regione Del Veneto Azienda ULSS N 7 Pedemontana
- Department Name
- UO Oncoematologia
- Principal Investigator Name
- Eros Di Bona
- Principal Investigator Email
- eros.dibona@aulss7.veneto.it
- Contact Person Name
- Eros Di Bona
- Contact Person Email
- eros.dibona@aulss7.veneto.it
- Site Name
- AORN San Giuseppe Moscati Avellino
- Department Name
- UO Ematologia
- Principal Investigator Name
- Camilla Frieri
- Principal Investigator Email
- camillafrieri@gmail.com
- Contact Person Name
- Camilla Frieri
- Contact Person Email
- camillafrieri@gmail.com
- Site Name
- Careggi University Hospital
- Department Name
- UO Ematologia
- Principal Investigator Name
- Barbara Scappini
- Principal Investigator Email
- scappinib@aou-careggi.toscana.it
- Contact Person Name
- Barbara Scappini
- Contact Person Email
- scappinib@aou-careggi.toscana.it
- Site Name
- Grande Ospedale Metropolitano Bianchi Melacrino Morelli
- Department Name
- UO Ematologia
- Principal Investigator Name
- Maria Bruna Greve
- Principal Investigator Email
- brunagreve@hotmail.it
- Contact Person Name
- Maria Bruna Greve
- Contact Person Email
- brunagreve@hotmail.it
- Site Name
- Regione Del Veneto Azienda ULSS N 7 Pedemontana (duplicate site listing?)
- Department Name
- UO Oncoematologia
- Principal Investigator Name
- Eros Di Bona
- Principal Investigator Email
- eros.dibona@aulss7.veneto.it
- Contact Person Name
- Eros Di Bona
- Contact Person Email
- eros.dibona@aulss7.veneto.it
Sponsor
Primary sponsor
- Full Name
- Alexion Pharmaceuticals Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Endpoint Clinical Inc.
- Responsibilities
- 3
- Name
- Icon Clinical Research Limited
- Responsibilities
- Site Training
- Name
- Medical Research Network Limited
- Responsibilities
- Non-Clinical Site Support Operative
- Name
- Medidata Solutions Inc.
- Responsibilities
- eCRF; 6; 7
- Name
- IQVIA Limited
- Responsibilities
- 1; 10; 12; patient reimbursement, site contract; 3; 6; 7; 9
- Name
- IQVIA RDS Hellas Single Member S.A.
- Responsibilities
- 1; 10; 12; patient reimbursement, site contracts; 3; 6; 7; 9
Third parties
- {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"3","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Site Training","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Medical Research Network Limited","duties_or_roles":"Non-Clinical Site Support Operative","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"eCRF; 6; 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"1; 10; 12; patient reimbursement, site contract; 3; 6; 7; 9","organisation_type":"Pharmaceutical company"}
- {"country":"Greece","full_name":"IQVIA RDS Hellas Single Member S.A.","duties_or_roles":"1; 10; 12; patient reimbursement, site contracts; 3; 6; 7; 9","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- ALXN 2040
- Active Substance
- DANICOPAN
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL USE
- Authorisation Status
- Investigational (no marketing authorisation listed)
- Orphan Designation
- Yes
- Maximum Dose
- 600 mg (maxDailyDoseAmount)
- Investigational Product Name
- Ultomiris 300 mg/30 mL concentrate for solution for infusion
- Active Substance
- RAVULIZUMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- INTRAVENOUS USE
- Route
- INTRAVENOUS USE
- Authorisation Status
- Authorised (marketing authorisation present)
- Maximum Dose
- 3600 mg (maxDailyDoseAmount)
- Investigational Product Name
- Soliris 300 mg concentrate for solution for infusion
- Active Substance
- ECULIZUMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- INTRAVENOUS USE
- Route
- INTRAVENOUS USE
- Authorisation Status
- Authorised (marketing authorisation present)
- Maximum Dose
- 900 mg (maxDailyDoseAmount)
- Combination Treatment
- Yes
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