Phase
Condition
Aplastic Anemia
Anemia
Bone Marrow Disorder
Treatment
Iptacopan
Clinical Study ID
Ages > 18 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
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 andwith a diagnosis of PNH confirmed by treating physician.
Stable regimen (dose and intervals) of anti-C5 antibody treatment (either eculizumabor ravulizumab) for at least 6 months prior to screening
Mean hemoglobin level ≥10 g/dL
Vaccination against Neisseria meningitidis and S. pneumoniae infection are requiredprior to the start of iptacopan treatment.
If not received previously, vaccination against Haemophilus influenzae infections isrecommended, if available and according to local regulations.
Ability to communicate well with the investigator, to understand and comply with therequirements of the study
Other protocol -defined inclusion criteria may apply at the end.
Exclusion
Exclusion Criteria:
Participation in any other investigational drug trial or use of otherinvestigational drugs at the time of enrollment
Patients requiring red blood cell transfusion in the 6 months prior to screening orduring screening
History of stem cell transplantation or any solid organ transplantation
Active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 daysprior to study drug administration
Presence of fever ≥ 38.0 °C (100.4 °F) within 7 days prior to study drugadministration
Human immunodeficiency virus (HIV) infection (known history of HIV or test positivefor 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, orunstable thrombotic event not amenable to active treatment as judged by theinvestigator 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 inthe trial.
Any medical condition deemed likely to interfere with the patient's participation inthe study
Female patients who are pregnant or breastfeeding, or intending to conceive duringthe course of the study
Study Design
Study Description
Connect with a study center
Novartis Investigative Site
Nantes Cedex 1, 44093
FranceSite Not Available
Novartis Investigative Site
Nice, 06202
FranceSite Not Available
Novartis Investigative Site
Paris 10, 75475
FranceSite Not Available
Novartis Investigative Site
Aachen, 52074
GermanySite Not Available
Novartis Investigative Site
Dresden, 01307
GermanySite Not Available
Novartis Investigative Site
Essen, 45147
GermanySite Not Available
Novartis Investigative Site
Ulm, 89081
GermanySite Not Available
Novartis Investigative Site
Avellino, AV 83100
ItalySite Not Available
Novartis Investigative Site
San Giovanni Rotondo, FG 71013
ItalySite Not Available
Novartis Investigative Site
Firenze, FI 50134
ItalySite Not Available
Novartis Investigative Site
Bassano Del Grappa, Vicenza 36061
ItalySite Not Available
Novartis Investigative Site
Seoul, 06351
Korea, Republic ofSite Not Available
Novartis Investigative Site
Barcelona, Catalunya 08036
SpainSite Not Available
Novartis Investigative Site
Aydin, 09100
TurkeySite Not Available
Novartis Investigative Site
Istanbul, 34093
TurkeySite Not Available
Novartis Investigative Site
Leeds, West Yorkshire LS9 7TF
United KingdomSite Not Available
Novartis Investigative Site
London, SE5 9RS
United KingdomSite Not Available
Novartis Investigative Site
Cerritos, California 90703
United StatesSite Not Available
City Of Hope National Med Center
Duarte, California 91010
United StatesSite Not Available
City Of Hope National Med Center City of Hope Medical Center
Duarte, California 91010
United StatesSite Not Available
Novartis Investigative Site
Duarte, California 91010
United StatesActive - Recruiting
Novartis Investigative Site
Los Angeles, California 90033
United StatesActive - Recruiting
USC Norris Cancer Center
Los Angeles, California 90033
United StatesSite Not Available
Novartis Investigative Site
Torrance, California 90509-2910
United StatesSite Not Available
Lakes Research
Miami Lakes, Florida 33014
United StatesSite Not Available
Novartis Investigative Site
Miami Lakes, Florida 33014
United StatesSite Not Available
Novartis Investigative Site
Augusta, Georgia 30912
United StatesSite Not Available
Mass Gen Hosp Cancer Center
Boston, Massachusetts 02114
United StatesSite Not Available
Novartis Investigative Site
Boston, Massachusetts 02114
United StatesActive - Recruiting
Novartis Investigative Site
Worcester, Massachusetts 01665
United StatesSite Not Available
Novartis Investigative Site
Minneapolis, Minnesota 55455
United StatesActive - Recruiting
University Of Minnesota
Minneapolis, Minnesota 55455
United StatesSite Not Available
Novartis Investigative Site
Saint Louis, Missouri 63110
United StatesSite Not Available
Montefiore Medical Center
Bronx, New York 10461
United StatesSite Not Available
Montefiore Medical Center .
Bronx, New York 10461
United StatesSite Not Available
Novartis Investigative Site
Bronx, New York 10461
United StatesActive - Recruiting
Novartis Investigative Site
Durham, North Carolina 27710
United StatesSite Not Available
Cleveland Clinic Foundation
Cleveland, Ohio 44195
United StatesSite Not Available
Novartis Investigative Site
Cleveland, Ohio 44195
United StatesActive - Recruiting
Novartis Investigative Site
Greenville, South Carolina 29615
United StatesActive - Recruiting
Prisma Health Upstate
Greenville, South Carolina 29615
United StatesSite Not Available
Huntsman Cancer Institute Univ of Utah
Salt Lake City, Utah 84112 0550
United StatesSite Not Available
Huntsman Cancer Institute Univ of Utah .
Salt Lake City, Utah 84112 0550
United StatesSite Not Available
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