Clinical Trial Goal
To find out if iptacopan is safe and works well to treat PNH in children and teens
You may be able to join this trial if you:
Your child:
- Is 2 - 18 years old
- Has PNH
- Have not had a blood or marrow transplant (BMT) in the last 1 year
- You agree to have other standard tests done to see if your child can be in the clinical trial
Trial Details
Iptacopan is a small molecule inhibitor of complement factor B.
Your child will get:
Your child will get:
- Iptacopan – A pill that your child takes by mouth. The dose your child will get depends on when they start the trial and how safe it has been
The clinical trial doctors will check your child's health for up to 1 year.
The Food and Drug Administration (FDA) has approved iptacopan to treat PNH in adults. Using it in this way to treat PNH in children and teens is new and unproven.
Contacts
Novartis Pharmaceuticals, 1-888-669-6682, novartis.email@novartis.com
Novartis Pharmaceuticals, +41613241111
Locations
Childrens Healthcare of AtlantaRECRUITING
Atlanta, Georgia
Robert Uddo, robert.uddo@choa.org
Cancer Institute of New JerseyRECRUITING
New Brunswick, New Jersey
Serban Morojanu, +1 732 235 7577, moroiasa@cinj.rutgers.edu
Childrens Hospital of PhiladelphiaRECRUITING
Philadelphia, Pennsylvania
Peter Nicholas, +1 267 425 2136, nicholasp@email.chop.edu
St Jude Childrens Research HospitalRECRUITING
Memphis, Tennessee
Michelle Brignac, michelle.brignac@stjude.org
Sponsors
lead: Novartis Pharmaceuticals
