Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis)
Status:
Terminated
Trial end date:
2008-12-01
Target enrollment:
Participant gender:
Summary
The purpose of this research study is to test a new combination of medicines, Phenylbutyrate
and Genistein, to determine if they could be used to treat cystic fibrosis (CF). The most
common genetic mutation found in patients with CF is called Delta F508. Due to this mutation,
there is a lack of salt (chloride) movement in your nose, sinuses, lungs, intestines,
pancreas and sweat glands. This lack of movement causes the clinical manifestations of the
disease.
Although Phenylbutyrate has been extensively used to treat patients with rare metabolic
diseases, Phenylbutyrate is an investigational drug for the purpose of this study. Genistein
is a naturally occurring substance that is found in food products such as soy and tofu, but
is also an investigational drug for this study. When used together, both drugs may be able to
restore normal chloride and salt (water) movements in body organs and glands in people with
CF.
We will be studying salt and water movement in the nose by a technique called nasal
transepithelial potential difference (NPD).
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Children's Hospital of Philadelphia
Collaborators:
Cystic Fibrosis Foundation Cystic Fibrosis Foundation Therapeutics