Phenylbutyrate/Genistein Duotherapy in Delta F508-Homozygous(for Cystic Fibrosis)
Status:
Completed
Trial end date:
2005-10-01
Target enrollment:
Participant gender:
Summary
We are testing a new combination of medicines, to determine if they could be used to treat
cystic fibrosis (CF). Subjects with CF who have two copies of the most common mutation
(change) found in patients with CF called DF508. CF is caused by a lack of chloride movement
in the nose, sinuses, lungs, intestines, pancreas and sweat glands. We are conducting this
study to determine the safety of using a combination of two medicines, Phenylbutyrate and
Genistein, to improve the ability of the cells lining the nose to regulate movement of salt
(chloride) and water in people with CF.
Phenylbutyrate has been extensively used to treat patients with rare metabolic diseases
(which are very different from CF), 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. Both drugs may be
able to restore normal chloride movements in body organs and glands. We will be studying salt
and water in the nose movement 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 National Center for Research Resources (NCRR)