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Phenylbutyrate/Genistein Duotherapy in Delta F508-Homozygous(for Cystic Fibrosis)

NCT00016744 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2009-01-09

No results posted yet for this study

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).

Conditions

Interventions

DRUG

Sodium 4-Phenylbutyrate (4PBA)

The standard oral adult dose is 20g/day (tablets) for 4 days.

DRUG

Unconjugated Isoflavones 100 (PTI G-4660, 87% Genistein)

Every participant will be administered a perfusion of 50 MicroM of Genistein during the modified NPD procedure.

DRUG

Placebo

The placebo dose will match the oral tablets in arm 1, maintaining the study blind.

Sponsors & Collaborators

Principal Investigators

  • Ronald Rubenstein, M.D., PhD. · Children's Hospital of Philadelphia

Study Design

Allocation
RANDOMIZED
Purpose
BASIC_SCIENCE
Masking
QUADRUPLE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2001-09-30
Primary Completion
2005-10-31
Completion
2005-10-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00016744 on ClinicalTrials.gov