Ignite Creation Date:
2024-05-05 @ 11:23 AM
Last Modification Date:
2024-10-26 @ 9:06 AM
Study NCT ID:
NCT00016744
Status:
COMPLETED
Last Update Posted:
2009-01-09
First Post:
2001-05-31
Brief Title:
PhenylbutyrateGenistein Duotherapy in Delta F508-Homozygousfor Cystic Fibrosis
Sponsor:
Childrens Hospital of Philadelphia
Organization:
Childrens Hospital of Philadelphia
Study Overview
Official Title:
A Pilot Trial of PhenylbutyrateGenistein Duotherapy in Delta F508-Homozygous Cystic Fibrosis Patients
Status:
COMPLETED
Status Verified Date:
2009-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
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
Detailed Description:
Subjects will be randomized to receive either the Phenylbutyrate or placebo tablets for 1 week with a 2 out of 3 chance of receiving Phenylbutyrate at a standard adult dose with visits on study days 1 4 and 7 The study will last an additional 2 weeks to determine whether the effects of the Phenylbutyrate or Placebo persist for any length of time with visits on study days 14 and 21 Every participant will receive the Genistein during the NPD on days 1 and 7 The dose of Genistein used will not be escalated and will be the same for every participant
Safety evaluations at each visit will include a history and physical exam and mental status exam blood and urine tests and lung function tests The main physiologic outcome of the trial will be the assessment of salt and water transport NPD In this way we will assess whether genistein enhances the effect of phenylbutyrate to improve chloride transport in the nose by topical application of genistein to the nasal mucosa during the NPD procedure on study days 1 and 7
Safety evaluations at each visit will include a history and physical exam and mental status exam blood and urine tests and lung function tests The main physiologic outcome of the trial will be the assessment of salt and water transport NPD In this way we will assess whether genistein enhances the effect of phenylbutyrate to improve chloride transport in the nose by topical application of genistein to the nasal mucosa during the NPD procedure on study days 1 and 7
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| IRB2000-10-2189 | None | None | None |