Status:
COMPLETED
Efficacy and Safety Study of EGCG/Tocotrienol in 18 Patients With Splicing-mutation-mediated Cystic Fibrosis (CF)
Lead Sponsor:
Hadassah Medical Organization
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
18+ years
Phase:
NA
Brief Summary
* Working Hypothesis: EGCG and Tocotrienol can act as genetic modifiers and increase the level of correctly spliced CFTR transcripts. * Aims of the Study: To determine in patients with CF if oral admi...
Detailed Description
Background: Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF trans-membrane conductance regulator protein. Despite substantial progress achieved in the understandi...
Eligibility Criteria
Inclusion
- Confirmed diagnosis of CF.
- Abnormal chloride secretion as measured by TEPD (a less than -5 mV TEPD assessment of chloride secretion with chloride-free amiloride and isoproterenol).
- Presence of a splicing mutation in at least one allele of the CFTR gene.
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures (including TEPD measurements, clinical laboratory tests,), and study restrictions.
- Ability to provide written informed consent.
- Evidence of personally signed and dated informed consent document indicating that the patient has been informed of all pertinent aspects of the trial.
Exclusion
- Prior or ongoing medical condition (e.g., concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the patient, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
- Ongoing acute illness including acute upper or lower respiratory infections within 2 weeks before start of study treatment.
- History of major complications of lung disease (including recent massive hemoptysis or pneumothorax) within 2 months prior to start of study treatment.
- Abnormalities on screening chest x-ray suggesting clinically significant active pulmonary disease other than CF, or new, significant abnormalities such as atelectasis or pleural effusion which may be indicative of clinically significant active pulmonary involvement secondary to CF.
- Abnormal liver function (serum ALT, AST, GGT, alkaline phosphatase, LDH, or total bilirubin \> 2 times upper limit of normal). Abnormal renal function (serum creatinine \>1.5 times upper limit of normal).
- Pregnancy or breast-feeding.
- History of solid organ or hematological transplantation.
- Ongoing participation in any other therapeutic clinical trial or exposure to another investigational drug within 14 days prior to start of study treatment.
- Change in intranasal medications (including use of corticosteroids, cromolyn, ipratropium bromide, phenylephrine, or oxymetazoline) within 14 days prior to start of study treatment.
- Change in treatment with systemic or inhaled corticosteroids within 14 days prior to start of study treatment.
Key Trial Info
Start Date :
November 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 16 2015
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT00889434
Start Date
November 1 2011
End Date
April 16 2015
Last Update
November 30 2017
Active Locations (1)
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1
Hadassah Medical Organization, Mount Scopus
Jerusalem, Israel, 91240