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Efficacy and Safety Study of EGCG/Tocotrienol in 18 Patients With Splicing-mutation-mediated Cystic Fibrosis (CF)

NCT00889434 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2017-11-30

No results posted yet for this study

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 administration of EGCG and Tocotrienol, both separate and in combination, modify CFTR splicing towards normal splicing as evaluated by improved Transepithelial Potential Difference (TEPD) assessment of chloride secretion.

To assess the effect of EGCG and Tocotrienol, both separate and in combination, on (1) additional TEPD measures of ion channel activity, (2) levels of correctly spliced CFTR mRNA in nasal mucosa, (3) cytokine levels in sputum and (4) changes in pulmonary function over the course of the study.

* Potential Implications to Medicine: Alternative splicing mechanisms are a common cause of genetic disease as \~15% of all known human mutations result in defective pre-mRNA splicing. Therapies based on augmenting the levels of full length or fully functioning proteins may have a substantial impact on the treatment of patients with genetic diseases.
* Contribution of the expected outcome to society Today genetic diseases can be treated but not healed. This proposal may be a step in the direction of finding a cure for patients carrying splicing mutations.

Conditions

Interventions

DIETARY_SUPPLEMENT

ECGC

1 cycle will comprise 28 days of continuous daily treatment with EGCG given once/day(total 375 mg) /day in the morning with food followed by a 14 day wash out period without drug.

DIETARY_SUPPLEMENT

Tocotrienol

One cycle will comprise 28 days of continuous daily treatment with Tocotrienol given 2 times/6 soft gel capsules (total 600 mg) /day (BID) in the morning and in the evening with food followed by a 14 day wash out period without drug.

DIETARY_SUPPLEMENT

EGCG + Tocotrienol

combination of both arms 375 mg EGCG + 600mg/day Tocotrienol

Sponsors & Collaborators

  • Hadassah Medical Organization

    lead OTHER

Principal Investigators

  • Eitan Kerem, MD · Hadassah Medical Organization

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
CROSSOVER

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-11-01
Primary Completion
2015-04-16
Completion
2015-04-16

Countries

  • Israel

Study Locations

More Related Trials

Entities

Diseases

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 NCT00889434 on ClinicalTrials.gov