NCT00889434

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

Completed NA Last updated 30 November 2017

What this trial tests

NA trial testing ECGC in Cystic Fibrosis in 7 participants. Completed in 16 April 2015.

Timeline

1 November 2011

Primary endpoint
16 April 2015

16 April 2015

Quick facts

Lead sponsor	Hadassah Medical Organization
Phase	NA
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	none
Primary purpose	treatment
Enrollment	7
Start date	1 November 2011
Primary completion	16 April 2015
Estimated completion	16 April 2015
Sites	1 location across Israel

Drugs / interventions tested

• ECGC
• Tocotrienol — full drug profile →
• EGCG + Tocotrienol

Conditions studied

• Cystic Fibrosis — all drugs for Cystic Fibrosis →

Sponsor

Hadassah Medical Organization — full company profile →

Who can join

18 and older, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

* 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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.