Single-site, Open-label, Dose-ranging, Efficacy, and Safety Study of EGCG/Tocotrienol in 18 Patients With Splicing-mutation-mediated CF

Eitan Kerem, MD Hadassah Medical Organization

Ongoing

Objective:
  To determine in patients with CF if oral administration of epigallocatechin gallate (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.

Study Type: Interventional

Study Design:
Randomized, Open Label, Cross-Over Study

Subjects: Patients with cystic fibrosis

Intervention:
1) Dietary Supplement– ECGC
2) Dietary Supplement — Tocotrienol
3) Dietary Supplement — EGCG + Tocotrienol

Primary Outcome:

  • Safety and Efficacy. Changes in nasal chloride secretion as assessed by TEPD,

Secondary Outcome:

  • Pulmonary function
  • Levels of CFTR mRNA
  • Cytokine levels in sputum ­­­­­­­­­
  • TEPD measures of ion channel activity

 Methodology: Patients with CF carrying splicing mutations will be treated with EGCG 200 mg/day, Tocotrienol 600mg/day or both for 28 day cycles. Clinical parameters (TEPD, FEV1 and cytokine levels in sputum) and molecular parameters (mRNA levels,) will be analyzed to determine the effectiveness of the treatment.  In vitro studies with cell cultures derived from CF patients have shown positive results; therefore an improvement in TEPD will be an indication for CFTR expression. An increase in mRNA levels and changes in FEV1, and cytokine levels will confirm the results.

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