Phase II clinical study for the therapy of cystic fibrosis patients with aspecific mutatio

• Conditions: Cystic fibrosis patients with F508del-CFTR in homozygous or compound heterozygous with Class I or II mutations; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2013-001258-82-IT
• Lead Sponsor: European Institute for Cystic Fibrosis Research (IERFC)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-07-29
• Last Update Posted: 18 January 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

-Diagnosis of cystic fibrosis by sweat test with chloride level=60 mEq/l and confirmed by genetic analysis in order to search cystic fibrosis F508del-CFTR homozigotic mutation or F508del-CFTR combined mutations of class I and II
- males or females aged at least 6 years old.
- fertile females should accept a contraceptive method.
- FEV1 =40% as compared to expected value for age and sex.
- At least 2 sweat tests within the 3 months before Meeting 1 to
establish the interindividual variation of chloride level in the sweat.
-Informed agreement
- All subjects must be able to understand the aim of the study
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 7
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Treatment with glucocorticoids per os or via inhalationat Meeting 1 or within 4 weeks before meeting 1.
- Treatment with oxygen, over day or over night.
- Other experimental drugs.
- Hypersensitivity (local or general) to cysteamine or penicillamine.
- Modifications in the therapy with macrolides, ant-asmatic, mucolytic
drugs, Dornase alfa, and/or FANS, DHA within 28 days before Meeting 1.

However, ita can be accepted the use of some drugs (for example FANS).
- Lung/hepatic/other organ transplantation.
- Kidney or hepatic alterations at Meeting 1:
-AST, ALT > 5 fold normal value . -
Creatinine > 2 fold normal value (ULN).
- Pregnancy
- Nursing.
- No contraceptive method.
- Neurologic and psychiatric pathologies that, on the basis of the Researcher
experience, could interfere with protocol

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified