A study to identify whether it is safe, if it works, and how much and how often cysteamine should be given to adult patients with Cystic Fibrosis (CF) who are being treated for a worsening of CF associated lung disease.

A Randomized, Double-Blind, Parallel Group, Placebo-Controlled Study Investigating the Optimal Dose Regimen, Efficacy, and Safety of Adding Oral Cysteamine in Adult Patients with Cystic Fibrosis (CF) Being Treated for an Exacerbation of CF-associated Lung Disease.

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-004986-99-IT

Enrollment

120

Registered

2016-09-06

Start date

2017-01-11

Completion date

Unknown

Last updated

2018-02-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

exacerbation of Cystic Fibrosis MedDRA version: 19.0 Level: PT Classification code 10070608 Term: Infective pulmonary exacerbation of cystic fibrosis System Organ Class: 10021881 - Infections and infestations

Interventions

Product Name: cysteamine bitartrate Pharmaceutical Form: Capsule, hard Pharmaceutical form of the placebo: Capsule, hard Route of administration of the placebo: Oral use

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. CF-associated lung disease with documented history of chronic infection with Gram-negative organism(s) 2. Established patient of the Principal Investigator's CF Multi-Disciplinary team 3. Age equal or greater than 18 years 4. Weight equal or more than 40 Kg 5. FEV1 more than 30% of predicted within the 6 months prior to study exacerbation 6. At baseline visit: experiencing a new exacerbation of CF associated lung disease requiring treatment that includes an aminoglycoside antibiotic Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 120 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: 1. Hypersensitive to cysteamine or to any of the excipients 2. Hypersensitive to penicillamine 3. Transplant recipient

Design outcomes

Primary

Measure	Time frame
Main Objective: - to determine the optimal dose and frequency of cysteamine in exacerbations of CF-associated lung disease - to determine the best questionnaire for evaluation of clinical benefit arising from use of cysteamine in exacerbations of CF-associated lung disease - to determine the effects of treatment with cysteamine on safety parameters;Secondary Objective: to determine the effects of treatment with cysteamine on an exacerbation of CF-associated lung disease for each of the following: - sputum IL8 and neutrophil elastase levels - forced expiratory volume in the first second (FEV1) - weight - C-reactive protein (CRP) - blood leukocyte count - assessment of blood and sputum cysteamine levels;Primary end point(s): - Change from baseline in patient health-related questionnaires (CFRSD-CRISS, Jarad and Sequeiros Smptom Score Questionnaire - Change from baseline in sputum bacterial load of (a) total CFU per ml and per mg and (b) gram negative CFU per ml and per mg at Day 7, Day 14 and Day 21 following a CF exacerbation - Change from baseline in sputum IL8 and neutrophil elastase levels at Day 7, Day 14 and Day 21 following a CF exacerbation - Change from baseline to Day 7, Day 14 and Day 21 in FEV1, weight, CRP, blood leucocyte count and CFQ-R - Assessment of blood and sputum cysteamine levels at Day 14 - Patient Global Assessment of Exacerbation outcome;Timepoint(s) of evaluation of this end point: See section E.5.1. Primary End Points	—

Measure

Time frame

Main Objective: - to determine the optimal dose and frequency of cysteamine in exacerbations of CF-associated lung disease - to determine the best questionnaire for evaluation of clinical benefit arising from use of cysteamine in exacerbations of CF-associated lung disease - to determine the effects of treatment with cysteamine on safety parameters;Secondary Objective: to determine the effects of treatment with cysteamine on an exacerbation of CF-associated lung disease for each of the following: - sputum IL8 and neutrophil elastase levels - forced expiratory volume in the first second (FEV1) - weight - C-reactive protein (CRP) - blood leukocyte count - assessment of blood and sputum cysteamine levels;Primary end point(s): - Change from baseline in patient health-related questionnaires (CFRSD-CRISS, Jarad and Sequeiros Smptom Score Questionnaire - Change from baseline in sputum bacterial load of (a) total CFU per ml and per mg and (b) gram negative CFU per ml and per mg at Day 7, Day 14 and Day 21 following a CF exacerbation - Change from baseline in sputum IL8 and neutrophil elastase levels at Day 7, Day 14 and Day 21 following a CF exacerbation - Change from baseline to Day 7, Day 14 and Day 21 in FEV1, weight, CRP, blood leucocyte count and CFQ-R - Assessment of blood and sputum cysteamine levels at Day 14 - Patient Global Assessment of Exacerbation outcome;Timepoint(s) of evaluation of this end point: See section E.5.1. Primary End Points

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Secondary

Measure	Time frame
Secondary end point(s): 1. Assessment of blood and sputum cysteamine levels at Day 14 2. Change from baseline in sputum bacterial load of (a) total CFU per ml and per mg and (b) gram negative CFU per ml and per mg at Day 7, Day 14 and Day 21 following a CF exacerbation 3 .Change from baseline in sputum IL8 and neutrophil elastase levels at Day 7, Day 14 and Day 21 following a CF exacerbation 4. Change from baseline to Day 7, Day 14 and Day 21 in FEV1, weight, CRP, blood leucocyte count and CFQ-R 5. Patient Global Assessment of Exacerbation outcome ;Timepoint(s) of evaluation of this end point: 1. Day 14 2. Day 7, 14, 21 3. Day 7, 14, 21 4. Day 7, 14, 21 5. EoS	—

Measure

Time frame

Secondary end point(s): 1. Assessment of blood and sputum cysteamine levels at Day 14 2. Change from baseline in sputum bacterial load of (a) total CFU per ml and per mg and (b) gram negative CFU per ml and per mg at Day 7, Day 14 and Day 21 following a CF exacerbation 3 .Change from baseline in sputum IL8 and neutrophil elastase levels at Day 7, Day 14 and Day 21 following a CF exacerbation 4. Change from baseline to Day 7, Day 14 and Day 21 in FEV1, weight, CRP, blood leucocyte count and CFQ-R 5. Patient Global Assessment of Exacerbation outcome ;Timepoint(s) of evaluation of this end point: 1. Day 14 2. Day 7, 14, 21 3. Day 7, 14, 21 4. Day 7, 14, 21 5. EoS

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Countries

Italy, Netherlands, United Kingdom, United States

Contacts

Public ContactDeborah O'Neil

NovaBiotics, Ltd.

Deborah@novabiotics.co.uk441224711377

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026