Cysteamine in Cystic Fibrosis

An open label investigation of the tolerability and pharmacokinetics of oral cysteamine in adults with Cystic Fibrosis. - Cysteamine in Cystic Fibrosis (2)

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-000284-40-GB

Enrollment

Registered

2014-04-29

Start date

2014-06-05

Completion date

Unknown

Last updated

2017-04-10

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

Conditions

Cystic Fibrosis MedDRA version: 16.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Cystagon (cysteamine bitartrate) immediate-release capsules Product Name: Cystagon (cysteamine bitartrate) immediate-release capsules Pharmaceutical Form: Capsule, hard INN or Proposed INN

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. CF related suppurative lung disease who expectorate sputum 2. Clinically stable for >4 weeks, 3. Aged =18 years, 4. Weight >50kg. 5. Female participants of child bearing potential should be using a reliable form of contraception. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 11 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1

Exclusion criteria

Exclusion criteria: 1. Hypersensitivity to the active substance, any form of cysteamine, or to any of the excipients. 2. Hypersensitivity to penicillamine. 3. Lung, liver transplant, on active transplant list. 4. For women, current pregnancy or breast-feeding, or planned pregnancy during the study. 5. Any other significant disease/disorder which, in the investigator’s opinion, either puts the patient at risk because of study participation or may influence the results of the study or the patient's ability to participate in the study.

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective of this study is to characterise the blood pharmacokinetic profile of oral immediate-release cysteamine (Cystagon) when administered to patients with Cystic Fibrosis at the dose licensed for use in cystinosis.;Secondary Objective: The secondary objectives are to demonstrate that when oral immediate-release cysteamine (Cystagon) is administered to patients with Cystic Fibrosis at the dose licensed for use in cystinosis: 1. Cysteamine enters the bronchial secretions 2. Cysteamine is tolerated A further secondary objective is for the Contract Research Organisation (CRO) to establish a method to quantify cysteamine in the sputum of patients with CF.;Primary end point(s): The primary outcome will be blood cysteamine measured at baseline, 30mins, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, and 24 hours after oral administration of immediate-release cysteamine (Cystagon) to patients with Cystic Fibrosis at the dose licensed for use in cystinosis. ;Timepoint(s) of evaluation of this end point: Baseline, 30mins, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, and 24 hours after oral administration of immediate-release cysteamine (Cystagon)	—

Measure

Time frame

Main Objective: The primary objective of this study is to characterise the blood pharmacokinetic profile of oral immediate-release cysteamine (Cystagon) when administered to patients with Cystic Fibrosis at the dose licensed for use in cystinosis.;Secondary Objective: The secondary objectives are to demonstrate that when oral immediate-release cysteamine (Cystagon) is administered to patients with Cystic Fibrosis at the dose licensed for use in cystinosis: 1. Cysteamine enters the bronchial secretions 2. Cysteamine is tolerated A further secondary objective is for the Contract Research Organisation (CRO) to establish a method to quantify cysteamine in the sputum of patients with CF.;Primary end point(s): The primary outcome will be blood cysteamine measured at baseline, 30mins, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, and 24 hours after oral administration of immediate-release cysteamine (Cystagon) to patients with Cystic Fibrosis at the dose licensed for use in cystinosis. ;Timepoint(s) of evaluation of this end point: Baseline, 30mins, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, and 24 hours after oral administration of immediate-release cysteamine (Cystagon)

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Secondary

Measure	Time frame
Secondary end point(s): 1. Sputum concentration of cysteamine 3 hours after final dosing. 2. Adverse events (tolerability) 3. Lung function (FEV1, FVC) 4. Weight 5. Disease specific health status using the CFQ-R 6. Quantitative sputum microbiology, sputum rheology, bacterial sensitivities ;Timepoint(s) of evaluation of this end point: CFQ-R will be assessed at recruitment and after 5 weeks sputum, adverse events, lung function, weight and sputum microbiology will be assessed at recruitment, 1,2,3 and 5 weeks	—

Measure

Time frame

Secondary end point(s): 1. Sputum concentration of cysteamine 3 hours after final dosing. 2. Adverse events (tolerability) 3. Lung function (FEV1, FVC) 4. Weight 5. Disease specific health status using the CFQ-R 6. Quantitative sputum microbiology, sputum rheology, bacterial sensitivities ;Timepoint(s) of evaluation of this end point: CFQ-R will be assessed at recruitment and after 5 weeks sputum, adverse events, lung function, weight and sputum microbiology will be assessed at recruitment, 1,2,3 and 5 weeks

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Countries

United Kingdom

Outcome results

None listed

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