Cysteamine in Cystic Fibrosis

• Register: EUCTR
• Last refreshed on: 10 April 2017
• Main ID: EUCTR2014-000284-40-GB
• Date of registration: 29/04/2014
• Prospective Registration: Yes
• Primary sponsor: University of Aberdeen
• Public title: Cysteamine in Cystic Fibrosis
• Scientific title: An open label investigation of the tolerability and pharmacokinetics of oral cysteamine in adults with Cystic Fibrosis. - Cysteamine in Cystic Fibrosis (2)
• Date of first enrolment: 05/06/2014
• Target sample size: 12
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-000284-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

United Kingdom

Key inclusion & exclusion 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:
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.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

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

Intervention(s)

Trade Name: Cystagon (cysteamine bitartrate) immediate-release capsules
Product Name: Cystagon (cysteamine bitartrate) immediate-release capsules
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: cysteamine bitartrate
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)

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)

Secondary Outcome(s)

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

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

Secondary ID(s)

3/001/14

Source(s) of Monetary Support

Cystic Fibrosis Trust

Health Sciences Scotland

Novabiotics Ltd

Secondary Sponsor(s)

NHS Grampian