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.

• Register: EUCTR
• Last refreshed on: 20 July 2020
• Main ID: EUCTR2015-004986-99-GB
• Date of registration: 06/09/2016
• Prospective Registration: Yes
• Primary sponsor: NovaBiotics, Ltd
• Public title: 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.
• Scientific title: 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.
• Date of first enrolment: 31/10/2016
• Target sample size: 120
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-004986-99
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 6
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Italy, Netherlands, United Kingdom, United States

Contacts

• Name: Deborah O'Neil
• Affiliation: NovaBiotics, Ltd.

• Name: Deborah O'Neil
• Affiliation: NovaBiotics, Ltd.

Key inclusion & exclusion 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
7) Females of childbearing potential will be included if they are
either sexually inactive (sexually abstinent for 14 days prior to the
first study drug dose continuing through 28 days after the last
study drug dose, or using one of the following highly effective
contraceptive (i.e. results in <1% failure rate when used
consistently and correctly) methods in this trial:
a. intrauterine device (IUD);
b. surgical sterilization of the partner (vasectomy for 6 months
minimum);
c. combined (estrogen or progestogen containing) hormonal
contraception associated with the inhibition of ovulation
(either oral, intravaginal, or transdermal);
d. progestogen only hormonal contraception associated with the
inhibition of ovulation (either oral, injectable, or
implantable);
e. intrauterine hormone releasing system (IUS);
f. bilateral tubal occlusion.
Females of childbearing potential agree to remain sexually inactive or to keep the same birth control method for at least 28 days following
the last dose.
9) A female of non-childbearing potential must have undergone one
of the following sterilization procedures at least 6 months prior to
the first study drug dose:
a. hysteroscopic sterilization;
b. bilateral tubal ligation or bilateral salpingectomy;
c. hysterectomy;
d. bilateral oophorectomy;
or be postmenopausal with amenorrhea for at least 1 year prior
to the first study drug dose and follicle stimulating hormone
(FSH) serum levels consistent with postmenopausal status.
10) A non-vasectomized male subject agrees to use a condom with
spermicide or abstain from sexual intercourse during the study
until 90 days beyond the last dose of study medication and the
female partner agrees to comply with inclusion 7 or 9. For a
vasectomized male who has had his vasectomy 6 months or more
prior to study start, it is required that they use a condom during
sexual intercourse. A male who has been vasectomized less than 6
months prior to study start must follow the same restrictions as a
non-vasectomized male.
11) If male, agrees not to donate sperm from the first study drug dose until 90 days after dosing.
12) Willing and able to comply with all protocol requirements and
procedures, including induction of sputum, if necessary
13) Willing and able to provide signed and dated informed consent
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:
1. Hypersensitive to cysteamine or to any of the excipients
2. Hypersensitive to penicillamine
3. Transplant recipient


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

Health Condition(s) or Problem(s) studied

exacerbation of Cystic Fibrosis

Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]

Intervention(s)

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

Primary Outcome(s)

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

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

Timepoint(s) of evaluation of this end point: See section E.5.1. Primary End Points

Secondary Outcome(s)

Secondary end point(s): See section E.5.1 Primary end points

Timepoint(s) of evaluation of this end point: See section E.5.1 Primary End Points

Secondary ID(s)

2015-004986-99-IT

NBTCS02

Source(s) of Monetary Support

NovaBiotics, Ltd.

Ethics review

Status: Approved
Approval date: 22/09/2016
Contact: