A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations
NCT ID: NCT03000348
Last Updated: 2021-04-14
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE2
91 participants
INTERVENTIONAL
2016-12-31
2018-04-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
First Study of Oral Cysteamine in Cystic Fibrosis
NCT02212431
Study to Assess Efficacy of AZD1236 in Patients With Cystic Fibrosis
NCT00812045
Study to Assess Amphotericin B Cystetic for Inhalation (ABCI) Doses in Healthy Volunteers & People with Cystic Fibrosis
NCT05802264
A Phase II , Placebo-controlled Study to Assess Efficacy of 28 Day Oral AZD9668 in Patients With Cystic Fibrosis
NCT00757848
Dose Escalation Study of KB001 in Cystic Fibrosis Patients Infected With Pseudomonas Aeruginosa
NCT00638365
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
High Dose, Once per day
Patient takes one oral dose of Cysteamine (high dose) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening.
Cysteamine
Oral Cysteamine Capsule
Placebo Oral Capsule
Placebo Oral Capsule
High Dose, Twice per day
Patient takes two oral doses of Cysteamine (high dose) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day.
Cysteamine
Oral Cysteamine Capsule
Placebo Oral Capsule
Placebo Oral Capsule
High Dose, Three times per day
Patient takes three oral doses of Cysteamine (high dose) per day, one in the morning, one at mid-day and one in the evening.
Cysteamine
Oral Cysteamine Capsule
Placebo
Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening.
Placebo Oral Capsule
Placebo Oral Capsule
Low Dose, Three times per day
Patient takes three oral doses of Cysteamine (low dose) per day, one in the morning, one at mid-day and one in the evening.
Cysteamine
Oral Cysteamine Capsule
Placebo Oral Capsule
Placebo Oral Capsule
Mid-Range Dose, Three times per day
Patient takes three oral doses of Cysteamine (mid-range dose) per day, one in the morning, one at mid-day and one in the evening.
Cysteamine
Oral Cysteamine Capsule
Placebo Oral Capsule
Placebo Oral Capsule
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Cysteamine
Oral Cysteamine Capsule
Placebo Oral Capsule
Placebo Oral Capsule
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. Established patient of the Principal Investigator's CF Multi Disciplinary Team (MDT)
3. Age ≥18 years
4. Weight \>40 kg
5. FEV1 \>30% of predicted within the 6 months prior to study exacerbation
6. At the baseline visit: experiencing a new exacerbation of CF-associated lung disease (based on Investigator assessment of ≥4 symptoms present on the Fuchs' criteria) 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:
1. intrauterine device (IUD);
2. surgical sterilization of the partner (vasectomy for 6 months minimum);
3. combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);
4. progestogen only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable);
5. intrauterine hormone releasing system (IUS);
6. bilateral tubal occlusion.
8. 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:
1. hysteroscopic sterilization;
2. bilateral tubal ligation or bilateral salpingectomy;
3. hysterectomy;
4. 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
Exclusion Criteria
2. Hypersensitive to penicillamine
3. Transplant recipient
4. Participation in any other interventional clinical research study (participation in observational studies is not exclusionary) within 30 days of Baseline (Day 0), and any planned participation in an interventional clinical research study for the duration of this study
5. If female, pregnancy, planned pregnancy, or breast-feeding
6. Any other significant disease/disorder which, in the Investigator's opinion, either puts the patient at risk due to study participation, or may influence the results of the study or the patient's ability to participate in the study
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Agility Clinical, Inc.
INDUSTRY
PSR Group B.V.
INDUSTRY
NovaBiotics Ltd.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Banner University of Arizona Medical Center
Tucson, Arizona, United States
San Francisco Critical Care Medical Group California Pacific Medical Center
San Francisco, California, United States
University of Florida
Gainesville, Florida, United States
Central Florida Pulmonary
Orlando, Florida, United States
Albany Medical College
Albany, New York, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
West Virginia University
Morgantown, West Virginia, United States
The Medical College of Wisconsin/Froedtert Hospital
Milwaukee, Wisconsin, United States
Ospedale Padiatrico Bambino Gesu Centro Fibrosi Cistica
Roma, , Italy
Azienda Ospedaliera Universitaria Integrato di Verona Borgo Trento Centro Fibrosi Cistica
Verona, , Italy
Aberdeen Royal Infirmary
Aberdeen, Scotland, United Kingdom
Ninewells Hospital Scottish Adult Cystic Fibrosis Service
Dundee, , United Kingdom
Western General Hospital Edinburgh, CF Adults / CF Unit
Edinburgh, , United Kingdom
NHS GGC
Glasgow, , United Kingdom
Raigmore Hospital
Inverness, , United Kingdom
St. James University Hospital
Leeds, , United Kingdom
Royal Victoria Infirmary Adult CF Centre
Newcastle upon Tyne, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Devereux G, Wrolstad D, Bourke SJ, Daines CL, Doe S, Dougherty R, Franco R, Innes A, Kopp BT, Lascano J, Layish D, MacGregor G, Murray L, Peckham D, Lucidi V, Lovie E, Robertson J, Fraser-Pitt DJ, O'Neil DA. Oral cysteamine as an adjunct treatment in cystic fibrosis pulmonary exacerbations: An exploratory randomized clinical trial. PLoS One. 2020 Dec 28;15(12):e0242945. doi: 10.1371/journal.pone.0242945. eCollection 2020.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol and Statistical Analysis Plan
Related Links
Access external resources that provide additional context or updates about the study.
NovaBiotics Website
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
NBTCS02
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.