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A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations (CARE-CF1)

This study is currently recruiting participants.
Verified August 2017 by NovaBiotics Ltd.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03000348
First Posted: December 22, 2016
Last Update Posted: August 4, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
Agility Clinical, Inc.
PSR Group B.V.
Information provided by (Responsible Party):
NovaBiotics Ltd.
  Purpose
This study investigates the use of cysteamine in the treatment of adults with Cystic Fibrosis who are experiencing an exacerbation of CF-associated lung disease. There are six different potential dosing regimens, including one that is placebo.

Condition Intervention Phase
Cystic Fibrosis Drug: Cysteamine Drug: Placebo Oral Capsule Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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 Being Treated for an Exacerbation of CF-associated Lung Disease

Resource links provided by NLM:


Further study details as provided by NovaBiotics Ltd.:

Primary Outcome Measures:
  • Change from baseline in CFRSD-CRISS [ Time Frame: Baseline through Day 21/End of Study ]
  • Change from baseline in CFQ-R [ Time Frame: Baseline through Day 21/End of Study ]
  • Change from baseline in Jarad and Sequeiros Symptom Score Questionnaire [ Time Frame: Baseline through Day 21/End of Study ]
  • Change from baseline in sputum bacterial load [ Time Frame: Baseline through Day 21/End of Study ]
  • Patient Global Assessment of Exacerbation outcome [ Time Frame: Day 14 ]
  • Safety and tolerability assessed by the number of subjects with Adverse Events [ Time Frame: Baseline through Day 21/End of Study ]
    Assessed by variables such as adverse events (AEs), laboratory assessments, physical examinations, and vital signs.


Secondary Outcome Measures:
  • Change from baseline in neutrophil elastase levels [ Time Frame: Baseline through Day 21/End of Study ]
  • Change from baseline in sputum IL8 [ Time Frame: Baseline through Day 21/End of Study ]
  • Change from baseline in FEV1 [ Time Frame: Baseline through Day 21/End of Study ]
  • Change from baseline in Weight [ Time Frame: Baseline through Day 21/End of Study ]
  • Change from baseline in C-Reactive Protein [ Time Frame: Baseline through Day 21/End of Study ]
  • Change from baseline in blood leukocyte count [ Time Frame: Baseline through Day 21/End of Study ]
  • Assessment of blood cysteamine levels [ Time Frame: Day 14 ]
  • Assessment of sputum cysteamine levels [ Time Frame: Day 14 ]

Estimated Enrollment: 120
Study Start Date: December 2016
Estimated Study Completion Date: November 2017
Estimated Primary Completion Date: November 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 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.
Drug: Cysteamine
Oral Cysteamine Capsule
Other Names:
  • Lynovex
  • NM001
  • Lynovex Oral
Drug: Placebo Oral Capsule
Placebo Oral Capsule
Active Comparator: 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.
Drug: Cysteamine
Oral Cysteamine Capsule
Other Names:
  • Lynovex
  • NM001
  • Lynovex Oral
Drug: Placebo Oral Capsule
Placebo Oral Capsule
Active Comparator: 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.
Drug: Cysteamine
Oral Cysteamine Capsule
Other Names:
  • Lynovex
  • NM001
  • Lynovex Oral
Placebo Comparator: Placebo
Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening.
Drug: Placebo Oral Capsule
Placebo Oral Capsule
Active Comparator: 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.
Drug: Cysteamine
Oral Cysteamine Capsule
Other Names:
  • Lynovex
  • NM001
  • Lynovex Oral
Drug: Placebo Oral Capsule
Placebo Oral Capsule
Active Comparator: 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.
Drug: Cysteamine
Oral Cysteamine Capsule
Other Names:
  • Lynovex
  • NM001
  • Lynovex Oral
Drug: Placebo Oral Capsule
Placebo Oral Capsule

Detailed Description:
This is a multicenter, double-blind, randomized, placebo-controlled, 6-arm study to investigate the optimal dose regimen, efficacy, and safety of cysteamine in the treatment of adult patients with CF who are experiencing an exacerbation of CF-associated lung disease. Patients will be screened for the study and eligible patients will be randomized to receive either cysteamine or placebo as add-on therapy to their standard of care treatment for CF-associated lung disease.
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 (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. Women of childbearing potential (not surgically sterile or 2 years postmenopausal) and men with a female partner of childbearing potential must use a medically accepted method of contraception and must agree to continue use of this method for the duration of the study and for 30 days after participation in the study. Acceptable methods of contraception for men and women include abstinence, barrier method (condom or diaphragm) with spermicide, intrauterine device (IUD), vasectomy in conjunction with barrier method, or steroidal contraceptive (oral, transdermal, implanted, injected) in conjunction with a barrier method.
  8. Willing and able to comply with all protocol requirements and procedures, including induction of sputum, if necessary
  9. Willing and able to provide signed and dated informed consent

Exclusion Criteria:

  1. Hypersensitive to cysteamine or to any of the excipients
  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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03000348


Contacts
Contact: Agility Clinical LYNOVEX-CARE-CF1@agility-clinical.com
Contact: Deborah O'Neil, PhD ++44 (0) 1224 711377 deborah@novabiotics.co.uk

Locations
United States, Arizona
Banner University of Arizona Medical Center Recruiting
Tucson, Arizona, United States, 85724
Contact: Elizabeth Ryan, RRT    520-626-3125    Elizabeth.ryan@email.arizona.edu   
Contact: Osmara Molina de Rodriguez, MS    520-626-3910    omolina@email.arizona.edu   
Principal Investigator: Cori Daines, MD         
United States, California
San Francisco Critical Care Medical Group California Pacific Medical Center Recruiting
San Francisco, California, United States, 94115
Contact: Anna von Bakonyi    415-600-1107    vonBakA@cpmcri.org   
Principal Investigator: Ryan Dougherty, MD FCCP         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Noni Graham    352-294-5195    noni.graham@medicine.ufl.edu   
Principal Investigator: Jorge Lascano, MD         
Central Florida Pulmonary Recruiting
Orlando, Florida, United States, 32803
Contact: Bert Kesser    407-841-1100 ext 118    bkesser@cfpulmonary.com   
Contact: Billy Fortson    407-841-1100 ext 173    bfortson@cfpulmonary.com   
Principal Investigator: Daniel T. Layish, MD         
United States, New York
Albany Medical College Recruiting
Albany, New York, United States, 12208
Contact: Anne M DeNero, RN, BSN    518-262-6608    deneroa@mail.amc.edu   
Principal Investigator: Thomas Smith, MD         
United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Elham Alqudah, CRC    614-722-6842    Elham.alqudah@nationwidechildrens.org   
Contact: Clinical Research Services    614.722.2650      
Principal Investigator: Benjamin Kopp, MD         
United States, West Virginia
West Virginia University Recruiting
Morgantown, West Virginia, United States, 26506
Contact: Tammy Clark, BSN, RN    304-293-5264    tclark@hsc.wvu.edu   
Principal Investigator: Kathryn Moffett, MD         
United States, Wisconsin
The Medical College of Wisconsin/Froedtert Hospital Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Erin Hubertz    414-955-7019    ehubertz@mcw.edu   
Contact: Ashley Wuerl    (414) 955-7036    awuerl@mcw.edu   
Principal Investigator: Rose Franco, MD         
Sub-Investigator: Julie Biller, MD         
Italy
Ospedale Padiatrico Bambino Gesu Centro Fibrosi Cistica Recruiting
Roma, Italy, 00165
Contact: Vincenza Lucidi    0039 06 6859 2045    vincenzina.lucidi@opbg.net   
Contact: Enza Montemitro       enza.montemitro@opbg.net   
Principal Investigator: Vincenza Lucidi         
Sub-Investigator: Enza Montemitro         
Azienda Ospedaliera Universitaria Integrato di Verona Borgo Trento Centro Fibrosi Cistica Recruiting
Verona, Italy, 37126
Contact: Sonia Volpi, MD    0039 045 812 3025    sonia.volpi@ospedaleuniverona.it   
Contact: Marianna Passiu       marianna.passiu@ospedaleuniverona.it   
Principal Investigator: Sonia Volpi, MD         
Sub-Investigator: Elena Spinelli         
United Kingdom
Aberdeen Royal Infirmary Recruiting
Aberdeen, Scotland, United Kingdom, AB25 2ZN
Contact: Graham Devereux, MD, PhD    0044 1224 438 476    graham.devereux@nhs.net   
Contact: Kairen Griffiths, RGN MSc    0044 1224 551 228    kairen.griffiths@nhs.net   
Principal Investigator: Graham Devereux, MD, PhD         
Ninewells Hospital Scottish Adult Cystic Fibrosis Service Recruiting
Dundee, United Kingdom, DD1 9SY
Contact: Tom Fardon    0044 1382 496 578    tom.fardon@nhs.net   
Principal Investigator: Tom Fardon         
Western General Hospital Edinburgh, CF Adults / CF Unit Recruiting
Edinburgh, United Kingdom, EH4 3HE
Contact: Helen Rodgers, MD    0044 1315 372 348    Helen.Rodgers@nhslothian.scot.nhs.uk   
Principal Investigator: Helen Rodgers, MD         
NHS GGC Recruiting
Glasgow, United Kingdom, G51 4TF
Contact: Gordon Macgregor    0044 1414 516 094/6095    gordon.macgregor@ggc.scot.nhs.uk   
Contact: Margaret McFadden    0044 1412 327 600    Margaret.mcfadden@ggc.scot.nhs.uk   
Principal Investigator: Gordon Macgregor, MD         
Royal Victoria Infirmary Adult CF Centre Recruiting
Newcastle upon Tyne, United Kingdom, NE1 4LP
Contact: Simon Doe       Simon.Doe@nuth.nhs.uk   
Contact: Rebecca Johnson         
Principal Investigator: Simon Doe         
Sub-Investigator: Stephen Bourke         
Norfolk and Norwich University Hospital Withdrawn
Norfolk, United Kingdom, NR47UY
Sponsors and Collaborators
NovaBiotics Ltd.
Agility Clinical, Inc.
PSR Group B.V.
  More Information

Additional Information:
Responsible Party: NovaBiotics Ltd.
ClinicalTrials.gov Identifier: NCT03000348     History of Changes
Other Study ID Numbers: NBTCS02
First Submitted: December 11, 2016
First Posted: December 22, 2016
Last Update Posted: August 4, 2017
Last Verified: August 2017

Keywords provided by NovaBiotics Ltd.:
Exacerbation
CF
Lung disease
Lung infection
Gram negative
Bacterial Infection
pneumonia
bronchitis

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Cysteamine
Cystine Depleting Agents
Molecular Mechanisms of Pharmacological Action