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

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ClinicalTrials.gov Identifier: NCT03000348
Recruitment Status : Completed
First Posted : December 22, 2016
Last Update Posted : June 26, 2018
Sponsor:
Collaborators:
Agility Clinical, Inc.
PSR Group B.V.
Information provided by (Responsible Party):
NovaBiotics Ltd.

Brief Summary:
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 or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Cysteamine Drug: Placebo Oral Capsule Phase 2

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 91 participants
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
Study Start Date : December 2016
Actual Primary Completion Date : April 2018
Actual Study Completion Date : April 2018


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Change from baseline in sputum bacterial load [ Time Frame: Baseline through Day 21/End of Study ]
  2. 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 :
  1. Change from baseline in neutrophil elastase levels [ Time Frame: Baseline through Day 21/End of Study ]
  2. Change from baseline in sputum IL8 [ Time Frame: Baseline through Day 21/End of Study ]
  3. Change from baseline in FEV1 [ Time Frame: Baseline through Day 21/End of Study ]
  4. Change from baseline in Weight and BMI [ Time Frame: Baseline through Day 21/End of Study ]
  5. Change from baseline in C-Reactive Protein [ Time Frame: Baseline through Day 21/End of Study ]
  6. Change from baseline in blood leukocyte count [ Time Frame: Baseline through Day 21/End of Study ]
  7. Assessment of blood cysteamine levels [ Time Frame: Day 14 ]
  8. Assessment of sputum cysteamine levels [ Time Frame: Day 14 ]
  9. Change from baseline in CFRSD-CRISS [ Time Frame: Baseline through Day 21/End of Study ]
  10. Change from baseline in CFQ-R [ Time Frame: Baseline through Day 21/End of Study ]
  11. Change from baseline in Jarad and Sequeiros Symptom Score Questionnaire [ Time Frame: Baseline through Day 21/End of Study ]
  12. Patient Global Assessment of Exacerbation outcome [ Time Frame: Day 14 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
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. 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:

  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

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


Locations
United States, Arizona
Banner University of Arizona Medical Center
Tucson, Arizona, United States, 85724
United States, California
San Francisco Critical Care Medical Group California Pacific Medical Center
San Francisco, California, United States, 94115
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
Central Florida Pulmonary
Orlando, Florida, United States, 32803
United States, New York
Albany Medical College
Albany, New York, United States, 12208
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, West Virginia
West Virginia University
Morgantown, West Virginia, United States, 26506
United States, Wisconsin
The Medical College of Wisconsin/Froedtert Hospital
Milwaukee, Wisconsin, United States, 53226
Italy
Ospedale Padiatrico Bambino Gesu Centro Fibrosi Cistica
Roma, Italy, 00165
Azienda Ospedaliera Universitaria Integrato di Verona Borgo Trento Centro Fibrosi Cistica
Verona, Italy, 37126
United Kingdom
Aberdeen Royal Infirmary
Aberdeen, Scotland, United Kingdom, AB25 2ZN
Ninewells Hospital Scottish Adult Cystic Fibrosis Service
Dundee, United Kingdom, DD1 9SY
Western General Hospital Edinburgh, CF Adults / CF Unit
Edinburgh, United Kingdom, EH4 3HE
NHS GGC
Glasgow, United Kingdom, G51 4TF
Raigmore Hospital
Inverness, United Kingdom, IV2 3UJ
St. James University Hospital
Leeds, United Kingdom, LS9 7TF
Royal Victoria Infirmary Adult CF Centre
Newcastle upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
NovaBiotics Ltd.
Agility Clinical, Inc.
PSR Group B.V.

Additional Information:
Responsible Party: NovaBiotics Ltd.
ClinicalTrials.gov Identifier: NCT03000348     History of Changes
Other Study ID Numbers: NBTCS02
First Posted: December 22, 2016    Key Record Dates
Last Update Posted: June 26, 2018
Last Verified: June 2018

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