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

April 13, 2021 updated by: NovaBiotics Ltd.

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
91

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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: Placebo Oral Capsule
Placebo Oral Capsule
Drug: Cysteamine
Oral Cysteamine Capsule
Other Names:
  • Lynovex
  • NM001
  • Lynovex Oral
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: Placebo Oral Capsule
Placebo Oral Capsule
Drug: Cysteamine
Oral Cysteamine Capsule
Other Names:
  • Lynovex
  • NM001
  • Lynovex Oral
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: Placebo Oral Capsule
Placebo Oral Capsule
Drug: Cysteamine
Oral Cysteamine Capsule
Other Names:
  • Lynovex
  • NM001
  • Lynovex Oral
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: Placebo Oral Capsule
Placebo Oral Capsule
Drug: Cysteamine
Oral Cysteamine Capsule
Other Names:
  • Lynovex
  • NM001
  • Lynovex Oral

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Sputum Bacterial Load
Time Frame: Baseline through Day 21/End of Study
Change from baseline through to Day 21 in log10 cfu/ml transformed total gram negative sputum bacterial load
Baseline through Day 21/End of Study
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.
Baseline through Day 21/End of Study

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Neutrophil Elastase Levels
Time Frame: Baseline through Day 21/End of Study
Actual values and change from baseline in neutrophil elastase levels were summarized using descriptive statistics by visit for each treatment group and each TDD group for the ITT Population.
Baseline through Day 21/End of Study
Change From Baseline in Sputum IL8
Time Frame: Baseline through Day 21/End of Study
Sputum IL-8 Levels by Visit - Covariate Adjusted ANCOVA with Observed Data ITT Population
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 FEV1 Percent Predicted (%) - Covariate Adjusted ANCOVA with Observed Data ITT Population
Baseline through Day 21/End of Study
Change From Baseline in BMI
Time Frame: Baseline through Day 21/End of Study
BMI (kg/m^2) by Visit - ANCOVA with Observed Data ITT Population
Baseline through Day 21/End of Study
Change From Baseline in C-Reactive Protein
Time Frame: Baseline through Day 21
Change from baseline in C-Reactive Protein at visits 7, 14 and 21
Baseline through Day 21
Change From Baseline in Blood Leukocyte Count
Time Frame: Baseline through Day 21/End of Study
Blood Leukocyte Count (10^9 leucocytes/L) by Visit - ANCOVA with Observed Data ITT Population
Baseline through Day 21/End of Study
Assessment of Blood Cysteamine Levels
Time Frame: Day 14
Study Drug Plasma at Day 14 Safety Population
Day 14
Assessment of Sputum Cysteamine Levels
Time Frame: Day 14
Study Drug Sputum Concentrations at Day 14 Safety Population
Day 14
Change From Baseline in CFRSD-CRISS
Time Frame: Baseline through to Day 21
Mean Change from Baseline in Cystic Fibrosis Respiratory Symptom Diary (CFRSD)-Chronic Respiratory Infection Symptom Scale (CRISS) CRFSD-CRISS:The CFRSD is a 16-item PROM to evaluate the effect of treatment on the severity of symptoms of acute respiratory infections associated with CF (i.e., CFRSD-CRISS) and to assess the emotional and activity impacts of these symptoms. The overall CRISS score range is 0-100 with 100 being the most severe symptoms.The CFRSD-CRISS is a validated unidimensional scale based on a subset of 8 items from the CFRSD questionnaire that quantifies symptom severity for the previous 24 hours to capture the magnitude of symptoms in stable CF, during medically treated CF exacerbations, and during recover from an exacerbation. The 8 items on the CFRSD-CRISS were scored using a 5-point Likert scale ranging from 0 (no symptom) to 4 (the highest magnitude of severity). So score range of 0-32.
Baseline through to Day 21
Change From Baseline in CFQ-R
Time Frame: Baseline through Day 21/End of Study
The CFQ-R is a disease-specific HRQOL (Health related quality of life) measure containing both generic and CF-specific scales and measures functioning during the previous 2 weeks. Each CFQ-R scale yielded standardized scores ranging from 0 to 100; higher scores indicated better HRQOL
Baseline through Day 21/End of Study
Change From Baseline in Jarad and Sequeiros Symptom Score Questionnaire
Time Frame: changes from baseline at day 7 and day 14
The Jarad and Sequeiros Symptom Questionnaire (Jarad, 2012) is a simple participant-completed questionnaire that assesses and evaluates change in participant symptoms related to different aspects of respiratory function during a CF exacerbation. The questionnaire consists of 4 questions, each answered on a 4-point scale ranging from 1 (best) to 4 (worst). A range of minimum 4 to maximum16.Jarad and Sequeiros Questionnaire Score - changes from baseline at day 7 and day 14
changes from baseline at day 7 and day 14
Change From Baseline in Weight
Time Frame: Baseline through Day 21/End of Study
Weight (kg) by visit - ANCOVA with observed data
Baseline through Day 21/End of Study

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
NovaBiotics Ltd.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Agility Clinical, Inc.
PSR Group B.V.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 1, 2016

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 1, 2018

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 1, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 11, 2016

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 19, 2016

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 22, 2016

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 14, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 13, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NBTCS02

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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