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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03000348
Other study ID # NBTCS02
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date December 2016
Est. completion date April 2018

Study information

Verified date April 2021
Source NovaBiotics Ltd.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 91
Est. completion date April 2018
Est. primary completion date April 2018
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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 Design


Related Conditions & MeSH terms


Intervention

Drug:
Cysteamine
Oral Cysteamine Capsule
Placebo Oral Capsule
Placebo Oral Capsule

Locations

Country Name City State
Italy Ospedale Padiatrico Bambino Gesu Centro Fibrosi Cistica Roma
Italy Azienda Ospedaliera Universitaria Integrato di Verona Borgo Trento Centro Fibrosi Cistica Verona
United Kingdom 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 States Albany Medical College Albany New York
United States Nationwide Children's Hospital Columbus Ohio
United States University of Florida Gainesville Florida
United States The Medical College of Wisconsin/Froedtert Hospital Milwaukee Wisconsin
United States West Virginia University Morgantown West Virginia
United States Central Florida Pulmonary Orlando Florida
United States San Francisco Critical Care Medical Group California Pacific Medical Center San Francisco California
United States Banner University of Arizona Medical Center Tucson Arizona

Sponsors (3)

Lead Sponsor Collaborator
NovaBiotics Ltd. Agility Clinical, Inc., PSR Group B.V.

Countries where clinical trial is conducted

United States,  Italy,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change From Baseline in Sputum Bacterial Load 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
Primary Safety and Tolerability Assessed by the Number of Subjects With Adverse Events Assessed by variables such as adverse events (AEs), laboratory assessments, physical examinations, and vital signs. Baseline through Day 21/End of Study
Secondary Change From Baseline in Neutrophil Elastase Levels 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
Secondary Change From Baseline in Sputum IL8 Sputum IL-8 Levels by Visit - Covariate Adjusted ANCOVA with Observed Data ITT Population Baseline through Day 21/End of Study
Secondary Change From Baseline in FEV1 Change from Baseline in FEV1 Percent Predicted (%) - Covariate Adjusted ANCOVA with Observed Data ITT Population Baseline through Day 21/End of Study
Secondary Change From Baseline in BMI BMI (kg/m^2) by Visit - ANCOVA with Observed Data ITT Population Baseline through Day 21/End of Study
Secondary Change From Baseline in C-Reactive Protein Change from baseline in C-Reactive Protein at visits 7, 14 and 21 Baseline through Day 21
Secondary Change From Baseline in Blood Leukocyte Count Blood Leukocyte Count (10^9 leucocytes/L) by Visit - ANCOVA with Observed Data ITT Population Baseline through Day 21/End of Study
Secondary Assessment of Blood Cysteamine Levels Study Drug Plasma at Day 14 Safety Population Day 14
Secondary Assessment of Sputum Cysteamine Levels Study Drug Sputum Concentrations at Day 14 Safety Population Day 14
Secondary Change From Baseline in CFRSD-CRISS 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
Secondary Change From Baseline in CFQ-R 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
Secondary Change From Baseline in Jarad and Sequeiros Symptom Score Questionnaire 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
Secondary Change From Baseline in Weight Weight (kg) by visit - ANCOVA with observed data Baseline through Day 21/End of Study
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