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CompletedNCT03000348CARE-CF1Updated Apr 14, 2021Results posted

A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations

A Phase 2 interventional study of Cysteamine and Placebo Oral Capsule in Cystic Fibrosis, sponsored by NovaBiotics Ltd.. Completed at 17 sites in 3 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-04-14.

Sponsored by NovaBiotics Ltd. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
91
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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.

Read the 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.

02

Conditions studied

  • Cystic Fibrosis

Keywords

  • Exacerbation
  • CF
  • Lung disease
  • Lung infection
  • Gram negative
  • Bacterial Infection
  • pneumonia
  • bronchitis
03

In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 91 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

This is the only study on the registry with NovaBiotics Ltd. as lead sponsor.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
91 participants (actual)

Study arms

  • 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 · Drug: 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 · Drug: 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

  • 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

  • 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 · Drug: 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 · Drug: Placebo Oral Capsule

Interventions

  • DrugCysteamine

    Oral Cysteamine Capsule

    Also known as: Lynovex, NM001, Lynovex Oral

  • DrugPlacebo Oral Capsule

    Placebo Oral Capsule

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: Baseline through Day 21/End of Study

  2. 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.

    Time frame: Baseline through Day 21/End of Study

Secondary outcomes

  1. 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.

    Time frame: Baseline through Day 21/End of Study

  2. Change From Baseline in Sputum IL8

    Sputum IL-8 Levels by Visit - Covariate Adjusted ANCOVA with Observed Data ITT Population

    Time frame: Baseline through Day 21/End of Study

  3. Change From Baseline in FEV1

    Change from Baseline in FEV1 Percent Predicted (%) - Covariate Adjusted ANCOVA with Observed Data ITT Population

    Time frame: Baseline through Day 21/End of Study

  4. Change From Baseline in BMI

    BMI (kg/m\^2) by Visit - ANCOVA with Observed Data ITT Population

    Time frame: Baseline through Day 21/End of Study

  5. Change From Baseline in C-Reactive Protein

    Change from baseline in C-Reactive Protein at visits 7, 14 and 21

    Time frame: Baseline through Day 21

  6. Change From Baseline in Blood Leukocyte Count

    Blood Leukocyte Count (10\^9 leucocytes/L) by Visit - ANCOVA with Observed Data ITT Population

    Time frame: Baseline through Day 21/End of Study

  7. Assessment of Blood Cysteamine Levels

    Study Drug Plasma at Day 14 Safety Population

    Time frame: Day 14

  8. Assessment of Sputum Cysteamine Levels

    Study Drug Sputum Concentrations at Day 14 Safety Population

    Time frame: Day 14

  9. 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.

    Time frame: Baseline through to Day 21

  10. 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

    Time frame: Baseline through Day 21/End of Study

  11. 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

    Time frame: changes from baseline at day 7 and day 14

  12. Change From Baseline in Weight

    Weight (kg) by visit - ANCOVA with observed data

    Time frame: Baseline through Day 21/End of Study

07

Results

Posted Apr 14, 2021

Participant flow

91 adult CF patients experiencing a pulmonary exacerbation were enrolled across 15 US and EU centres. 89 patients were randomised and 78 completed the 14 day treatment period of the study. First Patient first visit was 12 Jan 2017 and Last Patient last visit was 11 April 2018.

Participant flow — Overall Study
MilestonePlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Started171115151615
Day 14 treatment period171013141314
Completed14711121113
Not completed344352
Withdrew: Adverse event111111
Withdrew: Lost to follow-up001000
Withdrew: Withdrawal by subject010000
Withdrew: Consent000010
Withdrew: Failure to expectorate sputum101010
Withdrew: No gram negative121111
Withdrew: Patient had no transport000100
Withdrew: Dosing000010

Outcome measures

PrimaryChange From Baseline in Sputum Bacterial Load

Change from baseline through to Day 21 in log10 cfu/ml transformed total gram negative sputum bacterial load

Time frame:
Baseline through Day 21/End of Study
Reported as:
Mean · log 10 cfu/ml
Change From Baseline in Sputum Bacterial Load
log 10 cfu/mlPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per DayHigh Dose, Three Times Per Day
Day 7 change from baseline-2.1 ± 2.887-1.03 ± 3.124-1.28 ± 1.978-1.7 ± 2.439-1.03 ± 1.997-0.25 ± 2.228
Day 14 change from baseline-1.38 ± 2.2910.12 ± 2.045-1.24 ± 2.686-1.32 ± 2.298-0.98 ± 1.8940.33 ± 2.270
Day 21 change from baseline-0.18 ± 1.238-1.22 ± 2.760-0.26 ± 1.639-1.04 ± 2.891-0.87 ± 1.6770.93 ± 2.348
PrimarySafety 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.

Time frame:
Baseline through Day 21/End of Study
Reported as:
Number · participants
Safety and Tolerability Assessed by the Number of Subjects With Adverse Events
participantsPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Number of AEs302940334536
Number of SAEs112101
1 or more AEs91011121013
Severity of AE mild765647
Severity of AE moderate246556
Severity of AE severe000110
AEs leading to drug discontinuation111111
Nausea035353
Vomiting022221
Insomnia002012
Rash001130
Arthralgia011101
Decreased appetite001021
Haemoptysis201120
Oropharyngeal pain011011
Abdominal pain101020
Breath odour000102
SecondaryChange 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.

Time frame:
Baseline through Day 21/End of Study
Reported as:
Mean · ng/mL
Change From Baseline in Neutrophil Elastase Levels
ng/mLPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Day 7 change from baseline-4659 ± 3833523431 ± 53111704 ± 36395-19623 ± 35548-15769 ± 408995135 ± 27791
Day 14 change from baseline-3706 ± 42879-380 ± 157122-7208 ± 19210-12014 ± 23269-14083 ± 4121514660 ± 35539
Day 21 change from baseline-1000 ± 598564853 ± 18616171 ± 19229-21002 ± 2472-14591 ± 39179-2558 ± 20068
SecondaryChange From Baseline in Sputum IL8

Sputum IL-8 Levels by Visit - Covariate Adjusted ANCOVA with Observed Data ITT Population

Time frame:
Baseline through Day 21/End of Study
Reported as:
Mean · pg/mL
Change From Baseline in Sputum IL8
pg/mLPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Day 7 change from baseline-26088 ± 55506-17864 ± 33785-5511 ± 46204111574 ± 70338-2663 ± 41802-25785 ± 53721
Day 14 Change from baseline-28856 ± 39456-26528 ± 46737-33653 ± 553023167 ± 28204-6136 ± 53611221 ± 30639
Day 21 change from baseline9576 ± 66157-16043 ± 34551-19778 ± 493062831 ± 23050631 ± 32350-10671 ± 65656
SecondaryChange From Baseline in FEV1

Change from Baseline in FEV1 Percent Predicted (%) - Covariate Adjusted ANCOVA with Observed Data ITT Population

Time frame:
Baseline through Day 21/End of Study
Reported as:
Mean · percentage of predicted
Change From Baseline in FEV1
percentage of predictedPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Day 7 change from baseline7.9 ± 13.674.4 ± 5.526.9 ± 8.8511.8 ± 9.964.9 ± 5.483.7 ± 8.65
Day 14 change from baseline9.2 ± 14.034.0 ± 5.148.9 ± 10.8713.6 ± 10.835.3 ± 6.657.5 ± 7.07
Day 21 change from baseline9.5 ± 13.54.7 ± 5.036.5 ± 7.258.9 ± 10.666.4 ± 8.186.3 ± 11.34
SecondaryChange From Baseline in BMI

BMI (kg/m\^2) by Visit - ANCOVA with Observed Data ITT Population

Time frame:
Baseline through Day 21/End of Study
Reported as:
Mean · kg/m^2 for BMI
Change From Baseline in BMI
kg/m^2 for BMIPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
BMI Day 7 change from baseline0.39 ± 0.3710.32 ± 0.4980.28 ± 0.30.24 ± 0.4280.15 ± 0.5860.15 ± 0.589
BMI Day 14 change from baseline0.34 ± 0.4520.64 ± 0.5050.34 ± 0.4770.37 ± 0.4880.23 ± 0.7050.32 ± 0.893
BMI Day 21 change from baseline0.54 ± 0.5370.55 ± 0.5530.47 ± 0.5640.18 ± 0.478-0.02 ± 0.9800.12 ± 0.828
SecondaryChange From Baseline in C-Reactive Protein

Change from baseline in C-Reactive Protein at visits 7, 14 and 21

Time frame:
Baseline through Day 21
Reported as:
Mean · nmol/L
Change From Baseline in C-Reactive Protein
nmol/LPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Day 7 change from baseline-182.828 ± 188.1222-285.006 ± 295.0114-229.821 ± 590.1267-318.933 ± 344.9138-120.193 ± 200.2307-281.752 ± 293.2595
Day 14 change from baseline-75.671 ± 331.2220-312.435 ± 323.8782-216.105 ± 617.1174-215.242 ± 225.7293-111.272 ± 124.8394-294.13 ± 509.9255
Day 21 change from baseline-44.341 ± 296.4493-237.062 ± 378.4493-116.442 ± 730.9709-198.759 ± 235.0256-23.421 ± 157.4203-225.787 ± 543.1070
SecondaryChange From Baseline in Blood Leukocyte Count

Blood Leukocyte Count (10\^9 leucocytes/L) by Visit - ANCOVA with Observed Data ITT Population

Time frame:
Baseline through Day 21/End of Study
Reported as:
Mean · 10^9 leucocytes/L
Change From Baseline in Blood Leukocyte Count
10^9 leucocytes/LPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Day 7 change from baseline-2.553 ± 4.617-1.836 ± 2.0566-2.808 ± 3.595-2.482 ± 3.4502-2.575 ± 3.1189-2.863 ± 2.9932
Day 14 change from baseline-1.568 ± 4.7176-2.220 ± 2.3451-2.648 ± 3.9122-3.419 ± 3.5734-0.193 ± 3.8376-4.066 ± 2.4102
Day 21 change from baseline-1.870 ± 4.6187-1.757 ± 2.6670-1.880 ± 3.0935-1.763 ± 3.3193-0.127 ± 2.5869-3.316 ± 3.6322
SecondaryAssessment of Blood Cysteamine Levels

Study Drug Plasma at Day 14 Safety Population

Time frame:
Day 14
Reported as:
Mean · ng/ml
Assessment of Blood Cysteamine Levels
ng/mlPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Assessment of Blood Cysteamine Levels10.0 ± 0.00515.11 ± 683.956102.45 ± 144.863347.76 ± 507.747256.85 ± 377.262256.84 ± 251.593
SecondaryAssessment of Sputum Cysteamine Levels

Study Drug Sputum Concentrations at Day 14 Safety Population

Time frame:
Day 14
Reported as:
Mean · ng/ml
Assessment of Sputum Cysteamine Levels
ng/mlPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Assessment of Sputum Cysteamine Levels150.0 ± 0.00682.8 ± 1009.8150.0 ± 0.0316.4 ± 1423.6739.7 ± 1423.6811.1 ± 917.49
SecondaryChange 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.

Time frame:
Baseline through to Day 21
Reported as:
Mean · units on a scale
Change From Baseline in CFRSD-CRISS
units on a scalePlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Day 7 change from baseline-16.4 ± 8.31-18.1 ± 10.19-11.9 ± 7.98-19.1 ± 10.65-10.5 ± 5.91-17.8 ± 12.67
Day 14 change from baseline-16.3 ± 15.0-24.3 ± 16.35-15.5 ± 12.48-28.1 ± 16.88-14.8 ± 8.53-23.9 ± 16.41
Day 21 change from baseline-18.3 ± 11.7-23.2 ± 13.36-15.8 ± 11.68-19.9 ± 19.23-12.9 ± 6.3722.0 ± 17.71
SecondaryChange 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

Time frame:
Baseline through Day 21/End of Study
Reported as:
Mean · units on a scale
Change From Baseline in CFQ-R
units on a scalePlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Day 7 Change from baseline14.7 ± 18.2112.2 ± 13.8112.0 ± 16.3212.1 ± 14.9515.9 ± 14.119.6 ± 16.11
Day 14 change from baseline22.9 ± 21.5124.4 ± 14.1519.7 ± 16.7631.3 ± 17.3821.8 ± 14.0728.6 ± 15.69
Day 21 change from baseline24.3 ± 19.3430.6 ± 17.6225.2 ± 19.1931.3 ± 30.0824.8 ± 14.8537.3 ± 23.61
SecondaryChange 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

Time frame:
changes from baseline at day 7 and day 14
Reported as:
Mean · units on a scale
Change From Baseline in Jarad and Sequeiros Symptom Score Questionnaire
units on a scalePlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
change from baseline to day 7-2.6 ± 1.97-1.9 ± 2.38-1.4 ± 1.85-3.3 ± 2.35-2.4 ± 2.21-3.1 ± 3.33
change from baseline to day 14-3.2 ± 2.9-2.9 ± 2.6-2.0 ± 2.48-4.3 ± 2.16-3.1 ± 2.63-4.2 ± 3.19
SecondaryChange From Baseline in Weight

Weight (kg) by visit - ANCOVA with observed data

Time frame:
Baseline through Day 21/End of Study
Reported as:
Mean · kg
Change From Baseline in Weight
kgPlacebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per Day
Day 70.96 ± 1.0840.83 ± 1.2410.79 ± 1.1450.67 ± 1.1820.25 ± 1.5470.36 ± 1.74
Day 140.85 ± 1.3551.71 ± 1.2700.96 ± 1.3681.04 ± 1.4450.55 ± 1.8720.89 ± 2.621
Day 211.37 ± 1.4861.50 ± 1.6391.30 ± 1.5030.52 ± 1.295-0.28 ± 2.7280.34 ± 2.477

Adverse events

Collected over Adverse Events were collected from baseline (day 0, day study treatment commences) until day 21 (end of study/follow up). Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Placebo0/17 (0%)1/17 (5.9%)9/17 (52.9%)
High Dose, Once Per Day0/11 (0%)1/11 (9.1%)10/11 (90.9%)
Low Dose, Three Times Per Day0/15 (0%)2/15 (13.3%)11/15 (73.3%)
High Dose, Twice Per Day0/15 (0%)1/15 (6.7%)12/15 (80%)
Mid-Range Dose, Three Times Per Day0/16 (0%)0/16 (0%)10/16 (62.5%)
High Dose, Three Times Per Day0/15 (0%)1/15 (6.7%)13/15 (86.7%)
Most frequent serious events
Most frequent serious events
EventPlaceboHigh Dose, Once Per DayLow Dose, Three Times Per DayHigh Dose, Twice Per DayMid-Range Dose, Three Times Per DayHigh Dose, Three Times Per Day
Axillary vein thrombosisVascular disorders0/171/110/150/150/160/15
NephrolithiasisRenal and urinary disorders0/170/110/150/150/161/15
DepressionPsychiatric disorders0/170/111/150/150/160/15
Infective pulmonary exacerbation of CFInfections and infestations0/170/110/151/150/160/15
Campylobacter sepsisInfections and infestations0/170/111/150/150/160/15
HaemoptysisRespiratory, thoracic and mediastinal disorders1/170/110/150/150/160/15
Most frequent other events
Showing 10 of 103
Most frequent other events
EventPlaceboHigh Dose, Once Per DayLow Dose, Three Times Per DayHigh Dose, Twice Per DayMid-Range Dose, Three Times Per DayHigh Dose, Three Times Per Day
HeadacheNervous system disorders1/175/111/153/152/161/15
NauseaGastrointestinal disorders—3/115/153/155/163/15
RashSkin and subcutaneous tissue disorders0/170/111/151/153/160/15
vomitingGastrointestinal disorders0/172/112/152/152/161/15
DizzinessNervous system disorders1/172/111/150/150/160/15
Breath odourGastrointestinal disorders0/170/110/151/150/162/15
DysguesiaNervous system disorders1/171/110/152/150/160/15
PainGeneral disorders0/170/112/150/150/160/15
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations1/170/111/152/150/160/15
InsomniaPsychiatric disorders0/170/112/150/151/162/15

Baseline characteristics

Age, Continuous
Age, Continuous(years)Placebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per DayTotal
Mean27.2 ± 5.6427.5 ± 6.7732.5 ± 12.732.3 ± 9.7831.4 ± 12.027.5 ± 7.8929.8 ± 9.59
Sex: Female, Male
Sex: Female, Male(Participants)Placebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per DayTotal
Female86588843
Male951078746
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Placebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per DayTotal
Hispanic or Latino0001001
Not Hispanic or Latino15101312151378
Unknown or Not Reported21221210
BMI
BMI(kg/m^2)Placebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per DayTotal
Mean20.2 ± 2.2320.3 ± 3.0320.7 ± 2.4121.5 ± 2.2120.5 ± 3.0321.7 ± 2.8420.8 ± 2.61
Age at CF Diagnosis
Age at CF Diagnosis(years)Placebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per DayTotal
Mean0.9 ± 2.413.9 ± 0.240.3 ± 0.491.6 ± 4.934.8 ± 11.451.8 ± 5.630.9 ± 2.41
Duration of CF years
Duration of CF years(years)Placebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per DayTotal
Mean26.36 ± 5.6923.7 ± 9.2632.45 ± 12.4431.11 ± 10.9226.76 ± 11.6925.88 ± 7.0727.85 ± 9.97
FEV1 Percent predicted (%)
FEV1 Percent predicted (%)(litres per second)Placebo450mg, Once Per Day150mg, Three Times Per Day450mg, Twice Per Day300mg, Three Times Per Day450mg, Three Times Per DayTotal
Mean41.5 ± 15.3139.4 ± 19.8148.0 ± 18.2646.1 ± 22.7537.7 ± 13.4446.9 ± 20.5843.3 ± 18.34
08

Study locations

17 sites
  • Banner University of Arizona Medical Center
    Tucson, Arizona 85724, United States
  • San Francisco Critical Care Medical Group California Pacific Medical Center
    San Francisco, California 94115, United States
  • University of Florida
    Gainesville, Florida 32610, United States
  • Central Florida Pulmonary
    Orlando, Florida 32803, United States
  • Albany Medical College
    Albany, New York 12208, United States
  • Nationwide Children's Hospital
    Columbus, Ohio 43205, United States
  • West Virginia University
    Morgantown, West Virginia 26506, United States
  • The Medical College of Wisconsin/Froedtert Hospital
    Milwaukee, Wisconsin 53226, United States
  • Ospedale Padiatrico Bambino Gesu Centro Fibrosi Cistica
    Roma, 00165, Italy
  • Azienda Ospedaliera Universitaria Integrato di Verona Borgo Trento Centro Fibrosi Cistica
    Verona, 37126, Italy
  • Aberdeen Royal Infirmary
    Aberdeen, Scotland AB25 2ZN, United Kingdom
  • Ninewells Hospital Scottish Adult Cystic Fibrosis Service
    Dundee, DD1 9SY, United Kingdom
  • Western General Hospital Edinburgh, CF Adults / CF Unit
    Edinburgh, EH4 3HE, United Kingdom
  • NHS GGC
    Glasgow, G51 4TF, United Kingdom
  • Raigmore Hospital
    Inverness, IV2 3UJ, United Kingdom
  • St. James University Hospital
    Leeds, LS9 7TF, United Kingdom
  • Royal Victoria Infirmary Adult CF Centre
    Newcastle upon Tyne, NE1 4LP, United Kingdom
09

References and documents

Publications

  • 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. PubMed 33370348 ↗

Study documents

  • Protocol and statistical analysis plan · Oct 18, 2017

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 14, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03000348
Lead sponsor
NovaBiotics Ltd.
Collaborators
Agility Clinical, Inc., PSR Group B.V.
Responsible party
Sponsor
First posted
Dec 22, 2016
Start date
Dec 2016
Primary completion
Apr 2018
Completion
Apr 2018
Results posted
Apr 14, 2021
Last update
Apr 14, 2021

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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