Trial · NCT03000348
A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations
NCT03000348, Phase 2, Completed, NovaBiotics Ltd.; results first posted 2021-04-14
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations. Trials, record NCT03000348. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03000348
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations
- NCT number
- NCT03000348
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 91 participants, actual
- Lead sponsor
- NovaBiotics Ltd.
- Conditions
- Cystic Fibrosis
- Interventions
- Cysteamine, Placebo Oral Capsule
- First posted
- 2016-12-22
- Last updated
- 2021-04-14
- Results first posted
- 2021-04-14
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Sputum Bacterial Load | 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 |
| Primary | Safety and Tolerability Assessed by the Number of Subjects With Adverse Events | Baseline through Day 21/End of Study | Assessed by variables such as adverse events (AEs), laboratory assessments, physical examinations, and vital signs. |
| Secondary | Change From Baseline in Neutrophil Elastase Levels | 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. |
| Secondary | Change From Baseline in Sputum IL8 | Baseline through Day 21/End of Study | Sputum IL-8 Levels by Visit - Covariate Adjusted ANCOVA with Observed Data ITT Population |
| Secondary | Change From Baseline in FEV1 | Baseline through Day 21/End of Study | Change from Baseline in FEV1 Percent Predicted (%) - Covariate Adjusted ANCOVA with Observed Data ITT Population |
| Secondary | Change From Baseline in BMI | Baseline through Day 21/End of Study | BMI (kg/m\^2) by Visit - ANCOVA with Observed Data ITT Population |
| Secondary | Change From Baseline in C-Reactive Protein | Baseline through Day 21 | Change from baseline in C-Reactive Protein at visits 7, 14 and 21 |
| Secondary | Change From Baseline in Blood Leukocyte Count | Baseline through Day 21/End of Study | Blood Leukocyte Count (10\^9 leucocytes/L) by Visit - ANCOVA with Observed Data ITT Population |
| Secondary | Assessment of Blood Cysteamine Levels | Day 14 | Study Drug Plasma at Day 14 Safety Population |
| Secondary | Assessment of Sputum Cysteamine Levels | Day 14 | Study Drug Sputum Concentrations at Day 14 Safety Population |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Sputum Bacterial Load | Baseline through Day 21/End of Study | Intention to treat population | log 10 cfu/ml | MEAN |
| PRIMARY | Safety and Tolerability Assessed by the Number of Subjects With Adverse Events | Baseline through Day 21/End of Study | Summary of participant reported adverse events (AEs) by study group | participants | NUMBER |
| SECONDARY | Change From Baseline in Neutrophil Elastase Levels | Baseline through Day 21/End of Study | Neutrophil Elastase Levels by Visit - Covariate Adjusted ANCOVA with Observed Data ITT Population | ng/mL | MEAN |
| SECONDARY | Change From Baseline in Sputum IL8 | Baseline through Day 21/End of Study | ITT Population | pg/mL | MEAN |
| SECONDARY | Change From Baseline in FEV1 | Baseline through Day 21/End of Study | Change from Baseline in FEV1 Percent Predicted (%) - Covariate Adjusted ANCOVA with Observed Data ITT Population | percentage of predicted | MEAN |
| SECONDARY | Change From Baseline in BMI | Baseline through Day 21/End of Study | ITT | kg/m^2 for BMI | MEAN |
| SECONDARY | Change From Baseline in C-Reactive Protein | Baseline through Day 21 | CRP by visit ANCOVA with observed data ITT population | nmol/L | MEAN |
| SECONDARY | Change From Baseline in Blood Leukocyte Count | Baseline through Day 21/End of Study | ITT | 10^9 leucocytes/L | MEAN |
| SECONDARY | Assessment of Blood Cysteamine Levels | Day 14 | Safety population | ng/ml | MEAN |
| SECONDARY | Assessment of Sputum Cysteamine Levels | Day 14 | Safety Population - there are fewer numbers of patients due to the inability of some patients to provide sputum samples | ng/ml | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group306
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Sputum Bacterial Load | Placebo | Day 7 change from baseline | -2.1 | Standard Deviation 2.887 | log 10 cfu/ml |
| Change From Baseline in Sputum Bacterial Load | 450mg, Once Per Day | Day 7 change from baseline | -1.03 | Standard Deviation 3.124 | log 10 cfu/ml |
| Change From Baseline in Sputum Bacterial Load | 150mg, Three Times Per Day | Day 7 change from baseline | -1.28 | Standard Deviation 1.978 | log 10 cfu/ml |
| Change From Baseline in Sputum Bacterial Load | 450mg, Twice Per Day | Day 7 change from baseline | -1.7 | Standard Deviation 2.439 | log 10 cfu/ml |
| Change From Baseline in Sputum Bacterial Load | 300mg, Three Times Per Day | Day 7 change from baseline | -1.03 | Standard Deviation 1.997 | log 10 cfu/ml |
| Change From Baseline in Sputum Bacterial Load | High Dose, Three Times Per Day | Day 7 change from baseline | -0.25 | Standard Deviation 2.228 | log 10 cfu/ml |
| Change From Baseline in Sputum Bacterial Load | Placebo | Day 14 change from baseline | -1.38 | Standard Deviation 2.291 | log 10 cfu/ml |
| Change From Baseline in Sputum Bacterial Load | 450mg, Once Per Day | Day 14 change from baseline | 0.12 | Standard Deviation 2.045 | log 10 cfu/ml |
| Change From Baseline in Sputum Bacterial Load | 150mg, Three Times Per Day | Day 14 change from baseline | -1.24 | Standard Deviation 2.686 | log 10 cfu/ml |
| Change From Baseline in Sputum Bacterial Load | 450mg, Twice Per Day | Day 14 change from baseline | -1.32 | Standard Deviation 2.298 | log 10 cfu/ml |
1 to 10 of 306Source ClinicalTrials.gov
See all 306 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 17 | |
| Overall Study | STARTED | 450mg, Once Per Day | 11 | |
| Overall Study | STARTED | 150mg, Three Times Per Day | 15 | |
| Overall Study | STARTED | 450mg, Twice Per Day | 15 | |
| Overall Study | STARTED | 300mg, Three Times Per Day | 16 | |
| Overall Study | STARTED | 450mg, Three Times Per Day | 15 | |
| Overall Study | Day 14 Treatment Period | Placebo | 17 | |
| Overall Study | Day 14 Treatment Period | 450mg, Once Per Day | 10 | |
| Overall Study | Day 14 Treatment Period | 150mg, Three Times Per Day | 13 | |
| Overall Study | Day 14 Treatment Period | 450mg, Twice Per Day | 14 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events654
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Nausea | Gastrointestinal disorders | Placebo | 0 | 0 | 0 |
| other | Nausea | Gastrointestinal disorders | High Dose, Once Per Day | 3 | 11 | 3 |
| other | Nausea | Gastrointestinal disorders | Low Dose, Three Times Per Day | 5 | 15 | 5 |
| other | Nausea | Gastrointestinal disorders | High Dose, Twice Per Day | 3 | 15 | 3 |
| other | Nausea | Gastrointestinal disorders | Mid-Range Dose, Three Times Per Day | 5 | 16 | 5 |
| other | Nausea | Gastrointestinal disorders | High Dose, Three Times Per Day | 3 | 15 | 3 |
| other | vomiting | Gastrointestinal disorders | Placebo | 0 | 17 | 0 |
| other | vomiting | Gastrointestinal disorders | High Dose, Once Per Day | 2 | 11 | 2 |
| other | vomiting | Gastrointestinal disorders | Low Dose, Three Times Per Day | 2 | 15 | 2 |
| other | vomiting | Gastrointestinal disorders | High Dose, Twice Per Day | 2 | 15 | 2 |
1 to 10 of 654Source ClinicalTrials.gov
See all 654 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 17 |
| Total, all-cause mortality | High Dose, Once Per Day | 0 | 11 |
| Total, all-cause mortality | Low Dose, Three Times Per Day | 0 | 15 |
| Total, all-cause mortality | High Dose, Twice Per Day | 0 | 15 |
| Total, all-cause mortality | Mid-Range Dose, Three Times Per Day | 0 | 16 |
| Total, all-cause mortality | High Dose, Three Times Per Day | 0 | 15 |
| Total, other adverse events | Placebo | 9 | 17 |
| Total, other adverse events | High Dose, Once Per Day | 10 | 11 |
| Total, other adverse events | Low Dose, Three Times Per Day | 11 | 15 |
| Total, other adverse events | High Dose, Twice Per Day | 12 | 15 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 33370348 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03000348
GET https://conductscience.org/api/trials/v1/NCT03000348/results?page=2