A Study of the Dosing, Efficacy, and Safety of Oral Cyste... | NCT03000348

NCT03000348

Status Module

Sponsor/Collaborators Module

Name	Class
Agility Clinical, Inc.	INDUSTRY
PSR Group B.V.	INDUSTRY

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
High Dose, Once per day	Active Comparator	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
High Dose, Twice per day	Active Comparator	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
High Dose, Three times per day	Active Comparator	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	Placebo Comparator	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening.	Drug: Placebo Oral Capsule
Low Dose, Three times per day	Active Comparator	Patient takes three oral doses of Cysteamine (low dose) per day, one in the morning, one at mid-day and one in the evening.

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Cysteamine	Drug	Oral Cysteamine Capsule	High Dose, Once per day High Dose, Three times per day High Dose, Twice per day Low Dose, Three times per day

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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
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 Outcomes

Measure	Description	Time Frame
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
Change From Baseline in Sputum IL8

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

CF-associated lung disease with documented history of chronic infection with Gram-negative organism(s)
Established patient of the Principal Investigator's CF Multi Disciplinary Team (MDT)
Age ≥18 years
Weight >40 kg
FEV1 >30% of predicted within the 6 months prior to study exacerbation
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
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.
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.
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.
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.
If male, agrees not to donate sperm from the first study drug dose until 90 days after dosing.
Willing and able to comply with all protocol requirements and procedures, including induction of sputum, if necessary
Willing and able to provide signed and dated informed consent

Exclusion Criteria:

Hypersensitive to cysteamine or to any of the excipients
Hypersensitive to penicillamine
Transplant recipient
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
If female, pregnancy, planned pregnancy, or breast-feeding
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

Contacts/Locations Module

Facility	Status	City	State	ZIP	Country	Contacts
Banner University of Arizona Medical Center		Tucson	Arizona	85724	United States
San Francisco Critical Care Medical Group California Pacific Medical Center

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
33370348	Derived	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.

Label	URL
NovaBiotics Website	View source

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
FG001	450mg, Once Per Day

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Uploaded Document Information

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot_SAP	Yes	Yes	No	Study Protocol and Statistical Analysis Plan	Oct 18, 2017

Derived Section

Miscellaneous Info Module

Submission Tracking

No data available

No data is available for this block.

Condition Browse Module

Intervention Browse Module

Masking Info

Mid-Range Dose, Three times per day

Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening.

Cysteamine: Oral Cysteamine Capsule

Placebo Oral Capsule: Placebo Oral Capsule

Type of Units Analyzed

Arm/Group Information

ID	Title	Description
BG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
BG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
BG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
BG003	450mg, Twice Per Day	Patient takes two oral doses of Cysteamine (450mg) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
BG004	300mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (300mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
BG005	450mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule
BG006	Total	Total of all reporting groups

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	BG00027.2± 5.64 BG00127.5± 6.77 BG00232.5± 12.7 BG003

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG0008 BG0016 BG002

Ethnicity (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Hispanic or Latino	BG0000 BG0010 BG002

BMI

Mean

Standard Deviation

kg/m^2

Title

Denominators

Categories

Title	Measurements
	BG00020.2± 2.23 BG00120.3± 3.03 BG002

Age at CF Diagnosis

Age at CF diagnosis was calculated as the date of CF diagnosis-date of birth

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	BG0000.9± 2.41 BG0013.9± 0.24

Duration of CF years

Duration of CF was calculated as the informed consent date - the date of diagnosis/365.25

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	BG00026.36± 5.69 BG00123.7± 9.26

FEV1 Percent predicted (%)

Mean

Standard Deviation

litres per second

Title

Denominators

Categories

Title	Measurements
	BG00041.5± 15.31 BG00139.4± 19.81 BG002

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

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

Intention to treat population

Posted

Mean

Standard Deviation

log 10 cfu/ml

Baseline through Day 21/End of Study

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (l150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day	Patient takes two oral doses of Cysteamine (450mg) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG004	300mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (300mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG005	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. Cysteamine: Oral Cysteamine Capsule

Units	Counts
Participants	OG00017 OG00111 OG00215 OG003

Title	Denominators	Categories
Day 7 change from baseline	ParticipantsOG00017 ParticipantsOG0019 ParticipantsOG00213 ParticipantsOG003

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.

Summary of participant reported adverse events (AEs) by study group

Posted

Number

participants

Baseline through Day 21/End of Study

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day

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.

Neutrophil Elastase Levels by Visit - Covariate Adjusted ANCOVA with Observed Data ITT Population

Posted

Mean

Standard Deviation

ng/mL

Baseline through Day 21/End of Study

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule

Secondary

Change From Baseline in Sputum IL8

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

ITT Population

Posted

Mean

Standard Deviation

pg/mL

Baseline through Day 21/End of Study

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day	Patient takes two oral doses of Cysteamine (450mg) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule

Secondary

Change From Baseline in FEV1

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

Posted

Mean

Standard Deviation

percentage of predicted

Baseline through Day 21/End of Study

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day

Secondary

Change From Baseline in BMI

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

ITT

Posted

Mean

Standard Deviation

kg/m^2 for BMI

Baseline through Day 21/End of Study

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (l150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day	Patient takes two oral doses of Cysteamine (450mg) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule

Secondary

Change From Baseline in C-Reactive Protein

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

CRP by visit ANCOVA with observed data ITT population

Posted

Mean

Standard Deviation

nmol/L

Baseline through Day 21

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day

Secondary

Change From Baseline in Blood Leukocyte Count

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

ITT

Posted

Mean

Standard Deviation

10^9 leucocytes/L

Baseline through Day 21/End of Study

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day

Secondary

Assessment of Blood Cysteamine Levels

Study Drug Plasma at Day 14 Safety Population

Safety population

Posted

Mean

Standard Deviation

ng/ml

Day 14

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day	Patient takes two oral doses of Cysteamine (450mg) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule

Secondary

Assessment of Sputum Cysteamine Levels

Study Drug Sputum Concentrations at Day 14 Safety Population

Safety Population - there are fewer numbers of patients due to the inability of some patients to provide sputum samples

Posted

Mean

Standard Deviation

ng/ml

Day 14

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day

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.

Mean Change from Baseline in CRFSD-CRISS - Linear MMRM Model with Observed Data (ITT Population)

Posted

Mean

Standard Deviation

units on a scale

Baseline through to Day 21

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule

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

Change from Baseline to Day 14 in CFQ-R Respiratory Score: ANCOVA with Observed Data ITT Population

Posted

Mean

Standard Deviation

units on a scale

Baseline through Day 21/End of Study

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule

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

ITT Linear MMRM Model with Observed Data

Posted

Mean

Standard Deviation

units on a scale

changes from baseline at day 7 and day 14

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule

Secondary

Change From Baseline in Weight

Weight (kg) by visit - ANCOVA with observed data

ITT

Posted

Mean

Standard Deviation

Baseline through Day 21/End of Study

ID	Title	Description
OG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule
OG001	450mg, Once Per Day	Patient takes one oral dose of Cysteamine (450mg) per day, in the morning. The patient takes two oral placebo doses, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG002	150mg, Three Times Per Day	Patient takes three oral doses of Cysteamine (l150mg) per day, one in the morning, one at mid-day and one in the evening. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule
OG003	450mg, Twice Per Day	Patient takes two oral doses of Cysteamine (450mg) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule

Arm/Groups

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Placebo	Patient takes three oral doses of placebo, one in the morning, one at mid-day and one in the evening. Placebo Oral Capsule: Placebo Oral Capsule	17	1	17	9	17
EG001	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. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule	11	1	11	10	11
EG002	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. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule	15	2	15	11	15
EG003	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. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule	15	1	15	12	15
EG004	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. Cysteamine: Oral Cysteamine Capsule Placebo Oral Capsule: Placebo Oral Capsule	16	0	16	10	16
EG005	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. Cysteamine: Oral Cysteamine Capsule	15	1	15	13	15

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Haemoptysis	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	Prolongation of hospitalisation	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG0030 events0 affected15 at risk
Axillary vein thrombosis	Vascular disorders	MedDRA (20.0)	Systematic Assessment	Right axillary deep vein thrombosis	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Nephrolithiasis	Renal and urinary disorders	MedDRA (20.0)	Systematic Assessment	Kidney Stone	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Depression	Psychiatric disorders	MedDRA (20.0)	Systematic Assessment	Depression exacerbation manifesting in suicidal and homicdal ideation	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Infective pulmonary exacerbation of CF	Infections and infestations	MedDRA (20.0)	Systematic Assessment	Pulmonary exacerbation hospitalisation prolongation of hospitalisation	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Campylobacter sepsis	Infections and infestations	MedDRA (20.0)	Systematic Assessment	Campylobacter septicaemia hospitalisation prolongation of hospitalisation	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Nausea	Gastrointestinal disorders	MedDRA (Unspecified)	Systematic Assessment	EG0000 events0 affected0 at risk EG0013 events3 affected11 at risk EG0025 events5 affected15 at risk EG0033 events3 affected15 at risk EG0045 events5 affected16 at risk EG0053 events3 affected15 at risk
vomiting	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0012 events2 affected11 at risk EG0022 events2 affected15 at risk EG003
Abdominal pain	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Diarrhoea	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0002 events2 affected17 at risk EG0011 events1 affected11 at risk EG0021 events1 affected15 at risk EG003
Breath odour	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Constipation	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Abdominal discomfort	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Abdominal pain upper	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Dry mouth	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Dyspepsia	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Gingival recession	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Hypoaesthesia oral	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Post-tussive vomiting	Gastrointestinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Headache	Nervous system disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0015 events5 affected11 at risk EG0021 events1 affected15 at risk EG003
Dizziness	Nervous system disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0012 events2 affected11 at risk EG0021 events1 affected15 at risk EG003
Dysguesia	Nervous system disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Intention tremor	Nervous system disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Migraine	Nervous system disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Sciatica	Nervous system disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Sinus headache	Nervous system disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Haemoptysis	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0002 events2 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0021 events1 affected15 at risk EG003
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Sinus Pain	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Dry throat	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Dysphonia	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Hypoxia	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Increased viscosity of bronchial secretion	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Pleuritic pain	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Sinus congestion	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Sputum retention	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Wheezing	Respiratory, thoracic and mediastinal disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
pyrexia	General disorders	MedDRA (20.0)	Systematic Assessment	EG0002 events2 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Fatigue	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Oedema peripheral	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Pain	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0022 events2 affected15 at risk EG003
Administration site pain	General disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Adminstration site reaction	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Asthenia	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Catheter site pain	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Chest discomfort	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Crepitations	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Infusion site extravasation	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Infusion site pain	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Non-cardiac chest pain	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Peripheral swelling	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Sensation of foreign body	General disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Infective pulmonary exacerbation of cystic fibrosis	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Urinary tract infection (bacterial)	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Vulvovaginal candidiasis	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Vulvovaginal mycotic infection	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0002 events2 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Campylobacter sepsis	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Clostridium difficle colitis	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Clostridium difficle infection	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Laryngitis	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Oral candidiasis	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Oral herpes	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Pharyngitis	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Tooth abcess	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Viral infection	Infections and infestations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Pulmonary function test decreased	Investigations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Alanine aminotransferase increased	Investigations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Aspartate aminotransferase increased	Investigations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Blood alkaline phosphatase increased	Investigations	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
C-reactive protein increased	Investigations	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Carbon dioxide	Investigations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Colonoscopy	Investigations	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Eosinophil count increased	Investigations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Mycobacterium tuberculosis complex test positive	Investigations	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0021 events1 affected15 at risk EG003
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Back pain	Musculoskeletal and connective tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Groin pain	Musculoskeletal and connective tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Musculoskeletal chest pain	Musculoskeletal and connective tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Rash	Skin and subcutaneous tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Dermatitis allergic	Musculoskeletal and connective tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Pruritus allergic	Skin and subcutaneous tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Pruritus generalised	Skin and subcutaneous tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Skin odour abnormal	Skin and subcutaneous tissue disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Decreased appetite	Metabolism and nutrition disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Hyperglycaemia	Metabolism and nutrition disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Hyperkalaemia	Metabolism and nutrition disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Hypoglycaemia	Metabolism and nutrition disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Insomnia	Psychiatric disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0022 events2 affected15 at risk EG003
Anxiety	Psychiatric disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Depression	Psychiatric disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Axillary vein thrombosis	Vascular disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Deep vein thrombosis	Vascular disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Haematoma	Vascular disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Hypotension	Vascular disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Phlebitis	Vascular disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0011 events1 affected11 at risk EG0020 events0 affected15 at risk EG003
Contusion	Injury, poisoning and procedural complications	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Infusion site reaction	Injury, poisoning and procedural complications	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Stoma site reaction	Injury, poisoning and procedural complications	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Nephrolithiasis	Renal and urinary disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Renal colic	Renal and urinary disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Hypoacusis	Ear and labyrinth disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0021 events1 affected15 at risk EG003
Vision blurred	Eye disorders	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Oligomenorrhoea	Reproductive system and breast disorders	MedDRA (20.0)	Systematic Assessment	EG0000 events0 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003
Sinus operation	Surgical and medical procedures	MedDRA (20.0)	Systematic Assessment	EG0001 events1 affected17 at risk EG0010 events0 affected11 at risk EG0020 events0 affected15 at risk EG003

ID	Term
D003550	Cystic Fibrosis
D008171	Lung Diseases
D001424	Bacterial Infections
D011014	Pneumonia
D001991	Bronchitis

Ancestor Terms

ID	Term
D010182	Pancreatic Diseases
D004066	Digestive System Diseases
D012140	Respiratory Tract Diseases
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D007232	Infant, Newborn, Diseases
D001423	Bacterial Infections and Mycoses
D007239	Infections
D012141	Respiratory Tract Infections
D001982	Bronchial Diseases
D008173	Lung Diseases, Obstructive

ID	Term
D003543	Cysteamine

Patient takes two oral doses of Cysteamine (450mg) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day.

Cysteamine: Oral Cysteamine Capsule

Placebo Oral Capsule: Placebo Oral Capsule

Patient takes two oral doses of Cysteamine (450mg) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day.

Cysteamine: Oral Cysteamine Capsule

Placebo Oral Capsule: Placebo Oral Capsule

Patient takes two oral doses of Cysteamine (450mg) per day, one in the morning and one in the evening. The patient takes one oral placebo dose, at mid-day.

Cysteamine: Oral Cysteamine Capsule

Placebo Oral Capsule: Placebo Oral Capsule

EG0050 events0 affected15 at risk