Trial Outcomes & Findings for An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome (NCT NCT02117713)
NCT ID: NCT02117713
Last Updated: 2021-07-01
Results Overview
This primacy efficacy endpoint is the mean change from MRX baseline to week 48 in fasting sBA levels.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
34 participants
Primary outcome timeframe
Baseline to Week 48
Results posted on
2021-07-01
Participant Flow
Participant milestones
| Measure |
LUM001 (Maralixibat)
Participant received LUM001 also known as Maralixibat (MRX) administered orally once per day for up to 216 weeks.
|
|---|---|
|
Week 48
STARTED
|
34
|
|
Week 48
COMPLETED
|
26
|
|
Week 48
NOT COMPLETED
|
8
|
|
Long Term Follow-up Treatment
STARTED
|
26
|
|
Long Term Follow-up Treatment
COMPLETED
|
20
|
|
Long Term Follow-up Treatment
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
LUM001 (Maralixibat)
Participant received LUM001 also known as Maralixibat (MRX) administered orally once per day for up to 216 weeks.
|
|---|---|
|
Week 48
Adverse Event
|
4
|
|
Week 48
Withdrawal by caregiver
|
2
|
|
Week 48
Did not consent to Protocol Amendment 4
|
2
|
|
Long Term Follow-up Treatment
Adverse Event
|
3
|
|
Long Term Follow-up Treatment
Progressive Disease
|
2
|
|
Long Term Follow-up Treatment
Non-Compliance with study drug
|
1
|
Baseline Characteristics
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
Baseline characteristics by cohort
| Measure |
Core Study Period
n=34 Participants
Participant received LUM001 also known as Maralixibat (MRX) administered orally once per day.
|
|---|---|
|
Age, Continuous
|
7 years of age
STANDARD_DEVIATION 4.55 • n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
27 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
4 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
30 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 48Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This primacy efficacy endpoint is the mean change from MRX baseline to week 48 in fasting sBA levels.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=26 Participants
Week 48 values for participants.
|
|---|---|---|
|
Change From MRX Baseline to Week 48 in Fasting Serum Bile Acid (sBA)
|
223.62 μmol/L
Standard Deviation 205.041
|
157.35 μmol/L
Standard Deviation 178.538
|
SECONDARY outcome
Timeframe: Baseline to week 216Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
The secondary endpoint of this study was the mean change from MRX baseline to week 216 fasting in sBA levels.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=10 Participants
Week 48 values for participants.
|
|---|---|---|
|
Change From MRX Baseline to Week 216 in Fasting Serum Bile Acid (sBA)
|
223.62 μmol/L
Standard Deviation 205.041
|
118.81 μmol/L
Standard Deviation 126.878
|
SECONDARY outcome
Timeframe: Baseline to Week 218Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This secondary efficacy endpoint is the mean change from MRX baseline over time to week 218 in pruritus as measured by ItchRO(Obs) weekly average morning severity score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe). Results reported here are the long-term results.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=6 Participants
Week 48 values for participants.
|
|---|---|---|
|
Change From Baseline to Week 218 in Pruritus
|
2.533 score on a scale
Standard Deviation 0.8423
|
0.429 score on a scale
Standard Deviation 0.8571
|
SECONDARY outcome
Timeframe: Baseline to week 216Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in ALT levels.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=7 Participants
Week 48 values for participants.
|
|---|---|---|
|
Change From Baseline to Week 216 in Alanine Aminotransferase
|
152.9 U/L
Standard Deviation 82.26
|
222.4 U/L
Standard Deviation 126.53
|
SECONDARY outcome
Timeframe: Baseline to Week 216Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in ALP levels.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=7 Participants
Week 48 values for participants.
|
|---|---|---|
|
Change From Baseline to End of Treatment in Alkaline Phosphatase
|
598.8 U/L
Standard Deviation 199.89
|
528.3 U/L
Standard Deviation 221.93
|
SECONDARY outcome
Timeframe: Baseline to week 216Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in AST levels.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=7 Participants
Week 48 values for participants.
|
|---|---|---|
|
Change From MRX Baseline to Week 216 in Aspartate Aminotransferase
|
149.7 U/L
Standard Deviation 88.27
|
231.4 U/L
Standard Deviation 122.26
|
SECONDARY outcome
Timeframe: Baseline to week 216Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in clinician xanthoma severity scores. It is based on a 0-4 scale to rate the number of lesions present and the degree to which the lesions interfere or limit activities. Clinician xanthoma severity scores range from 0 to 4, with a score of zero representing no evidence of xanthomatosis and a score of 4 representing xanthoma so severe that it is disabling.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=7 Participants
Week 48 values for participants.
|
|---|---|---|
|
Change From MRX Baseline to Week 216 in Clinician Xanthoma Severity Score
|
0.9 Points
Standard Deviation 1.33
|
1.1 Points
Standard Deviation 1.68
|
SECONDARY outcome
Timeframe: Baseline to Week 216Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This secondary efficacy endpoint is the mean change from MRX baseline over time to week 216/LOCF in pruritus as measured by the Clinician Scratch Scale (CSS). The Clinician Scratch Scale uses a 5-point scale, where 0 = none; 1 = rubbing or mild scratching when undistracted; 2 = active scratching without evident skin abrasions; 3 = abrasion evident; 4 = cutaneous mutilation, haemorrhage and scarring evident.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=7 Participants
Week 48 values for participants.
|
|---|---|---|
|
Change From Baseline to Week 216/LOFC Clinician Scratch Scale (CSS) Score
|
2.9 Points
Standard Deviation 1.08
|
1.1 Points
Standard Deviation 1.07
|
SECONDARY outcome
Timeframe: Baseline to Week 216Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in GGT.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=7 Participants
Week 48 values for participants.
|
|---|---|---|
|
Change From MRX Baseline to Week 216 in Gamma Glutamyltransferase
|
493.5 U/L
Standard Deviation 380.29
|
431 U/L
Standard Deviation 235.35
|
SECONDARY outcome
Timeframe: Baseline to week 216Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in total bilirubin.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=7 Participants
Week 48 values for participants.
|
|---|---|---|
|
Mean Change From MRX Baseline to Week 216 in Total Bilirubin
|
5.62 mg/dL
Standard Deviation 6.420
|
5.39 mg/dL
Standard Deviation 5.181
|
SECONDARY outcome
Timeframe: Baseline to week 216Population: Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm.
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in direct bilirubin.
Outcome measures
| Measure |
MRX Baseline Value
n=34 Participants
These are the MRX baseline values for participants
|
Maralixibat Week 48 Values
n=7 Participants
Week 48 values for participants.
|
|---|---|---|
|
Mean Change From MRX Baseline to Week 216 in Direct Bilirubin
|
3.541 mg/dL
Standard Deviation 3.6433
|
3.714 mg/dL
Standard Deviation 3.8369
|
Adverse Events
LUM001 (Maralixibat)
Serious events: 6 serious events
Other events: 34 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
LUM001 (Maralixibat)
n=34 participants at risk
Participant received LUM001 (also known as Maralixibat or MRX) as oral solution once daily based on participant's weight. The dose was escalated from 14, 35, 70, 140 and 280 microgram per kilogram per day (mcg/kg/day) for 4-week dose escalation period. During 8-weeks of dose optimization period, drug was adjusted in titrated manner and dosing was continued to complete the stable dosing and safety monitoring periods for up to 96 weeks of cumulative LUM001 exposure in this study. Dosing during long-term optional follow-up treatment periods 1 and 2 was maintained at the same dose levels as at weeks 96 and 144 for participants rolling over into these treatment periods respectively.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Haematemesis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Haematochezia
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Hepatobiliary disorders
Autoimmune hepatitis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Hepatobiliary disorders
Chronic hepatic failure
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Fungal infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Metabolism and nutrition disorders
Malnutrition
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
Other adverse events
| Measure |
LUM001 (Maralixibat)
n=34 participants at risk
Participant received LUM001 (also known as Maralixibat or MRX) as oral solution once daily based on participant's weight. The dose was escalated from 14, 35, 70, 140 and 280 microgram per kilogram per day (mcg/kg/day) for 4-week dose escalation period. During 8-weeks of dose optimization period, drug was adjusted in titrated manner and dosing was continued to complete the stable dosing and safety monitoring periods for up to 96 weeks of cumulative LUM001 exposure in this study. Dosing during long-term optional follow-up treatment periods 1 and 2 was maintained at the same dose levels as at weeks 96 and 144 for participants rolling over into these treatment periods respectively.
|
|---|---|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Blood and lymphatic system disorders
Splenic infarction
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Congenital, familial and genetic disorders
Bicuspid aortic valve
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Congenital, familial and genetic disorders
Non-compaction cardiomyopathy
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Ear and labyrinth disorders
Ear pain
|
8.8%
3/34 • Number of events 5 • Baseline to End of Treatment (Week 216)
|
|
Ear and labyrinth disorders
Eustachian tube dysfunction
|
2.9%
1/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Ear and labyrinth disorders
Excessive cerumen production
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Ear and labyrinth disorders
Tympanic membrane perforation
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Ear and labyrinth disorders
Vertigo
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Eye disorders
Asthenopia
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Eye disorders
Eye pruritus
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Eye disorders
Vision blurred
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Abdominal discomfort
|
8.8%
3/34 • Number of events 4 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Abdominal distension
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Abdominal pain
|
17.6%
6/34 • Number of events 39 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Abdominal pain lower
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Abdominal pain upper
|
17.6%
6/34 • Number of events 18 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Aphthous ulcer
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Dental caries
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Diarrhoea
|
38.2%
13/34 • Number of events 37 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Dry mouth
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Eosinophilic oesophagitis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Faecaloma
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
11.8%
4/34 • Number of events 4 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Haematochezia
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Inguinal hernia
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Nausea
|
8.8%
3/34 • Number of events 4 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Post-tussive vomiting
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Toothache
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Gastrointestinal disorders
Vomiting
|
29.4%
10/34 • Number of events 17 • Baseline to End of Treatment (Week 216)
|
|
General disorders
Asthenia
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
General disorders
Catheter site haemorrhage
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
General disorders
Catheter site pain
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
General disorders
Chest pain
|
5.9%
2/34 • Number of events 3 • Baseline to End of Treatment (Week 216)
|
|
General disorders
Fatigue
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
General disorders
Influenza like illness
|
17.6%
6/34 • Number of events 22 • Baseline to End of Treatment (Week 216)
|
|
General disorders
Medical device pain
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
General disorders
Peripheral swelling
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
General disorders
Pyrexia
|
44.1%
15/34 • Number of events 39 • Baseline to End of Treatment (Week 216)
|
|
Hepatobiliary disorders
Hepatobiliary disease
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Hepatobiliary disorders
Hepatosplenomegaly
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Hepatobiliary disorders
Jaundice
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Immune system disorders
Seasonal allergy
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Adenovirus infection
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Anorectal infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Bacterial infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Bronchitis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Conjunctivitis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Conjunctivitis viral
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Ear infection
|
20.6%
7/34 • Number of events 15 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Ear infection bacterial
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Eye infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Fungal skin infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Gastroenteritis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Gastroenteritis viral
|
11.8%
4/34 • Number of events 5 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Gastrointestinal viral infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Gingivitis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
2.9%
1/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Herpes simplex
|
2.9%
1/34 • Number of events 5 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Influenza
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Kidney infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Localised infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Nasopharyngitis
|
29.4%
10/34 • Number of events 24 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Oral herpes
|
2.9%
1/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Otitis media
|
2.9%
1/34 • Number of events 3 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Parainfluenzae virus infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Pharyngitis streptococcal
|
14.7%
5/34 • Number of events 10 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Pneumonia
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Pneumonia bacterial
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Respiratory tract infection viral
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Sinusitis
|
17.6%
6/34 • Number of events 11 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Staphylococcal skin infection
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Tinea capitis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Upper respiratory tract infection
|
47.1%
16/34 • Number of events 63 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Urinary tract infection
|
8.8%
3/34 • Number of events 5 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Viral infection
|
11.8%
4/34 • Number of events 5 • Baseline to End of Treatment (Week 216)
|
|
Infections and infestations
Viral upper respiratory tract infection
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Arterial injury
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Arthropod sting
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Concussion
|
8.8%
3/34 • Number of events 3 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Contusion
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Hand fracture
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Head injury
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Joint injury
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Limb injury
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Muscle injury
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Procedural anxiety
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Procedural haemorrhage
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Procedural pain
|
11.8%
4/34 • Number of events 8 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Rib fracture
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Skin laceration
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Sports injury
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Sunburn
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Alanine aminotransferase abnormal
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Alanine aminotransferase increased
|
29.4%
10/34 • Number of events 14 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Aspartate aminotransferase abnormal
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Aspartate aminotransferase increased
|
23.5%
8/34 • Number of events 8 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Blood albumin increased
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Blood alkaline phosphatase increased
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Blood bicarbonate decreased
|
2.9%
1/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Blood bilirubin increased
|
5.9%
2/34 • Number of events 3 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Gamma-glutamyltransferase abnormal
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Gamma-glutamyltransferase increased
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Heart rate irregular
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
International normalised ratio abnormal
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
International normalised ratio increased
|
20.6%
7/34 • Number of events 9 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Neutrophil count decreased
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Platelet count decreased
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Ultrasound liver abnormal
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Investigations
Vitamin D decreased
|
5.9%
2/34 • Number of events 3 • Baseline to End of Treatment (Week 216)
|
|
Investigations
White blood cell count increased
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Metabolism and nutrition disorders
Decreased appetite
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
14.7%
5/34 • Number of events 6 • Baseline to End of Treatment (Week 216)
|
|
Metabolism and nutrition disorders
Vitamin E deficiency
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Metabolism and nutrition disorders
Vitamin K deficiency
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.8%
3/34 • Number of events 3 • Baseline to End of Treatment (Week 216)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.7%
5/34 • Number of events 7 • Baseline to End of Treatment (Week 216)
|
|
Musculoskeletal and connective tissue disorders
Growth retardation
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
11.8%
4/34 • Number of events 9 • Baseline to End of Treatment (Week 216)
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatic adenoma
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm skin
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Nervous system disorders
Carotid artery stenosis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Nervous system disorders
Dizziness
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Nervous system disorders
Febrile convulsion
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Nervous system disorders
Headache
|
35.3%
12/34 • Number of events 16 • Baseline to End of Treatment (Week 216)
|
|
Nervous system disorders
Migraine
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Nervous system disorders
Restless legs syndrome
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Nervous system disorders
Seizure
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Nervous system disorders
Somnolence
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Psychiatric disorders
Anxiety
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Psychiatric disorders
Depression
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Psychiatric disorders
Insomnia
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Renal and urinary disorders
Chromaturia
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Renal and urinary disorders
Dysuria
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Renal and urinary disorders
Haematuria
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Renal and urinary disorders
Proteinuria
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Renal and urinary disorders
Renal tubular acidosis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Adenoidal disorder
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
38.2%
13/34 • Number of events 18 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
20.6%
7/34 • Number of events 84 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
17.6%
6/34 • Number of events 9 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
26.5%
9/34 • Number of events 12 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary artery stenosis
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
20.6%
7/34 • Number of events 9 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Acne
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Alopecia areata
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Erythema
|
5.9%
2/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Exfoliative rash
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Miliaria
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Papule
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
23.5%
8/34 • Number of events 11 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Rash
|
20.6%
7/34 • Number of events 10 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Skin hypertrophy
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Skin and subcutaneous tissue disorders
Xanthoma
|
2.9%
1/34 • Number of events 2 • Baseline to End of Treatment (Week 216)
|
|
Vascular disorders
Flushing
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
|
Vascular disorders
Hypertension
|
2.9%
1/34 • Number of events 1 • Baseline to End of Treatment (Week 216)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place