Trial Outcomes & Findings for Derazantinib in Subjects With FGFR2 Gene Fusion-, Mutation- or Amplification- Positive Inoperable or Advanced Intrahepatic Cholangiocarcinoma (NCT NCT03230318)
NCT ID: NCT03230318
Last Updated: 2023-12-19
Results Overview
ORR was defined as the proportion of patients who achieved a confirmed clinical response (CR) or partial response (PR) by blinded independent central review using the internationally recognized criteria for the radiological assessment in tumor response of solid tumors (RECIST) Version 1.1
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
148 participants
Primary outcome timeframe
From first dose and up to 54 months
Results posted on
2023-12-19
Participant Flow
A total of 729 patients underwent molecular screening, and 148 were enrolled and assigned treatment. One patient was subsequently not confirmed to have fibroblast growth factor receptor 2 (FGFR2) fusion, and was therefore excluded from the Safety/ITT population, meaning that for all Safety/ITT population analyses 147 patients were included (Substudy 1 = 103, Substudy 2 = 44).
Participant milestones
| Measure |
Substudy 1
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
Substudy 2 comprised of patients with with inoperable or advanced iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily
|
|---|---|---|
|
Overall Study
STARTED
|
104
|
44
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
104
|
44
|
Reasons for withdrawal
| Measure |
Substudy 1
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
Substudy 2 comprised of patients with with inoperable or advanced iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily
|
|---|---|---|
|
Overall Study
Radiographic disease progression
|
72
|
19
|
|
Overall Study
Clinical progression
|
17
|
7
|
|
Overall Study
Adverse Event
|
5
|
3
|
|
Overall Study
Physician Decision
|
2
|
3
|
|
Overall Study
Death
|
3
|
2
|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
1
|
3
|
|
Overall Study
Other
|
3
|
7
|
Baseline Characteristics
Derazantinib in Subjects With FGFR2 Gene Fusion-, Mutation- or Amplification- Positive Inoperable or Advanced Intrahepatic Cholangiocarcinoma
Baseline characteristics by cohort
| Measure |
Substudy 1
n=103 Participants
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
n=44 Participants
Substudy 2 comprised of patients with inoperable or advanced iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily
|
Total
n=147 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
56.5 years
STANDARD_DEVIATION 12.28 • n=5 Participants
|
61.3 years
STANDARD_DEVIATION 11.75 • n=7 Participants
|
58.0 years
STANDARD_DEVIATION 12.28 • n=5 Participants
|
|
Sex: Female, Male
Female
|
67 Participants
n=5 Participants
|
25 Participants
n=7 Participants
|
92 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
36 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
55 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
8 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
86 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
123 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From first dose and up to 54 monthsPopulation: Intent-to-treat (ITT) Population: included all patients who received any amount of study drug
ORR was defined as the proportion of patients who achieved a confirmed clinical response (CR) or partial response (PR) by blinded independent central review using the internationally recognized criteria for the radiological assessment in tumor response of solid tumors (RECIST) Version 1.1
Outcome measures
| Measure |
Substudy 1
n=103 Participants
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
Substudy 2 comprised of patients with iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily one hour prior to or two hours after a meal
|
|---|---|---|
|
Substudy 1: Objective Response Rate (ORR)
|
22.3 Percentage of participants
Interval 14.7 to 31.6
|
—
|
PRIMARY outcome
Timeframe: From first dose and up to 3 monthsPopulation: Modified intent-to-treat (mITT) Population: included all patients in the Safety/ITT population, who had at least one post-baseline disease assessment (at least one post-baseline imaging assessment in accordance with RECIST 1.1, or documented clinical progression \[every effort was made to objectively assess radiographic progression\]), or reported death during the treatment period.
PFS3 was defined as the proportion of patients who have progression-free survival at 3 months from the first date of receiving study drug as assessed by survival status and blinded independent central review as per RECIST 1.1.
Outcome measures
| Measure |
Substudy 1
n=43 Participants
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
Substudy 2 comprised of patients with iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily one hour prior to or two hours after a meal
|
|---|---|---|
|
Substudy 2: Progression Free Survival at 3 Months (PFS 3)
|
62.8 Percentage of participants
Interval 46.7 to 77.0
|
—
|
SECONDARY outcome
Timeframe: From first dose and up to 54 monthsPopulation: mITT Population: included all patients in the Safety/ITT population, who had at least one post-baseline disease assessment (at least one post-baseline imaging assessment in accordance with RECIST 1.1, or documented clinical progression \[every effort was made to objectively assess radiographic progression\]), or reported death during the treatment period.
PFS was calculated from the first date of receiving study drug until radiographic disease progression by blinded independent central review or death.
Outcome measures
| Measure |
Substudy 1
n=103 Participants
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
n=44 Participants
Substudy 2 comprised of patients with iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily one hour prior to or two hours after a meal
|
|---|---|---|
|
PFS
|
8.1 Months
Interval 5.5 to 8.3
|
8.3 Months
Interval 3.5 to
Not evaluable due to the insufficient number of events
|
SECONDARY outcome
Timeframe: From first dose and up to 54 monthsPopulation: ITT population: included all patients who received any amount of study drug
OS was calculated from the first date of receiving study drug until death
Outcome measures
| Measure |
Substudy 1
n=103 Participants
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
n=44 Participants
Substudy 2 comprised of patients with iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily one hour prior to or two hours after a meal
|
|---|---|---|
|
Overall Survival (OS)
|
17.2 Months
Interval 12.5 to 22.4
|
11.9 Months
Interval 8.4 to 15.9
|
SECONDARY outcome
Timeframe: From first dose and up to 54 monthsPopulation: ITT Population: included all patients who received any amount of study drug
DoR was calculated from the first date of documented tumor response to disease progression by blinded independent central review per RECIST version 1.1 DoR was derived only for patients who have the best overall response of CR or PR
Outcome measures
| Measure |
Substudy 1
n=103 Participants
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
n=44 Participants
Substudy 2 comprised of patients with iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily one hour prior to or two hours after a meal
|
|---|---|---|
|
Duration of Response (DoR)
|
6.5 Months
Interval 4.6 to 9.2
|
NA Months
Not evaluable due to the insufficient number of events
|
SECONDARY outcome
Timeframe: From first dose and up to 54 monthsPopulation: mITT Population: included all patients in the Safety/ITT population, who had at least one post-baseline disease assessment (at least one post-baseline imaging assessment in accordance with RECIST 1.1, or documented clinical progression \[every effort was made to objectively assess radiographic progression\]), or reported death during the treatment period.
ORR was defined as the proportion of patients who achieved a confirmed clinical response (CR) or partial response (PR) by blinded independent central review using the internationally recognized criteria for the radiological assessment in tumor response of solid tumors (RECIST) Version 1.1
Outcome measures
| Measure |
Substudy 1
n=43 Participants
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
Substudy 2 comprised of patients with iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily one hour prior to or two hours after a meal
|
|---|---|---|
|
Substudy 2: Objective Response Rate
|
9.3 Percentage of participants
Interval 2.6 to 22.1
|
—
|
SECONDARY outcome
Timeframe: TEAEs were defined as all events occurring after drug treatment began and up to 30 days after last study drug administrationPopulation: Safety / ITT population included all patients who received any amount of study drug
Number of patients experiencing TEAE of Grade 3 to 5 according to Common Terminology Criteria for Adverse Events (CTCAE)
Outcome measures
| Measure |
Substudy 1
n=103 Participants
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
n=44 Participants
Substudy 2 comprised of patients with iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily one hour prior to or two hours after a meal
|
|---|---|---|
|
Number of Patients With Grade 3-5 Treatment-emergent Adverse Events (TEAEs)
Number of patients without TEAEs of Grade 3-5
|
37 Participants
|
18 Participants
|
|
Number of Patients With Grade 3-5 Treatment-emergent Adverse Events (TEAEs)
Number of patients with only unrelated TEAEs of Grade 3-5
|
31 Participants
|
16 Participants
|
|
Number of Patients With Grade 3-5 Treatment-emergent Adverse Events (TEAEs)
Number of patients with related TEAEs of Grade 3-5
|
35 Participants
|
10 Participants
|
Adverse Events
Substudy 1
Serious events: 37 serious events
Other events: 103 other events
Deaths: 74 deaths
Substudy 2
Serious events: 16 serious events
Other events: 43 other events
Deaths: 25 deaths
Serious adverse events
| Measure |
Substudy 1
n=103 participants at risk
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
n=44 participants at risk
Substudy 2 comprised of patients with with inoperable or advanced iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily
|
|---|---|---|
|
Investigations
Alanine aminotransferase increased
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Aspartate aminotransferase increased
|
1.9%
2/103 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Blood bilirubin increased
|
1.9%
2/103 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Cardiac disorders
Atrioventricular block second degree
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Cardiac disorders
Bundle branch block left
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Nervous system disorders
Encephalopathy
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Nervous system disorders
Hepatic encephalopathy
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Nervous system disorders
Presyncope
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Nervous system disorders
Tremor
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Eye disorders
Visual acuity reduced
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial obstruction
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal oedema
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Abdominal pain
|
5.8%
6/103 • Number of events 7 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Ascites
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Diverticular perforation
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Gastric perforation
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Nausea
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Oesophageal varices haemorrhage
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Vomiting
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Renal and urinary disorders
Acute kidney injury
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Renal and urinary disorders
Renal tubular necrosis
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
1.9%
2/103 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Arthritis bacterial
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Bacteraemia
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
COVID-19 pneumonia
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Intervertebral discitis
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Meningitis bacterial
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Peritonitis
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Pneumonia
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Sepsis
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Sialoadenitis
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Subcutaneous abscess
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Urinary tract infection
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Injury, poisoning and procedural complications
Fall
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Injury, poisoning and procedural complications
Traumatic intracranial haematoma
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumour haemorrhage
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Surgical and medical procedures
Liver transplant
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Vascular disorders
Peripheral arterial occlusive disease
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Vascular disorders
Vena cava thrombosis
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
Chest pain
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
Death
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
Disease progression
|
8.7%
9/103 • Number of events 9 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
General physical health deterioration
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
Pyrexia
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Hepatobiliary disorders
Cholangitis
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Hepatobiliary disorders
Hepatic failure
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Hepatobiliary disorders
Jaundice cholestatic
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Reproductive system and breast disorders
Prostatitis
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Product Issues
Device occlusion
|
0.00%
0/103 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
Other adverse events
| Measure |
Substudy 1
n=103 participants at risk
Substudy 1 comprised of patients with inoperable or advanced iCCA with FGFR2 fusions
Derazantinib was administered orally at 300 mg once daily
|
Substudy 2
n=44 participants at risk
Substudy 2 comprised of patients with with inoperable or advanced iCCA with FGFR2 mutations or amplifications
Derazantinib was administered orally at 300 mg once daily
|
|---|---|---|
|
Nervous system disorders
Dysgeusia
|
19.4%
20/103 • Number of events 20 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
25.0%
11/44 • Number of events 11 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Nervous system disorders
Headache
|
11.7%
12/103 • Number of events 14 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
11.4%
5/44 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
0.97%
1/103 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Nervous system disorders
Tremor
|
7.8%
8/103 • Number of events 9 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Eye disorders
Cornea verticillata
|
6.8%
7/103 • Number of events 7 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Eye disorders
Dry eye
|
19.4%
20/103 • Number of events 20 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
15.9%
7/44 • Number of events 7 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Eye disorders
Keratitis
|
1.9%
2/103 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Eye disorders
Vision blurred
|
25.2%
26/103 • Number of events 26 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
13.6%
6/44 • Number of events 8 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Eye disorders
Visual acuity reduced
|
6.8%
7/103 • Number of events 7 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Eye disorders
Visual impairment
|
4.9%
5/103 • Number of events 5 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 4 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Eye disorders
Xerophthalmia
|
8.7%
9/103 • Number of events 11 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
11.4%
5/44 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.6%
14/103 • Number of events 15 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
15.9%
7/44 • Number of events 7 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
9.7%
10/103 • Number of events 10 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
4.5%
2/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
8.7%
9/103 • Number of events 9 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Abdominal distension
|
2.9%
3/103 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Abdominal pain
|
25.2%
26/103 • Number of events 30 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
13.6%
6/44 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
9.7%
10/103 • Number of events 12 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Constipation
|
27.2%
28/103 • Number of events 31 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
18.2%
8/44 • Number of events 10 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Diarrhoea
|
33.0%
34/103 • Number of events 52 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
31.8%
14/44 • Number of events 21 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Dry mouth
|
29.1%
30/103 • Number of events 32 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
38.6%
17/44 • Number of events 17 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Blood phosphorus increased
|
12.6%
13/103 • Number of events 20 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
4.5%
2/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Neutrophil count decreased
|
3.9%
4/103 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Platelet count decreased
|
9.7%
10/103 • Number of events 20 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
11.4%
5/44 • Number of events 5 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Weight decreased
|
16.5%
17/103 • Number of events 17 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
13.6%
6/44 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
White blood cell count decreased
|
5.8%
6/103 • Number of events 11 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Blood and lymphatic system disorders
Anaemia
|
15.5%
16/103 • Number of events 18 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
13.6%
6/44 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
6.8%
7/103 • Number of events 8 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Nervous system disorders
Dizziness
|
12.6%
13/103 • Number of events 13 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Alanine aminotransferase increased
|
35.0%
36/103 • Number of events 43 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
29.5%
13/44 • Number of events 15 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Aspartate aminotransferase increased
|
44.7%
46/103 • Number of events 52 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
29.5%
13/44 • Number of events 15 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Blood alkaline phosphatase increased
|
16.5%
17/103 • Number of events 18 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
20.5%
9/44 • Number of events 11 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Investigations
Blood creatinine increased
|
16.5%
17/103 • Number of events 18 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
15.9%
7/44 • Number of events 8 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Dyspepsia
|
9.7%
10/103 • Number of events 10 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
4.5%
2/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
5.8%
6/103 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Nausea
|
39.8%
41/103 • Number of events 51 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
40.9%
18/44 • Number of events 23 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Gastrointestinal disorders
Vomiting
|
29.1%
30/103 • Number of events 43 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
34.1%
15/44 • Number of events 23 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
15.5%
16/103 • Number of events 17 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
13.6%
6/44 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
13.6%
14/103 • Number of events 14 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Skin and subcutaneous tissue disorders
Nail discolouration
|
1.9%
2/103 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.6%
13/103 • Number of events 13 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
14.6%
15/103 • Number of events 15 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
19.4%
20/103 • Number of events 23 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
20.5%
9/44 • Number of events 9 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Metabolism and nutrition disorders
Dehydration
|
5.8%
6/103 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
4.5%
2/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
10.7%
11/103 • Number of events 11 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Metabolism and nutrition disorders
Hyperphosphataemia
|
29.1%
30/103 • Number of events 36 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
27.3%
12/44 • Number of events 14 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
6.8%
7/103 • Number of events 9 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
4.5%
2/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
7.8%
8/103 • Number of events 8 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
9.1%
4/44 • Number of events 4 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
5.8%
6/103 • Number of events 6 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
4.5%
2/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Infections and infestations
Urinary tract infection
|
8.7%
9/103 • Number of events 9 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
4.5%
2/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Vascular disorders
Hypertension
|
23.3%
24/103 • Number of events 30 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
11.4%
5/44 • Number of events 7 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
Asthenia
|
24.3%
25/103 • Number of events 27 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
13.6%
6/44 • Number of events 8 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
Fatigue
|
33.0%
34/103 • Number of events 37 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
29.5%
13/44 • Number of events 16 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
Mucosal inflammation
|
2.9%
3/103 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
6.8%
3/44 • Number of events 3 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
Oedema peripheral
|
4.9%
5/103 • Number of events 5 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
9.1%
4/44 • Number of events 5 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
General disorders
Pyrexia
|
6.8%
7/103 • Number of events 9 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
4.5%
2/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Psychiatric disorders
Anxiety
|
7.8%
8/103 • Number of events 8 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
4.5%
2/44 • Number of events 2 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Psychiatric disorders
Depression
|
6.8%
7/103 • Number of events 7 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
2.3%
1/44 • Number of events 1 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
|
Psychiatric disorders
Insomnia
|
14.6%
15/103 • Number of events 15 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
0.00%
0/44 • All AEs were assessed from first day of treatment and until 28-35-day post-treatment follow-up period. The AE assessment period for the whole study lasted up to 50 months.
|
Additional Information
Dr Manuel Häckl, MD
Basilea Pharmaceutica International Ltd, Allschwil
Phone: +41 76 302 53 10
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60