Trial Outcomes & Findings for A Study to Evaluate the Safety and Effect of CFZ533 on Patients With Graves' Disease (NCT NCT02713256)
NCT ID: NCT02713256
Last Updated: 2021-01-05
Results Overview
Normalization of TSH is defined as TSH level greater than 0.35 mU/L after 12 week treatment (Day 85)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
12 week (DAY 85)
Results posted on
2021-01-05
Participant Flow
Participant milestones
| Measure |
CFZ533 10 mg/kg
CFZ533 intravenously over approximately one hour
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Evaluate the Safety and Effect of CFZ533 on Patients With Graves' Disease
Baseline characteristics by cohort
| Measure |
CFZ533 10 mg/kg
n=15 Participants
CFZ533 intravenously over approximately one hour
|
|---|---|
|
Age, Continuous
|
44.3 years
STANDARD_DEVIATION 12.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 week (DAY 85)Population: Pharmacodynamic (PD) Analysis Set- all patients in the study was included in this analysis set. Patients who discontinue before 4 weeks of treatment for any reason are not counted for the calculation of responders.
Normalization of TSH is defined as TSH level greater than 0.35 mU/L after 12 week treatment (Day 85)
Outcome measures
| Measure |
CFZ533 10 mg/kg
n=13 Participants
CFZ533 intravenously over approximately one hour
|
|---|---|
|
Percentage of Participants Whose Thyroid Stimulating Hormone (TSH) Levels Normalize After 12 Week Treatment
|
0 percentage of participants
|
PRIMARY outcome
Timeframe: 12 week (DAY 85)Population: Pharmacodynamic (PD) Analysis Set- all patients in the study was included in this analysis set. Patients who discontinue before 4 weeks of treatment for any reason are not counted for the calculation of responders.
Percentage of participants whose total triiodothyronine (total T3) levels decrease after 12 week treatment. A decrease is when total T3 level is below Upper limit of normal (ULN) ≤ 2.79 nmol/L
Outcome measures
| Measure |
CFZ533 10 mg/kg
n=13 Participants
CFZ533 intravenously over approximately one hour
|
|---|---|
|
Percentage of Participants Whose Total Triiodothyronine (Total T3) Levels Decrease After 12 Week Treatment
|
38.5 percentage of participants
|
PRIMARY outcome
Timeframe: 12 week (DAY 85)Population: Pharmacodynamic (PD) Analysis Set- all patients in the study was included in this analysis set. Patients who discontinue before 4 weeks of treatment for any reason are not counted for the calculation of responders.
Percentage of participants whose free thyroxine (free T4) levels decrease after 12 weeks of treatment (DAY85). A decrease is when free T4 level is below Upper limit of normal (ULN) ≤ 22.7 pmol/L)
Outcome measures
| Measure |
CFZ533 10 mg/kg
n=13 Participants
CFZ533 intravenously over approximately one hour
|
|---|---|
|
Percentage of Participants Whose Free Thyroxine (Free T4) Levels Decrease After 12 Week Treatment
|
30.8 percentage of participants
|
Adverse Events
CFZ533 10 mg/kg
Serious events: 1 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
CFZ533 10 mg/kg
n=15 participants at risk
CFZ533 intravenously over approximately one hour
|
|---|---|
|
Cardiac disorders
TACHYCARDIA
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
Other adverse events
| Measure |
CFZ533 10 mg/kg
n=15 participants at risk
CFZ533 intravenously over approximately one hour
|
|---|---|
|
Cardiac disorders
PALPITATIONS
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Ear and labyrinth disorders
VERTIGO
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Gastrointestinal disorders
CONSTIPATION
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Gastrointestinal disorders
DIARRHOEA
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Gastrointestinal disorders
DRY MOUTH
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Gastrointestinal disorders
NAUSEA
|
13.3%
2/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Gastrointestinal disorders
VOMITING
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
General disorders
FATIGUE
|
13.3%
2/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Infections and infestations
CYSTITIS
|
20.0%
3/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Infections and infestations
SKIN INFECTION
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
13.3%
2/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Infections and infestations
VIRAL UPPER RESPIRATORY TRACT INFECTION
|
13.3%
2/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Investigations
BLOOD CREATINE PHOSPHOKINASE INCREASED
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Investigations
CARDIAC MURMUR
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Investigations
ELECTROCARDIOGRAM QT PROLONGED
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Investigations
HEPATIC ENZYME INCREASED
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Musculoskeletal and connective tissue disorders
MUSCULOSKELETAL PAIN
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Musculoskeletal and connective tissue disorders
OSTEOPENIA
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Nervous system disorders
HEADACHE
|
13.3%
2/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Psychiatric disorders
INSOMNIA
|
13.3%
2/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Psychiatric disorders
SLEEP DISORDER
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Skin and subcutaneous tissue disorders
ALOPECIA
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Skin and subcutaneous tissue disorders
HYPERHIDROSIS
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Skin and subcutaneous tissue disorders
PRURITUS
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
|
Skin and subcutaneous tissue disorders
URTICARIA
|
6.7%
1/15 • Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
- Publication restrictions are in place
Restriction type: OTHER