Trial Outcomes & Findings for A Study With TEPEZZA in Patients With Diffuse Cutaneous Systemic Sclerosis (dcSSc) (NCT NCT04478994)
NCT ID: NCT04478994
Last Updated: 2024-06-28
Results Overview
An adverse event (AE) is any untoward medical occurrence, which does not necessarily have a causal relationship with treatment. TEAEs are those that occurred after the first dose of trial drug.
Recruitment status
TERMINATED
Study phase
PHASE1
Target enrollment
3 participants
Primary outcome timeframe
From first dose of trial drug up to Week 24
Results posted on
2024-06-28
Participant Flow
Participant milestones
| Measure |
TEPEZZA 20 mg/kg
Participants receive 8 infusions of TEPEZZA every 3 weeks (q3W) for a total of 21 weeks: TEPEZZA 10 mg/kg administered on Day 1 and TEPEZZA 20 mg/kg administered q3W for the remaining 7 infusions.
|
Placebo
Participants receive 8 infusions of placebo q3W for a total of 21 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
2
|
|
Overall Study
COMPLETED
|
0
|
1
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
TEPEZZA 20 mg/kg
Participants receive 8 infusions of TEPEZZA every 3 weeks (q3W) for a total of 21 weeks: TEPEZZA 10 mg/kg administered on Day 1 and TEPEZZA 20 mg/kg administered q3W for the remaining 7 infusions.
|
Placebo
Participants receive 8 infusions of placebo q3W for a total of 21 weeks.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
Baseline Characteristics
A Study With TEPEZZA in Patients With Diffuse Cutaneous Systemic Sclerosis (dcSSc)
Baseline characteristics by cohort
| Measure |
TEPEZZA 20 mg/kg
n=1 Participants
Participants receive 8 infusions of TEPEZZA every 3 weeks (q3W) for a total of 21 weeks: TEPEZZA 10 mg/kg administered on Day 1 and TEPEZZA 20 mg/kg administered q3W for the remaining 7 infusions.
|
Placebo
n=2 Participants
Participants receive 8 infusions of placebo q3W for a total of 21 weeks.
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From first dose of trial drug up to Week 24Population: All randomized participants
An adverse event (AE) is any untoward medical occurrence, which does not necessarily have a causal relationship with treatment. TEAEs are those that occurred after the first dose of trial drug.
Outcome measures
| Measure |
TEPEZZA 20 mg/kg
n=1 Participants
Participants receive 8 infusions of TEPEZZA every 3 weeks (q3W) for a total of 21 weeks: TEPEZZA 10 mg/kg administered on Day 1 and TEPEZZA 20 mg/kg administered q3W for the remaining 7 infusions.
|
Placebo
n=2 Participants
Participants receive 8 infusions of placebo q3W for a total of 21 weeks.
|
|---|---|---|
|
Number of Participants Experiencing a Treatment Emergent Adverse Event (TEAE) Through Week 24
|
0 Participants
|
2 Participants
|
Adverse Events
TEPEZZA 20 mg/kg
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Placebo
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
TEPEZZA 20 mg/kg
n=1 participants at risk
Participants receive 8 infusions of TEPEZZA every 3 weeks (q3W) for a total of 21 weeks: TEPEZZA 10 mg/kg administered on Day 1 and TEPEZZA 20 mg/kg administered q3W for the remaining 7 infusions.
|
Placebo
n=2 participants at risk
Participants receive 8 infusions of placebo q3W for a total of 21 weeks.
|
|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Chronic lymphocytic leukaemia
|
0.00%
0/1 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
50.0%
1/2 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
Other adverse events
| Measure |
TEPEZZA 20 mg/kg
n=1 participants at risk
Participants receive 8 infusions of TEPEZZA every 3 weeks (q3W) for a total of 21 weeks: TEPEZZA 10 mg/kg administered on Day 1 and TEPEZZA 20 mg/kg administered q3W for the remaining 7 infusions.
|
Placebo
n=2 participants at risk
Participants receive 8 infusions of placebo q3W for a total of 21 weeks.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/1 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
50.0%
1/2 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/1 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
50.0%
1/2 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/1 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
50.0%
1/2 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
|
General disorders
Fatigue
|
0.00%
0/1 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
50.0%
1/2 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/1 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
50.0%
1/2 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
|
Investigations
Weight decreased
|
0.00%
0/1 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
50.0%
1/2 • From enrollment (all-cause mortality) or first dose of trial drug on Day 1 (adverse events) until 3 weeks (21 days) after last dose of trial drug, up to Week 48.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Horizon requests that any investigator/institution that plans on presenting/publishing results provide written notification of their request 60 days prior to their presentation/publication. Horizon requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if Horizon needs to secure patent or proprietary protection.
- Publication restrictions are in place
Restriction type: OTHER