Trial Outcomes & Findings for Nelfinavir in Systemic Lupus Erythematosus (NCT NCT02066311)
NCT ID: NCT02066311
Last Updated: 2020-02-24
Results Overview
Change in serum anti-dsDNA titer from baseline to Day 56; a decrease in titer ≥ 35% was considered a positive response
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
baseline to Day 56
Results posted on
2020-02-24
Participant Flow
Participant milestones
| Measure |
Open-label Study With the Simon Two-Stage Trial Design
All subjects will be dosed with nelfinavir 750 mg orally three times daily. This dose may be decreased to 750 mg twice daily if not tolerated. For both stages of the trial the dosing period is 8 weeks followed by a 4 week observation period.
A maximum of 13 subjects will be enrolled in Stage 1. From the 13 subjects enrolled in Stage 1; if 3 or fewer subjects achieve a Response (a ≥35% reduction in serum anti-dsDNA antibody titer), the trial will be terminated. If 4 or more subjects achieve a Response, the study will be expanded to enroll an additional maximum of 30 patients in Stage 2.
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Open-label Study With the Simon Two-Stage Trial Design
All subjects will be dosed with nelfinavir 750 mg orally three times daily. This dose may be decreased to 750 mg twice daily if not tolerated. For both stages of the trial the dosing period is 8 weeks followed by a 4 week observation period.
A maximum of 13 subjects will be enrolled in Stage 1. From the 13 subjects enrolled in Stage 1; if 3 or fewer subjects achieve a Response (a ≥35% reduction in serum anti-dsDNA antibody titer), the trial will be terminated. If 4 or more subjects achieve a Response, the study will be expanded to enroll an additional maximum of 30 patients in Stage 2.
|
|---|---|
|
Overall Study
Adverse Event
|
4
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Nelfinavir in Systemic Lupus Erythematosus
Baseline characteristics by cohort
| Measure |
Open-label Study With the Simon Two-Stage Trial Design
n=15 Participants
All subjects will be dosed with nelfinavir 750 mg orally three times daily. This dose may be decreased to 750 mg twice daily if not tolerated. For both stages of the trial the dosing period is 8 weeks followed by a 4 week observation period.
A maximum of 13 subjects will be enrolled in Stage 1. From the 13 subjects enrolled in Stage 1; if 3 or fewer subjects achieve a Response (a ≥35% reduction in serum anti-dsDNA antibody titer), the trial will be terminated. If 4 or more subjects achieve a Response, the study will be expanded to enroll an additional maximum of 30 patients in Stage 2.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
9 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=93 Participants
|
|
Serum anti-dsDNA antibody titer
|
313.8 IU/mL
STANDARD_DEVIATION 299.7 • n=93 Participants
|
PRIMARY outcome
Timeframe: baseline to Day 56Population: The number of participants whose anti-dsDNA antibody titer decreased by ≥ 35% from baseline to Day 56
Change in serum anti-dsDNA titer from baseline to Day 56; a decrease in titer ≥ 35% was considered a positive response
Outcome measures
| Measure |
Open-label Study With the Simon Two-Stage Trial Design
n=10 Participants
All subjects will be dosed with nelfinavir 750 mg orally three times daily. This dose may be decreased to 750 mg twice daily if not tolerated. For both stages of the trial the dosing period is 8 weeks followed by a 4 week observation period.
A maximum of 13 subjects will be enrolled in Stage 1. From the 13 subjects enrolled in Stage 1; if 3 or fewer subjects achieve a Response (a ≥35% reduction in serum anti-dsDNA antibody titer), the trial will be terminated. If 4 or more subjects achieve a Response, the study will be expanded to enroll an additional maximum of 30 patients in Stage 2.
|
|---|---|
|
Inhibition of Anti-dsDNA Binding
|
1 Participants
|
Adverse Events
Open-label Study With the Simon Two-Stage Trial Design
Serious events: 2 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Open-label Study With the Simon Two-Stage Trial Design
n=15 participants at risk
All subjects will be dosed with nelfinavir 750 mg orally three times daily. This dose may be decreased to 750 mg twice daily if not tolerated. For both stages of the trial the dosing period is 8 weeks followed by a 4 week observation period.
A maximum of 13 subjects will be enrolled in Stage 1. From the 13 subjects enrolled in Stage 1; if 3 or fewer subjects achieve a Response (a ≥35% reduction in serum anti-dsDNA antibody titer), the trial will be terminated. If 4 or more subjects achieve a Response, the study will be expanded to enroll an additional maximum of 30 patients in Stage 2.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Abscess
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Immune system disorders
Pyrexia
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
Other adverse events
| Measure |
Open-label Study With the Simon Two-Stage Trial Design
n=15 participants at risk
All subjects will be dosed with nelfinavir 750 mg orally three times daily. This dose may be decreased to 750 mg twice daily if not tolerated. For both stages of the trial the dosing period is 8 weeks followed by a 4 week observation period.
A maximum of 13 subjects will be enrolled in Stage 1. From the 13 subjects enrolled in Stage 1; if 3 or fewer subjects achieve a Response (a ≥35% reduction in serum anti-dsDNA antibody titer), the trial will be terminated. If 4 or more subjects achieve a Response, the study will be expanded to enroll an additional maximum of 30 patients in Stage 2.
|
|---|---|
|
Gastrointestinal disorders
Abdominal tenderness
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Infections and infestations
Abscess
|
13.3%
2/15 • Number of events 2 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Immune system disorders
Alopecia
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Blood and lymphatic system disorders
Anemia
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
20.0%
3/15 • Number of events 3 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Cardiac disorders
Chest Pain
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Gastrointestinal disorders
Dehydration
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Gastrointestinal disorders
Diarrhea
|
60.0%
9/15 • Number of events 9 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Blood and lymphatic system disorders
Eosinophilia
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Gastrointestinal disorders
Gastroenteritis
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Nervous system disorders
Headache
|
13.3%
2/15 • Number of events 2 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Hepatobiliary disorders
Hepatic Enzyme increased
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Cardiac disorders
Hypotension
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Musculoskeletal and connective tissue disorders
Joint Pain
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Immune system disorders
Lupus Flare
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Infections and infestations
Lymphadentiis
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasm
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Renal and urinary disorders
Nephrolithiasis
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Infections and infestations
Oropharyngeal pain
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Gastrointestinal disorders
Pancreatitis
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Immune system disorders
Pyrexia
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Skin and subcutaneous tissue disorders
Rash
|
26.7%
4/15 • Number of events 4 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Infections and infestations
Sinusitis
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Musculoskeletal and connective tissue disorders
Tendonitis
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Infections and infestations
Upper Respiratory tract infection
|
20.0%
3/15 • Number of events 3 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Infections and infestations
Viral Infection
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
|
Gastrointestinal disorders
vomiting
|
6.7%
1/15 • Number of events 1 • Day 1 to Day 84- 84 days
Adverse events were assessed systematically by regular investigator assessments and regular laboratory testing.
|
Additional Information
Dr. Meggan Mackay
The Feinstein Institute for Medical Research
Phone: 516-562-3838
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place