Trial Outcomes & Findings for Safety and Efficacy of Topical R333 in Patients With Discoid Lupus Erythematosus (DLE) and Systemic Lupus Erythematosus (SLE) Lesions (NCT NCT01597050)

Last Updated: 2016-07-14

Results Overview

Percentage of patients who achieved at least a 50% decrease from baseline in the total combined Erythema and Scaling score of all treated lesions at Week 4. A decrease is an improvement in measurement of erythema and scaling of the lesions.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

54 participants

Primary outcome timeframe

Up to Week 4

Results posted on

2016-07-14

Participant Flow

Participant milestones

Participant milestones
Measure	Drug: R932333 R333 6% (60 mg/g), bid R932333: R393233 6% (60 mg/g), bid	Placebo Placebo, bid Placebo: Placebo, bid
Overall Study STARTED	36	18
Overall Study COMPLETED	35	18
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Drug: R932333 R333 6% (60 mg/g), bid R932333: R393233 6% (60 mg/g), bid	Placebo Placebo, bid Placebo: Placebo, bid
Overall Study Withdrawal by Subject	1	0

Baseline Characteristics

Safety and Efficacy of Topical R333 in Patients With Discoid Lupus Erythematosus (DLE) and Systemic Lupus Erythematosus (SLE) Lesions

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Drug: R932333 n=36 Participants R333 6% (60 mg/g), bid R932333: R393233 6% (60 mg/g), bid	Placebo n=18 Participants Placebo, bid Placebo: Placebo, bid	Total n=54 Participants Total of all reporting groups
Age, Continuous	46.1 years STANDARD_DEVIATION 11.3 • n=5 Participants	48.3 years STANDARD_DEVIATION 12.57 • n=7 Participants	46.8 years STANDARD_DEVIATION 11.69 • n=5 Participants
Sex: Female, Male Female	28 Participants n=5 Participants	16 Participants n=7 Participants	44 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	2 Participants n=7 Participants	10 Participants n=5 Participants
Region of Enrollment Canada	7 participants n=5 Participants	2 participants n=7 Participants	9 participants n=5 Participants
Region of Enrollment United States	29 participants n=5 Participants	16 participants n=7 Participants	45 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to Week 4

Population: Per-protocol population all patients who had no major protocol deviations and were present at all scheduled visits up to and including Week 4.

Outcome measures

Outcome measures
Measure	Drug: R932333 n=36 Participants R333 6% (60 mg/g), bid R932333: R393233 6% (60 mg/g), bid	Placebo n=18 Participants Placebo, bid Placebo: Placebo, bid
Decrease in the Total Combined Erythema and Scaling Score (Minimum of 0 and Maximum of 65) of All Treated Lesions.	22.2 percentage of subjects	27.8 percentage of subjects

Adverse Events

Drug: R932333

Serious events: 0 serious events

Other events: 16 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Anne-Marie Duliege, MD

Rigel

Phone: 650-624-1100

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60

Measure	Drug: R932333 n=36 participants at risk R333 6% (60 mg/g), bid R932333: R393233 6% (60 mg/g), bid	Placebo n=18 participants at risk Placebo, bid Placebo: Placebo, bid
Infections and infestations Upper Respiratory Track Infection	8.3% 3/36 • Number of events 3 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	16.7% 3/18 • Number of events 3 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Gastrointestinal disorders Vomiting	11.1% 4/36 • Number of events 4 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Skin and subcutaneous tissue disorders Pruritus	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	11.1% 2/18 • Number of events 2 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
General disorders Application site pain	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Nervous system disorders Headache	8.3% 3/36 • Number of events 3 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Respiratory, thoracic and mediastinal disorders Sinus congestion	8.3% 3/36 • Number of events 3 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Blood and lymphatic system disorders Leukopenia	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Investigations Blood bilirubin increased	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Eye disorders Eyelid margin crusting	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Psychiatric disorders Insomnia	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Infections and infestations Sinusitis	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	11.1% 2/18 • Number of events 2 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Infections and infestations Laryngitis	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Gastrointestinal disorders Nausea	5.6% 2/36 • Number of events 2 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Gastrointestinal disorders Abdominal distension	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Gastrointestinal disorders Diarrhoea	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Gastrointestinal disorders Gastrooesophageal reflux disease	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Gastrointestinal disorders Stomatitis	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Skin and subcutaneous tissue disorders Skin lesion	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	11.1% 2/18 • Number of events 2 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Skin and subcutaneous tissue disorders Erythema	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Skin and subcutaneous tissue disorders Rash	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
General disorders Application site pruritus	5.6% 2/36 • Number of events 2 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
General disorders Chest pain	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
General disorders Fatigue	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
General disorders Pain	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
General disorders Pyrexia	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Nervous system disorders Migraine	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	5.6% 2/36 • Number of events 2 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Respiratory, thoracic and mediastinal disorders Respiratory tract congestion	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Blood and lymphatic system disorders Lymphadenopathy	0.00% 0/36 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	5.6% 1/18 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Blood and lymphatic system disorders Mean cell volume increased	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Blood and lymphatic system disorders Neutrophil count increased	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Blood and lymphatic system disorders White blood cell count increased	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Musculoskeletal and connective tissue disorders Pain in extremity	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.
Eye disorders Lacrimation increased	2.8% 1/36 • Number of events 1 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.	0.00% 0/18 • The AE reporting period begins with the first dose of double blind study drug and ends with the final study (follow-up) visit at week 6.