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

• Sponsor: Rigel Pharmaceuticals (Industry)
• Overall Status: Completed
• CT.gov ID: NCT01597050
• Collaborator: (none)
• Enrollment: 54
• Locations: 15
• Arms: 2
• Duration (Months): 13
• Patients Per Site: 3.6
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

The purpose of this study is to determine the safety, efficacy and tolerability of topical R333 ointment in Discoid Lupus Erythematosus (DLE) and Systemic Lupus Erythematosus (SLE) patients with active discoid lesions.

Condition or Disease	Intervention/Treatment	Phase
Lupus Erythematosus, Discoid; Lupus Erythematosus, Systemic	Drug: R932333; Drug: Placebo	Phase 2

Detailed Description

This is a Phase 2, multi-center, randomized, double-blind, placebo-controlled study to evaluate the preliminary efficacy, safety, tolerability, and pharmacokinetics of topical R333 ointment formulated at 6% (60 mg/g) in DLE and SLE patients with active discoid lesions.

Study Design

• Study Type: Interventional
• Actual Enrollment: 54 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of R333 6% Ointment Administered Topically to Discoid Lupus Erythematosus (DLE) and Systemic Lupus Erythematosus (SLE) Patients With Active Cutaneous Discoid Lesions
• Study Start Date: Aug 1, 2012
• Actual Primary Completion Date: Sep 1, 2013
• Actual Study Completion Date: Sep 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Drug: R932333 R333 6% (60 mg/g), bid	Drug: R932333; R393233 6% (60 mg/g), bid; Other Names: R333
Placebo Comparator: Placebo Placebo, bid	Drug: Placebo; Placebo, bid

Outcome Measures

Primary Outcome Measures

1. Decrease in the Total Combined Erythema and Scaling Score (Minimum of 0 and Maximum of 65) of All Treated Lesions. [Up to Week 4]

   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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 75 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Diagnosis of SLE or DLE (DLE confirmed histologically prior to randomization).

• At least 2 active discoid lesions secondary to SLE or DLE prior to study entry, each with a minimum Erythema Rating Score of ≥ 2. At least 1 of the active discoid lesions must have been present (by history) for ≥ 3 weeks prior to screening.

• Patients who are taking azathioprine, hydroxychloroquine, chloroquine, quinacrine, methotrexate, and/ or oral glucocorticoids, must be receiving a stable daily dose ≥ 4 weeks prior to randomization and must remain on the same dose throughout the study. Azathioprine, hydroxychloroquine, chloroquine, quinacrine, or methotrexate must be initiated ≥ 8 weeks prior to randomization.

Exclusion Criteria:

• Congenital or acquired immunodeficiency including: HIV infection, agammaglobulinemias, T cell deficiencies or HTLV-1 infection at any time prior to the study.

• Lymphoproliferative disease or previous total lymphoid irradiation.

• Uncontrolled or poorly controlled hypertension.

• History of psoriasis, eczema, or relevant atopy.

• Exposure to excessive or chronic UV radiation (e.g., tanning beds, sunbathing, solarium, phototherapy) within 2 weeks prior to randomization or during the study period.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Wallace Rheumatic Study Center	Los Angeles	California	United States	90027
2	Stanford Dermatology	Redwood City	California	United States	94063
3	Memorial Medical Group Clinical Research Institute	South Bend	Indiana	United States	46601
4	North Shore Long Island Health System	Lake Success	New York	United States	11042
5	Columbia University Medical Center	New York	New York	United States	10032
6	Wake Forest University Health Sciences	Winston-Salem	North Carolina	United States	27104
7	Oklahoma Medical Research Foundation	Oklahoma City	Oklahoma	United States	73104
8	University of Pennsylvania-Dermatology Research Office	Philadelphia	Pennsylvania	United States	19104
9	Metroplex Clinical Research Center	Dallas	Texas	United States	75231
10	University of Texas Medical School at Houston	Houston	Texas	United States	77030
11	University of Utah Department of Dermatology	Salt Lake City	Utah	United States	84132
12	Virginia Clinical Research, Inc	Norfolk	Virginia	United States	23507
13	University of British Columbia, Vancouver Dermatology Clinical Trials Unit	Vancouver	British Columbia	Canada	V5Z 4E8
14	Dermadvances Research	Winnipeg	Manitoba	Canada	R3C 1R4
15	Lynderm Research, Inc	Markham	Ontario	Canada	L3P 1A8

Sponsors and Collaborators

• Rigel Pharmaceuticals

Investigators

• Study Director: Daniel Magilavy, MD, Rigel Pharmaceuticals, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Rigel Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT01597050
• Other Study ID Numbers: C-932333-002
• First Posted: May 11, 2012
• Last Update Posted: Jul 14, 2016
• Last Verified: Jun 1, 2016

• Keywords provided by Rigel Pharmaceuticals: Discoid Lupus Erythematosus; Systemic Lupus Erythematosus
• Additional relevant MeSH terms: Lupus Erythematosus, Systemic; Lupus Erythematosus, Discoid

Study Results

Participant Flow

Arm/Group Title	Drug: R932333	Placebo
Arm/Group Description	R333 6% (60 mg/g), bid R932333: R393233 6% (60 mg/g), bid	Placebo, bid Placebo: Placebo, bid
Period Title: Overall Study
STARTED	36	18
COMPLETED	35	18
NOT COMPLETED	1	0

Baseline Characteristics

Arm/Group Title	Drug: R932333	Placebo	Total
Arm/Group Description	R333 6% (60 mg/g), bid R932333: R393233 6% (60 mg/g), bid	Placebo, bid Placebo: Placebo, bid	Total of all reporting groups
Overall Participants	36	18	54
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	46.1 (11.3)	48.3 (12.57)	46.8 (11.69)
Sex: Female, Male (Count of Participants)
Female	28 77.8%	16 88.9%	44 81.5%
Male	8 22.2%	2 11.1%	10 18.5%
Region of Enrollment (participants) [Number]
Canada	7 19.4%	2 11.1%	9 16.7%
United States	29 80.6%	16 88.9%	45 83.3%

Outcome Measures

1. Primary Outcome

Title	Decrease in the Total Combined Erythema and Scaling Score (Minimum of 0 and Maximum of 65) of All Treated Lesions.
Description	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.
Time Frame	Up to Week 4

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Drug: R932333	Placebo
Arm/Group Description	R333 6% (60 mg/g), bid R932333: R393233 6% (60 mg/g), bid	Placebo, bid Placebo: Placebo, bid
Measure Participants	36	18
Number [percentage of subjects]	22.2	27.8

Adverse Events

Time Frame	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.
Arm/Group Title	Drug: R932333		Placebo
Arm/Group Description	R333 6% (60 mg/g), bid R932333: R393233 6% (60 mg/g), bid		Placebo, bid Placebo: Placebo, bid
All Cause Mortality
	Drug: R932333		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Drug: R932333		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/36 (0%)		0/18 (0%)
Other (Not Including Serious) Adverse Events
	Drug: R932333		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	16/36 (44.4%)		10/18 (55.6%)
Blood and lymphatic system disorders
Leukopenia	0/36 (0%)	0	1/18 (5.6%)	1
Lymphadenopathy	0/36 (0%)	0	1/18 (5.6%)	1
Mean cell volume increased	1/36 (2.8%)	1	0/18 (0%)	0
Neutrophil count increased	1/36 (2.8%)	1	0/18 (0%)	0
White blood cell count increased	1/36 (2.8%)	1	0/18 (0%)	0
Eye disorders
Eyelid margin crusting	1/36 (2.8%)	1	0/18 (0%)	0
Lacrimation increased	1/36 (2.8%)	1	0/18 (0%)	0
Gastrointestinal disorders
Vomiting	4/36 (11.1%)	4	0/18 (0%)	0
Nausea	2/36 (5.6%)	2	0/18 (0%)	0
Abdominal distension	1/36 (2.8%)	1	0/18 (0%)	0
Diarrhoea	1/36 (2.8%)	1	0/18 (0%)	0
Gastrooesophageal reflux disease	1/36 (2.8%)	1	0/18 (0%)	0
Stomatitis	1/36 (2.8%)	1	0/18 (0%)	0
General disorders
Application site pain	1/36 (2.8%)	1	1/18 (5.6%)	1
Application site pruritus	2/36 (5.6%)	2	0/18 (0%)	0
Chest pain	1/36 (2.8%)	1	0/18 (0%)	0
Fatigue	0/36 (0%)	0	1/18 (5.6%)	1
Pain	1/36 (2.8%)	1	0/18 (0%)	0
Pyrexia	0/36 (0%)	0	1/18 (5.6%)	1
Infections and infestations
Upper Respiratory Track Infection	3/36 (8.3%)	3	3/18 (16.7%)	3
Sinusitis	0/36 (0%)	0	2/18 (11.1%)	2
Laryngitis	0/36 (0%)	0	1/18 (5.6%)	1
Investigations
Blood bilirubin increased	1/36 (2.8%)	1	0/18 (0%)	0
Musculoskeletal and connective tissue disorders
Musculoskeletal pain	0/36 (0%)	0	1/18 (5.6%)	1
Pain in extremity	1/36 (2.8%)	1	0/18 (0%)	0
Nervous system disorders
Headache	3/36 (8.3%)	3	1/18 (5.6%)	1
Migraine	0/36 (0%)	0	1/18 (5.6%)	1
Psychiatric disorders
Insomnia	1/36 (2.8%)	1	0/18 (0%)	0
Respiratory, thoracic and mediastinal disorders
Sinus congestion	3/36 (8.3%)	3	0/18 (0%)	0
Oropharyngeal pain	2/36 (5.6%)	2	0/18 (0%)	0
Respiratory tract congestion	1/36 (2.8%)	1	0/18 (0%)	0
Skin and subcutaneous tissue disorders
Pruritus	1/36 (2.8%)	1	2/18 (11.1%)	2
Skin lesion	0/36 (0%)	0	2/18 (11.1%)	2
Erythema	1/36 (2.8%)	1	0/18 (0%)	0
Rash	0/36 (0%)	0	1/18 (5.6%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.