Clinical trial NCT01294774: Safety and Efficacy of KRP203 in Subacute Cutaneous Lupus Erythematosus
Official title: A Multi-center, Double-blind, Placebo-controlled, Proof-of-concept Study to Evaluate the Efficacy and Tolerability of KRP203 in Patients With Active Subacute Cutaneous Lupus Erythematosus
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Subacute Cutaneous Lupus Erythematosus
Keywords: Lupus erythematosus
skin lupus
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2011-02-11
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will assess the safety and efficacy of KRP203 in clinically active subacute cutaneous lupus erythematosus patients, who have demonstrated inadequate response to standard treatment, such as antimalarials.


Eligibility criteria: Inclusion Criteria:

* Male and female patients,18 to 65 years of age inclusive, who have been defined as having SCLE based on the typical clinical picture and the characteristic histopathological features as described by Sontheimer et al. at least three months before study entry (screening)

Exclusion Criteria:

1. Patients with preexisting nephritis, central nervous or pulmonary involvement or any major internal organ damage, either related or unrelated to lupus, which are deemed by the Investigator to be clinically significant. Patients having signs or symptoms of other autoimmune diseases such as systemic lupus erythematosus or Sjogren's syndrome are allowed to enter the study at the Investigator's discretion.
2. Patients who have been treated with:

   * immunoglobulins and/or monoclonal antibodies within 6 months prior to randomization.
   * rituximab, cyclophosphamide, or other immunosuppressive treatments with effects potentially lasting over 6 months, within 12 months prior to randomization.
   * a medium or high dose (≥ 1 mg prednisone or equivalent per body weight kg) corticosteroid therapy in the last 8 weeks prior to randomization.
   * antimalarial agents (hydroxychloroquine, chloroquine or quinacrine) in the last 6 weeks prior to randomization.
   * biologic therapies, such as etanercept, within the last 4 weeks prior to randomization.
   * any other immunosuppressive or immunomodulatory therapy such as methotrexate, azathioprine, cyclosporin A or mycophenolate, thalidomide, retinoids or dapsone in the last 4 weeks prior to randomization.
   * total lymphoid irradiation or bone marrow transplantation.
3. Pregnant, planning to get pregnant, and/or lactating females or males planning to father a child within time period of the study or subsequent exclusionary period.

Other protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: Efficacy of KRP203 in reduction of severity of symptoms, as measured using the activity score of the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI)
timeFrame: 12 weeks
Secondary outcomes:
measure: Safety and tolerability of oral KRP203 in patients with subacute cutaneous lupus erythematosus
timeFrame: 12 weeks
measure: Steady-state blood concentrations of KRP203 and KRP203-Phosphate (KRP203-P) in SCLE patients
timeFrame: 12 weeks
measure: Changes in the activity of SCLE using visual analogue scales for global skin health as assessed by the physician and the patient
timeFrame: 12 weeks
measure: Measure the systemic features of SCLE using the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Bochum
country: Germany
facility: Novartis Investigative Site
city: Bonn
country: Germany
facility: Novartis Investigative Site
city: Frankfurt am Main
country: Germany
facility: Novartis Investigative Site
city: Tuebingen
country: Germany
facility: Novartis Investigative Site
city: Athens
country: Greece
facility: Novartis Investigative Site
state: GR
city: Thessaloniki
country: Greece
facility: Novartis Investigative Site
state: GR
city: Genova
country: Italy
facility: Novartis Investigative Site
state: GE
city: Siena
country: Italy
facility: Novartis Investigative Site
state: SI
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: