Study to assess the safety and efficacy of KRP203 in active subacute cutaneous lupus erythematosus patients, who have shown poor response to standard treatment.

• Register: EUCTR
• Last refreshed on: 15 April 2013
• Main ID: EUCTR2010-019689-10-GR
• Date of registration: 06/07/2011
• Prospective Registration: Yes
• Primary sponsor: Novartis Services AG
• Public title: Study to assess the safety and efficacy of KRP203 in active subacute cutaneous lupus erythematosus patients, who have shown poor response to standard treatment.
• Scientific 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 - A2202
• Date of first enrolment: 09/08/2011
• Target sample size: 24
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-019689-10
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 1

Countries of recruitment

Germany, Greece, Italy

Key inclusion & exclusion criteria

Inclusion criteria:
- Male and female (non-pregnant, non-lactating female)patients between 18-65 years of age. Having SCLE as described by Sontheimer et al (see Section 6.2) for at least 3 months before screening.

- Moderate to severe active skin disease at baseline by having an activity score of CLASI ³ 6, has to be demontrated, with at least 2 points in at least 3 different anatomical locations for erythema or scale/hypertrophy.

Other protocol-defined inclusion criteria may apply

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 24
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- Patients with preexisting nephritis, central nervous or pulmonary involvement or any major internal organ damage, either related or unrelated to lupus. Patients having signs or symptoms of other autoimmune diseases such as systemic lupus erythematosus or Sjogren`s syndrome are allowed to enter the study
- 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 exclusion criteria may apply.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Subacute cutaneous lupus erythematosus, in patients who do not respond to first line therapies (i.e. antimalrials)
MedDRA version: 14.0 Level: HLT Classification code 10025135 Term: Lupus erythematosus (incl subtypes) System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Intervention(s)

Product Name: KRP203
Product Code: KRP203
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Not established
CAS Number: 509088-69-1
Current Sponsor code: KRP203
Concentration unit: mg/g milligram(s)/gram
Concentration type: range
Concentration number: 0.1-1
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: Assess preliminary clinical efficacy of oral KRP203 in treatment refractory patients with subacute cutaneous lupus erythematosus (SCLE) after 12 weeks of treatment with KPR203 versus placebo.

Primary end point(s): Mean difference from baseline in the activity score of CLASI (from now on referred to only as CLASI).

Secondary Objective: • Assess safety and tolerability of oral KRP203 in patients with SCLE
• Assess steady-state blood concentrations of KRP203 and KRP203-Phosphate (KRP203-P) in SCLE patients
• Assess changes in the activity of SCLE using visual analogue scales for global skin health as assessed by the physician and the patient
• Assess the systemic features of SCLE using the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)

Timepoint(s) of evaluation of this end point: For the purposes of assessing the sample size, efficacy would be demonstrated if there would be at least 70% confidence that the true improvement induced by KRP203 in mean CLASI at 12 weeks is at least 5 points and at least 70% confidence that KRP203 is superior to placebo in terms of the primary endpoint.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: End of study

Secondary end point(s): - Safety and tolerability of KRP203A as assessed by standard safety evaluations and the use of SLEDAI
- Steady state PK blood concentrations of KRP203 and KRP203-p assessed by LC-MS/MS method wit a LLQ of 0.05 ng/mL
- Assess changes in the activity of SCLE using visual analogue scales for global skin health as assessed by the physician and the patient

Secondary ID(s)

2010-019689-10-DE

CKRP203A2202

Source(s) of Monetary Support

Novartis Pharma AG