Clinical trial NCT02472795: Clinical Study to Investigate the Biological Activity, Safety, Tolerability, and Pharmacokinetics of ACT-334441 in Subjects With Systemic Lupus Erythematosus
Official title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Dose-response Study to Investigate the Biological Activity, Safety, Tolerability, and Pharmacokinetics of ACT-334441 in Subjects With Systemic Lupus Erythematosus
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Systemic Lupus Erythematosus
Keywords: Systemic lupus erythematosus
Lead sponsor: Idorsia Pharmaceuticals Ltd.
Sponsor class: INDUSTRY
First posted: 2015-06-16
Enrollment: 105
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: International trial to evaluate the biological activity and safety of cenerimod (ACT-334441) in systemic lupus erythematosus (SLE) patients.

Detailed description: This multicentre, double-blind, placebo-controlled study will have a staggered approach (Part A and B).

In part A, eligible patients will be randomly assigned (1:1:1:1) to once daily oral administration of cenerimod (0.5, 1, 2 mg) or placebo. After all patients have completed 4 weeks of treatment during part A, an Independent Data Monitoring Committee will review non-blinded data in an interim analysis to evaluate the safety profile of cenerimod and recommend whether the study could proceed to part B.

In part B, additional patients will be randomized (3:1) to once daily oral administration of cenerimod 4 mg or placebo.

All participants will receive study medication for 12 weeks.

Eligibility criteria: Inclusion Criteria:

* Male and female participants aged 18 to 65 years with established SLE. Participants must have active SLE, Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) score of at least 2 points for musculoskeletal or mucocutaneous manifestations and history or presence at screening of positive anti-nuclear antibodies (ANA) or anti-double-stranded DNA (anti-dsDNA) antibodies.
* Enrolled participants must be treated with background SLE medications.

Exclusion Criteria:

* Participants with significant medical conditions or therapies for such conditions (e.g., cardiovascular, pulmonary, immunological, hepatic, ophthalmological, infection and infection risks, history or presence of malignancy, history or presence of bone marrow or solid organ transplantation) or lactating or pregnant women.
* Participants with severe SLE disease or with clinically relevant medical or surgical conditions that, in the opinion of the investigator, would put the subject at risk by participating in the study.

Primary outcomes:
measure: Change in Total Lymphocyte Count From Baseline to End-of-treatment (EOT)
timeFrame: Baseline to end-of-treatment (EOT) (up to 12 weeks)
measure: Change in Total Lymphocyte Count From Baseline to Each Post-baseline Assessment
timeFrame: Baseline, Week 2, Week 4, Week 8, Week 12, end-of-treatment Visit (up to 12 weeks)
Secondary outcomes:
Other outcomes:
Locations:
city: Anniston
country: United States
facility: Investigator Site
state: Alabama
city: Clearwater
country: United States
facility: Investigator Site
state: Florida
city: Minsk
country: Belarus
facility: Investigator Site
city: Minsk
country: Belarus
facility: Investigator Site
city: Vitebsk
country: Belarus
facility: Investigator Site
city: Plovdiv
country: Bulgaria
facility: Investigator Site
city: Plovdiv
country: Bulgaria
facility: Investigator Site
city: Sofia
country: Bulgaria
facility: Investigator Site
city: Tbilisi
country: Georgia
facility: Investigator Site
city: Kemerovo
country: Russian Federation
facility: Investigator Site
city: Kursk
country: Russian Federation
facility: Investigator Site
city: Omsk
country: Russian Federation
facility: Investigator Site
city: Orenburg
country: Russian Federation
facility: Investigator Site
city: Smolensk
country: Russian Federation
facility: Investigator Site
city: Vladimir
country: Russian Federation
facility: Investigator Site
city: Vinnytsya
country: Ukraine
facility: Investigator Site
city: Vinnytsya
country: Ukraine
facility: Investigator Site
city: Zaporizhia
country: Ukraine
facility: Investigator Site
Study officials:
affiliation: Idorsia Pharmaceuticals Ltd.
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Hermann V, Batalov A, Smakotina S, Juif PE, Cornelisse P. First use of cenerimod, a selective S1P1 receptor modulator, for the treatment of SLE: a double-blind, randomised, placebo-controlled, proof-of-concept study. Lupus Sci Med. 2019 Nov 9;6(1):e000354. doi: 10.1136/lupus-2019-000354. eCollection 2019.