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Clinical Study to Investigate the Biological Activity, Safety, Tolerability, and Pharmacokinetics of ACT-334441 in Subjects With Systemic Lupus Erythematosus

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ClinicalTrials.gov Identifier: NCT02472795
Recruitment Status : Completed
First Posted : June 16, 2015
Results First Posted : February 26, 2020
Last Update Posted : February 26, 2020
Sponsor:
Information provided by (Responsible Party):
Idorsia Pharmaceuticals Ltd.

Brief Summary:
International trial to evaluate the biological activity and safety of cenerimod (ACT-334441) in systemic lupus erythematosus (SLE) patients.

Condition or disease Intervention/treatment Phase
Systemic Lupus Erythematosus Drug: Matching placebo Drug: Cenerimod Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 105 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : June 1, 2015
Actual Primary Completion Date : January 23, 2017
Actual Study Completion Date : February 28, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Lupus

Arm Intervention/treatment
Experimental: Cenerimod 0.5 mg (Part A)
Participants will receive cenerimod 0.5 mg capsules orally once daily for 12 weeks.
Drug: Cenerimod
One capsule of cenerimod to be taken once daily, irrespective of food intake. The capsule is to be swallowed whole. The capsule should be taken each day at approximately the same time (preferably each morning).
Other Name: ACT-334441

Experimental: Cenerimod 1 mg (Part A)
Participants will receive cenerimod 1 mg capsules orally once daily for 12 weeks.
Drug: Cenerimod
One capsule of cenerimod to be taken once daily, irrespective of food intake. The capsule is to be swallowed whole. The capsule should be taken each day at approximately the same time (preferably each morning).
Other Name: ACT-334441

Experimental: Cenerimod 2 mg (Part A)
Participants will receive cenerimod 2 mg capsules orally once daily for 12 weeks.
Drug: Cenerimod
One capsule of cenerimod to be taken once daily, irrespective of food intake. The capsule is to be swallowed whole. The capsule should be taken each day at approximately the same time (preferably each morning).
Other Name: ACT-334441

Experimental: Cenerimod 4 mg (Part B)
Participants will received cenerimod 4 mg capsules orally once daily for 12 weeks. This treatment arm will start after all patients in Part A have completed 4 weeks of placebo, 0.5 mg, 1 mg and 2 mg cenerimod treatment.
Drug: Cenerimod
One capsule of cenerimod to be taken once daily, irrespective of food intake. The capsule is to be swallowed whole. The capsule should be taken each day at approximately the same time (preferably each morning).
Other Name: ACT-334441

Placebo Comparator: Matching placebo (Part A and B)
Capsules of matching placebo taken orally once daily for 12 weeks.
Drug: Matching placebo
One capsule of cenerimod to be taken once daily, irrespective of food intake. The capsule is to be swallowed whole. The capsule should be taken each day at approximately the same time (preferably each morning).
Other Name: Placebo




Primary Outcome Measures :
  1. Change in Total Lymphocyte Count From Baseline to End-of-treatment (EOT) [ Time Frame: Baseline to end-of-treatment (EOT) (up to 12 weeks) ]

    The primary objective of the clinical study was to asses whether cenerimod could reduce the number of circulating lymphocytes in the bloodstream of people with systemic lupus erythematosus (SLE).

    The change was defined as: Total lymphocyte count at end-of-treatment (EOT) minus total lymphocyte count at baseline.

    A negative change over time indicates that the number of peripheral circulating lymphocytes has decreased.

    The reduction of the total lymphocyte count over a treatment period indicates a pharmacodynamic effect.

    The value at baseline was defined as the last non-missing value obtained from a sample taken prior to the first study treatment intake.

    End-of-treatment (EOT) was defined as the last post-baseline value with treatment for at least 21 days up to Week 12.


  2. Change in Total Lymphocyte Count From Baseline to Each Post-baseline Assessment [ Time Frame: Baseline, Week 2, Week 4, Week 8, Week 12, end-of-treatment Visit (up to 12 weeks) ]

    The primary objective of the clinical study was to assess whether cenerimod could reduce the number of circulating lymphocytes in the bloodstream of people with systemic lupus erythematosus (SLE).

    The change was defined as: Total lymphocyte count at visit minus total lymphocyte count at baseline.

    A negative change over time indicates that the number of peripheral circulating lymphocytes has decreased.

    The reduction of the total lymphocyte count over a treatment period indicates a pharmacodynamic effect.

    The value at baseline was defined as the last non-missing value obtained from a sample taken prior to the first study treatment intake.

    End-of-treatment (EOT) was defined as the last post-baseline value with treatment for at least 21 days up to Week 12.




Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02472795


Locations
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United States, Alabama
Investigator Site
Anniston, Alabama, United States, 36207
United States, Florida
Investigator Site
Clearwater, Florida, United States, 33765
Belarus
Investigator Site
Minsk, Belarus, 220116
Investigator Site
Minsk, Belarus, 223041
Investigator Site
Vitebsk, Belarus, 210037
Bulgaria
Investigator Site
Plovdiv, Bulgaria, 4000
Investigator Site
Plovdiv, Bulgaria, 4002
Investigator Site
Sofia, Bulgaria, 1612
Georgia
Investigator Site
Tbilisi, Georgia, 0186
Russian Federation
Investigator Site
Kemerovo, Russian Federation, 650066
Investigator Site
Kursk, Russian Federation, 305007
Investigator Site
Omsk, Russian Federation, 644111
Investigator Site
Orenburg, Russian Federation, 460018
Investigator Site
Smolensk, Russian Federation, 214025
Investigator Site
Vladimir, Russian Federation, 600023
Ukraine
Investigator Site
Vinnytsya, Ukraine, 21018
Investigator Site
Vinnytsya, Ukraine, 21029
Investigator Site
Zaporizhia, Ukraine, 69600
Sponsors and Collaborators
Idorsia Pharmaceuticals Ltd.
Investigators
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Study Director: Study Director Idorsia Pharmaceuticals Ltd.
  Study Documents (Full-Text)

Documents provided by Idorsia Pharmaceuticals Ltd.:
Study Protocol  [PDF] March 25, 2015
Statistical Analysis Plan  [PDF] April 20, 2017

Additional Information:
Publications of Results:
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Responsible Party: Idorsia Pharmaceuticals Ltd.
ClinicalTrials.gov Identifier: NCT02472795    
Other Study ID Numbers: AC-064A201
First Posted: June 16, 2015    Key Record Dates
Results First Posted: February 26, 2020
Last Update Posted: February 26, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Idorsia Pharmaceuticals Ltd.:
Systemic lupus erythematosus
Additional relevant MeSH terms:
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Lupus Erythematosus, Systemic
Connective Tissue Diseases
Autoimmune Diseases
Immune System Diseases