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

Primary Purpose

Systemic Lupus Erythematosus

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Matching placebo
Cenerimod
Sponsored by
Idorsia Pharmaceuticals Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Systemic Lupus Erythematosus focused on measuring Systemic lupus erythematosus

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Investigator Site
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Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Experimental

Experimental

Experimental

Placebo Comparator

Arm Label

Cenerimod 0.5 mg (Part A)

Cenerimod 1 mg (Part A)

Cenerimod 2 mg (Part A)

Cenerimod 4 mg (Part B)

Matching placebo (Part A and B)

Arm Description

Participants will receive cenerimod 0.5 mg capsules orally once daily for 12 weeks.

Participants will receive cenerimod 1 mg capsules orally once daily for 12 weeks.

Participants will receive cenerimod 2 mg capsules orally once daily for 12 weeks.

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.

Capsules of matching placebo taken orally once daily for 12 weeks.

Outcomes

Primary Outcome Measures

Change in Total Lymphocyte Count From Baseline to End-of-treatment (EOT)
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.
Change in Total Lymphocyte Count From Baseline to Each Post-baseline Assessment
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.

Secondary Outcome Measures

Full Information

First Posted
May 14, 2015
Last Updated
February 12, 2020
Sponsor
Idorsia Pharmaceuticals Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT02472795
Brief Title
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

2. Study Status

Record Verification Date
February 2020
Overall Recruitment Status
Completed
Study Start Date
June 1, 2015 (Actual)
Primary Completion Date
January 23, 2017 (Actual)
Study Completion Date
February 28, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Idorsia Pharmaceuticals Ltd.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Systemic Lupus Erythematosus
Keywords
Systemic lupus erythematosus

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
105 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cenerimod 0.5 mg (Part A)
Arm Type
Experimental
Arm Description
Participants will receive cenerimod 0.5 mg capsules orally once daily for 12 weeks.
Arm Title
Cenerimod 1 mg (Part A)
Arm Type
Experimental
Arm Description
Participants will receive cenerimod 1 mg capsules orally once daily for 12 weeks.
Arm Title
Cenerimod 2 mg (Part A)
Arm Type
Experimental
Arm Description
Participants will receive cenerimod 2 mg capsules orally once daily for 12 weeks.
Arm Title
Cenerimod 4 mg (Part B)
Arm Type
Experimental
Arm Description
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.
Arm Title
Matching placebo (Part A and B)
Arm Type
Placebo Comparator
Arm Description
Capsules of matching placebo taken orally once daily for 12 weeks.
Intervention Type
Drug
Intervention Name(s)
Matching placebo
Other Intervention Name(s)
Placebo
Intervention Description
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).
Intervention Type
Drug
Intervention Name(s)
Cenerimod
Other Intervention Name(s)
ACT-334441
Intervention Description
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).
Primary Outcome Measure Information:
Title
Change in Total Lymphocyte Count From Baseline to End-of-treatment (EOT)
Description
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.
Time Frame
Baseline to end-of-treatment (EOT) (up to 12 weeks)
Title
Change in Total Lymphocyte Count From Baseline to Each Post-baseline Assessment
Description
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.
Time Frame
Baseline, Week 2, Week 4, Week 8, Week 12, end-of-treatment Visit (up to 12 weeks)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Idorsia Pharmaceuticals Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
Investigator Site
City
Anniston
State/Province
Alabama
ZIP/Postal Code
36207
Country
United States
Facility Name
Investigator Site
City
Clearwater
State/Province
Florida
ZIP/Postal Code
33765
Country
United States
Facility Name
Investigator Site
City
Minsk
ZIP/Postal Code
220116
Country
Belarus
Facility Name
Investigator Site
City
Minsk
ZIP/Postal Code
223041
Country
Belarus
Facility Name
Investigator Site
City
Vitebsk
ZIP/Postal Code
210037
Country
Belarus
Facility Name
Investigator Site
City
Plovdiv
ZIP/Postal Code
4000
Country
Bulgaria
Facility Name
Investigator Site
City
Plovdiv
ZIP/Postal Code
4002
Country
Bulgaria
Facility Name
Investigator Site
City
Sofia
ZIP/Postal Code
1612
Country
Bulgaria
Facility Name
Investigator Site
City
Tbilisi
ZIP/Postal Code
0186
Country
Georgia
Facility Name
Investigator Site
City
Kemerovo
ZIP/Postal Code
650066
Country
Russian Federation
Facility Name
Investigator Site
City
Kursk
ZIP/Postal Code
305007
Country
Russian Federation
Facility Name
Investigator Site
City
Omsk
ZIP/Postal Code
644111
Country
Russian Federation
Facility Name
Investigator Site
City
Orenburg
ZIP/Postal Code
460018
Country
Russian Federation
Facility Name
Investigator Site
City
Smolensk
ZIP/Postal Code
214025
Country
Russian Federation
Facility Name
Investigator Site
City
Vladimir
ZIP/Postal Code
600023
Country
Russian Federation
Facility Name
Investigator Site
City
Vinnytsya
ZIP/Postal Code
21018
Country
Ukraine
Facility Name
Investigator Site
City
Vinnytsya
ZIP/Postal Code
21029
Country
Ukraine
Facility Name
Investigator Site
City
Zaporizhia
ZIP/Postal Code
69600
Country
Ukraine

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
31798918
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. Erratum In: Lupus Sci Med. 2020 May;7(1):
Results Reference
result
Links:
URL
https://lupus.bmj.com/content/lupusscimed/suppl/2019/11/12/6.1.e000354.DC4/lupus-2019-000354_lay_summary.pdf
Description
Lay summary on results reference by Hermann V et al. First use of cenerimod, a selective S1P(1) 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.

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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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