A Multi-Site, Open-Label Extension Trial of Oral RPC1063 in Relapsing Multiple Sclerosis

Study at a glance

From the registry record, last updated by the sponsor on 30 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

A Phase 3, Multi-Center, Randomized, Double-Blind, Double-Dummy, Active Controlled, Parallel Group Study To Evaluate The Efficacy And Safety Of RPC1063 Administered Orally To Relapsing Multiple Sclerosis Patients

ClinicalTrials.gov ID

NCT02576717

Acronym / sponsor ID

RPC01-3001

Conditions

Multiple Sclerosis

Keywords

MS, RMS, Multiple Sclerosis, Relapsing Multiple Sclerosis

Phase

Phase 3

Study type

Interventional

Recruitment status

Completed

Enrolment

2,494 participants (actual)

Lead sponsor

CelgeneIndustry (pharma, biotech, devices)

Study start

16 Oct 2015 (actual)

Primary completion

5 Jan 2023 (actual)

Study completion

5 Jan 2023 (actual)

First posted

15 Oct 2015 (estimated)

Results

Posted 30 Jan 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose

Treatment

Allocation

Not applicable

Intervention model

Single group

Masking

None (open label)

Number of arms

1

Who may qualify

Ages

18 to 55 years

Sex

All

Healthy volunteers

Not accepted

Age groups

Adult

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. Number of Participants Experiencing Adverse Events (AEs)

   Time frame: From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

   An AE is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporarily associated with the use of medicinal product, whether or not considered related to the investigational medicinal product.

2. Number of Participants Experiencing Serious Adverse Events (SAEs)

   Time frame: From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

   A Serious Adverse Event (SAE) is any untoward medical occurrence that at any dose results in death, is life-threatening (defined as an event in which the subject was at risk of death at the time of the event; it does not refer to an event which hypothetically might have caused death if it were more severe), requires inpatient hospitalization or causes prolongation of existing hospitalization.

3. Number of Participants Experiencing Adverse Events (AEs) Leading to Discontinuation

   Time frame: From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

   An AE is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporarily associated with the use of medicinal product, whether or not considered related to the investigational medicinal product.

4. Number of Participants Experiencing Adverse Events (AEs) Leading to Withdrawal

   Time frame: From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

   An AE is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporarily associated with the use of medicinal product, whether or not considered related to the investigational medicinal product.

5. Number of Participants Experiencing Adverse Events (AEs) of Special Interest

   Time frame: From first dose to 90-days post last dose (an average of 65 months up to a max of 85 months)

   An AE is any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease temporarily associated with the use of medicinal product, whether or not considered related to the investigational medicinal product.

6. Number of Participants With Abnormalities in Blood Absolute Lymphocyte Count (ALC)

   Time frame: From first dose up until last dose of study treatment (up to approximately 82 months)

   An absolute lymphocyte count (ALC) is a part of a blood test that measures the number of lymphocytes, a type of white blood cell, in the blood. Lymphocytes help fight infections and diseases. Reductions in ALC levels for participants in this study is expected and is a primary pharmacodynamic effect of RPC1063. LLN = Lower limit of normal

7. Number of Participants With Abnormalities in White Blood Cell Count (WBC)

   Time frame: From first dose up until last dose of study treatment (up to approximately 82 months)

   A white blood cell count is a part of a blood test that measures the number of white blood cells in the blood. White blood cells help fight infections and diseases. LLN = Lower limit of normal

8. Number of Participants With Abnormalities in Blood Absolute Neutrophil Count (ANC)

   Time frame: From first dose up until last dose of study treatment (up to approximately 82 months)

   An absolute neutrophil count is a part of a blood test that measures the number of neutrophils, a type of white blood cell, in the blood. Neutrophils help fight infections and diseases.

9. Number of Participants With Abnormalities in Specific Liver Function Tests

   Time frame: From first dose up until last dose of study treatment (up to approximately 82 months)

   The number of participants with laboratory abnormalities in specific liver tests above ULN by category. ULN = Upper Limit of Normal

10. Number of Participants With Electrocardiogram (ECG) Result Abnormalities

    Time frame: From first dose to 28-days post last dose (an average of 63 months up to a max of 83 months)

    An electrocardiogram (ECG) measures electrical activity of the heart to detect cardiac problems.

11. Number of Participants With Clinically Relevant Abnormalities in Vital Signs

    Time frame: At baseline and 60 months after first dose of study therapy

    Vital signs included body temperature, sitting heart rate/pulse (HR), sitting systolic blood pressure (SBP), sitting diastolic blood pressure (DBP). Baseline refers to assessments made on or before the first day participants received study treatment.

12. Number of Participants With Physical Examination Abnormalities

    Time frame: At baseline and every 12 months thereafter up until 84 months post first dose.

    The number of participants with abnormal physical examination results. The assessments included abdominal, extremity, head, heart, lungs, neck, neurological non-MS, other and skin assessments. Baseline refers to assessments made on or before the first day participants received study treatment.

13. Number of Participants Self-Identifying Suicidality by Columbia-Suicide Severity Rating Scale (C-SSRS)

    Time frame: At baseline and every 3 months thereafter up until 78 months post first dose.

    The Columbia-Suicide Severity Rating Scale (C-SSRS) is a unique suicide risk assessment tool that supports suicide risk assessment through a series of simple, plain-language questions. The answers help users identify whether someone is at risk for suicide, assess the severity and immediacy of that risk, and gauge the level of support that the person needs. Results are displayed as the number of participants who answered "Yes" to at least one of the 10 questions in the suicidal ideation or suicidal behavior section. Ideation from 1 (wishing to be dead) - 5 (Active suicidal ideation with specific plan and intent) Behavior from 6 (Preparatory acts or behavior) - 10 (Completed suicide). Baseline refers to assessments made on or before the first day participants received study treatment.

14. Number of Participants With Changes in Suicidality From Last Day on Treatment Per the Columbia-Suicide Severity Rating Scale (C-SSRS)

    Time frame: 1, 4, 7, 14, 21, 28, and 90 days post last dose.

    The Columbia-Suicide Severity Rating Scale (C-SSRS) is a unique suicide risk assessment tool that supports suicide risk assessment through a series of simple, plain-language questions. The answers help users identify whether someone is at risk for suicide, assess the severity and immediacy of that risk, and gauge the level of support that the person needs. Results are displayed as the number of participants who answered "Yes" to at least one of the 10 questions in the suicidal ideation or suicidal behavior section. Ideation from 1 (wishing to be dead) - 5 (Active suicidal ideation with specific plan and intent) Behavior from 6 (Preparatory acts or behavior) - 10 (Completed suicide).

15. Change in Physician's Withdrawal Checklist (PWC-20) Total Score From Last Day on Treatment

    Time frame: 1, 4, 7, 14, 21, and 90 days post last dose.

    The PWC-20 is a rater-administered 20-item scale to assess signs and symptoms of withdrawal. Twenty items are rated on a 4-point scale as not present (0 points), mild (1 point), moderate (2 points), or severe (3 points). The points from all items are calculated as a total score. Higher scores indicate more severe withdrawal symptoms.

16. Change in Hospital Anxiety and Depression Scale (HADS) Score From Last Day on Treatment

    Time frame: 1, 4, 7, 14, 21, and 90 days post last dose.

    The HADS is a validated patient reported outcome for assessing anxiety and depression. It consists of 14 items in total, 7 items related to anxiety and 7 items related to depression. For each item patients select a statement (valued at 0 to 3 points) that closest matches their own feeling over the past week. Separate total scores for anxiety and depression are derived by adding up points. Total scores can range from 0 to 21 points. Higher scores indicate more severe anxiety and depression and scores of 8 to 10 are generally considered indicative of borderline anxiety/depression disorders and scores of 11 and higher are generally considered indicative of anxiety/depression disorders.

17. Changes in Epworth Sleepiness Scale (ESS) Score From Last Day on Treatment

    Time frame: 1, 4, 7, 14, 21, and 90 days post last dose.

    The ESS is a validated self administered questionnaire with 8 questions. Respondents rate on a 4-point scale (0 to 3) their chances of dozing off or falling asleep while engaged in 8 different activities. The ESS score is the sum of 8 item scores and can range from 0 to 24 points. Higher scores indicate more daytime sleepiness.

18. Changes in Vital Sign Values From Last Day on Treatment

    Time frame: 1, 4, 7, 14, 21, 28, and 90 days post last dose.

    Vital signs included sitting systolic blood pressure (SBP), sitting diastolic blood pressure (DBP).

Results and safety

Loading the posted results from ClinicalTrials.gov…

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2024.
• Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
• Derived from this study: PubMed 41677183Rubin DT, Danese S, Nakase H, Ungaro RC, Wolf DC, Alekseeva O, Petersen A, Liu Z, Mehra D, Jain A, Osterman MT, Krakovich A, Riolo JV, DeBoer E, Appio J, Sinh P, Cree BAC, Cohen JA, Irving P. Integrated long-term safety of 10-year ozanimod treatment: results from clinical trials in patients with moderate-to-severe ulcerative colitis or relapsing multiple sclerosis. Inflamm Bowel Dis. 2026 May 1;32(5):954-962. doi: 10.1093/ibd/izaf319.
• Derived from this study: PubMed 41190505Selmaj KW, Steinman L, Comi G, Bar-Or A, Arnold DL, Hartung HP, Montalban X, Havrdova EK, Sheffield JK, Krakovich A, Cheng CY, Riolo JV, Pachai C, Thorpe A, DeBoer E, Kappos L, Cohen JA, Cree BA. Long-term safety and efficacy of ozanimod in relapsing multiple sclerosis: Final analysis of the DAYBREAK open-label extension trial. Mult Scler. 2025 Nov;31(13):1557-1571. doi: 10.1177/13524585251382796. Epub 2025 Nov 5.
• Derived from this study: PubMed 40698614Cohen JA, Arnold DL, DeLuca J, Hartung HP, Kappos L, Comi G, Selmaj K, Steinman L, Bar-Or A, Montalban X, Havrdova EK, Sheffield JK, Pachai C, Cheng CY, Riolo JV, Cree BA. Brain atrophy and associations with long-term disability and cognitive function in participants with relapsing multiple sclerosis treated with ozanimod: Results from phase 3 and open-label extension trials. Mult Scler. 2025 Sep;31(10):1218-1230. doi: 10.1177/13524585251355842. Epub 2025 Jul 23.
• Derived from this study: PubMed 39018016Regueiro M, Siegmund B, Horst S, Moslin R, Charles L, Petersen A, Tatosian D, Wu H, Lawlor G, Fischer M, D'Haens G, Colombel JF. Concomitant Administration of Ozanimod and Serotonergic Antidepressants in Patients With Ulcerative Colitis or Relapsing Multiple Sclerosis. Inflamm Bowel Dis. 2025 Apr 10;31(4):1010-1017. doi: 10.1093/ibd/izae136.
• Derived from this study: PubMed 38130041Naismith RT, Cohen JA, Bar-Or A, Comi G, Selmaj KW, Hartung HP, Sheffield JK, Krakovich A, Tatosian D, Cheng CY, Reardon J, Khaychuk V, Riolo JV, Silva D, Cree BA. Concurrent administration of serotonergic antidepressants and ozanimod in participants with relapsing multiple sclerosis from the open-label extension DAYBREAK trial. Mult Scler. 2024 Feb;30(2):177-183. doi: 10.1177/13524585231216854. Epub 2023 Dec 21.
• Derived from this study: PubMed 37550942Cree BAC, Maddux R, Bar-Or A, Hartung HP, Kaur A, Brown E, Li Y, Hu Y, Sheffield JK, Silva D, Harris S. SARS-CoV-2 vaccination and infection in ozanimod-treated participants with relapsing multiple sclerosis. Ann Clin Transl Neurol. 2023 Oct;10(10):1725-1737. doi: 10.1002/acn3.51862. Epub 2023 Aug 7.
• BMS Clinical Trial InformationLink provided by the sponsor.
• BMS Clinical Trial Patient RecruitingLink provided by the sponsor.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

175 sites in 26 countries: see every site and its status.