Phase 3
N=2,494
A Multi-Site, Open-Label Extension Trial of Oral RPC1063 in Relapsing Multiple Sclerosis
Multiple Sclerosis
Bottom Line
View on ClinicalTrials.gov: NCT02576717 ↗Enrolled (actual)
2,494
Serious AEs
15.3%
Results posted
Jan 2024
Primary outcome: Primary: Number of Participants Experiencing Adverse Events (AEs) — 668; 775; 776 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- RPC1063 (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Celgene
- Primary completion
- Jan 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Experiencing Adverse Events (AEs) |
668; 775; 776 | — |
| PRIMARY Number of Participants Experiencing Serious Adverse Events (SAEs) |
108; 139; 134 | — |
| PRIMARY Number of Participants Experiencing Adverse Events (AEs) Leading to Discontinuation |
35; 35; 28 | — |
| PRIMARY Number of Participants Experiencing Adverse Events (AEs) Leading to Withdrawal |
32; 35; 28 | — |
| PRIMARY Number of Participants Experiencing Adverse Events (AEs) of Special Interest |
19; 26; 19; 9; 14; 16 | — |
| PRIMARY Number of Participants With Abnormalities in Blood Absolute Lymphocyte Count (ALC) |
33; 33; 27; 265; 323; 339 | — |
| PRIMARY Number of Participants With Abnormalities in White Blood Cell Count (WBC) |
0; 0; 2; 27; 48; 43 | — |
| PRIMARY Number of Participants With Abnormalities in Blood Absolute Neutrophil Count (ANC) |
0; 2; 1 | — |
| PRIMARY Number of Participants With Abnormalities in Specific Liver Function Tests |
311; 353; 353; 90; 100; 107 | — |
| PRIMARY Number of Participants With Electrocardiogram (ECG) Result Abnormalities |
6; 5; 3; 1; 0; 0 | — |
| PRIMARY Number of Participants With Clinically Relevant Abnormalities in Vital Signs |
0; 0; 0; 15; 19; 26 | — |
| PRIMARY Number of Participants With Physical Examination Abnormalities |
0; 0; 1; 3; 2; 8 | — |
| PRIMARY Number of Participants Self-Identifying Suicidality by Columbia-Suicide Severity Rating Scale (C-SSRS) |
0; 2; 0; 2; 1; 1 | — |
| PRIMARY Number of Participants With Changes in Suicidality From Last Day on Treatment Per the Columbia-Suicide Severity Rating Scale (C-SSRS) |
3; 0; 0; 0; 0; 0 | — |
| PRIMARY Change in Physician's Withdrawal Checklist (PWC-20) Total Score From Last Day on Treatment |
-0.1; -0.6; -0.5; -1.1; -0.4; -1.2 | — |
| PRIMARY Change in Hospital Anxiety and Depression Scale (HADS) Score From Last Day on Treatment |
0.3; -0.1; -0.1; -0.2; 0.1; 0.1 | — |
| PRIMARY Changes in Epworth Sleepiness Scale (ESS) Score From Last Day on Treatment |
0.4; -0.6; -0.5; -0.7; -0.9; -0.9 | — |
| PRIMARY Changes in Vital Sign Values From Last Day on Treatment |
-1.77; 0.63; -0.04; 0.82; -0.4; 1.25 | — |
| SECONDARY Annualized Relapse Rate (ARR) |
0.097; 0.108; 0.090 | — |
| SECONDARY Time to First Relapse (TFR) |
NA; NA; NA | — |
| SECONDARY Number of Participants Who Were Relapse Free |
513; 605; 605 | — |
| SECONDARY Average Number of New or Enlarging Hyperintense T2-Weighted Brain MRI Lesions Per Scan at Each Visit |
1.532; 1.163; 1.302; 1.254; 1.005; 1.190 | — |
| SECONDARY Average Number of Gadolinium-Enhanced (GdE) Brain MRI Lesions Per Scan at Each Visit |
0.460; 0.244; 0.177; 0.106; 0.130; 0.205 | — |
| SECONDARY Time to Onset of Disability Progression as Defined by a Sustained Worsening in Expanded Disability Status Scale (EDSS) |
NA; NA; NA; NA; NA; NA | — |
| SECONDARY Number of Participants Free of Gadolinium-Enhanced (GdE) Brain Lesions at Each Visit |
538; 609; 662; 622; 644; 627 | — |
| SECONDARY Number of Participants Free of New or Enlarging T2 Lesions at Each Visit |
322; 400; 419; 260; 323; 338 | — |
| SECONDARY Percent Change in Normalized Brain Volume (Atrophy) on Brain MRI Scans From Baseline at Each Visit |
-0.407; -0.359; -0.385; -0.702; -0.657; -0.671 | — |
| SECONDARY Change in Multiple Sclerosis Functional Composite (MSFC) Score From Baseline at Each Applicable Visit |
0.058; -0.085; -0.064; -0.072; -0.082; -0.086 | — |
| SECONDARY Change in Multiple Sclerosis Quality of Life 54 Score From Baseline at Each Applicable Visit |
0.1; -0.1; 0.2; 0.8; -0.6; -0.3 | — |
| SECONDARY Change From Baseline in Volume of Gadolinium Enhanced T1 Lesions |
-0.092; -0.015; -0.004; -0.099; -0.017; 0.009 | — |
| SECONDARY Change From Baseline in Volume of T2 Lesions |
0.190; 0.174; 0.278; 0.307; 0.303; 0.518 | — |
| SECONDARY Change From Baseline in Volume of Unenhancing T1 Lesions |
-0.625; -0.772; -0.760; -0.192; -0.465; -0.455 | — |
| SECONDARY Cumulative Number of New Unenhancing T1 Lesions |
1.9; 1.1; 1.2; 2.7; 1.8; 2.2 | — |
Summary
The purpose of the trial is to determine the safety and efficacy of RPC1063 in patients with relapsing multiple sclerosis.
Eligibility Criteria
Eligibility Criteria:
To be eligible to participate in this trial, patients must meet all of the following criteria:
- Completed one of the parent trials
- Does not have a condition that would require withdrawal from one of the parent trials
- Has no conditions requiring treatment with a prohibited concomitant medication
- Is not receiving treatment with any of the following drugs or interventions within the corresponding timeframe:
At Baseline (Day 1)
- CYP2C8 inhibitors (eg, gemfibrozil or clopidogrel) or inducers (eg, rifampicin) Two weeks prior to Baseline (Day 1)
- Monoamine oxidase inhibitors (eg, selegiline, phenelzine)
- Ability to provide written informed consent and to be compliant with the schedule of protocol assessments
- Female patients of childbearing potential:
Must agree to practice a highly effective method of contraception throughout the study until completion of the 90-day Safety Follow-up Visit. Highly effective methods of contraception are those that alone or in combination result in a failure rate of a Pearl index of less than 1% per year when used consistently and correctly.
Acceptable methods of birth control in this study are the following:
- Combined hormonal (estrogen and progestogen containing) contraception, which may be oral, intravaginal, or transdermal
- Progestogen-only hormonal contraception associated with inhibition of ovulation, which may be oral, injectable, or implantable
- Placement of an intrauterine device (IUD)
- Placement of an intrauterine hormone-releasing system (IUS)
- Bilateral tubal occlusion
- Vasectomised partner
- Sexual abstinence.
Exclusion Criteria
Data sourced from ClinicalTrials.gov (NCT02576717). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.