Multiple Sclerosis
Conditions
Brief summary
The purpose of this trial is to test the Single-Use Autoinjector for a) ease of use; b) multiple domains related to subject's acceptability and satisfaction, and c) reliability of the correct function for self-injection of Rebif® 44 mcg sc tiw in subjects with relapsing multiple sclerosis (RMS).
Interventions
DEVICERebidose®
The device is a ready to use single use auto injector containing Rebif® 44 mcg in a 0.5 mL prefilled syringe for sc injection
Sponsors
EMD Serono
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Male and female subjects, 18-65 years of age, inclusive, at the time of informed consent signature 2. RMS diagnosed according to the McDonald criteria 3. Currently receiving Rebif® 44mcg sc tiw using manual injections and/or Rebiject II autoinjector for greater than twelve weeks 4. Capable of self-injecting using the single-use autoinjector 5. Be willing and able to comply with the study procedures for the duration of the trial 6. Have given written informed consent and signed Health Insurance Portability and Accountability Act (HIPAA) Authorization before any study-related activities are carried out 7. Female subjects must not be either pregnant or breast-feeding and must lack childbearing potential
Exclusion criteria
1. Have used any other injectable medications (i.e. for pain) on a regular basis during the week prior to Screening or throughout the duration of the trial (the administration of a single injection for treatment or prophylaxis of a condition unrelated to MS or Rebif® therapy (e.g., influenza or pneumococcus vaccination) will be acceptable) 2. Have received MS therapy other than Rebif® within twelve weeks prior to Screening or at any time during the trial (e.g., other disease modifying drugs, Rebif® New Formulation, combination therapy, immunomodulatory and/or immunosuppressive agents, including but not limited to any interferon, glatiramer acetate (Copolymer I), cyclophosphamide, cyclosporine, methotrexate, linomide, azathioprine, mitoxantrone, teriflunomide, laquinimod, cladribine, total lymphoid irradiation, anti-lymphocyte monoclonal antibody treatment (e.g. natalizumab, alemtuzumab/Campath, anti- CD4), Intravenous immunoglobulin (IVIg), cytokines or anti-cytokine therapy) and telbivudine 3. Have inadequate liver function, defined by a alanine aminotransferase (ALT) \> 2.5x upper limit of normal (ULN), or alkaline phosphatase \> 2.5x ULN, or total bilirubin \> 2.5x ULN 4. Have inadequate bone marrow reserve, defined as a total white blood cell count \< 3.0x 109/L, platelet count \< 75x109/L, haemoglobin \< 100g/L 5. Have complete transverse myelitis or bilateral optic neuritis 6. Have a history of alcohol or drug abuse 7. Have thyroid dysfunction 8. Have moderate to severe renal impairment 9. Have a major medical or psychiatric illness that in the opinion of the investigator creates undue risk to the subject or could affect compliance with the study protocol 10. Have a history of seizures not adequately controlled by treatment 11. Have serious or acute cardiac disease, such as uncontrolled dysrhythmias, uncontrolled angina pectoris, cardiomyopathy, or uncontrolled congestive heart failure 12. Have, in the opinion of the investigator, any visual, physical or cognitive impairment that would preclude the subject from self-injecting with the single-use autoinjector 13. Have a known hypersensitivity or allergy to interferon-beta or any of the excipients 14. Have participated in another clinical trial within the past thirty days 15. Are pregnant or attempting to conceive
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Relapsing Multiple Sclerosis (RMS) Subjects Rating the Single-use Autoinjector as 'Easy to Use' or 'Very Easy to Use' for Self-injection in a User Trial Questionnaire | at 12 weeks | Data from the User Trial Questionnaire, Question 14 (Overall, how do you rate your experience with using the injection device?) Mean and confidence interval refer to proportion of subjects responding positively to question. Missing values were replaced with worst case response. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | at 12 weeks | The User Trial Questionnaire was used to assess the ease of use, functional reliability, overall satisfaction with device attributes, convenience, safety and portability of the device. Mean and confidence intervals refer to proportion of subjects responding positively to question. Secondary endpoints presented for decriptive purposes only thus no statistical analysis performed. |
Countries
United States
Participant flow
Recruitment details
109 subjects were recruited from 12 US clinics in the US during the trial period Oct 2009 to April 2010.
Pre-assignment details
Subjects who signed informed consent and satisfied the eligibility critera at screening returned to the clinic on study day 1 to begin treatment. All subjects participating in the trial received Rebif 44 mcg subcutaneious (sc, three times per week (tiw) using the single-use autoinjector (SA).
Participants by arm
| Arm | Count |
|---|---|
| RMS Subject Disposition Intent to Treat Analysis (ITT) includes all subjects who provide written informed consent and received at least one dose of trial medication | 109 |
| Total | 109 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Subject found device difficult to use | 1 |
Baseline characteristics
| Characteristic | RMS Subject Disposition |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 109 Participants |
| Age Continuous | 46.0 years STANDARD_DEVIATION 9.32 |
| Region of Enrollment United States | 109 participants |
| Sex: Female, Male Female | 76 Participants |
| Sex: Female, Male Male | 33 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 83 / 109 |
| serious Total, serious adverse events | 4 / 109 |
Outcome results
Primary
Proportion of Relapsing Multiple Sclerosis (RMS) Subjects Rating the Single-use Autoinjector as 'Easy to Use' or 'Very Easy to Use' for Self-injection in a User Trial Questionnaire
Data from the User Trial Questionnaire, Question 14 (Overall, how do you rate your experience with using the injection device?) Mean and confidence interval refer to proportion of subjects responding positively to question. Missing values were replaced with worst case response.
Time frame: at 12 weeks
Population: Subjects in the all enrolled analysis set who received at least one dose of investigational medicinal product (IMP) were included in the ITT analysis set. This analysis set was used to analyze the primary and secondary variables and all safety data.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| RMS Subject Disposition | Proportion of Relapsing Multiple Sclerosis (RMS) Subjects Rating the Single-use Autoinjector as 'Easy to Use' or 'Very Easy to Use' for Self-injection in a User Trial Questionnaire | 0.86 Proportion of subjects |
95% CI: [0.8, 0.93]
Secondary
Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes.
The User Trial Questionnaire was used to assess the ease of use, functional reliability, overall satisfaction with device attributes, convenience, safety and portability of the device. Mean and confidence intervals refer to proportion of subjects responding positively to question. Secondary endpoints presented for decriptive purposes only thus no statistical analysis performed.
Time frame: at 12 weeks
Population: Subjects in the all enrolled analysis set who received at least one dose of IMP were included in the ITT analysis set. This analysis set was used to analyze the primary and secondary variables and all safety data.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Device makes injections simple | 0.83 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Helps me self-administer injections | 0.83 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Able to often or always administer full injection | 0.96 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Satisfaction with ease of removing cap from device | 0.87 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Satisfaction with ease of activating button | 0.78 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Satisfaction with time to inject medicine | 0.83 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Satisfaction with ability to see post injection | 0.81 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Rate how reliable injection device was | 0.79 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Allows easy access to skin injection sites | 0.78 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Ease of pushing device against skin | 0.76 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Ease of holding device during injection | 0.77 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Satisfaction with size of device | 0.61 Proportion of subjects |
| RMS Subject Disposition | Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes. | Rate convenience of device | 0.74 Proportion of subjects |