The Multicenter, Open-label, Single-use Autoinjector Convenience Study

• ClinicalTrials.gov Identifier: NCT00958009
• Recruitment Status: Completed
• First Posted: August 13, 2009
• Results First Posted: December 30, 2010
• Last Update Posted: August 7, 2013
• Sponsor: EMD Serono
• Information provided by: EMD Serono

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label)
• Condition: Multiple Sclerosis
• Intervention: Device: Rebidose®
• Enrollment: 109

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).

Arm/Group Title	RMS Subject Disposition
Arm/Group Description	Intent to Treat Analysis (ITT) includes all subjects who provide written informed consent and received at least one dose of trial medication
Period Title: Overall Study
Started	109
Completed	107
Not Completed	2
Reason Not Completed
Adverse Event	1
Subject found device difficult to use	1

Baseline Characteristics

Arm/Group Title		RMS Subject Disposition
Arm/Group Description		Intent to Treat Analysis (ITT) includes all subjects who provide written informed consent and received at least one dose of trial medication
Overall Number of Baseline Participants		109
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	109 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	109 100.0%
	>=65 years	0 0.0%
Age Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	109 participants
		46.0 (9.32)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	109 participants
	Female	76 69.7%
	Male	33 30.3%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United States	Number Analyzed	109 participants
		109

Outcome Measures

1. Primary Outcome

Title	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
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	RMS Subject Disposition
Arm/Group Description:	Intent to Treat Analysis (ITT) includes all subjects who provide written informed consent and received at least one dose of trial medication
Overall Number of Participants Analyzed	109
Mean (95% Confidence Interval); Unit of Measure: Proportion of subjects
	0.86; (0.80 to 0.93)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	RMS Subject Disposition
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	mean positive response
	Estimated Value	0.86
	Confidence Interval	(2-Sided) 95%; 0.80 to 0.93
	Estimation Comments	Confidence Interval calculated from normal approximation to the binomial. The primary objective was met if the lower bound of the 95% confidence interval is >0.50.

2. Secondary Outcome

Title	Multiple Secondary Endpoints Were Assessed, Based on Questions From the User Trial Questionnaire Related to the Single-use Autoinjector Device Use-related Outcomes.
Description	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

Outcome Measure Data

Analysis Population Description

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/Group Title	RMS Subject Disposition
Arm/Group Description:	Intent to Treat Analysis (ITT) includes all subjects who provide written informed consent and received at least one dose of trial medication
Overall Number of Participants Analyzed	109
Mean (95% Confidence Interval); Unit of Measure: Proportion of subjects
Device makes injections simple	0.83; (0.77 to 0.90)
Allows easy access to skin injection sites	0.78; (0.70 to 0.86)
Helps me self-administer injections	0.83; (0.75 to 0.90)
Ease of pushing device against skin	0.76; (0.68 to 0.84)
Ease of holding device during injection	0.77; (0.69 to 0.85)
Able to often or always administer full injection	0.96; (0.93 to 1.00)
Satisfaction with size of device	0.61; (0.51 to 0.70)
Satisfaction with ease of removing cap from device	0.87; (0.81 to 0.93)
Satisfaction with ease of activating button	0.78; (0.70 to 0.86)
Satisfaction with time to inject medicine	0.83; (0.75 to 0.90)
Satisfaction with ability to see post injection	0.81; (0.73 to 0.88)
Rate convenience of device	0.74; (0.66 to 0.83)
Rate how reliable injection device was	0.79; (0.71 to 0.87)

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	RMS Subject Disposition
Arm/Group Description	Intent to Treat Analysis (ITT) includes all subjects who provide written informed consent and received at least one dose of trial medication
All-Cause Mortality
	RMS Subject Disposition
	Affected / at Risk (%)
Total	--/--
Serious Adverse Events
	RMS Subject Disposition
	Affected / at Risk (%)	# Events
Total	4/109 (3.67%)
Cardiac disorders
Angina pectoris † 1	1/109 (0.92%)	1
Gastrointestinal disorders
Pancreatitis acute † 1	1/109 (0.92%)	1
General disorders
Chest Pain † 1	1/109 (0.92%)	1
Nervous system disorders
Convulsion † 1	1/109 (0.92%)	1
Vascular disorders
Hypertension † 1	1/109 (0.92%)	1
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDra 13.0
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	RMS Subject Disposition
	Affected / at Risk (%)	# Events
Total	83/109 (76.15%)
General disorders
Influenza like illness † 1	26/109 (23.85%)	26
Injection site erythema † 1	39/109 (35.78%)	39
Injection site haematoma † 1	13/109 (11.93%)	13
Injection site pain † 1	27/109 (24.77%)	27
Injection site swelling † 1	8/109 (7.34%)	8
Infections and infestations
Upper respiratory tract infection † 1	6/109 (5.50%)	6
Nervous system disorders
Headache † 1	7/109 (6.42%)	7
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDra 13.0

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Neither Institution nor any Principal Investigators shall publish or present any results from such Study to any third parties until: (i) EMD Serono publishes the results from all sites participating in such Study; (ii) Institution receives notification from EMD Serono that publication of the multi-site results is no longer planned; or (iii) twenty-four (24) months following the completion of the multi-site study at all sites, whichever occurs first.

Results Point of Contact

• Name/Title: Fernando Dangond, MD
• Organization: EMD Serono, Inc.
• Phone: 781-681-2348
• EMail: Fernando.dangond@emdserono.com

• Responsible Party: Fernando Dangond, , Senior Director, Medical Affairs Neurology, EMD Serono, Inc.
• ClinicalTrials.gov Identifier: NCT00958009
• Other Study ID Numbers: 29651
• First Submitted: August 12, 2009
• First Posted: August 13, 2009
• Results First Submitted: December 1, 2010
• Results First Posted: December 30, 2010
• Last Update Posted: August 7, 2013