Trial Outcomes & Findings for Rebif New Formulation (RNF) Quality of Life (QOL) Study (NCT NCT00472797)
NCT ID: NCT00472797
Last Updated: 2013-08-07
Results Overview
The MSTCQ Global Side Effect domain assesses the degree of satisfaction on global side effect questions 9, 10 \& 11 on a scale from 3 (not at all satisfied) to 15 (extremely satisfied). Percent change calculated as 100% \* (score at week 12 - score at baseline) / score at baseline.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
232 participants
Primary outcome timeframe
% change from Baseline to Week 12
Results posted on
2013-08-07
Participant Flow
232 subjects were recruited from 25 Multiple Sclerosis (MS) Clinics in the US from April 2007 through November 2007.
Run-in period (up to 30 days) on Rebif® 44 mcg tiw: The run-in period included a Screening Visit; assessments consisted of informed consent, medical/disease history, physical exam and laboratory assessments which were performed in the two-week period prior to Study Day 1, (prior to the first dose of a new formulation of rebif)
Participant milestones
| Measure |
New Formulation of Rebif - Non-Titrated
The new formulation of rebif is not approved and under investigation in the US
|
New Formulation of Rebif - Titrated
The new frmulation of rebif is not approved and under investigation in the US
|
|---|---|---|
|
Overall Study
STARTED
|
119
|
113
|
|
Overall Study
COMPLETED
|
115
|
112
|
|
Overall Study
NOT COMPLETED
|
4
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Rebif New Formulation (RNF) Quality of Life (QOL) Study
Baseline characteristics by cohort
| Measure |
New Formulation of Rebif - Non-Titrated
n=119 Participants
|
New Formulation of Rebif - Titrated
n=113 Participants
|
Total
n=232 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0.0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
119 Participants
n=5 Participants
|
113 Participants
n=7 Participants
|
232.0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0.0 Participants
n=5 Participants
|
|
Age Continuous
|
44.6 years
STANDARD_DEVIATION 9.0 • n=5 Participants
|
42.8 years
STANDARD_DEVIATION 9.4 • n=7 Participants
|
43.7 years
STANDARD_DEVIATION 9.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
91 Participants
n=5 Participants
|
89 Participants
n=7 Participants
|
180.0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
52.0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
119 participants
n=5 Participants
|
113 participants
n=7 Participants
|
232.0 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: % change from Baseline to Week 12Population: Safety: This population includes all subjects who received at least one dose of study drug. To explain difference in number, 1 subject lost to follow-up, 1 subject withdrew consent
The MSTCQ Global Side Effect domain assesses the degree of satisfaction on global side effect questions 9, 10 \& 11 on a scale from 3 (not at all satisfied) to 15 (extremely satisfied). Percent change calculated as 100% \* (score at week 12 - score at baseline) / score at baseline.
Outcome measures
| Measure |
All New Formulation of Rebif Subjects
n=230 Participants
All subjects combined in Intent to Treat (ITT) Population
|
Titrated
New formulation of rebif
|
All New Formulation of Rebif Subjects
All subjects combined in Intent to Treat (ITT) Population
|
|---|---|---|---|
|
Percent Change in Global Side Effects (GSE) on Multiple Sclerosis Treatment Concerns Quesionnaire (MSTCQ)
|
5.0 percent change
Standard Deviation 17.7
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Intent to Treat (ITT) and Last Observation Carried Forward (LOCF) Higher scores indicate a more favorable response
The MSTCQ Global Side Effect domain assesses the subjects degree of satisfaction on global side effect questions 9, 10 \& 11 on a scale from 3 (not at all satisfied) to 15 (extremely satisfied).
Outcome measures
| Measure |
All New Formulation of Rebif Subjects
n=117 Participants
All subjects combined in Intent to Treat (ITT) Population
|
Titrated
n=113 Participants
New formulation of rebif
|
All New Formulation of Rebif Subjects
n=230 Participants
All subjects combined in Intent to Treat (ITT) Population
|
|---|---|---|---|
|
Total Score for Global Side Effects on Multiple Sclerosis Treatment Concerns Questionnaire (MSTCQ)
Mean GSE at Baseline
|
13.4 score on scale
Standard Deviation 1.7
|
13.4 score on scale
Standard Deviation 1.8
|
13.4 score on scale
Standard Deviation 1.7
|
|
Total Score for Global Side Effects on Multiple Sclerosis Treatment Concerns Questionnaire (MSTCQ)
Mean GSE at Week 12 (LOCF)
|
14.0 score on scale
Standard Deviation 1.6
|
13.8 score on scale
Standard Deviation 1.6
|
13.9 score on scale
Standard Deviation 1.6
|
|
Total Score for Global Side Effects on Multiple Sclerosis Treatment Concerns Questionnaire (MSTCQ)
Percent Change - Baseline to Week 12 (LOCF)
|
5.5 score on scale
Standard Deviation 16.5
|
4.5 score on scale
Standard Deviation 18.8
|
5.0 score on scale
Standard Deviation 17.7
|
SECONDARY outcome
Timeframe: Baseline to Week 12The MSTCQ in all domains assesses quality of life. The score for all domains other than the Global Side Effect ranges from 17 (most favorable) to 85 (least favorable). Change calculated as (score at week 12 - score at baseline.
Outcome measures
| Measure |
All New Formulation of Rebif Subjects
n=117 Participants
All subjects combined in Intent to Treat (ITT) Population
|
Titrated
n=113 Participants
New formulation of rebif
|
All New Formulation of Rebif Subjects
n=230 Participants
All subjects combined in Intent to Treat (ITT) Population
|
|---|---|---|---|
|
Change in Score From Baseline to Week 12 for All Domains Other Than Global Side Effects on Multiple Sclerosis Treatment Concerns Questionnaire (MSTCQ)
Injection Site Reactions Domain
|
-1.9 score on scale
Standard Deviation 3.2
|
-1.3 score on scale
Standard Deviation 2.9
|
-1.6 score on scale
Standard Deviation 3.1
|
|
Change in Score From Baseline to Week 12 for All Domains Other Than Global Side Effects on Multiple Sclerosis Treatment Concerns Questionnaire (MSTCQ)
Injection System Satisfaction Domain
|
-3.2 score on scale
Standard Deviation 4.0
|
-2.9 score on scale
Standard Deviation 4.1
|
-3.1 score on scale
Standard Deviation 4.1
|
|
Change in Score From Baseline to Week 12 for All Domains Other Than Global Side Effects on Multiple Sclerosis Treatment Concerns Questionnaire (MSTCQ)
Side Effects Domain
|
-3.3 score on scale
Standard Deviation 6.4
|
-2.3 score on scale
Standard Deviation 5.7
|
-2.8 score on scale
Standard Deviation 6.1
|
|
Change in Score From Baseline to Week 12 for All Domains Other Than Global Side Effects on Multiple Sclerosis Treatment Concerns Questionnaire (MSTCQ)
Flu-Like Systems Domain
|
-0.9 score on scale
Standard Deviation 3.6
|
-0.8 score on scale
Standard Deviation 3.6
|
-0.8 score on scale
Standard Deviation 3.6
|
|
Change in Score From Baseline to Week 12 for All Domains Other Than Global Side Effects on Multiple Sclerosis Treatment Concerns Questionnaire (MSTCQ)
Total Scores in all above Domains
|
-6.7 score on scale
Standard Deviation 8.8
|
-5.3 score on scale
Standard Deviation 7.6
|
-6.0 score on scale
Standard Deviation 8.3
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: ITT and LOCF
The SF-MPQ assesses Tolerability of Pain with 15 questions to evaluate the type and severity of pain experienced 60 minutes after an injection of study drug. Scores range from 0 (no pain) to 45 (severe pain).
Outcome measures
| Measure |
All New Formulation of Rebif Subjects
n=117 Participants
All subjects combined in Intent to Treat (ITT) Population
|
Titrated
n=113 Participants
New formulation of rebif
|
All New Formulation of Rebif Subjects
n=229 Participants
All subjects combined in Intent to Treat (ITT) Population
|
|---|---|---|---|
|
Total Score on Short-Form McGill Pain Questionnaire (SF-MPQ): Change in Baseline to Wk 12
|
0.7 score on scale
Standard Deviation 3.4
|
1.1 score on scale
Standard Deviation 3.9
|
0.9 score on scale
Standard Deviation 3.7
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: ITT
The SF-MPQ included a visual analog scale, ranging from 0 to 100 mm, on which subjects rate pain from no pain (0 mm) to worst possible pain (100 mm). A rating of \<5 mm was considered pain-free.
Outcome measures
| Measure |
All New Formulation of Rebif Subjects
n=119 Participants
All subjects combined in Intent to Treat (ITT) Population
|
Titrated
n=113 Participants
New formulation of rebif
|
All New Formulation of Rebif Subjects
n=232 Participants
All subjects combined in Intent to Treat (ITT) Population
|
|---|---|---|---|
|
Tolerability in Pain Using Visual Analog Scale (VAS)
Baseline - No. of Subjects Pain Free
|
51 Participants
|
48 Participants
|
101 Participants
|
|
Tolerability in Pain Using Visual Analog Scale (VAS)
Week 12 - No. of Subjects Pain Free
|
53 Participants
|
48 Participants
|
101 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 12 (LOCF)Population: ITT and LOCF
Change in Baseline to Week 12 Last Observation Carried Forward(LOCF)- A blinded assessment of injection site redness was conducted by a health care professional (1-72 hours) after the most recent injection measuring redness at its widest diameter in mm. Diameter of Redness, lower is better.
Outcome measures
| Measure |
All New Formulation of Rebif Subjects
n=114 Participants
All subjects combined in Intent to Treat (ITT) Population
|
Titrated
n=110 Participants
New formulation of rebif
|
All New Formulation of Rebif Subjects
n=224 Participants
All subjects combined in Intent to Treat (ITT) Population
|
|---|---|---|---|
|
Tolerability - Redness at Injection Site
|
-2.3 mm
Standard Deviation 27.7
|
-3.1 mm
Standard Deviation 28.2
|
-2.7 mm
Standard Deviation 27.9
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Change from Baseline to Each VisitPopulation: ITT
Change from Baseline to each visit for Physical and Mental Component scores for SF-36. Score is norm-based with a mean of 50 and a standard deviation of 10.
Outcome measures
| Measure |
All New Formulation of Rebif Subjects
n=119 Participants
All subjects combined in Intent to Treat (ITT) Population
|
Titrated
n=113 Participants
New formulation of rebif
|
All New Formulation of Rebif Subjects
n=232 Participants
All subjects combined in Intent to Treat (ITT) Population
|
|---|---|---|---|
|
SF-36 Physical and Mental Component Scores
Physical Component Score - Baseline
|
43.4 Score
Standard Deviation 9.4
|
43.6 Score
Standard Deviation 9.3
|
43.5 Score
Standard Deviation 9.3
|
|
SF-36 Physical and Mental Component Scores
Physical Component -Change from Baseline to Wk 12
|
1.8 Score
Standard Deviation 5.7
|
2.0 Score
Standard Deviation 4.3
|
1.9 Score
Standard Deviation 5.0
|
|
SF-36 Physical and Mental Component Scores
Physical Component -Change from Baseline to Wk 36
|
-0.2 Score
Standard Deviation 7.7
|
1.5 Score
Standard Deviation 6.0
|
0.6 Score
Standard Deviation 7.0
|
|
SF-36 Physical and Mental Component Scores
Physical Component -Baseline to Ext. Visit 1
|
2.0 Score
Standard Deviation 6.5
|
1.7 Score
Standard Deviation 6.6
|
1.9 Score
Standard Deviation 6.5
|
|
SF-36 Physical and Mental Component Scores
Physical Component -Baseline to Ext Visit 3
|
1.0 Score
Standard Deviation 6.5
|
2.0 Score
Standard Deviation 6.4
|
1.5 Score
Standard Deviation 6.4
|
|
SF-36 Physical and Mental Component Scores
Physical Component -Baseline to Exit Visit/LOCF
|
0.4 Score
Standard Deviation 6.4
|
1.4 Score
Standard Deviation 6.4
|
0.9 Score
Standard Deviation 6.4
|
|
SF-36 Physical and Mental Component Scores
Mental Component - Baseline
|
46.1 Score
Standard Deviation 10.9
|
46.1 Score
Standard Deviation 10.9
|
46.1 Score
Standard Deviation 10.9
|
|
SF-36 Physical and Mental Component Scores
Mental Component - Change from Baseline to Week 12
|
2.5 Score
Standard Deviation 8.7
|
1.3 Score
Standard Deviation 9.1
|
1.9 Score
Standard Deviation 8.9
|
|
SF-36 Physical and Mental Component Scores
Mental Component - Change from Baseline to Week 36
|
1.8 Score
Standard Deviation 10.6
|
-0.8 Score
Standard Deviation 10.0
|
0.5 Score
Standard Deviation 10.4
|
|
SF-36 Physical and Mental Component Scores
Mental Component - Baseline to Ext Visit 1
|
0.3 Score
Standard Deviation 10.7
|
-1.2 Score
Standard Deviation 8.9
|
-0.4 Score
Standard Deviation 9.8
|
|
SF-36 Physical and Mental Component Scores
Mental Component - Baseline to Ext Visit 3
|
1.3 Score
Standard Deviation 11.2
|
0.1 Score
Standard Deviation 10.4
|
0.7 Score
Standard Deviation 10.8
|
|
SF-36 Physical and Mental Component Scores
Mental Component - Baseline to Exit/LOCF
|
-0.1 Score
Standard Deviation 11.2
|
0.1 Score
Standard Deviation 9.7
|
0.0 Score
Standard Deviation 10.5
|
Adverse Events
Non-Titrated
Serious events: 10 serious events
Other events: 90 other events
Deaths: 0 deaths
Titrated
Serious events: 6 serious events
Other events: 78 other events
Deaths: 0 deaths
All New Formulation of Rebif Subjects
Serious events: 16 serious events
Other events: 168 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Non-Titrated
n=118 participants at risk
New formulation of rebif
|
Titrated
n=113 participants at risk
New formulation of rebif
|
All New Formulation of Rebif Subjects
n=231 participants at risk
All subjects combined in Intent to Treat (ITT) Population
|
|---|---|---|---|
|
Cardiac disorders
Artrial fibrillation
|
0.00%
0/118
|
0.88%
1/113 • Number of events 1
|
100.0%
1/1 • Number of events 1
|
|
Cardiac disorders
Cardiomiopathy
|
0.00%
0/118
|
0.88%
1/113 • Number of events 1
|
100.0%
1/1 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
0.00%
0/118
|
0.88%
1/113 • Number of events 1
|
100.0%
1/1 • Number of events 1
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/118
|
0.88%
1/113 • Number of events 1
|
100.0%
1/1 • Number of events 1
|
|
Cardiac disorders
Coronary artery disease
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Vascular disorders
Deep vein thrombosis
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Metabolism and nutrition disorders
Dehydration
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Injury, poisoning and procedural complications
Facial bones fracture
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Injury, poisoning and procedural complications
Fall
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Haemangiopericytoma
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc protrusion
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Reproductive system and breast disorders
Menorrhagia
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
0.00%
0/118
|
0.88%
1/113 • Number of events 1
|
100.0%
1/1 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Renal and urinary disorders
Renal Artery Fibromuscular Dysplasia
|
0.00%
0/118
|
0.88%
1/113 • Number of events 1
|
100.0%
1/1 • Number of events 1
|
|
Gastrointestinal disorders
Retroperitoneal haematoma
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Vascular disorders
Secondary Hypertension
|
0.00%
0/118
|
0.88%
1/113 • Number of events 1
|
100.0%
1/1 • Number of events 1
|
|
Infections and infestations
Sinusitis
|
0.00%
0/118
|
0.88%
1/113 • Number of events 1
|
100.0%
1/1 • Number of events 1
|
|
Social circumstances
Suicidal ideation
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
|
Endocrine disorders
Thyrotoxic crisis
|
0.85%
1/118 • Number of events 1
|
0.00%
0/113
|
100.0%
1/1 • Number of events 1
|
Other adverse events
| Measure |
Non-Titrated
n=118 participants at risk
New formulation of rebif
|
Titrated
n=113 participants at risk
New formulation of rebif
|
All New Formulation of Rebif Subjects
n=231 participants at risk
All subjects combined in Intent to Treat (ITT) Population
|
|---|---|---|---|
|
General disorders
Chills
|
7.6%
9/118 • Number of events 9
|
3.5%
4/113 • Number of events 4
|
5.6%
13/231 • Number of events 13
|
|
Nervous system disorders
Headache
|
32.2%
38/118 • Number of events 38
|
24.8%
28/113 • Number of events 28
|
28.6%
66/231 • Number of events 66
|
|
General disorders
Influenza-like Illness
|
9.3%
11/118 • Number of events 11
|
8.0%
9/113 • Number of events 9
|
8.7%
20/231 • Number of events 20
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
11.0%
13/118 • Number of events 13
|
0.88%
1/113 • Number of events 1
|
6.1%
14/231 • Number of events 14
|
|
Respiratory, thoracic and mediastinal disorders
Nasopharyngitis
|
7.6%
9/118 • Number of events 9
|
8.0%
9/113 • Number of events 9
|
7.8%
18/231 • Number of events 18
|
|
General disorders
Pain
|
16.9%
20/118 • Number of events 20
|
14.2%
16/113 • Number of events 16
|
15.6%
36/231 • Number of events 36
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Tract Infection
|
6.8%
8/118 • Number of events 8
|
3.5%
4/113 • Number of events 4
|
5.2%
12/231 • Number of events 12
|
|
Renal and urinary disorders
Urinary tract infection
|
5.1%
6/118 • Number of events 6
|
8.0%
9/113 • Number of events 9
|
6.5%
15/231 • Number of events 15
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER