Trial Outcomes & Findings for Comparative Effectiveness Study for Bipolar Disorder (NCT NCT01331304)

NCT ID: NCT01331304

Last Updated: 2018-04-26

Results Overview

The CGI-EI integrates benefits and harms and yields a score that can be compared across interventions. It is made up of 2 subscales: therapeutic effects and side effects. Each rating is on a scale from 1 to 4. To combine these two subscales into the CGI-EI we report as our primary outcome, we subtracted the side effects subscale from the therapeutic effects subscale. Thus, the CGI-EI we report ranges the integers from -3 to +3 (i.e. possible scores are -3,-2,-1,0,1,2,3). A score of -3 is the most burdensome side effect score (4) and the least therapeutic effect score (1) and a score of +3 is the least burdensome side effect score (1) and the highest therapeutic effect score (4). Higher CGI-EI signifies better outcome (minimal side effects, maximal therapeutic effect). Lower CGI-EI signifies worse outcome (maximal side effects, minimal therapeutic effect).To compute CGI-EI score, we subtract the side effect score from the therapeutic effect score.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

482 participants

Primary outcome timeframe

Average 6 month score minus Average baseline score

Results posted on

2018-04-26

Participant Flow

Participant milestones

Participant milestones
Measure
Li + APT
Study participants will take lithium in addition to any other medications recommended by the study physician. Lithium: 600-1200mg per day over 6 months
QTP + APT
Study participants will take quetiapine in addition to any other medications recommended by the study physician. Quetiapine: 100-800mg a day over 6 months
Overall Study
STARTED
240
242
Overall Study
COMPLETED
182
182
Overall Study
NOT COMPLETED
58
60

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Comparative Effectiveness Study for Bipolar Disorder

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Li + APT
n=240 Participants
Study participants will take lithium in addition to any other medications recommended by the study physician. Lithium: 600-1200mg per day over 6 months
QTP + APT
n=242 Participants
Study participants will take quetiapine in addition to any other medications recommended by the study physician. Quetiapine: 100-800mg a day over 6 months
Total
n=482 Participants
Total of all reporting groups
Age, Continuous
38.6 Years
STANDARD_DEVIATION 12.1 • n=93 Participants
39.1 Years
STANDARD_DEVIATION 12.2 • n=4 Participants
38.9 Years
STANDARD_DEVIATION 12.1 • n=27 Participants
Sex: Female, Male
Female
140 Participants
n=93 Participants
143 Participants
n=4 Participants
283 Participants
n=27 Participants
Sex: Female, Male
Male
100 Participants
n=93 Participants
99 Participants
n=4 Participants
199 Participants
n=27 Participants
Region of Enrollment
United States
240 participants
n=93 Participants
242 participants
n=4 Participants
482 participants
n=27 Participants

PRIMARY outcome

Timeframe: Average 6 month score minus Average baseline score

The CGI-EI integrates benefits and harms and yields a score that can be compared across interventions. It is made up of 2 subscales: therapeutic effects and side effects. Each rating is on a scale from 1 to 4. To combine these two subscales into the CGI-EI we report as our primary outcome, we subtracted the side effects subscale from the therapeutic effects subscale. Thus, the CGI-EI we report ranges the integers from -3 to +3 (i.e. possible scores are -3,-2,-1,0,1,2,3). A score of -3 is the most burdensome side effect score (4) and the least therapeutic effect score (1) and a score of +3 is the least burdensome side effect score (1) and the highest therapeutic effect score (4). Higher CGI-EI signifies better outcome (minimal side effects, maximal therapeutic effect). Lower CGI-EI signifies worse outcome (maximal side effects, minimal therapeutic effect).To compute CGI-EI score, we subtract the side effect score from the therapeutic effect score.

Outcome measures

Outcome measures
Measure
Li + APT
n=240 Participants
Study participants will take lithium in addition to any other medications recommended by the study physician. Lithium: 600-1200mg per day over 6 months
QTP + APT
n=242 Participants
Study participants will take quetiapine in addition to any other medications recommended by the study physician. Quetiapine: 100-800mg a day over 6 months
Clinical Global Impression-Efficacy Index (CGI-EI)
1.58 Units on the scale
Interval 1.32 to 1.84
1.52 Units on the scale
Interval 1.26 to 1.78

PRIMARY outcome

Timeframe: 6 Months

Necessary Clinical Adjustment (NCA): The Medication Recommendation Tracking Form was developed and successfully implemented in a previous study to capture recommended medication changes at each study visit 17. Clinicians record dosage changes, missed doses, new medications added or discontinued, and specify the reason for each change. Any change in psychotropic medications, or medications used to treat side effects, is coded along with the reason for the change. NCAs include those changes made for lack of effectiveness or intolerance, but not changes for planned dose titrations.

Outcome measures

Outcome measures
Measure
Li + APT
n=240 Participants
Study participants will take lithium in addition to any other medications recommended by the study physician. Lithium: 600-1200mg per day over 6 months
QTP + APT
n=242 Participants
Study participants will take quetiapine in addition to any other medications recommended by the study physician. Quetiapine: 100-800mg a day over 6 months
Necessary Clinical Adjustments
.8 Mean NCAs per month
Standard Deviation .8
.9 Mean NCAs per month
Standard Deviation 1.0

SECONDARY outcome

Timeframe: Average baseline score minus Average 6 month score

The Framingham risk score captures the classic risk factors for cardiovascular disease, including age, sex, systolic blood pressure, total and high density lipoprotein cholesterol, diabetes mellitus, and smoking. The Framingham risk score is used as a simple predictive tool to determine 10-year (short term) risk for developing cardiovascular disease (CHD), with higher scores indicating higher risk. Established benchmarks exist for scores from 0 to 25--though it can exceed this value--that are meant to translate to the probability of developing heart disease.

Outcome measures

Outcome measures
Measure
Li + APT
n=240 Participants
Study participants will take lithium in addition to any other medications recommended by the study physician. Lithium: 600-1200mg per day over 6 months
QTP + APT
n=242 Participants
Study participants will take quetiapine in addition to any other medications recommended by the study physician. Quetiapine: 100-800mg a day over 6 months
Risk of Cardiovascular Disease - Framingham Risk Score
-0.26 units on a scale
Interval -0.66 to 0.14
0.17 units on a scale
Interval -0.21 to 0.54

SECONDARY outcome

Timeframe: Average baseline score minus Average 6-month score

The LIFE-RIFT asses the extent to which psychopathology has impacted current functioning in work, household chores, interpersonal relationships with partner, family, and friends, recreational activities, and life, satisfaction, leisure activities and social relationships. Summary scores can range from 4 to 20, with higher scores indicating greater functional impairment.

Outcome measures

Outcome measures
Measure
Li + APT
n=240 Participants
Study participants will take lithium in addition to any other medications recommended by the study physician. Lithium: 600-1200mg per day over 6 months
QTP + APT
n=242 Participants
Study participants will take quetiapine in addition to any other medications recommended by the study physician. Quetiapine: 100-800mg a day over 6 months
Longitudinal Interval Follow up Evaluation Range of Impaired Functioning Tool (LIFE-RIFT)
-3.74 units on a scale
Interval -4.29 to -3.19
-3.61 units on a scale
Interval -4.15 to -3.07

Adverse Events

Li + APT

Serious events: 36 serious events
Other events: 210 other events
Deaths: 0 deaths

QTP + APT

Serious events: 27 serious events
Other events: 221 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Li + APT
n=240 participants at risk
Study participants will take lithium in addition to any other medications recommended by the study physician. Lithium: 600-1200mg per day over 6 months
QTP + APT
n=242 participants at risk
Study participants will take quetiapine in addition to any other medications recommended by the study physician. Quetiapine: 100-800mg a day over 6 months
Pregnancy, puerperium and perinatal conditions
Pregnancy
1.2%
3/240 • Number of events 3 • Six months
0.41%
1/242 • Number of events 1 • Six months
Psychiatric disorders
Psychiatric Hospitalization
7.5%
18/240 • Number of events 23 • Six months
5.8%
14/242 • Number of events 19 • Six months
Injury, poisoning and procedural complications
Car/Bike Accident
0.83%
2/240 • Number of events 2 • Six months
0.41%
1/242 • Number of events 1 • Six months
Psychiatric disorders
Suicide
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
Cardiac disorders
Death
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
Surgical and medical procedures
Laparoscopic Cholecystectomy
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
General disorders
Alcoholism Treatment
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
Gastrointestinal disorders
Gastrointestinal Issues
0.83%
2/240 • Number of events 2 • Six months
0.00%
0/242 • Six months
Renal and urinary disorders
Kidney Infection
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
General disorders
Heroin Overdose
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
Skin and subcutaneous tissue disorders
Eczema Outbreak
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
General disorders
Hospitalization reason unknown
0.00%
0/240 • Six months
0.41%
1/242 • Number of events 1 • Six months
Injury, poisoning and procedural complications
Broken Bones
0.00%
0/240 • Six months
0.83%
2/242 • Number of events 2 • Six months
Injury, poisoning and procedural complications
Fall
0.00%
0/240 • Six months
0.83%
2/242 • Number of events 2 • Six months
Infections and infestations
Flu Hospitalization
0.00%
0/240 • Six months
0.41%
1/242 • Number of events 1 • Six months
General disorders
Extreme Sedation Hospitalization
0.00%
0/240 • Six months
0.41%
1/242 • Number of events 1 • Six months
Musculoskeletal and connective tissue disorders
Muscle Spasms Hospitalization
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
Hepatobiliary disorders
Biliary Cirrhosis
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
General disorders
Study Medication Overdose
0.42%
1/240 • Number of events 1 • Six months
0.83%
2/242 • Number of events 2 • Six months
Nervous system disorders
Pseudoseizure
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Cyst
0.00%
0/240 • Six months
0.41%
1/242 • Number of events 1 • Six months
Infections and infestations
Staph Infection
0.00%
0/240 • Six months
0.41%
1/242 • Number of events 1 • Six months
General disorders
Dizziness
0.42%
1/240 • Number of events 1 • Six months
0.00%
0/242 • Six months

Other adverse events

Other adverse events
Measure
Li + APT
n=240 participants at risk
Study participants will take lithium in addition to any other medications recommended by the study physician. Lithium: 600-1200mg per day over 6 months
QTP + APT
n=242 participants at risk
Study participants will take quetiapine in addition to any other medications recommended by the study physician. Quetiapine: 100-800mg a day over 6 months
General disorders
Excessive sleepiness/daytime somnolence
28.3%
68/240 • Number of events 96 • Six months
76.9%
186/242 • Number of events 249 • Six months
Nervous system disorders
Headache
20.0%
48/240 • Number of events 61 • Six months
13.6%
33/242 • Number of events 43 • Six months
General disorders
Dry Mouth
12.1%
29/240 • Number of events 32 • Six months
21.1%
51/242 • Number of events 58 • Six months
Gastrointestinal disorders
Nausea
24.2%
58/240 • Number of events 70 • Six months
6.2%
15/242 • Number of events 17 • Six months
General disorders
Increased Weight
10.4%
25/240 • Number of events 31 • Six months
18.6%
45/242 • Number of events 55 • Six months
Musculoskeletal and connective tissue disorders
Tremor
26.2%
63/240 • Number of events 69 • Six months
5.4%
13/242 • Number of events 13 • Six months
General disorders
Increased Appetite
8.3%
20/240 • Number of events 22 • Six months
22.7%
55/242 • Number of events 56 • Six months
Nervous system disorders
Memory/concentration problems
11.2%
27/240 • Number of events 32 • Six months
8.7%
21/242 • Number of events 25 • Six months
Nervous system disorders
Dizziness/Lightheaded
8.8%
21/240 • Number of events 24 • Six months
9.5%
23/242 • Number of events 30 • Six months
Gastrointestinal disorders
Upset stomach/stomach pain/bloating
12.9%
31/240 • Number of events 36 • Six months
5.0%
12/242 • Number of events 13 • Six months
Gastrointestinal disorders
Diarrhea
13.3%
32/240 • Number of events 38 • Six months
2.9%
7/242 • Number of events 10 • Six months
Gastrointestinal disorders
Constipation
7.9%
19/240 • Number of events 23 • Six months
9.1%
22/242 • Number of events 23 • Six months
General disorders
Increased Thirst
12.1%
29/240 • Number of events 32 • Six months
1.7%
4/242 • Number of events 4 • Six months
Renal and urinary disorders
Increased Urinary Frequency
13.3%
32/240 • Number of events 33 • Six months
0.83%
2/242 • Number of events 3 • Six months
Musculoskeletal and connective tissue disorders
Stiffness/muscle aches
5.4%
13/240 • Number of events 14 • Six months
5.8%
14/242 • Number of events 14 • Six months
Musculoskeletal and connective tissue disorders
Weakness/muscle fatigue
4.6%
11/240 • Number of events 12 • Six months
5.8%
14/242 • Number of events 15 • Six months
General disorders
Restless/fidgety (akathisia)
2.9%
7/240 • Number of events 7 • Six months
6.2%
15/242 • Number of events 17 • Six months
Respiratory, thoracic and mediastinal disorders
Respiratory infection or other cold-like symptoms
5.0%
12/240 • Number of events 12 • Six months
5.0%
12/242 • Number of events 12 • Six months
Musculoskeletal and connective tissue disorders
Muscle twitching (myoclonus)
2.5%
6/240 • Number of events 6 • Six months
5.8%
14/242 • Number of events 14 • Six months
Skin and subcutaneous tissue disorders
Hair Loss
6.7%
16/240 • Number of events 16 • Six months
1.7%
4/242 • Number of events 4 • Six months
Eye disorders
Blurred Vision
5.0%
12/240 • Number of events 15 • Six months
2.5%
6/242 • Number of events 7 • Six months
General disorders
Insomnia
5.0%
12/240 • Number of events 12 • Six months
2.1%
5/242 • Number of events 7 • Six months

Additional Information

Dr. Andrew Nierenberg

Massachusetts General Hospital

Phone: 617-724-0837

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place