Trial Outcomes & Findings for Kuvan in People With Schizophrenia and Schizoaffective Disorder (NCT NCT01706965)
NCT ID: NCT01706965
Last Updated: 2017-09-13
Results Overview
This is a is a 30 item rating scale widely used in the assessment of schizophrenia ranging from 30-210. Higher scores are worse
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
27 participants
Primary outcome timeframe
Baseline (start of Kuvan) and at six weeks of treatment
Results posted on
2017-09-13
Participant Flow
Participant milestones
| Measure |
Kuvan
Kuvan once-daily dosing initiated with 10mg/kg for the first week followed by 20mg/kg for the remaining weeks of the study
Kuvan: 20mg/kg using a daily dosing schedule. To increase tolerability, treatment will be initiated at 10mg/kg for the first week of the study. Given the short length of the trial, no dose adjustments will be made for changes in weight
|
Multivitamin
Daily multivitamin tablet
Multivitamin: Multi-vitamin will be used as an active control in this study. Will be dosed daily
|
|---|---|---|
|
Overall Study
STARTED
|
14
|
13
|
|
Overall Study
COMPLETED
|
13
|
12
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Kuvan in People With Schizophrenia and Schizoaffective Disorder
Baseline characteristics by cohort
| Measure |
Kuvan
n=13 Participants
Kuvan once-daily dosing initiated with 10mg/kg for the first week followed by 20mg/kg for the remaining weeks of the study
Kuvan: 20mg/kg using a daily dosing schedule. To increase tolerability, treatment will be initiated at 10mg/kg for the first week of the study. Given the short length of the trial, no dose adjustments will be made for changes in weight
|
Multivitamin
n=12 Participants
Daily multivitamin tablet
Multivitamin: Multi-vitamin will be used as an active control in this study. Will be dosed daily
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
40.15 years
STANDARD_DEVIATION 12.1 • n=5 Participants
|
46.1 years
STANDARD_DEVIATION 8.0 • n=7 Participants
|
43.0 years
STANDARD_DEVIATION 10.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline (start of Kuvan) and at six weeks of treatmentThis is a is a 30 item rating scale widely used in the assessment of schizophrenia ranging from 30-210. Higher scores are worse
Outcome measures
| Measure |
Kuvan
n=13 Participants
Kuvan once-daily dosing initiated with 10mg/kg for the first week followed by 20mg/kg for the remaining weeks of the study
Kuvan: 20mg/kg using a daily dosing schedule. To increase tolerability, treatment will be initiated at 10mg/kg for the first week of the study. Given the short length of the trial, no dose adjustments will be made for changes in weight
|
Multivitamin
n=12 Participants
Daily multivitamin tablet
Multivitamin: Multi-vitamin will be used as an active control in this study. Will be dosed daily
|
|---|---|---|
|
Final Positive and Negative Symptom Scale (PANSS)
|
54.4 PANSS Total
Standard Deviation 10.9
|
60.2 PANSS Total
Standard Deviation 13.7
|
PRIMARY outcome
Timeframe: Baseline to week 6MATRICS assessing 7 domains (Speed of Processing, Attention/Vigilance, Working Memory, Verbal Learning, Visual Learning, Reasoning and Problem Solving, and Social Cognition. Raw scores are converted into a composite T-score (normative mean = 50; standard deviation = 10), where higher values indicated less impairment.
Outcome measures
| Measure |
Kuvan
n=13 Participants
Kuvan once-daily dosing initiated with 10mg/kg for the first week followed by 20mg/kg for the remaining weeks of the study
Kuvan: 20mg/kg using a daily dosing schedule. To increase tolerability, treatment will be initiated at 10mg/kg for the first week of the study. Given the short length of the trial, no dose adjustments will be made for changes in weight
|
Multivitamin
n=12 Participants
Daily multivitamin tablet
Multivitamin: Multi-vitamin will be used as an active control in this study. Will be dosed daily
|
|---|---|---|
|
Final MATRICS Cognitive Battery
|
33.92 Total MATRICS score
Standard Deviation 17.1
|
35.0 Total MATRICS score
Standard Deviation 12.5
|
Adverse Events
Kuvan
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Multivitamin
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Kuvan
n=13 participants at risk
Kuvan once-daily dosing initiated with 10mg/kg for the first week followed by 20mg/kg for the remaining weeks of the study
Kuvan: 20mg/kg using a daily dosing schedule. To increase tolerability, treatment will be initiated at 10mg/kg for the first week of the study. Given the short length of the trial, no dose adjustments will be made for changes in weight
|
Multivitamin
n=12 participants at risk
Daily multivitamin tablet
Multivitamin: Multi-vitamin will be used as an active control in this study. Will be dosed daily
|
|---|---|---|
|
Gastrointestinal disorders
constipation
|
15.4%
2/13 • Number of events 2
|
25.0%
3/12 • Number of events 3
|
|
General disorders
Dizziness
|
30.8%
4/13 • Number of events 4
|
8.3%
1/12 • Number of events 1
|
|
Renal and urinary disorders
nocturnal enuresis
|
7.7%
1/13 • Number of events 1
|
0.00%
0/12
|
|
Gastrointestinal disorders
Stomach disconfort
|
15.4%
2/13 • Number of events 2
|
0.00%
0/12
|
|
Skin and subcutaneous tissue disorders
bruising
|
7.7%
1/13 • Number of events 1
|
0.00%
0/12
|
|
Gastrointestinal disorders
dry mouth
|
7.7%
1/13 • Number of events 1
|
8.3%
1/12 • Number of events 1
|
|
Gastrointestinal disorders
nausea
|
7.7%
1/13 • Number of events 1
|
8.3%
1/12 • Number of events 1
|
|
Psychiatric disorders
restlessnes
|
7.7%
1/13 • Number of events 1
|
16.7%
2/12 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
stiffness
|
7.7%
1/13 • Number of events 1
|
0.00%
0/12
|
|
Infections and infestations
URI
|
7.7%
1/13 • Number of events 1
|
0.00%
0/12
|
|
Respiratory, thoracic and mediastinal disorders
Difficulty speaking
|
7.7%
1/13 • Number of events 1
|
0.00%
0/12
|
|
Respiratory, thoracic and mediastinal disorders
sore throat
|
7.7%
1/13 • Number of events 1
|
16.7%
2/12 • Number of events 2
|
|
Gastrointestinal disorders
diarrhea
|
0.00%
0/13
|
25.0%
3/12 • Number of events 3
|
|
Gastrointestinal disorders
headache
|
0.00%
0/13
|
16.7%
2/12 • Number of events 2
|
|
Immune system disorders
urtiacia
|
0.00%
0/13
|
8.3%
1/12 • Number of events 1
|
|
Reproductive system and breast disorders
Vaginal Yeast infection
|
0.00%
0/13
|
8.3%
1/12 • Number of events 1
|
|
Eye disorders
Eye swelling
|
0.00%
0/13
|
8.3%
1/12 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
hand pain
|
0.00%
0/13
|
8.3%
1/12 • Number of events 1
|
|
Psychiatric disorders
auditory hallucinatiuons
|
0.00%
0/13
|
8.3%
1/12 • Number of events 1
|
|
Gastrointestinal disorders
anorexia
|
0.00%
0/13
|
8.3%
1/12 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place