Trial Outcomes & Findings for Rhode Island Diastolic Dysfunction - Heart Failure (NCT NCT02353312)
NCT ID: NCT02353312
Last Updated: 2025-07-03
Results Overview
The change from baseline in oxygen consumption during maximal bike exercise. This was measured using cardiopulmonary exercise tress test (CPET), recorded in milliliters per kilogram per minute (mL/kg/min). Higher oxygen consumption scores indicate better aerobic capacity and cardiovascular function. An increase in oxygen consumption from baseline to post-intervention signifies an improvement in these areas. Conversely, a decrease would indicate a decline in aerobic capacity and cardiovascular health.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
28 participants
Primary outcome timeframe
Baseline (period 0), 3 mos (period 1), 6 mos (period 2)
Results posted on
2025-07-03
Participant Flow
Participant milestones
| Measure |
Kuvan First, Then no Kuvan
Kuvan® supplementation in addition to standard care for heart failure for three months. At the end of three months, stop Kuvan®, patients will only receive Standard care for heart failure for another 3 months
Kuvan: Kuvan® (sapropterin dihydrochloride) will be initiated at 10mg/kg/day with meals for one week. After telephone contact on day 7, assuming no adverse effects are noted, the patient will be instructed to increase their daily dose to 20mg/kg/day with meals for the remainder of the 3 months.
|
No Kuvan First, Then Kuvan
Standard care for heart failure for three months. At the end of three months, Starting Kuvan® supplementation in addition to Standard care for heart failure for another 3 months
Kuvan: Kuvan® (sapropterin dihydrochloride) will be initiated at 10mg/kg/day with meals for one week. After telephone contact on day 7, assuming no adverse effects are noted, the patient will be instructed to increase their daily dose to 20mg/kg/day with meals for the remainder of the 3 months.
|
|---|---|---|
|
Overall Study
STARTED
|
16
|
12
|
|
Overall Study
COMPLETED
|
13
|
10
|
|
Overall Study
NOT COMPLETED
|
3
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Rhode Island Diastolic Dysfunction - Heart Failure
Baseline characteristics by cohort
| Measure |
Kuvan First, Then no Kuvan
n=16 Participants
Kuvan® supplementation in addition to standard care for heart failure for three months. At the end of three months, stop Kuvan®, patients will only receive Standard care for heart failure for another 3 months
Kuvan: Kuvan® (sapropterin dihydrochloride) will be initiated at 10mg/kg/day with meals for one week. After telephone contact on day 7, assuming no adverse effects are noted, the patient will be instructed to increase their daily dose to 20mg/kg/day with meals for the remainder of the 3 months.
|
No Kuvan First, Then Kuvan
n=12 Participants
Standard care for heart failure for three months. At the end of three months, Starting Kuvan® supplementation in addition to Standard care for heart failure for another 3 months
Kuvan: Kuvan® (sapropterin dihydrochloride) will be initiated at 10mg/kg/day with meals for one week. After telephone contact on day 7, assuming no adverse effects are noted, the patient will be instructed to increase their daily dose to 20mg/kg/day with meals for the remainder of the 3 months.
|
Total
n=28 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
70.4 years
STANDARD_DEVIATION 9.78 • n=5 Participants
|
72.1 years
STANDARD_DEVIATION 5.85 • n=7 Participants
|
71.4 years
STANDARD_DEVIATION 8.23 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
26 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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 (period 0), 3 mos (period 1), 6 mos (period 2)Population: All patients who had both baseline and final test.
The change from baseline in oxygen consumption during maximal bike exercise. This was measured using cardiopulmonary exercise tress test (CPET), recorded in milliliters per kilogram per minute (mL/kg/min). Higher oxygen consumption scores indicate better aerobic capacity and cardiovascular function. An increase in oxygen consumption from baseline to post-intervention signifies an improvement in these areas. Conversely, a decrease would indicate a decline in aerobic capacity and cardiovascular health.
Outcome measures
| Measure |
On Kuvan
n=20 Participants
Kuvan® supplementation in addition to standard care for heart failure in either the first or last three months. Kuvan® (sapropterin dihydrochloride) will be initiated at 10mg/kg/day with meals for one week. After telephone contact on day 7, assuming no adverse effects are noted, the patient will be instructed to increase their daily dose to 20mg/kg/day with meals for the remainder of the 3 months.
|
Not on Kuvan
n=22 Participants
Standard care for heart failure in either the first or last three months.
|
|---|---|---|
|
Change From Baseline in Oxygen Consumption During Maximal Bike Exercise
|
14.45 mL/kg/min
Standard Deviation 5.00
|
14.18 mL/kg/min
Standard Deviation 3.69
|
Adverse Events
On Kuvan
Serious events: 4 serious events
Other events: 28 other events
Deaths: 0 deaths
Off Kuvan
Serious events: 6 serious events
Other events: 28 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
On Kuvan
n=28 participants at risk
Kuvan® supplementation in addition to standard care for heart failure for three months. At the end of three months, stop Kuvan®, patients will only receive Standard care for heart failure for another 3 months
Kuvan: Kuvan® (sapropterin dihydrochloride) will be initiated at 10mg/kg/day with meals for one week. After telephone contact on day 7, assuming no adverse effects are noted, the patient will be instructed to increase their daily dose to 20mg/kg/day with meals for the remainder of the 3 months.
|
Off Kuvan
n=28 participants at risk
Standard care for heart failure for three months. At the end of three months, Starting Kuvan® supplementation in addition to Standard care for heart failure for another 3 months
|
|---|---|---|
|
Investigations
Hospitalizations
|
7.1%
2/28 • Number of events 2 • 6 months
There were 28 patients enrolled.
|
10.7%
3/28 • Number of events 3 • 6 months
There were 28 patients enrolled.
|
|
Investigations
ER visit
|
7.1%
2/28 • Number of events 2 • 6 months
There were 28 patients enrolled.
|
10.7%
3/28 • Number of events 5 • 6 months
There were 28 patients enrolled.
|
Other adverse events
| Measure |
On Kuvan
n=28 participants at risk
Kuvan® supplementation in addition to standard care for heart failure for three months. At the end of three months, stop Kuvan®, patients will only receive Standard care for heart failure for another 3 months
Kuvan: Kuvan® (sapropterin dihydrochloride) will be initiated at 10mg/kg/day with meals for one week. After telephone contact on day 7, assuming no adverse effects are noted, the patient will be instructed to increase their daily dose to 20mg/kg/day with meals for the remainder of the 3 months.
|
Off Kuvan
n=28 participants at risk
Standard care for heart failure for three months. At the end of three months, Starting Kuvan® supplementation in addition to Standard care for heart failure for another 3 months
|
|---|---|---|
|
Infections and infestations
Fever
|
3.6%
1/28 • Number of events 2 • 6 months
There were 28 patients enrolled.
|
10.7%
3/28 • Number of events 5 • 6 months
There were 28 patients enrolled.
|
|
Gastrointestinal disorders
Nausea
|
14.3%
4/28 • Number of events 5 • 6 months
There were 28 patients enrolled.
|
7.1%
2/28 • Number of events 3 • 6 months
There were 28 patients enrolled.
|
|
Gastrointestinal disorders
Emesis
|
0.00%
0/28 • 6 months
There were 28 patients enrolled.
|
0.00%
0/28 • 6 months
There were 28 patients enrolled.
|
|
Gastrointestinal disorders
Diarrhea
|
42.9%
12/28 • Number of events 20 • 6 months
There were 28 patients enrolled.
|
28.6%
8/28 • Number of events 17 • 6 months
There were 28 patients enrolled.
|
|
Blood and lymphatic system disorders
Rhinorrhea
|
50.0%
14/28 • Number of events 17 • 6 months
There were 28 patients enrolled.
|
25.0%
7/28 • Number of events 8 • 6 months
There were 28 patients enrolled.
|
|
General disorders
weight gain
|
42.9%
12/28 • Number of events 19 • 6 months
There were 28 patients enrolled.
|
42.9%
12/28 • Number of events 16 • 6 months
There were 28 patients enrolled.
|
|
Vascular disorders
JVD
|
14.3%
4/28 • Number of events 5 • 6 months
There were 28 patients enrolled.
|
21.4%
6/28 • Number of events 6 • 6 months
There were 28 patients enrolled.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary crackles
|
3.6%
1/28 • Number of events 1 • 6 months
There were 28 patients enrolled.
|
3.6%
1/28 • Number of events 1 • 6 months
There were 28 patients enrolled.
|
|
Vascular disorders
Peripheral abdominal edema
|
75.0%
21/28 • Number of events 34 • 6 months
There were 28 patients enrolled.
|
57.1%
16/28 • Number of events 27 • 6 months
There were 28 patients enrolled.
|
|
Vascular disorders
Other
|
53.6%
15/28 • Number of events 21 • 6 months
There were 28 patients enrolled.
|
60.7%
17/28 • Number of events 25 • 6 months
There were 28 patients enrolled.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place