Trial Outcomes & Findings for Desmopressin for Bedwetting in Children With SCD (NCT NCT04420585)
NCT ID: NCT04420585
Last Updated: 2024-05-07
Results Overview
Change in the percentage of nights with bedwetting episodes assessed at baseline based on participant recall over the previous 14 nights, as well as at \~1 month (30 nights) based on completion of a study (enuresis) diary. This is used to assess whether the use of desmopressin in patients with sickle cell disease and nocturnal enuresis will decrease the number of nighttime episodes of enuresis by 50% after initiating DDAVP at 0.4 mg nightly dose (with dose escalation as clinically appropriate). A negative value indicates a decrease in bedwetting episodes.
TERMINATED
PHASE4
8 participants
Baseline and ~1 month
2024-05-07
Participant Flow
Participant milestones
| Measure |
Treatment Group
Desmopressin 0.2mg tablets, dose titrated to effect
NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination.
Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if \<50 % improvement
|
|---|---|
|
Overall Study
STARTED
|
8
|
|
Overall Study
COMPLETED
|
5
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Treatment Group
Desmopressin 0.2mg tablets, dose titrated to effect
NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination.
Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if \<50 % improvement
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Never initiated medication
|
2
|
Baseline Characteristics
6 participants had Sickle Cell Anemia (Hb SS) and two participants had Sickle Hemoglobin-C (Hb SC)
Baseline characteristics by cohort
| Measure |
Treatment Group
n=8 Participants
Desmopressin 0.2mg tablets, dose titrated to effect
NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination.
Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if \<50 % improvement
|
|---|---|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Black or African American
|
8 Participants
n=8 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=8 Participants
|
|
Region of Enrollment
United States
|
8 Participants
n=8 Participants
|
|
Genotype of Sickle Cell Disease
Sickle Cell Anemia (Hb SS)
|
6 Participants
n=8 Participants
|
|
Genotype of Sickle Cell Disease
Sickle Hemoglobin-C Disease (Hb SC)
|
2 Participants
n=8 Participants
|
|
Number of Bedwetting Days Based on Disease Type
Sickle Cell Anemia (Hb SS)
|
10.5 days
STANDARD_DEVIATION 4.0 • n=6 Participants • 6 participants had Sickle Cell Anemia (Hb SS) and two participants had Sickle Hemoglobin-C (Hb SC)
|
|
Age, Continuous
|
12.1 years
STANDARD_DEVIATION 2.7 • n=8 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=8 Participants
|
|
Number of Bedwetting Days Based on Disease Type
Sickle Hemoglobin-C Disease (Hb SC)
|
8.5 days
STANDARD_DEVIATION 0.7 • n=2 Participants • 6 participants had Sickle Cell Anemia (Hb SS) and two participants had Sickle Hemoglobin-C (Hb SC)
|
PRIMARY outcome
Timeframe: Baseline and ~1 monthChange in the percentage of nights with bedwetting episodes assessed at baseline based on participant recall over the previous 14 nights, as well as at \~1 month (30 nights) based on completion of a study (enuresis) diary. This is used to assess whether the use of desmopressin in patients with sickle cell disease and nocturnal enuresis will decrease the number of nighttime episodes of enuresis by 50% after initiating DDAVP at 0.4 mg nightly dose (with dose escalation as clinically appropriate). A negative value indicates a decrease in bedwetting episodes.
Outcome measures
| Measure |
Treatment Group
n=5 Participants
Desmopressin 0.2mg tablets, dose titrated to effect
NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination.
Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if \<50 % improvement
|
|---|---|
|
Change in Bedwetting Episodes
|
-41 percentage of nights
Standard Deviation 26.2
|
SECONDARY outcome
Timeframe: Baseline and 4 weeksPopulation: Data was not collected for the Impact of Bedwetting on day-to-day activities.
To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have an improved quality of life compared to their baseline. This will be measured using the PedsQL Measurement Model which measures health related quality of life in children with acute and chronic health conditions, like sickle cell. The scales focuses on areas such as activities, feelings, and school performance.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and ~1 monthChange in the percentage of nights with awakenings over the previous 14 nights was assessed by summarizing and comparing baseline and 1 month nighttime awakening data. The percentage of nights with nighttime awakenings was evaluated at baseline based on recall and at 1 month (30 nights) based on completion of a study diary. This was used to determine whether the use of desmopressin in patients with sickle cell disease and nocturnal enuresis changes the rates of nighttime awakenings to urinate (nocturia), defined as episodes of nighttime awakening to void in children ≥5 years of age, compared to prior to initiating treatment with DDAVP. A positive value indicates an increase in nighttime awakenings
Outcome measures
| Measure |
Treatment Group
n=5 Participants
Desmopressin 0.2mg tablets, dose titrated to effect
NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination.
Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if \<50 % improvement
|
|---|---|
|
Change in Nighttime Awakenings
|
16.8 percentage of nights
Standard Deviation 25.3
|
SECONDARY outcome
Timeframe: Baseline and ~1 monthPopulation: 1 month fatigue data was not able to collected from one participant who did not complete the 1 month PROMIS Fatigue questionnaire. Accordingly, change results were not available.
Change in daytime fatigue will be assessed to determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have less daytime fatigue compared to their baseline data. The PROMIS Pediatric Fatigue Short Form (Version 2.0) will be used to compare levels of fatigue from baseline and \~1 month (30 nights) on the study medication. The PROMIS Fatigue scale, which utilizes a 7-day recall period, is a 10-item questionnaire consisting of 5 responses ranging from 1-5 with one indicative of "Never" and five indicative of "almost always" resulting in a raw scoring range of 10-50. Negative PROMIS change scores are associated with decreased levels of fatigue.
Outcome measures
| Measure |
Treatment Group
n=5 Participants
Desmopressin 0.2mg tablets, dose titrated to effect
NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination.
Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if \<50 % improvement
|
|---|---|
|
Change in Daytime Fatigue
|
-6.0 score on a scale
Interval -18.0 to 6.0
|
Adverse Events
Treatment Group
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment Group
n=8 participants at risk
Desmopressin 0.2mg tablets, dose titrated to effect
NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination.
Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if \<50 % improvement
|
|---|---|
|
Nervous system disorders
Dizziness
|
12.5%
1/8 • Number of events 1 • 4 weeks after initiation of medication
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place