Primary outcomeMean· Baseline and ~1 month
Change in Bedwetting Episodes
| Group | Value (percentage of nights) | Spread |
|---|---|---|
| Treatment Group | -41 | 26.2 |
Trial · Interventional
TerminatedPhase 4Results postedRegistry dataDesmopressin for Bedwetting in Children With SCD
NCTNCT04420585 (opens ClinicalTrials.gov in a new tab)Other id2020-11268
This trial was stopped early.
Reason given at the registry: Study was not able to meet recruitment after 9 years of trying to enroll patients on different versions of the protocol and changing recruitment strategies.
Results were posted for this trial.
Overview
- Status
- Terminated
- Phase
- Phase 4
- Enrollment
- 8 participants (actual)
- Sponsor
- Montefiore Medical Center · Other
- Design
- Allocation not applicable · Open label (no masking)
- Countries
1 country
United States
- Start
- 7 Jul 2020
- Primary completion
- 12 Jun 2023
- Completion
- 12 Jun 2023
- First posted
- 9 Jun 2020
- Last update posted
- 7 May 2024
- Conditions
- Nocturnal Enuresis · Anemia, Sickle Cell
Timeline
1 trial · 1 stopped early · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
20202021202220232024
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
Interventions
- DesmopressinDrug
Arms1
Experimental
Treatment Group
Desmopressin 0.2mg tablets, dose titrated to effect
Primary outcomes1
Outcomes the sponsor listed as primary.
Change in Bedwetting Episodes
Time frame · Baseline and ~1 month
Registry description
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.
Secondary outcomes(3)
Impact of Bedwetting on Day to Day Activities
Time frame · Baseline and 4 weeks
Registry description
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.
Change in Nighttime Awakenings
Time frame · Baseline and ~1 month
Registry description
Change 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
Change in Daytime Fatigue
Time frame · Baseline and ~1 month
Registry description
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.
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(3)
Secondary outcome· Baseline and 4 weeks
Impact of Bedwetting on Day to Day Activities
| Group | Value |
|---|---|
| Treatment Group | not posted |
Secondary outcomeMean· Baseline and ~1 month
Change in Nighttime Awakenings
| Group | Value (percentage of nights) | Spread |
|---|---|---|
| Treatment Group | 16.8 | 25.3 |
Secondary outcomeMean· Baseline and ~1 month
Change in Daytime Fatigue
| Group | Value (score on a scale) |
|---|---|
| Treatment Group | -6.0 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Treatment GroupDesmopressin 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 | 0 / 8 | 0 / 8 | 1 / 8 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 8 Years up to 21 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
1. Patients with Hemoglobin SS, SC, SB0thal or SB+thal
2. Patients with at least two episodes of primary nocturnal enuresis per week or four episodes over the two weeks prior to enrollment.
3. Patients with secondary enuresis who have been evaluated and cleared by a pediatric urologist as not having other etiologies of enuresis (e.g. overactive detrusor activity, a genitourinary anatomic abnormality)
Exclusion Criteria:
1. Patients with developmental delay or neurologic dysfunction secondary to stroke.
2. Patients with hypertension or underlying renal disease.
3. Patients with genitourinary anatomic abnormalities. Any prior renal ultrasound showing normal genitourinary anatomy is sufficient to clear a patient for the study.
4. Patients with daytime urinary incontinence
5. Patients with glucosuria on urinalysis.
6. Patients with secondary nocturnal enuresis who have not been evaluated by a pediatric urologist to rule out other etiologies of enuresis.
7. Patients who are pregnant.
8. Patients receiving another medicine for nocturnal enuresis (e.g. imipramine).
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