Desmopressin as a Therapy for Nocturnal Enuresis in Pediatric Patients With Sickle Cell Disease
Overview
- Phase
- Phase 4
- Intervention
- Desmopressin
- Conditions
- Nocturnal Enuresis
- Sponsor
- Montefiore Medical Center
- Enrollment
- 8
- Locations
- 1
- Primary Endpoint
- Change in Bedwetting Episodes
- Status
- Terminated
- Last Updated
- last year
Overview
Brief Summary
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
Detailed Description
Night time bedwetting is a common complication of sickle cell disease, and affects up to 30 % of children. Desmopressin is an oral medication that increases water reabsorption in the kidneys. Studies have shown that it is effective in decreasing bedwetting episodes in children without sickle cell disease. Chronic sickling episodes causing damage to the kidneys could cause permanent damage and may make this treatment ineffective in sickle cell disease. This trial will inform pediatric sickle cell doctors if desmopressin is an appropriate treatment for bed wetting in the investigators patients. This study expanded upon a previously terminated study which had separately registered (NCT02636387, ID: 2014-3768).
Investigators
Eligibility Criteria
Inclusion Criteria
- •Patients with Hemoglobin SS, SC, SB0thal or SB+thal
- •Patients with at least two episodes of primary nocturnal enuresis per week or four episodes over the two weeks prior to enrollment.
- •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
- •Patients with developmental delay or neurologic dysfunction secondary to stroke.
- •Patients with hypertension or underlying renal disease.
- •Patients with genitourinary anatomic abnormalities. Any prior renal ultrasound showing normal genitourinary anatomy is sufficient to clear a patient for the study.
- •Patients with daytime urinary incontinence
- •Patients with glucosuria on urinalysis.
- •Patients with secondary nocturnal enuresis who have not been evaluated by a pediatric urologist to rule out other etiologies of enuresis.
- •Patients who are pregnant.
- •Patients receiving another medicine for nocturnal enuresis (e.g. imipramine).
Arms & Interventions
Treatment Group
Desmopressin 0.2mg tablets, dose titrated to effect
Intervention: Desmopressin
Outcomes
Primary Outcomes
Change in Bedwetting Episodes
Time Frame: Baseline and ~1 month
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
- Impact of Bedwetting on Day to Day Activities(Baseline and 4 weeks)
- Change in Nighttime Awakenings(Baseline and ~1 month)
- Change in Daytime Fatigue(Baseline and ~1 month)