Desmopressin as a Therapy for Bedwetting in Children With Sickle Cell Disease

Terminated

• Conditions: Nocturnal Enuresis; Anemia, Sickle Cell
• Interventions: Drug: Desmopressin

• Registration Number: NCT02636387
• Lead Sponsor: Montefiore Medical Center

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 work is being continued on study ID: 2020-11268.

Study Timeline

• Study Start: 2015-08-26
• Study First Submitted: 2015-12-17
• Study First Submitted QC: 2015-12-18
• Study First Posted: 2015-12-21
• Primary Completion: 2018-10-27
• Study Completion: 2018-10-27
• Status Verified: 2022-05
• Last Update Submitted: 2022-05-25
• Last Update Posted: 2022-05-31

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 14

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).

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Desmopressin	Desmopressin	0.2mg tablets, dose titrated to effect

Primary Outcome Measures

Name	Time	Method
Reduction in Bedwetting episodes	Baseline and 4 weeks	To prospectively assess if 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 indicated compared to the control group.

Secondary Outcome Measures

Name	Time	Method
Quality of life measure	Baseline and 4 weeks	To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have an improved quality of life compared to the control group.
Reduction in Nighttime awakenings	Baseline and 4 weeks	To determine if the use of desmopressin in patients with nocturnal enuresis improves rates of nocturia, defined as episodes of nighttime awakening to void in children ≥5 years of age, compared to the control group.
Reduction in Daytime Fatigue	Baseline and 4 weeks	To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have less daytime fatigue compared to the control group.

Trial Locations

Locations (1)

Children's Hospital at Montefiore; 🇺🇸 Bronx, New York, United States