Desmopressin for Bedwetting in Children With SCD

Phase 4

Terminated

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

• Registration Number: NCT04420585
• 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 study expanded upon a previously terminated study which had separately registered (NCT02636387, ID: 2014-3768).

Study Timeline

• Study First Submitted: 2020-05-21
• Study First Submitted QC: 2020-06-04
• Study First Posted: 2020-06-09
• Study Start: 2020-07-07
• Primary Completion: 2023-06-12
• Study Completion: 2023-06-12
• Results First Submitted: 2024-02-22
• Status Verified: 2024-04
• Results First Submitted QC: 2024-04-10
• Last Update Submitted: 2024-04-10
• Results First Posted: 2024-05-07
• Last Update Posted: 2024-05-07

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 8

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)

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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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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Change in Bedwetting Episodes	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 Outcome Measures

Name	Time	Method
Impact of Bedwetting on Day to Day Activities	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 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	Baseline and ~1 month	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	Baseline and ~1 month	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.

Trial Locations

Locations (1)

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