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Desmopressin for Bedwetting in Children With SCD

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ClinicalTrials.gov Identifier: NCT04420585
Recruitment Status : Recruiting
First Posted : June 9, 2020
Last Update Posted : October 1, 2020
Sponsor:
Information provided by (Responsible Party):
Kerry Morrone, Montefiore Medical Center

Tracking Information
First Submitted Date  ICMJE May 21, 2020
First Posted Date  ICMJE June 9, 2020
Last Update Posted Date October 1, 2020
Actual Study Start Date  ICMJE July 7, 2020
Estimated Primary Completion Date December 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 4, 2020)
Change in Bedwetting episodes [ Time Frame: 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 appropriate.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 4, 2020)
  • Impact of Bedwetting on day to day activities [ Time Frame: 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 [ Time Frame: 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 prior to initiating treatment with DDAVP. Patient's will track their nighttime awakenings using a study diary.
  • Change in Daytime Fatigue [ Time Frame: Baseline and 4 weeks ]
    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 will be used to compare levels of fatigue from baseline to 4 weeks on the medication.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Desmopressin for Bedwetting in Children With SCD
Official Title  ICMJE Desmopressin as a Therapy for Nocturnal Enuresis in Pediatric Patients With Sickle Cell Disease
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Nocturnal Enuresis
  • Anemia, Sickle Cell
Intervention  ICMJE Drug: Desmopressin
Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement
Other Name: DDAVP
Study Arms  ICMJE Experimental: Treatment Group
Desmopressin 0.2mg tablets, dose titrated to effect
Intervention: Drug: Desmopressin
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 4, 2020)
60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2025
Estimated Primary Completion Date December 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Years to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Kerry Morrone, MD 718-741-2342 kmorrone@montefiore.org
Contact: Deepa Manwani, MD 718-741-2342 dmanwani@montefiore.org
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04420585
Other Study ID Numbers  ICMJE 2020-11268
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Kerry Morrone, Montefiore Medical Center
Study Sponsor  ICMJE Montefiore Medical Center
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Kerry Morrone, MD Montefiore Medical Center
PRS Account Montefiore Medical Center
Verification Date September 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP