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Desmopressin for Bedwetting in Children With SCD

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04420585
Recruitment Status : Recruiting
First Posted : June 9, 2020
Last Update Posted : May 31, 2022
Sponsor:
Information provided by (Responsible Party):
Kerry Morrone, Montefiore Medical Center

Study Description
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Brief Summary:
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.

Condition or disease Intervention/treatment Phase
Nocturnal Enuresis Anemia, Sickle Cell Drug: Desmopressin Phase 4

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 is an edited continuation of study ID: 2014-3768.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Desmopressin as a Therapy for Nocturnal Enuresis in Pediatric Patients With Sickle Cell Disease
Actual Study Start Date : July 7, 2020
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Treatment Group
Desmopressin 0.2mg tablets, dose titrated to effect
 Drug: Desmopressin
Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement
Other Name: DDAVP


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.


Eligibility Criteria
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Ages Eligible for Study:   8 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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).
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04420585


Contacts
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Contact: Kerry Morrone, MD 718-741-2342 kmorrone@montefiore.org
Contact: Deepa Manwani, MD 718-741-2342 dmanwani@montefiore.org

Locations
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United States, New York
Children's Hospital at Montefiore Recruiting
Bronx, New York, United States, 10467
Contact: Kerry Morrone, MD    718-741-2342    kmorrone@montefiore.org   
Contact: Deepa Morrone, MD    718-741-2342    dmanwani@montefiore.org   
Sponsors and Collaborators
Montefiore Medical Center
Investigators
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Principal Investigator: Kerry Morrone, MD Montefiore Medical Center
More Information
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Publications:

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Responsible Party: Kerry Morrone, Assistant Professor Pediatrics, Montefiore Medical Center
ClinicalTrials.gov Identifier: NCT04420585    
Other Study ID Numbers: 2020-11268
First Posted: June 9, 2020    Key Record Dates
Last Update Posted: May 31, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Additional relevant MeSH terms:
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Enuresis
Urinary Incontinence
Nocturnal Enuresis
Anemia, Sickle Cell
Urination Disorders
Urologic Diseases
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
Male Urogenital Diseases
Behavioral Symptoms
Elimination Disorders
Mental Disorders
Lower Urinary Tract Symptoms
 Urological Manifestations
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Deamino Arginine Vasopressin
Hemostatics
Coagulants
Antidiuretic Agents
Natriuretic Agents
Physiological Effects of Drugs