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

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02636387
Recruitment Status : Terminated (Low recruitment)
First Posted : December 21, 2015
Last Update Posted : May 26, 2020
Sponsor:
Information provided by (Responsible Party):
Kerry Morrone, Montefiore Medical Center

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
Nocturnal Enuresis Anemia, Sickle Cell Drug: Desmopressin

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.

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Study Type : Observational
Actual Enrollment : 14 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Desmopressin as a Therapy for Nocturnal Enuresis in Patients With Sickle Cell Disease
Actual Study Start Date : August 26, 2015
Actual Primary Completion Date : October 27, 2018
Actual Study Completion Date : October 27, 2018


Group/Cohort Intervention/treatment
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

Placebo
Placebo Comparator



Primary Outcome Measures :
  1. Reduction 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 indicated compared to the control group.


Secondary Outcome Measures :
  1. Quality of life measure [ 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 the control group.

  2. Reduction 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 the control group.

  3. Reduction 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 the control group.



Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study population will consist of children (ages 8-21) with Hemoglobin SS, SC, SB0thal or SB+thal and with Nocturnal Enuresis who are treated at the children's hospitals outpatient hematology clinic and/or the inpatient hematology unit.
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).

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): NCT02636387


Locations
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United States, New York
Children's Hospital at Montefiore
Bronx, New York, United States, 10467
Sponsors and Collaborators
Montefiore Medical Center
Investigators
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Principal Investigator: Kerry A Morrone, MD Children's Hospital at Montefiore
Publications:

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Responsible Party: Kerry Morrone, Assistant Professor of Pediatrics, Montefiore Medical Center
ClinicalTrials.gov Identifier: NCT02636387    
Other Study ID Numbers: 2014-3768
First Posted: December 21, 2015    Key Record Dates
Last Update Posted: May 26, 2020
Last Verified: May 2020
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
Additional relevant MeSH terms:
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Enuresis
Urinary Incontinence
Nocturnal Enuresis
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Urination Disorders
Urologic Diseases
Behavioral Symptoms
Elimination Disorders
Mental Disorders
Lower Urinary Tract Symptoms
Urological Manifestations
Signs and Symptoms
Deamino Arginine Vasopressin
Hemostatics
Coagulants
Antidiuretic Agents
Natriuretic Agents
Physiological Effects of Drugs