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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Desmopressin as a Therapy for Nocturnal Enuresis in 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. Half of the children will receive placebo and half of the children will receive desmopressin, with the option to prescribe desmopressin as soon as the study is completed.

Detailed Summary: 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. The investigators designed this trial to definitively answer this question by randomizing the patients to give half of the children placebo and the other half titrated doses of desmopressin. This trial will inform pediatric sickle cell doctors if desmopressin is an appropriate treatment for bed wetting in the investigators patients.
Sponsor: Montefiore Medical Center

Current Primary Outcome: Reduction in Bedwetting episodes [ Time Frame: Baseline and 4 weeks ]

The investigators primary endpoint is to detect a 50 % difference between placebo and desmopressin treatment groups in number of wet nights after treatment implementation. This will be assessed by patient study diary.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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. The investigators will assess quality of life by administering the Promis Fatigue Scale prior to administering the study drug and 4 weeks after the intervention.
  • 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, compared to placebo.


Original Secondary Outcome: Same as current

Information By: Montefiore Medical Center

Dates:
Date Received: December 17, 2015
Date Started: January 2016
Date Completion: June 2018
Last Updated: September 12, 2016
Last Verified: September 2016