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

• ClinicalTrials.gov Identifier: NCT02636387
• Recruitment Status: Terminated
• First Posted: December 21, 2015
• Last Update Posted: May 31, 2022
• Sponsor: Montefiore Medical Center
• Information provided by (Responsible Party): Kerry Morrone, Montefiore Medical Center

Tracking Information

• First Submitted Date: December 17, 2015
• First Posted Date: December 21, 2015
• Last Update Posted Date: May 31, 2022
• Actual Study Start Date: August 26, 2015
• Actual Primary Completion Date: October 27, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 21, 2020)

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.

Original Primary Outcome Measures (submitted: December 18, 2015)

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.

Current Secondary Outcome Measures (submitted: May 21, 2020)

• 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.
• 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.
• 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.

Original Secondary Outcome Measures (submitted: December 18, 2015)

• 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.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Desmopressin as a Therapy for Bedwetting in Children With Sickle Cell Disease
• 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.

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 work is being continued on study ID: 2020-11268.

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• 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.

Condition

• Nocturnal Enuresis
• Anemia, Sickle Cell

Intervention

Drug: Desmopressin

Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement

Other Name: DDAVP

Study Groups/Cohorts

• Desmopressin
  0.2mg tablets, dose titrated to effect
  Intervention: Drug: Desmopressin
• Placebo
  Placebo Comparator

Publications *

• Neveus T, von Gontard A, Hoebeke P, Hjalmas K, Bauer S, Bower W, Jorgensen TM, Rittig S, Walle JV, Yeung CK, Djurhuus JC. The standardization of terminology of lower urinary tract function in children and adolescents: report from the Standardisation Committee of the International Children's Continence Society. J Urol. 2006 Jul;176(1):314-24. doi: 10.1016/S0022-5347(06)00305-3.
• Barakat LP, Smith-Whitley K, Schulman S, Rosenberg D, Puri R, Ohene-Frempong K. Nocturnal enuresis in pediatric sickle cell disease. J Dev Behav Pediatr. 2001 Oct;22(5):300-5. doi: 10.1097/00004703-200110000-00004.
• Readett DR, Morris JS, Serjeant GR. Nocturnal enuresis in sickle cell haemoglobinopathies. Arch Dis Child. 1990 Mar;65(3):290-3. doi: 10.1136/adc.65.3.290.
• Field JJ, Austin PF, An P, Yan Y, DeBaun MR. Enuresis is a common and persistent problem among children and young adults with sickle cell anemia. Urology. 2008 Jul;72(1):81-4. doi: 10.1016/j.urology.2008.02.006. Epub 2008 Apr 2.
• Naitoh Y, Kawauchi A, Soh J, Kamoi K, Miki T. Health related quality of life for monosymptomatic enuretic children and their mothers. J Urol. 2012 Nov;188(5):1910-4. doi: 10.1016/j.juro.2012.07.012. Epub 2012 Sep 19.
• Glazener CM, Evans JH. Desmopressin for nocturnal enuresis in children. Cochrane Database Syst Rev. 2002;(3):CD002112. doi: 10.1002/14651858.CD002112.
• Becker AM. Sickle cell nephropathy: challenging the conventional wisdom. Pediatr Nephrol. 2011 Dec;26(12):2099-109. doi: 10.1007/s00467-010-1736-2. Epub 2011 Jan 4.
• Statius van Eps LW, Schouten H, Haar Romeny-Wachter CC, La Porte-Wijsman LW. The relation between age and renal concentrating capacity in sickle cell disease and hemoglobin C disease. Clin Chim Acta. 1970 Mar;27(3):501-11. doi: 10.1016/0009-8981(70)90305-0. No abstract available.
• Figueroa TE, Benaim E, Griggs ST, Hvizdala EV. Enuresis in sickle cell disease. J Urol. 1995 Jun;153(6):1987-9.
• Robson WL, Leung AK, Norgaard JP. The comparative safety of oral versus intranasal desmopressin for the treatment of children with nocturnal enuresis. J Urol. 2007 Jul;178(1):24-30. doi: 10.1016/j.juro.2007.03.015. Epub 2007 May 11.

Recruitment Information

• Recruitment Status: Terminated
• Actual Enrollment (submitted: May 21, 2020): 14
• Original Estimated Enrollment (submitted: December 18, 2015): 118
• Actual Study Completion Date: October 27, 2018
• Actual Primary Completion Date: October 27, 2018 (Final data collection date for primary outcome measure)

Eligibility 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).

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 8 Years to 21 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02636387
• Other Study ID Numbers: 2014-3768
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Kerry Morrone, Montefiore Medical Center
• Original Responsible Party: Same as current
• Current Study Sponsor: Montefiore Medical Center
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Kerry A Morrone, MD; Children's Hospital at Montefiore

• PRS Account: Montefiore Medical Center
• Verification Date: May 2022