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

Anemia, Sickle Cell Clinical Trial

Official title:

Desmopressin as a Therapy for Nocturnal Enuresis in Patients With Sickle Cell Disease

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02636387
• Other study ID #: 2014-3768
• Status: Terminated
• Start date: August 26, 2015
• Est. completion date: October 27, 2018

Study information

• Verified date: May 2022
• Source: Montefiore Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 14
• Est. completion date: October 27, 2018
• Est. primary completion date: October 27, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years to 21 Years

Eligibility

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).

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Enuresis
• Nocturnal Enuresis
• Urinary Incontinence

Intervention

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

Locations

Country	Name	City	State
United States	Children's Hospital at Montefiore	Bronx	New York

Sponsors (1)

Lead Sponsor	Collaborator
Montefiore Medical Center

Country where clinical trial is conducted

United States, 

References & Publications (10)

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. — View Citation

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. Review. — View Citation

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. — View Citation

Figueroa TE, Benaim E, Griggs ST, Hvizdala EV. Enuresis in sickle cell disease. J Urol. 1995 Jun;153(6):1987-9. — View Citation

Glazener CM, Evans JH. Desmopressin for nocturnal enuresis in children. Cochrane Database Syst Rev. 2002;(3):CD002112. Review. — View Citation

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. — View Citation

Nevéus T, von Gontard A, Hoebeke P, Hjälmås K, Bauer S, Bower W, Jørgensen 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. — View Citation

Readett DR, Morris JS, Serjeant GR. Nocturnal enuresis in sickle cell haemoglobinopathies. Arch Dis Child. 1990 Mar;65(3):290-3. — View Citation

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. Epub 2007 May 11. Review. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Reduction in Bedwetting episodes	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.	Baseline and 4 weeks
Secondary	Quality of life measure	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.	Baseline and 4 weeks
Secondary	Reduction in Nighttime awakenings	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.	Baseline and 4 weeks
Secondary	Reduction in Daytime Fatigue	To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have less daytime fatigue compared to the control group.	Baseline and 4 weeks