Desmopressin for Bedwetting in Children With SCD

NCT ID: NCT04420585

Last Updated: 2024-05-07

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE4

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-07-07

Study Completion Date

2023-06-12

Brief Summary

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

Detailed Description

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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 study expanded upon a previously terminated study which had separately registered (NCT02636387, ID: 2014-3768).

Conditions

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Nocturnal Enuresis Anemia, Sickle Cell

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment Group

Desmopressin 0.2mg tablets, dose titrated to effect

Group Type EXPERIMENTAL

Desmopressin

Intervention Type DRUG

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

Interventions

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Desmopressin

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

Intervention Type DRUG

Other Intervention Names

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DDAVP

Eligibility Criteria

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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).
Minimum Eligible Age

8 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Montefiore Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Kerry Morrone, MD

Role: PRINCIPAL_INVESTIGATOR

Montefiore Medical Center

Locations

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Children's Hospital at Montefiore

The Bronx, New York, United States

Site Status

Countries

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United States

References

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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.

Reference Type BACKGROUND
PMID: 16753432 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 11718233 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 2334206 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18384865 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 22999692 (View on PubMed)

Glazener CM, Evans JH. Desmopressin for nocturnal enuresis in children. Cochrane Database Syst Rev. 2002;(3):CD002112. doi: 10.1002/14651858.CD002112.

Reference Type BACKGROUND
PMID: 12137645 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21203778 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 5435231 (View on PubMed)

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

Reference Type BACKGROUND
PMID: 7752379 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 17574054 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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2020-11268

Identifier Type: -

Identifier Source: org_study_id

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