Desmopressin for Bedwetting in Children With SCD

NCT04420585 | Terminated Phase 4 Results FDA Drug

• 1 other identifier: 2020-11268
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Nocturnal Enuresis; Anemia, Sickle Cell

Outcome Measures

Primary Outcomes (1)

• Change in Bedwetting Episodes

  Change in the percentage of nights with bedwetting episodes assessed at baseline based on participant recall over the previous 14 nights, as well as at \~1 month (30 nights) based on completion of a study (enuresis) diary. This is used to assess whether 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 appropriate). A negative value indicates a decrease in bedwetting episodes.

  Baseline and ~1 month

Secondary Outcomes (3)

• Impact of Bedwetting on Day to Day Activities

  Baseline and 4 weeks

• Change in Nighttime Awakenings

  Baseline and ~1 month

• Change in Daytime Fatigue

  Baseline and ~1 month

Study Arms (1)

Treatment Group

EXPERIMENTAL

Desmopressin 0.2mg tablets, dose titrated to effect

Drug: Desmopressin

Interventions

Desmopressin DRUG

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

Also known as: DDAVP

Treatment Group

Eligibility Criteria

• Age: 8 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients with Hemoglobin SS, SC, SB0thal or SB+thal
• Patients with at least two episodes of primary nocturnal enuresis per week or four episodes over the two weeks prior to enrollment.
• 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)

You may not qualify if:

• Patients with developmental delay or neurologic dysfunction secondary to stroke.
• Patients with hypertension or underlying renal disease.
• Patients with genitourinary anatomic abnormalities. Any prior renal ultrasound showing normal genitourinary anatomy is sufficient to clear a patient for the study.
• Patients with daytime urinary incontinence
• Patients with glucosuria on urinalysis.
• Patients with secondary nocturnal enuresis who have not been evaluated by a pediatric urologist to rule out other etiologies of enuresis.
• Patients who are pregnant.
• Patients receiving another medicine for nocturnal enuresis (e.g. imipramine).

Sponsors & Collaborators

• Montefiore Medical Center: lead

Study Sites (1)

Children's Hospital at Montefiore

The Bronx, New York, 10467, United States

Location

Related Publications (10)

• 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.

  PMID: 16753432 BACKGROUND

• 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.

  PMID: 11718233 BACKGROUND

• 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.

  PMID: 2334206 BACKGROUND

• 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.

  PMID: 18384865 BACKGROUND

• 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.

  PMID: 22999692 BACKGROUND

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

  PMID: 12137645 BACKGROUND

• 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.

  PMID: 21203778 BACKGROUND

• 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.

  PMID: 5435231 BACKGROUND

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

  PMID: 7752379 BACKGROUND

• 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.

  PMID: 17574054 BACKGROUND

MeSH Terms

Conditions

Nocturnal Enuresis; Anemia, Sickle Cell

Interventions

Deamino Arginine Vasopressin

Condition Hierarchy (Ancestors)

Enuresis; Urination Disorders; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Behavioral Symptoms; Behavior; Elimination Disorders; Mental Disorders; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Arginine Vasopressin; Vasopressins; Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Oligopeptides; Nerve Tissue Proteins; Proteins

Results Point of Contact

• Title: Dr. Kerry Morrone
• Organization: Montefiore Medical Center

kmorrone@montefiore.org

718-741-2342

Study Officials

• Kerry Morrone, MD

  Montefiore Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 21, 2020
• First Posted: June 9, 2020
• Study Start: July 7, 2020
• Primary Completion: June 12, 2023
• Study Completion: June 12, 2023
• Last Updated: May 7, 2024
• Results First Posted: May 7, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)