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Study was not able to meet recruitment after 9 years of trying to enroll patients on different versions of the protocol and changing recruitment strategies.
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This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Group | Experimental | Desmopressin 0.2mg tablets, dose titrated to effect |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Desmopressin | Drug | Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Impact of Bedwetting on Day to Day Activities | To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have an improved quality of life compared to their baseline. This will be measured using the PedsQL Measurement Model which measures health related quality of life in children with acute and chronic health conditions, like sickle cell. The scales focuses on areas such as activities, feelings, and school performance. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Kerry Morrone, MD | Montefiore Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital at Montefiore | The Bronx | New York | 10467 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 16753432 | Background | 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. | |
| 11718233 |
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment Group | Desmopressin 0.2mg tablets, dose titrated to effect NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination. Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Treatment Group | Desmopressin 0.2mg tablets, dose titrated to effect NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination. Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Mean | Standard Deviation | percentage of nights | Baseline and ~1 month |
|
4 weeks after initiation of medication
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment Group | Desmopressin 0.2mg tablets, dose titrated to effect NOTE: While an option, doses were not titrated for any of the participants in this study prior to study termination. Desmopressin: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dizziness | Nervous system disorders | MedDRA (Unspecified) | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Kerry Morrone | Montefiore Medical Center | 718-741-2342 | kmorrone@montefiore.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 7, 2020 | Jan 18, 2024 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D053206 | Nocturnal Enuresis |
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D004775 | Enuresis |
| D014555 | Urination Disorders |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
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| ID | Term |
|---|---|
| D003894 | Deamino Arginine Vasopressin |
| ID | Term |
|---|---|
| D001127 | Arginine Vasopressin |
| D014667 | Vasopressins |
| D010909 | Pituitary Hormones, Posterior |
| D010907 | Pituitary Hormones |
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| Baseline and 4 weeks |
| Change in Nighttime Awakenings | Change in the percentage of nights with awakenings over the previous 14 nights was assessed by summarizing and comparing baseline and 1 month nighttime awakening data. The percentage of nights with nighttime awakenings was evaluated at baseline based on recall and at 1 month (30 nights) based on completion of a study diary. This was used to determine whether the use of desmopressin in patients with sickle cell disease and nocturnal enuresis changes the rates of nighttime awakenings to urinate (nocturia), defined as episodes of nighttime awakening to void in children ≥5 years of age, compared to prior to initiating treatment with DDAVP. A positive value indicates an increase in nighttime awakenings | Baseline and ~1 month |
| Change in Daytime Fatigue | Change in daytime fatigue will be assessed to determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have less daytime fatigue compared to their baseline data. The PROMIS Pediatric Fatigue Short Form (Version 2.0) will be used to compare levels of fatigue from baseline and ~1 month (30 nights) on the study medication. The PROMIS Fatigue scale, which utilizes a 7-day recall period, is a 10-item questionnaire consisting of 5 responses ranging from 1-5 with one indicative of "Never" and five indicative of "almost always" resulting in a raw scoring range of 10-50. Negative PROMIS change scores are associated with decreased levels of fatigue. | Baseline and ~1 month |
| 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. |
| 2334206 | 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. |
| 18384865 | 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. |
| 22999692 | 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. |
| 12137645 | Background | Glazener CM, Evans JH. Desmopressin for nocturnal enuresis in children. Cochrane Database Syst Rev. 2002;(3):CD002112. doi: 10.1002/14651858.CD002112. |
| 21203778 | 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. |
| 5435231 | 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. |
| 7752379 | Background | Figueroa TE, Benaim E, Griggs ST, Hvizdala EV. Enuresis in sickle cell disease. J Urol. 1995 Jun;153(6):1987-9. |
| 17574054 | 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. |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Genotype of Sickle Cell Disease | The number of participants with Sickle Cell Anemia (Hb SS) vs Sickle Hemoglobin-C (Hb SC) was determined. | Count of Participants | Participants |
|
| Number of Bedwetting Days Based on Disease Type | Participants were asked to recall the number of days during which a bedwetting episode was experienced over the prior 14 days. Mean number of days was reported by disease type. | 6 participants had Sickle Cell Anemia (Hb SS) and two participants had Sickle Hemoglobin-C (Hb SC) | Mean | Standard Deviation | days |
|
|
|
| Secondary | Impact of Bedwetting on Day to Day Activities | To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have an improved quality of life compared to their baseline. This will be measured using the PedsQL Measurement Model which measures health related quality of life in children with acute and chronic health conditions, like sickle cell. The scales focuses on areas such as activities, feelings, and school performance. | Data was not collected for the Impact of Bedwetting on day-to-day activities. | Posted | Baseline and 4 weeks |
|
|
| Secondary | Change in Nighttime Awakenings | Change in the percentage of nights with awakenings over the previous 14 nights was assessed by summarizing and comparing baseline and 1 month nighttime awakening data. The percentage of nights with nighttime awakenings was evaluated at baseline based on recall and at 1 month (30 nights) based on completion of a study diary. This was used to determine whether the use of desmopressin in patients with sickle cell disease and nocturnal enuresis changes the rates of nighttime awakenings to urinate (nocturia), defined as episodes of nighttime awakening to void in children ≥5 years of age, compared to prior to initiating treatment with DDAVP. A positive value indicates an increase in nighttime awakenings | Posted | Mean | Standard Deviation | percentage of nights | Baseline and ~1 month |
|
|
|
| Secondary | Change in Daytime Fatigue | Change in daytime fatigue will be assessed to determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have less daytime fatigue compared to their baseline data. The PROMIS Pediatric Fatigue Short Form (Version 2.0) will be used to compare levels of fatigue from baseline and ~1 month (30 nights) on the study medication. The PROMIS Fatigue scale, which utilizes a 7-day recall period, is a 10-item questionnaire consisting of 5 responses ranging from 1-5 with one indicative of "Never" and five indicative of "almost always" resulting in a raw scoring range of 10-50. Negative PROMIS change scores are associated with decreased levels of fatigue. | 1 month fatigue data was not able to collected from one participant who did not complete the 1 month PROMIS Fatigue questionnaire. Accordingly, change results were not available. | Posted | Mean | Full Range | score on a scale | Baseline and ~1 month |
|
|
|
| 0 |
| 8 |
| 0 |
| 8 |
| 1 |
| 8 |
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| D005261 |
| Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D001526 | Behavioral Symptoms |
| D001519 | Behavior |
| D019960 | Elimination Disorders |
| D001523 | Mental Disorders |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D036361 |
| Peptide Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D009479 | Neuropeptides |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D009842 | Oligopeptides |
| D009419 | Nerve Tissue Proteins |
| D011506 | Proteins |