<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT04420585</url>
  </required_header>
  <id_info>
    <org_study_id>2020-11268</org_study_id>
    <nct_id>NCT04420585</nct_id>
  </id_info>
  <brief_title>Desmopressin for Bedwetting in Children With SCD</brief_title>
  <official_title>Desmopressin as a Therapy for Nocturnal Enuresis in Pediatric Patients With Sickle Cell Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Montefiore Medical Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Montefiore Medical Center</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>Yes</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study assesses if using the medication desmopressin will decrease nightime bedwetting in&#xD;
      children with sickle cell disease.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Night time bedwetting is a common complication of sickle cell disease, and affects up to 30 %&#xD;
      of children . Desmopressin is an oral medication that increases water reabsorption in the&#xD;
      kidneys. Studies have shown that it is effective in decreasing bedwetting episodes in&#xD;
      children without sickle cell disease. Chronic sickling episodes causing damage to the kidneys&#xD;
      could cause permanent damage and may make this treatment ineffective in sickle cell disease.&#xD;
      This trial will inform pediatric sickle cell doctors if desmopressin is an appropriate&#xD;
      treatment for bed wetting in the investigators patients.&#xD;
&#xD;
      This is an edited continuation of study ID: 2014-3768.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    We were not able to meet recruitment after 9 years of trying to enroll patients on different&#xD;
    versions of the protocol and changing recruitment strategies.&#xD;
  </why_stopped>
  <start_date type="Actual">July 7, 2020</start_date>
  <completion_date type="Actual">June 12, 2023</completion_date>
  <primary_completion_date type="Actual">June 12, 2023</primary_completion_date>
  <phase>Phase 4</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in Bedwetting episodes</measure>
    <time_frame>Baseline and 4 weeks</time_frame>
    <description>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 appropriate.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Impact of Bedwetting on day to day activities</measure>
    <time_frame>Baseline and 4 weeks</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in Nighttime awakenings</measure>
    <time_frame>Baseline and 4 weeks</time_frame>
    <description>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 prior to initiating treatment with DDAVP. Patient's will track their nighttime awakenings using a study diary.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in Daytime Fatigue</measure>
    <time_frame>Baseline and 4 weeks</time_frame>
    <description>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 will be used to compare levels of fatigue from baseline to 4 weeks on the medication.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">7</enrollment>
  <condition>Nocturnal Enuresis</condition>
  <condition>Anemia, Sickle Cell</condition>
  <arm_group>
    <arm_group_label>Treatment Group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Desmopressin 0.2mg tablets, dose titrated to effect</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Desmopressin</intervention_name>
    <description>Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if &lt;50 % improvement</description>
    <arm_group_label>Treatment Group</arm_group_label>
    <other_name>DDAVP</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Patients with Hemoglobin SS, SC, SB0thal or SB+thal&#xD;
&#xD;
          2. Patients with at least two episodes of primary nocturnal enuresis per week or four&#xD;
             episodes over the two weeks prior to enrollment.&#xD;
&#xD;
          3. Patients with secondary enuresis who have been evaluated and cleared by a pediatric&#xD;
             urologist as not having other etiologies of enuresis (e.g. overactive detrusor&#xD;
             activity, a genitourinary anatomic abnormality)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Patients with developmental delay or neurologic dysfunction secondary to stroke.&#xD;
&#xD;
          2. Patients with hypertension or underlying renal disease.&#xD;
&#xD;
          3. Patients with genitourinary anatomic abnormalities. Any prior renal ultrasound showing&#xD;
             normal genitourinary anatomy is sufficient to clear a patient for the study.&#xD;
&#xD;
          4. Patients with daytime urinary incontinence&#xD;
&#xD;
          5. Patients with glucosuria on urinalysis.&#xD;
&#xD;
          6. Patients with secondary nocturnal enuresis who have not been evaluated by a pediatric&#xD;
             urologist to rule out other etiologies of enuresis.&#xD;
&#xD;
          7. Patients who are pregnant.&#xD;
&#xD;
          8. Patients receiving another medicine for nocturnal enuresis (e.g. imipramine).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>8 Years</minimum_age>
    <maximum_age>21 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Kerry Morrone, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Montefiore Medical Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's Hospital at Montefiore</name>
      <address>
        <city>Bronx</city>
        <state>New York</state>
        <zip>10467</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <reference>
    <citation>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.</citation>
    <PMID>16753432</PMID>
  </reference>
  <reference>
    <citation>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.</citation>
    <PMID>11718233</PMID>
  </reference>
  <reference>
    <citation>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.</citation>
    <PMID>2334206</PMID>
  </reference>
  <reference>
    <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.</citation>
    <PMID>18384865</PMID>
  </reference>
  <reference>
    <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.</citation>
    <PMID>22999692</PMID>
  </reference>
  <reference>
    <citation>Glazener CM, Evans JH. Desmopressin for nocturnal enuresis in children. Cochrane Database Syst Rev. 2002;(3):CD002112. doi: 10.1002/14651858.CD002112.</citation>
    <PMID>12137645</PMID>
  </reference>
  <reference>
    <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.</citation>
    <PMID>21203778</PMID>
  </reference>
  <reference>
    <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. doi: 10.1016/0009-8981(70)90305-0. No abstract available.</citation>
    <PMID>5435231</PMID>
  </reference>
  <reference>
    <citation>Figueroa TE, Benaim E, Griggs ST, Hvizdala EV. Enuresis in sickle cell disease. J Urol. 1995 Jun;153(6):1987-9.</citation>
    <PMID>7752379</PMID>
  </reference>
  <reference>
    <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. doi: 10.1016/j.juro.2007.03.015. Epub 2007 May 11.</citation>
    <PMID>17574054</PMID>
  </reference>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>May 21, 2020</study_first_submitted>
  <study_first_submitted_qc>June 4, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 9, 2020</study_first_posted>
  <last_update_submitted>June 12, 2023</last_update_submitted>
  <last_update_submitted_qc>June 12, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 15, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Montefiore Medical Center</investigator_affiliation>
    <investigator_full_name>Kerry Morrone</investigator_full_name>
    <investigator_title>Assistant Professor Pediatrics</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Enuresis</mesh_term>
    <mesh_term>Urinary Incontinence</mesh_term>
    <mesh_term>Nocturnal Enuresis</mesh_term>
    <mesh_term>Anemia, Sickle Cell</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Deamino Arginine Vasopressin</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT04420585
org study id: 2020-11268
nct id: NCT04420585
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
is us export: Yes
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 is an edited continuation of study ID: 2014-3768.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change in Bedwetting episodes
time frame: Baseline and 4 weeks
description: 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 appropriate.
measure: Impact of Bedwetting on day to day activities
time frame: Baseline and 4 weeks
description: 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.
measure: Change in Nighttime awakenings
time frame: Baseline and 4 weeks
description: 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 prior to initiating treatment with DDAVP. Patient's will track their nighttime awakenings using a study diary.
measure: Change in Daytime Fatigue
time frame: Baseline and 4 weeks
description: 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 will be used to compare levels of fatigue from baseline to 4 weeks on the medication.
arm group label: Treatment Group
arm group type: Experimental
description: Desmopressin 0.2mg tablets, dose titrated to effect
intervention type: Drug
intervention name: Desmopressin
description: Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement
arm group label: Treatment Group
other name: DDAVP
criteria: 
gender: All
minimum age: 8 Years
maximum age: 21 Years
healthy volunteers: No
last name: Kerry Morrone, MD
role: Principal Investigator
affiliation: Montefiore Medical Center
facility: 
country: United States
citation: 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
citation: 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
citation: 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
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.
PMID: 18384865
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.
PMID: 22999692
citation: Glazener CM, Evans JH. Desmopressin for nocturnal enuresis in children. Cochrane Database Syst Rev. 2002;(3):CD002112. doi: 10.1002/14651858.CD002112.
PMID: 12137645
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.
PMID: 21203778
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. doi: 10.1016/0009-8981(70)90305-0. No abstract available.
PMID: 5435231
citation: Figueroa TE, Benaim E, Griggs ST, Hvizdala EV. Enuresis in sickle cell disease. J Urol. 1995 Jun;153(6):1987-9.
PMID: 7752379
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. doi: 10.1016/j.juro.2007.03.015. Epub 2007 May 11.
PMID: 17574054
responsible party type: Principal Investigator
investigator affiliation: Montefiore Medical Center
investigator full name: Kerry Morrone
investigator title: Assistant Professor Pediatrics
mesh term: Enuresis
mesh term: Urinary Incontinence
mesh term: Nocturnal Enuresis
mesh term: Anemia, Sickle Cell
mesh term: Deamino Arginine Vasopressin
sharing ipd: No