Nocturnal EnuresisPhase 4NCT04420585
Desmopressin for Bedwetting in Children With SCD
A Phase 4 study testing Desmopressin in people with Nocturnal Enuresis and Anemia, Sickle Cell, run by Montefiore Medical Center at 1 site in United States.
Trial summary
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease. Read more about this study
- Who is it for?
- Ages 8 to 21 years
- Conditions the study is for
- Nocturnal Enuresis, Anemia, Sickle Cell
- What is being tested?
- Desmopressin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Jul 2020to 12 Jun 2023
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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)
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital at Montefiore
The Bronx, New York, 10467
About this study
Official title: Desmopressin as a Therapy for Nocturnal Enuresis in Pediatric Patients With Sickle Cell Disease
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
Read the detailed 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 study expanded upon a previously terminated study which had separately registered (NCT02636387, ID: 2014-3768).
- Conditions
- Nocturnal Enuresis, Anemia, Sickle Cell
- Being tested
- Desmopressin
- Medical subject headings
- Nocturnal Enuresis, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DesmopressinDrug
Also called DDAVP
Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | Desmopressin 0.2mg tablets, dose titrated to effect Receives: Drug: Desmopressin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline and ~1 month
Secondary outcomes (3)
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.
Measured: 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
Measured: 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.
Measured: Baseline and ~1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2020
- Study start7 Jul 2020
- Primary completion12 Jun 2023
- Study completion12 Jun 2023
Why it stopped: 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Nocturnal EnuresisStudies of the same condition, open and under way.
- More trials run by Montefiore Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Montefiore Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kerry Morrone, MD, Principal investigator, Montefiore Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04420585
- Sponsor's protocol ID
- 2020-11268
- Status last verified
- Apr 2024
- First submitted
- 21 May 2020
- Last updated
- 7 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 16753432Barakat 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.
PubMed 11718233Readett 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.
PubMed 2334206Field 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.
PubMed 18384865Naitoh 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.
PubMed 22999692Glazener CM, Evans JH. Desmopressin for nocturnal enuresis in children. Cochrane Database Syst Rev. 2002;(3):CD002112. doi: 10.1002/14651858.CD002112.
PubMed 12137645Becker 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.
PubMed 21203778Statius 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.
PubMed 5435231Figueroa TE, Benaim E, Griggs ST, Hvizdala EV. Enuresis in sickle cell disease. J Urol. 1995 Jun;153(6):1987-9.
PubMed 7752379Robson 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.
PubMed 17574054
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Desmopressin as a Therapy for Nocturnal Enuresis in Pediatric Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT04420585
- Acronym / sponsor ID
- 2020-11268
- Conditions
- Nocturnal Enuresis, Anemia, Sickle Cell
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: 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.
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Montefiore Medical CenterUniversity, hospital or other
- Study start
- 7 Jul 2020 (actual)
- Primary completion
- 12 Jun 2023 (actual)
- Study completion
- 12 Jun 2023 (actual)
- First posted
- 9 Jun 2020 (actual)
- Results
- Posted 7 May 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
Detailed 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 study expanded upon a previously terminated study which had separately registered (NCT02636387, ID: 2014-3768).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Drug: DesmopressinDesmopressin 0.2mg tablets, dose titrated to effect |
Who may qualify
- Ages
- 8 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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)
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DesmopressinAlso: DDAVPTwo desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement | Drug | Treatment Group |
Primary outcome
Change in Bedwetting Episodes
Time frame: Baseline and ~1 month
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.
3 secondary outcomes
Impact of Bedwetting on Day to Day Activities
Time frame: Baseline and 4 weeks
Change in Nighttime Awakenings
Time frame: Baseline and ~1 month
Change in Daytime Fatigue
Time frame: Baseline and ~1 month
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 16753432Neveus 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.
- Background: PubMed 11718233Barakat 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.
- Background: PubMed 2334206Readett 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.
- Background: PubMed 18384865Field 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.
- Background: PubMed 22999692Naitoh 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.
- Background: PubMed 12137645Glazener CM, Evans JH. Desmopressin for nocturnal enuresis in children. Cochrane Database Syst Rev. 2002;(3):CD002112. doi: 10.1002/14651858.CD002112.
- Background: PubMed 21203778Becker 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.
- Background: PubMed 5435231Statius 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.
- Background: PubMed 7752379Figueroa TE, Benaim E, Griggs ST, Hvizdala EV. Enuresis in sickle cell disease. J Urol. 1995 Jun;153(6):1987-9.
- Background: PubMed 17574054Robson 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kerry Morrone, MD principal investigator
Montefiore Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Desmopressin in people with Nocturnal Enuresis and Anemia, Sickle Cell, run by Montefiore Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 7 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Bedwetting Episodes (time frame: Baseline and ~1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04420585, which also lists 10 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 May 2024.