Anorectal MalformationMARQOLNCT02029248
National Study on the Quality of Life of Patients With Anorectal Malformation
An observational study of Anorectal Malformation, run by Nantes University Hospital at 37 sites in France.
Trial summary
Anorectal malformations, occurring approximately 1 in 5000 live births, mainly involve the distal anus and rectum, but also sometimes the urinary and genital tracts. Defects range from the minor and easily treated with an excellent functional prognosis, to those that are complex and often associated with a poor… Read more about this study
- Who is it for?
- Ages 6 to 30 years
- Conditions the study is for
- Anorectal Malformation
- What is being tested?
- Quality of life questionnaire
- How many people
- 762 participants
- Where
- 37 sitesFrance
- Study dates
- Apr 2012to Apr 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
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 6 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients initially treated in a french pediatric surgery service for anorectal malformation
Who may be able to join
- Patients who are between 6 and 30 years old
- Patients initially treated in a french pediatric surgery service for anorectal malformation
Who cannot join
- Patients lost from sight or dead
- Patients with concomitant malformations that can modify sphincter functions besides anorectal malformation (spina bifida, )
- Patients whose brain functions do not allow to reply to a questionnaire (mental retardation)
Trial locations and contacts
37 sites in 1 country.
37 sites in 1 country
France37 sites
CHU Amiens
Amiens, 80054
CHU Angers
Angers, 49033
CHU Besançon
Besançon, 25030
CHU Bordeaux
Bordeaux, 33076
CHU Brest
Brest, 29609
Hospices Civils de Lyon
Bron, 69677
CHU Caen
Caen, 14033
CHU Clermont-Ferrand
Clermont-Ferrand, 63000
Hôpitaux Civils de Colmar
Colmar, 68024
CHU Dijon
Dijon, 21079
CHU Fort-de-France
Fort-de-France (Martinique), 97261
CHU Grenoble
Grenoble, 38043
AP-HP Hôpital Bicêtre
Le Kremlin-Bicêtre, 94275
CH Le Mans
Le Mans, 72037
CHRU Lille
Lille, 59037
CHU Limoges
Limoges, 87042
AP-HM La Timone
Marseille, 13385
AP-HM Hôpital Nord
Marseille, 13915
CHU Montpellier
Montpellier, 34295
CHU de Nantes
Nantes, 44093
CHU Nice
Nice, 06200
CHR Orléans
Orléans, 45032
AP-HP Hôpital Trousseau
Paris, 75012
AP-HP Hôpital Necker
Paris, 75015
AP-HP Hôpital Robert Debré
Paris, 75019
CHU Pointe-à-Pitre
Pointe-à-Pitre (Guadeloupe), 97159
CHU Poitiers
Poitiers, 86021
CHU Reims
Reims, 51100
CHU Rennes
Rennes, 35203
CHU Rouen
Rouen, 76031
CHR La Réunion
Saint Denis (La Réunion), 97405
CH Saint Brieuc
Saint-Brieuc, 22027
CHU Saint Etienne
Saint-Etienne, 42055
CHRU Strasbourg
Strasbourg, 67098
CHU Toulouse
Toulouse, 31059
CHRU Tours
Tours, 37044
CHU Nancy
Vandœuvre-lès-Nancy, 54511
About this study
Official title: National Study on the Quality of Life of Patients With Anorectal Malformation (MARQOL)
Anorectal malformations, occurring approximately 1 in 5000 live births, mainly involve the distal anus and rectum, but also sometimes the urinary and genital tracts. Defects range from the minor and easily treated with an excellent functional prognosis, to those that are complex and often associated with a poor functional prognosis.
Despite the better knowledge of the anatomy and physiology and the improvement of surgical management after birth, fecal and urinary incontinence can occur, due mainly to deficient nerve supply.
The quality of life of such patients is largely unknown in this country. The aim of the investigators study is to propose specific and generic questionnaires to the patients registered in the national database, correlated to their anatomical and functional status. A better understanding of such correlations should allow improvements in their medical and social management.
Read the detailed description
Anorectal malformations (ARM), occurring approximately 1 in 5000 live births, mainly involve the distal anus and rectum, but also sometimes the urinary and genital tracts. Defects range from the minor and easily treated with an excellent functional prognosis, to those that are complex and often associated with a poor functional prognosis. Despite the better knowledge of the anatomy and physiology and the improvement of surgical management after birth, fecal and urinary incontinence can occur, due mainly to deficient nerve supply. A few studies and clinical practice have shown that disease-specific problems of ARM have an effect on somatic function, mental health and psychosocial functioning.
The assessment of quality of life requires a self-report questionnaire composed of items related to physical, emotional and social functioning, and disease-related symptoms. A specific ARM questionnaire, the HAQL, was developed by a Dutch team and published in 2001. Associated with generic questionnaire, it allowed quality of life assessment in Dutch population.
The quality of life of such patients is largely unknown in this larger country. The aim of the investigators study is to propose specific (HAQL translated from Dutch to French) and generic questionnaires to the patients registered in the national database, correlated to their anatomical and functional status.
A better understanding of such correlations should allow improvements in their medical and social management.
- Conditions
- Anorectal Malformation
- Being tested
- Quality of life questionnaire
- Keywords
- Anorectal malformation, fecal incontinence, quality of life, child
- Medical subject headings
- Anorectal Malformations, Fecal Incontinence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Quality of life questionnaireOther
| Study group | Role | What they receive |
|---|---|---|
| Children 6-7 | Patients who are between 6 and 7 years old Receives: Other: Quality of life questionnaire | |
| Children 8-11 | Patients who are between 8 and 11 years old Receives: Other: Quality of life questionnaire | |
| Children 12-16 | Patients who are between 12 and 16 years old Receives: Other: Quality of life questionnaire | |
| Patients 17-30 | Patients who are between 17 and 30 years old Receives: Other: Quality of life questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quality of life measured by HAQL specific questionnaire and two generic questionnaires: VSPA for children or WHOQOL-BREF for adults; functional status measured by Krickenbeck score
HAQL: between 35 and 48 items (depending on the age of the patients) composing 8 dimensions; VSPA: 39 items composing 8 dimensions; WHOQOL-BREF: 26 items composing 4 dimensions; Krickenbeck: 3 items.
Measured: At the time of the inclusion
Secondary outcomes (1)
Kind of malformation, associated malformation, sequelas, handicap degree, social integration
Clinical questionnaire
Measured: At the time of the inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2014 (estimated)
- Study startApr 2012
- Primary completionApr 2015
- Study completionApr 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Anorectal MalformationStudies of the same condition, open and under way.
- More trials run by Nantes University HospitalThe 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
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 762 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nantes University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- MAREP ( Center For Rare Diseases Center Ano-Rectal And Pelvic Abnormalities)
- Responsible party
- Sponsor
- Investigators
- Guillaume Podevin, MD, PhD, Principal investigator, CHU d'Angers (France)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02029248
- Sponsor's protocol ID
- BRD/10/06-R
- Acronym
- MARQOL
- Status last verified
- Jul 2017
- First submitted
- 2 Dec 2013
- Last updated
- 19 Jul 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Clermidi P, Podevin G, Cretolle C, Sarnacki S, Hardouin JB. The challenge of measuring quality of life in children with Hirschsprung's disease or anorectal malformation. J Pediatr Surg. 2013 Oct;48(10):2118-27. doi: 10.1016/j.jpedsurg.2013.03.071.
PubMed 24094967Mantoo S, Meurette G, Wyart V, Hardouin J, Cretolle C, Capito C, Sarnacki S, Podevin G, Lehur PA. The impact of anorectal malformations on anorectal function and social integration in adulthood: report from a national database. Colorectal Dis. 2013 Jun;15(6):e330-5. doi: 10.1111/codi.12186.
PubMed 23464709Podevin G, Petit T, Mure PY, Gelas T, Demarche M, Allal H, Becmeur F, Varlet F, Philippe P, Weil D, Heloury Y. Minimally invasive surgery for anorectal malformation in boys: a multicenter study. J Laparoendosc Adv Surg Tech A. 2009 Apr;19 Suppl 1:S233-5. doi: 10.1089/lap.2008.0137.supp.
PubMed 18973467Fleury J, Picherot G, Cretolle C, Podevin G, David A, Caillon J, Roze JC, Gras-le Guen C. Currarino syndrome as an etiology of a neonatal Escherichia coli meningitis. J Perinatol. 2007 Sep;27(9):589-91. doi: 10.1038/sj.jp.7211783.
PubMed 17724455
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- National Study on the Quality of Life of Patients With Anorectal Malformation (MARQOL)
- ClinicalTrials.gov ID
- NCT02029248
- Acronym / sponsor ID
- MARQOL / BRD/10/06-R
- Conditions
- Anorectal Malformation
- Keywords
- Anorectal malformation, fecal incontinence, quality of life, child
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 762 participants (actual)
- Lead sponsor
- Nantes University HospitalUniversity, hospital or other
- Collaborators
- MAREP ( Center For Rare Diseases Center Ano-Rectal And Pelvic Abnormalities)
- Study start
- Apr 2012
- Primary completion
- Apr 2015 (actual)
- Study completion
- Apr 2015 (actual)
- First posted
- 7 Jan 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Anorectal malformations, occurring approximately 1 in 5000 live births, mainly involve the distal anus and rectum, but also sometimes the urinary and genital tracts. Defects range from the minor and easily treated with an excellent functional prognosis, to those that are complex and often associated with a poor functional prognosis.
Despite the better knowledge of the anatomy and physiology and the improvement of surgical management after birth, fecal and urinary incontinence can occur, due mainly to deficient nerve supply.
The quality of life of such patients is largely unknown in this country. The aim of the investigators study is to propose specific and generic questionnaires to the patients registered in the national database, correlated to their anatomical and functional status. A better understanding of such correlations should allow improvements in their medical and social management.
Detailed description
Anorectal malformations (ARM), occurring approximately 1 in 5000 live births, mainly involve the distal anus and rectum, but also sometimes the urinary and genital tracts. Defects range from the minor and easily treated with an excellent functional prognosis, to those that are complex and often associated with a poor functional prognosis. Despite the better knowledge of the anatomy and physiology and the improvement of surgical management after birth, fecal and urinary incontinence can occur, due mainly to deficient nerve supply. A few studies and clinical practice have shown that disease-specific problems of ARM have an effect on somatic function, mental health and psychosocial functioning.
The assessment of quality of life requires a self-report questionnaire composed of items related to physical, emotional and social functioning, and disease-related symptoms. A specific ARM questionnaire, the HAQL, was developed by a Dutch team and published in 2001. Associated with generic questionnaire, it allowed quality of life assessment in Dutch population.
The quality of life of such patients is largely unknown in this larger country. The aim of the investigators study is to propose specific (HAQL translated from Dutch to French) and generic questionnaires to the patients registered in the national database, correlated to their anatomical and functional status.
A better understanding of such correlations should allow improvements in their medical and social management.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Children 6-7 | – | Other: Quality of life questionnairePatients who are between 6 and 7 years old |
| Children 8-11 | – | Other: Quality of life questionnairePatients who are between 8 and 11 years old |
| Children 12-16 | – | Other: Quality of life questionnairePatients who are between 12 and 16 years old |
| Patients 17-30 | – | Other: Quality of life questionnairePatients who are between 17 and 30 years old |
Who may qualify
- Ages
- 6 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients initially treated in a french pediatric surgery service for anorectal malformation
Key inclusion criteria (2 listed)
- Patients who are between 6 and 30 years old
- Patients initially treated in a french pediatric surgery service for anorectal malformation
Key exclusion criteria (3 listed)
- Patients lost from sight or dead
- Patients with concomitant malformations that can modify sphincter functions besides anorectal malformation (spina bifida, )
- Patients whose brain functions do not allow to reply to a questionnaire (mental retardation)
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 |
|---|---|---|
| Quality of life questionnaire | Other | Children 12-16, Children 6-7, Children 8-11, Patients 17-30 |
Primary outcome
Quality of life measured by HAQL specific questionnaire and two generic questionnaires: VSPA for children or WHOQOL-BREF for adults; functional status measured by Krickenbeck score
Time frame: At the time of the inclusion
HAQL: between 35 and 48 items (depending on the age of the patients) composing 8 dimensions; VSPA: 39 items composing 8 dimensions; WHOQOL-BREF: 26 items composing 4 dimensions; Krickenbeck: 3 items.
1 secondary outcome
Kind of malformation, associated malformation, sequelas, handicap degree, social integration
Time frame: At the time of the inclusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2017.
- Background: PubMed 24094967Clermidi P, Podevin G, Cretolle C, Sarnacki S, Hardouin JB. The challenge of measuring quality of life in children with Hirschsprung's disease or anorectal malformation. J Pediatr Surg. 2013 Oct;48(10):2118-27. doi: 10.1016/j.jpedsurg.2013.03.071.
- Background: PubMed 23464709Mantoo S, Meurette G, Wyart V, Hardouin J, Cretolle C, Capito C, Sarnacki S, Podevin G, Lehur PA. The impact of anorectal malformations on anorectal function and social integration in adulthood: report from a national database. Colorectal Dis. 2013 Jun;15(6):e330-5. doi: 10.1111/codi.12186.
- Background: PubMed 18973467Podevin G, Petit T, Mure PY, Gelas T, Demarche M, Allal H, Becmeur F, Varlet F, Philippe P, Weil D, Heloury Y. Minimally invasive surgery for anorectal malformation in boys: a multicenter study. J Laparoendosc Adv Surg Tech A. 2009 Apr;19 Suppl 1:S233-5. doi: 10.1089/lap.2008.0137.supp.
- Background: PubMed 17724455Fleury J, Picherot G, Cretolle C, Podevin G, David A, Caillon J, Roze JC, Gras-le Guen C. Currarino syndrome as an etiology of a neonatal Escherichia coli meningitis. J Perinatol. 2007 Sep;27(9):589-91. doi: 10.1038/sj.jp.7211783.
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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.
Guillaume Podevin, MD, PhD principal investigator
CHU d'Angers (France)
37 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Anorectal Malformation, run by Nantes University Hospital at 37 sites in France.
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 2 inclusion and 3 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: finished, as of the sponsor's last update on 19 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in France. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Quality of life measured by HAQL specific questionnaire and two generic questionnaires: VSPA for children or WHOQOL-BREF for adults; functional status measured by Krickenbeck score (time frame: At the time of the inclusion). 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 NCT02029248, which also lists 4 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 19 Jul 2017.