Hirschsprung DiseaseALOHANCT05450991
Long-term Qualitative and Quantitative Outcomes of Children With Hirschsprung's Disease and Anorectal Malformations
An observational study of Hirschsprung Disease and Anorectal Malformations, run by Alder Hey Children's NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
Approximately 340 babies are born with Hirschsprung's disease (HSCR) or an anorectal malformation (ARM) per year in the UK. Most require corrective surgery in the newborn or early infancy period. In both conditions, there is both variability in the severity of the condition and the type of operative technique used. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hirschsprung Disease, Anorectal Malformations
- What is being tested?
- Pull through
- Type of treatment
- Surgery or procedure
- How many people
- 1,200 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 25 Jan 2022to 1 Dec 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Children and adults diagnosed with Hirschsprung's disease or Anorectal Malformation since 1991 cared for in a single centre.
Who may be able to join
- All children treated at either center with histologically confirmed Hirschsprung's Disease since diagnosis from 1991
- All children diagnosed with anorectal malformation on the basis of position in relation to the external sphincter and size
Who cannot join
- Patients with non-histologically diagnosed Hirschsprung's Disease
- Patients whose initial primary treatment or majority of follow-up for either condition has been external to either center
- Patients with funnel anus
- Adults who are unable to consent for themselves
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United Kingdom1 site, 1 recruiting
- Recruiting
Alder Hey Children's Hospital
Liverpool, Merseyside, L12 2AP
SarahRachel Almond, 01512284811, sarah.almond@alderhey.nhs.uk
About this study
Approximately 340 babies are born with Hirschsprung's disease (HSCR) or an anorectal malformation (ARM) per year in the UK. Most require corrective surgery in the newborn or early infancy period. In both conditions, there is both variability in the severity of the condition and the type of operative technique used. Many children do well following surgery and have good continence in later life. However a significant proportion of children suffer from a lifetime of constipation or incontinence. This has a significant impact on their social and psychological welfare and is a significant burden on healthcare resources.
Due to the variation in practice, there are limited data on long-term outcomes following surgery for children with HSCR or ARMs. Where studies have been performed, they often include small numbers, non-standardised outcome measures and short follow-up periods. It is therefore difficult to ascertain the effectiveness of different management strategies. However, a recent Delphi process has been carried out to establish 10 core measures in HSCR to improve outcome reporting. This research group has a long track record in this research area, specifically in the determination of long-term outcomes of children with HSCR, leading to well cited papers within the literature. In the last 2 decades there has been a significant change in the surgical techniques used in HSCR, however the underlying evidence base for this is still lacking. This study seeks to follow-on from previous studies looking specifically at the long-term outcomes in children with HSCR. Furthermore, the aim is to widen the study to encompass all children treated at Alder Hey and also to use the same methodology to assess long-term outcomes for children with ARMs, as both groups of patients experience similar long term morbidity.
This will provide qualitative and quantitative data aiding counselling parents of children with HSCR/ARM.
Read the detailed description
Hirschsprung's disease (HSCR) affects 1 in 5000 live births in the UK and is caused by a failure of development of the enteric nervous system (aganglionosis) in the distal bowel to a variable extent. Short segment HSCR, in which the aganglionic segment is restricted to the rectosigmoid region, accounts for over 80% of cases, as opposed to the more extensive long-segment HSCR which can affect the entire colon resulting in total colonic aganglionosis.
Current surgical management requires resection of the affected bowel with anastomosis of normal bowel to the anus or rectum. There are significant differences in operative and management strategies nationally and globally which include open, laparoscopic and purely trans-anal approaches, and the different pull-through procedures include the Duhamel, Swenson and the Soave. Each approach has potential advantages and disadvantages however it is not possible to draw a conclusion as to which operative strategy is the 'gold standard'.
The reasons for the current lack of clarity are multifactorial. In particular, most studies are small single-center, observational, short duration and retrospective. There is also a lack of strong evidence from systematic reviews and meta-analyses. Furthermore, there have been changes in practice over time in addition to significant outcome reporting heterogeneity in the published literature. It is therefore extremely difficult to provide accurate prognostic data to parents when the newborn is diagnosed with HSCR and at the time of the definitive pull-through procedure.
The investigators have previously reported early adult functional outcomes and long-term functional outcomes following the Duhamel procedure. Functional outcome scores were calculated by using the Rintala scoring system, and individual scores improved with time. There has since been a change in practice at Alder Hey (AH) and the more common procedure now performed is a modification of the Soave procedure. It is therefore of importance to compare the functional outcomes of patients who have undergone different procedures and to assess the longer-term outcomes of the cohort previously assessed.
Eleven years ago the investigators also published the outcomes of children with anorectal malformations (ARMs) in children presenting between 1994 and 2000. At the time the Posterior-Sagittal Ano-RectoPlasty (PSARP) had recently become the widely accepted primary surgical approach to ARMs. The study unit established a follow-up clinic dedicated solely to patients with ARM in 1994 and consultants sub-specialised to prevent dilution of case-load.
Primary outcomes were functional outcomes and quality of life. These were studied in children aged 4 years and over by the end of the study period. 85 patients qualified for the study with an age range of 4.2 to 10.1 years (median 6.7 years). 76 patients were eligible to complete the functional questionnaire and there was a response rate of 76.3% (58/76). 77.5% (62) completed the QOL score. These were compared with an age and gender-matched control group.
The study found that children with a more severe lesion had a significantly lower functional outcome score, independent of age. However, their quality of life did not seem to be impacted upon by their condition.
The long-term outcome of ARMs is poorly described. Understanding the childhood impact of ARM on a child and family's function and QOL in the medium term is important. However, the long-term impact on adolescents and adults who have this condition may differ significantly. During this period there is a significant shift in the patient's understanding and management of their own condition, and other factors including sexual function and obstetric issues become more important.
Since this study period there has been an increase in the undertaking of surgery in the neonatal period in children with a 'low' ARM, in some cases avoiding colostomy in those who would previously have required one. This change in practice may alter the impact of an ARM on a child's QOL and function.
The NETS1HD study group have recently developed a core outcome set to be used in studies comparing interventions for children with Hirschsprung's Disease which consists of a group of agreed, standardised outcomes that have been identified by key stakeholders as being the most important in determining success of treatment.
By using this core outcome set to evaluate the short and long-term outcomes of children with HSCR and ARM in the North West region, this study will be able to provide important prognostic information to parents. As this is a heterogenous group of patients, the investigators will be looking to identify the differences in long-term functional outcomes between patient subgroups and whether particular operative strategies have more favourable long-term outcomes. This will allow counselling of parents appropriately at the time of diagnosis and will assist clinical decision-making regarding the management of individual patients.
- Conditions
- Hirschsprung Disease, Anorectal Malformations
- Being tested
- Pull through
- Medical subject headings
- Hirschsprung Disease, Anorectal Malformations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pull throughProcedure or surgery
Also called PSARP, Anoplasty, Soave Procedure, Swenson's Procedure
Children diagnosed with Hirschpsrung's Disease or an anorectal malformation, with or without operative intervention will be included
| Study group | Role | What they receive |
|---|---|---|
| Hirschsprung's Disease | Infants, children and adults cared for in Alder Hey Children's Hospital from the neonatal period onward with histologically confirmed Hirschsprung's disease. Receives: Procedure: Pull through | |
| Anorectal Malformations | Infants, children and adults cared for in Alder Hey Children's Hospital from the neonatal period onward with an anorectal malformation. Receives: Procedure: Pull through |
What is being measured
The primary outcome is the main question the study is designed to answer.
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
PedsQL score for children. Compared to pre-defined controls. Score 0 (best)-92 (worst)
Measured: 1 year of age
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
PedsQL score for children. Compared to pre-defined controls. Score 0 (best)-92 (worst)
Measured: 5 years of age
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
PedsQL score for children. Compared to pre-defined controls. Score 0 (best)-92 (worst)
Measured: 10 years of age
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
PedsQL score for children. Compared to pre-defined controls Score 0 (best)-92 (worst)
Measured: 15 years of age
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
GI-QOL score. Compared to pre-defined controls. 0 (worst)-144 (best)
Measured: 20 years of age
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
GI-QOL score. Compared to pre-defined controls. 0 (worst)-144 (best)
Measured: 30 years of age
Description of urinary function of children and adults with HSCR and ARM
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Measured: 5 years of age
Description of urinary function of children and adults with HSCR and ARM
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Measured: 10 years of age
Description of urinary function of children and adults with HSCR and ARM
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Measured: 15 years of age
Description of urinary function of children and adults with HSCR and ARM
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Measured: 20 years of age
Description of urinary function of children and adults with HSCR and ARM
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Measured: 30 years of age
Comparison of the bowel function of children and adults with HSCR and ARM to pre-defined controls
Paediatric incontinence and constipation score
Measured: 5 years 0 (worst)-52 (best)
Comparison of the bowel function of children and adults with HSCR and ARM to pre-defined controls
Paediatric incontinence and constipation score
Measured: 10 years 0 (worst)-52 (best)
Comparison of the bowel function of children and adults with HSCR and ARM to pre-defined controls
Paediatric incontinence and constipation score
Measured: 15 years 0 (worst)-52 (best)
Description of the Mortality rate of children and adults with ARM and HSCR
Death rate with cause given
Measured: 1year of age
Description of the Mortality rate of children and adults with ARM and HSCR
Death rate with cause given
Measured: 5 years of age
Description of the Mortality rate of children and adults with ARM and HSCR
Death rate with cause given
Measured: 10 years of age
Description of the Mortality rate of children and adults with ARM and HSCR
Death rate with cause given
Measured: 15 years of age
Description of the Mortality rate of children and adults with ARM and HSCR
Death rate with cause given
Measured: 20 years of age
Description of the Mortality rate of children and adults with ARM and HSCR
Death rate with cause given
Measured: 30 years of age
Secondary outcomes (2)
Comparison of the sexual health of adults with HSCR and ARM compared to pre-defined controls and obstetric health
Descriptive data
Measured: 30 years of age
Description of the obstetric health of adults with HSCR and ARM
Descriptive data
Measured: 30 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2022
- Study start25 Jan 2022
- Primary completion1 Aug 2028 (estimated)
- Study completion1 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hirschsprung DiseaseStudies of the same condition, open and under way.
- 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
- Cohort
- Time perspective
- Other
- Enrolment
- 1,200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alder Hey Children's NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05450991
- Sponsor's protocol ID
- 219338
- Acronym
- ALOHA
- Status last verified
- Jan 2024
- First submitted
- 18 Jun 2022
- Last updated
- 30 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Qualitative and Quantitative Outcomes of Children With Hirschsprung's Disease and Anorectal Malformations
- ClinicalTrials.gov ID
- NCT05450991
- Acronym / sponsor ID
- ALOHA / 219338
- Conditions
- Hirschsprung Disease, Anorectal Malformations
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,200 participants (estimated)
- Lead sponsor
- Alder Hey Children's NHS Foundation TrustUniversity, hospital or other
- Study start
- 25 Jan 2022 (actual)
- Primary completion
- 1 Aug 2028 (estimated)
- Study completion
- 1 Dec 2028 (estimated)
- First posted
- 11 Jul 2022 (actual)
- 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.
Approximately 340 babies are born with Hirschsprung's disease (HSCR) or an anorectal malformation (ARM) per year in the UK. Most require corrective surgery in the newborn or early infancy period. In both conditions, there is both variability in the severity of the condition and the type of operative technique used. Many children do well following surgery and have good continence in later life. However a significant proportion of children suffer from a lifetime of constipation or incontinence. This has a significant impact on their social and psychological welfare and is a significant burden on healthcare resources.
Due to the variation in practice, there are limited data on long-term outcomes following surgery for children with HSCR or ARMs. Where studies have been performed, they often include small numbers, non-standardised outcome measures and short follow-up periods. It is therefore difficult to ascertain the effectiveness of different management strategies. However, a recent Delphi process has been carried out to establish 10 core measures in HSCR to improve outcome reporting. This research group has a long track record in this research area, specifically in the determination of long-term outcomes of children with HSCR, leading to well cited papers within the literature. In the last 2 decades there has been a significant change in the surgical techniques used in HSCR, however the underlying evidence base for this is still lacking. This study seeks to follow-on from previous studies looking specifically at the long-term outcomes in children with HSCR. Furthermore, the aim is to widen the study to encompass all children treated at Alder Hey and also to use the same methodology to assess long-term outcomes for children with ARMs, as both groups of patients experience similar long term morbidity.
This will provide qualitative and quantitative data aiding counselling parents of children with HSCR/ARM.
Detailed description
Hirschsprung's disease (HSCR) affects 1 in 5000 live births in the UK and is caused by a failure of development of the enteric nervous system (aganglionosis) in the distal bowel to a variable extent. Short segment HSCR, in which the aganglionic segment is restricted to the rectosigmoid region, accounts for over 80% of cases, as opposed to the more extensive long-segment HSCR which can affect the entire colon resulting in total colonic aganglionosis.
Current surgical management requires resection of the affected bowel with anastomosis of normal bowel to the anus or rectum. There are significant differences in operative and management strategies nationally and globally which include open, laparoscopic and purely trans-anal approaches, and the different pull-through procedures include the Duhamel, Swenson and the Soave. Each approach has potential advantages and disadvantages however it is not possible to draw a conclusion as to which operative strategy is the 'gold standard'.
The reasons for the current lack of clarity are multifactorial. In particular, most studies are small single-center, observational, short duration and retrospective. There is also a lack of strong evidence from systematic reviews and meta-analyses. Furthermore, there have been changes in practice over time in addition to significant outcome reporting heterogeneity in the published literature. It is therefore extremely difficult to provide accurate prognostic data to parents when the newborn is diagnosed with HSCR and at the time of the definitive pull-through procedure.
The investigators have previously reported early adult functional outcomes and long-term functional outcomes following the Duhamel procedure. Functional outcome scores were calculated by using the Rintala scoring system, and individual scores improved with time. There has since been a change in practice at Alder Hey (AH) and the more common procedure now performed is a modification of the Soave procedure. It is therefore of importance to compare the functional outcomes of patients who have undergone different procedures and to assess the longer-term outcomes of the cohort previously assessed.
Eleven years ago the investigators also published the outcomes of children with anorectal malformations (ARMs) in children presenting between 1994 and 2000. At the time the Posterior-Sagittal Ano-RectoPlasty (PSARP) had recently become the widely accepted primary surgical approach to ARMs. The study unit established a follow-up clinic dedicated solely to patients with ARM in 1994 and consultants sub-specialised to prevent dilution of case-load.
Primary outcomes were functional outcomes and quality of life. These were studied in children aged 4 years and over by the end of the study period. 85 patients qualified for the study with an age range of 4.2 to 10.1 years (median 6.7 years). 76 patients were eligible to complete the functional questionnaire and there was a response rate of 76.3% (58/76). 77.5% (62) completed the QOL score. These were compared with an age and gender-matched control group.
The study found that children with a more severe lesion had a significantly lower functional outcome score, independent of age. However, their quality of life did not seem to be impacted upon by their condition.
The long-term outcome of ARMs is poorly described. Understanding the childhood impact of ARM on a child and family's function and QOL in the medium term is important. However, the long-term impact on adolescents and adults who have this condition may differ significantly. During this period there is a significant shift in the patient's understanding and management of their own condition, and other factors including sexual function and obstetric issues become more important.
Since this study period there has been an increase in the undertaking of surgery in the neonatal period in children with a 'low' ARM, in some cases avoiding colostomy in those who would previously have required one. This change in practice may alter the impact of an ARM on a child's QOL and function.
The NETS1HD study group have recently developed a core outcome set to be used in studies comparing interventions for children with Hirschsprung's Disease which consists of a group of agreed, standardised outcomes that have been identified by key stakeholders as being the most important in determining success of treatment.
By using this core outcome set to evaluate the short and long-term outcomes of children with HSCR and ARM in the North West region, this study will be able to provide important prognostic information to parents. As this is a heterogenous group of patients, the investigators will be looking to identify the differences in long-term functional outcomes between patient subgroups and whether particular operative strategies have more favourable long-term outcomes. This will allow counselling of parents appropriately at the time of diagnosis and will assist clinical decision-making regarding the management of individual patients.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hirschsprung's Disease | – | Procedure: Pull throughInfants, children and adults cared for in Alder Hey Children's Hospital from the neonatal period onward with histologically confirmed Hirschsprung's disease. |
| Anorectal Malformations | – | Procedure: Pull throughInfants, children and adults cared for in Alder Hey Children's Hospital from the neonatal period onward with an anorectal malformation. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Children and adults diagnosed with Hirschsprung's disease or Anorectal Malformation since 1991 cared for in a single centre.
Key inclusion criteria (2 listed)
- All children treated at either center with histologically confirmed Hirschsprung's Disease since diagnosis from 1991
- All children diagnosed with anorectal malformation on the basis of position in relation to the external sphincter and size
Key exclusion criteria (4 listed)
- Patients with non-histologically diagnosed Hirschsprung's Disease
- Patients whose initial primary treatment or majority of follow-up for either condition has been external to either center
- Patients with funnel anus
- Adults who are unable to consent for themselves
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 |
|---|---|---|
| Pull throughAlso: PSARP, Anoplasty, Soave Procedure, Swenson's ProcedureChildren diagnosed with Hirschpsrung's Disease or an anorectal malformation, with or without operative intervention will be included | Procedure or surgery | Anorectal Malformations, Hirschsprung's Disease |
Primary outcome
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
Time frame: 1 year of age
PedsQL score for children. Compared to pre-defined controls. Score 0 (best)-92 (worst)
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
Time frame: 5 years of age
PedsQL score for children. Compared to pre-defined controls. Score 0 (best)-92 (worst)
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
Time frame: 10 years of age
PedsQL score for children. Compared to pre-defined controls. Score 0 (best)-92 (worst)
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
Time frame: 15 years of age
PedsQL score for children. Compared to pre-defined controls Score 0 (best)-92 (worst)
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
Time frame: 20 years of age
GI-QOL score. Compared to pre-defined controls. 0 (worst)-144 (best)
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls
Time frame: 30 years of age
GI-QOL score. Compared to pre-defined controls. 0 (worst)-144 (best)
Description of urinary function of children and adults with HSCR and ARM
Time frame: 5 years of age
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Description of urinary function of children and adults with HSCR and ARM
Time frame: 10 years of age
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Description of urinary function of children and adults with HSCR and ARM
Time frame: 15 years of age
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Description of urinary function of children and adults with HSCR and ARM
Time frame: 20 years of age
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Description of urinary function of children and adults with HSCR and ARM
Time frame: 30 years of age
Proportion requiring urinary catheterisation or experiencing urinary incontinence
Comparison of the bowel function of children and adults with HSCR and ARM to pre-defined controls
Time frame: 5 years 0 (worst)-52 (best)
Paediatric incontinence and constipation score
Comparison of the bowel function of children and adults with HSCR and ARM to pre-defined controls
Time frame: 10 years 0 (worst)-52 (best)
Paediatric incontinence and constipation score
Comparison of the bowel function of children and adults with HSCR and ARM to pre-defined controls
Time frame: 15 years 0 (worst)-52 (best)
Paediatric incontinence and constipation score
Description of the Mortality rate of children and adults with ARM and HSCR
Time frame: 1year of age
Death rate with cause given
Description of the Mortality rate of children and adults with ARM and HSCR
Time frame: 5 years of age
Death rate with cause given
Description of the Mortality rate of children and adults with ARM and HSCR
Time frame: 10 years of age
Death rate with cause given
Description of the Mortality rate of children and adults with ARM and HSCR
Time frame: 15 years of age
Death rate with cause given
Description of the Mortality rate of children and adults with ARM and HSCR
Time frame: 20 years of age
Death rate with cause given
Description of the Mortality rate of children and adults with ARM and HSCR
Time frame: 30 years of age
Death rate with cause given
2 secondary outcomes
Comparison of the sexual health of adults with HSCR and ARM compared to pre-defined controls and obstetric health
Time frame: 30 years of age
Description of the obstetric health of adults with HSCR and ARM
Time frame: 30 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2024.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hirschsprung Disease and Anorectal Malformations, run by Alder Hey Children's NHS Foundation Trust at 1 site in United Kingdom.
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 4 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: looking for participants now, as of the sponsor's last update on 30 Jan 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 Kingdom. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls (time frame: 1 year of age). The study lists 20 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 30 Jan 2024.