Inflammatory Bowel DiseasesNCT03752372
Microbiome Alterations in IL10RA-deficient Patients After HSCT
An observational study of Inflammatory Bowel Diseases and Microbiota, run by Children's Hospital of Fudan University at 1 site in China.
Trial summary
To elucidate the longitudinal development of intestinal microbiota in patients with IL10RA deficiency after hematopoietic stem cell transplantation (HSCT). The investigators planned to collect fecal samples from IL10RA-deficient patients who received HSCT. Read more about this study
- Who is it for?
- Ages 1 day to 6 yearsHealthy volunteers can take part
- Conditions the study is for
- Inflammatory Bowel Diseases, Microbiota
- What is being tested?
- hematopoietic stem cell transplantation
- Type of treatment
- Injection or infusion, Surgery or procedure
- How many people
- 13 participants
- Where
- 1 siteChina
- Study dates
- 1 Oct 2017to 28 Feb 2020
- 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 1 day to 6 years
- All sexes
- Healthy volunteers accepted
- Child
Study population: Patients who were diagnosed with IL10RA deficiency and who received the hematopoietic stem cell transplantation in a tertiary hospital were enrolled.
Who may be able to join
- IL10RA gene mutations;
- Eligible for hematopoietic stem cell transplantation.
Who cannot join
- Unwilling to participate.
Trial locations and contacts
1 site in 1 country.
China1 site
Children's hospital of Fudan university
Shanghai, Shanghai Municipality, 201102
About this study
Official title: Longitudinal Development of Intestinal Microbiome in IL10RA-deficient Patients After Hematopoietic Stem Cell Transplantation
To elucidate the longitudinal development of intestinal microbiota in patients with IL10RA deficiency after hematopoietic stem cell transplantation (HSCT). The investigators planned to collect fecal samples from IL10RA-deficient patients who received HSCT. Samples were collected more than once every three days after engraftment in lamina flow ward and at least once a week before discharge. Microbial DNA was extracted from the fecal samples. And all analysis was based on the next generation sequencing data.
Read the detailed description
Hematopoietic stem cell transplantation is considered the only curative therapy for patients with interleukin-10 receptor-A(IL10RA) deficiency. The investigators aimed to collect the fecal samples of these patients throughout the conditioning, transplantation until discharge, providing a dense insight into the longitudinal development of intestinal microbiota.
- Conditions
- Inflammatory Bowel Diseases, Microbiota
- Being tested
- hematopoietic stem cell transplantation
- Keywords
- interleukin 10 receptor alpha gene
- Medical subject headings
- Inflammatory Bowel Diseases, Inflammatory Bowel Disease 28, Autosomal Recessive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hematopoietic stem cell transplantationProcedure or surgery
- Reduced intensity conditioning(RIC) chemotherapy regimen before transplantation
- All patients were cared for in single rooms ventilated with a highly effective particulate air filtration system. All patients received intravenous immunoglobulin and antimicrobial prophylaxis, which included antiviral, antifungal, and Pneumocystis jirovecii prophylaxis with ganciclovir and micafungin, as per routine clinical practice before transplantation.
- Umbilical cord blood transplantation
| Study group | Role | What they receive |
|---|---|---|
| HSCT cohort | IL10RA-deficient patients who received hematopoietic stem cell transplantation Receives: Procedure: hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Shannon indices
Shannon index is a popular diversity index in the ecological literature; minimum score is zero, higher values represent a better outcome
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
Simpson indices
Simpson index in ecology is to measure the degree of concentration when individuals are classified into types; scale ranges [0,1]; lower values represent a better outcome
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
relative abundance of Proteobacteria
describe the microbiome at the phylum level
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
relative abundance of Firmicutes
describe the microbiome at the phylum level
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
Clostridium sensus_stricto_1
describe the microbiome at the genus level
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
Escherichia - Shigella
describe the microbiome at the genus level
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
Secondary outcomes (6)
chimerism
The level of chimerism of each patient will be reported as percentages [0,100%]
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
manifestation of graft-versus-host disease
describe the graft-versus-host disease happened to each patient
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
Sobs indices
describe the the observed richness of each sample
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
Chao1 estimator
describe the operational taxonomic unit(OTU) numbers of each sample
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
neutrophil cell count
blood test results
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
platelet count
blood test results
Measured: during the hospitalization of each eligible patient, an average of 90 days per patient
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2018
- Study start1 Oct 2017
- Primary completion28 Feb 2020
- Study completion28 Feb 2020
Why it stopped: COVID-19
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Inflammatory Bowel DiseasesStudies 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
- Prospective
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ying HUANG, Children's Hospital of Fudan University
- Investigators
- Ying Huang, MD, Principal investigator, Children's Hospital of Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03752372
- Sponsor's protocol ID
- HSCT microbiomte
- Status last verified
- Aug 2020
- First submitted
- 19 Nov 2018
- Last updated
- 12 Aug 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Peng K, Qian X, Huang Z, Lu J, Wang Y, Zhou Y, Wang H, Wu B, Wang Y, Chen L, Zhai X, Huang Y. Umbilical Cord Blood Transplantation Corrects Very Early-Onset Inflammatory Bowel Disease in Chinese Patients With IL10RA-Associated Immune Deficiency. Inflamm Bowel Dis. 2018 Jun 8;24(7):1416-1427. doi: 10.1093/ibd/izy028.
PubMed 29788474
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Longitudinal Development of Intestinal Microbiome in IL10RA-deficient Patients After Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT03752372
- Acronym / sponsor ID
- HSCT microbiomte
- Conditions
- Inflammatory Bowel Diseases, Microbiota
- Keywords
- interleukin 10 receptor alpha gene
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: COVID-19
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Study start
- 1 Oct 2017 (actual)
- Primary completion
- 28 Feb 2020 (actual)
- Study completion
- 28 Feb 2020 (actual)
- First posted
- 26 Nov 2018 (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.
To elucidate the longitudinal development of intestinal microbiota in patients with IL10RA deficiency after hematopoietic stem cell transplantation (HSCT). The investigators planned to collect fecal samples from IL10RA-deficient patients who received HSCT. Samples were collected more than once every three days after engraftment in lamina flow ward and at least once a week before discharge. Microbial DNA was extracted from the fecal samples. And all analysis was based on the next generation sequencing data.
Detailed description
Hematopoietic stem cell transplantation is considered the only curative therapy for patients with interleukin-10 receptor-A(IL10RA) deficiency. The investigators aimed to collect the fecal samples of these patients throughout the conditioning, transplantation until discharge, providing a dense insight into the longitudinal development of intestinal microbiota.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HSCT cohort | – | Procedure: hematopoietic stem cell transplantationIL10RA-deficient patients who received hematopoietic stem cell transplantation |
Who may qualify
- Ages
- 1 day to 6 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- Patients who were diagnosed with IL10RA deficiency and who received the hematopoietic stem cell transplantation in a tertiary hospital were enrolled.
Key inclusion criteria (2 listed)
- IL10RA gene mutations;
- Eligible for hematopoietic stem cell transplantation.
Key exclusion criteria (1 listed)
- Unwilling to participate.
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 |
|---|---|---|
| hematopoietic stem cell transplantation1. Reduced intensity conditioning(RIC) chemotherapy regimen before transplantation 2. All patients were cared for in single rooms ventilated with a highly effective particulate air filtration system. All patients received intravenous immunoglobulin and antimicrobial prophylaxis, which included antiviral, antifungal, and Pneumocystis jirovecii prophylaxis with ganciclovir and micafungin, as per routine clinical practice before transplantation. 3. Umbilical cord blood transplantation | Procedure or surgery | HSCT cohort |
Primary outcome
Shannon indices
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
Shannon index is a popular diversity index in the ecological literature; minimum score is zero, higher values represent a better outcome
Simpson indices
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
Simpson index in ecology is to measure the degree of concentration when individuals are classified into types; scale ranges [0,1]; lower values represent a better outcome
relative abundance of Proteobacteria
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
describe the microbiome at the phylum level
relative abundance of Firmicutes
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
describe the microbiome at the phylum level
Clostridium sensus_stricto_1
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
describe the microbiome at the genus level
Escherichia - Shigella
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
describe the microbiome at the genus level
6 secondary outcomes
chimerism
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
manifestation of graft-versus-host disease
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
Sobs indices
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
Chao1 estimator
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
neutrophil cell count
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
platelet count
Time frame: during the hospitalization of each eligible patient, an average of 90 days per patient
Results and safety
The study was stopped early (reason given: COVID-19). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2020.
- Results publication: PubMed 29788474Peng K, Qian X, Huang Z, Lu J, Wang Y, Zhou Y, Wang H, Wu B, Wang Y, Chen L, Zhai X, Huang Y. Umbilical Cord Blood Transplantation Corrects Very Early-Onset Inflammatory Bowel Disease in Chinese Patients With IL10RA-Associated Immune Deficiency. Inflamm Bowel Dis. 2018 Jun 8;24(7):1416-1427. doi: 10.1093/ibd/izy028.
- 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.
Ying Huang, MD principal investigator
Children's Hospital of Fudan University
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 Inflammatory Bowel Diseases and Microbiota, run by Children's Hospital of Fudan University at 1 site in China.
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 1 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 12 Aug 2020. 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 China. 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 was stopped early (reason given: COVID-19). Results are not available in the source record.
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?
Shannon indices (time frame: during the hospitalization of each eligible patient, an average of 90 days per patient). The study lists 6 primary outcomes in all. 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 NCT03752372, which also lists 1 linked publication.
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 12 Aug 2020; AzTrials last refreshed its copy on 31 Oct 2026.