Crohn DiseaseNCT05262829
Clinical Study of Human Umbilical Cord Mesenchymal Stem Cells in the Treatment of Moderate and Severe Crohn's Disease
A study testing Human Umbilical Cord Mesenchymal Stem Cells in people with Crohn Disease, run by Shanghai East Hospital at 1 site in China.
Trial summary
This is a prospective, single-arm, open-ended study to evaluate the efficacy and safety of human umbilical cord MSCs in the treatment of refractory moderate-to-severe Crohn's disease. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Crohn Disease
- What is being tested?
- Human Umbilical Cord Mesenchymal Stem Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 28 Feb 2022to 31 Jan 2025
- Phase
- Interventional
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects aged between 18 and 70 years (including borderline values), of either sex.
- Subjects with a comprehensive diagnosis of Crohn's disease for more than 3 months based on the patient's clinical presentation, endoscopy, imaging and pathology, with reference to the Consensus Opinion on the Diagnosis and Treatment of Inflammatory Bowel Disease in China (2018-Beijing).
- Failure to respond to existing conventional therapy, or to primary or secondary treatment with TNF alpha monoclonal antibody, vedolizumab or ustekinumabd.
- Current Crohn's disease with a Crohn's Disease Activity Index (CDAI) score ≥ 220 and ≤ 450.
- Evidence of active inflammation and ulceration confirmed by endoscopy during screening.
- Subjects must be free of active, latent, or undertreated Mycobacterium tuberculosis infection.
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- All women of childbearing potential and all men must be willing to use at least one highly effective method of contraception at the time of signing the consent form and throughout the study period until 1 month after the last dose of study drug.
- Subjects who are willing and able to undergo treatment and follow-up, laboratory tests, and other study procedures as planned.
- Subjects who are able to sign (and date) an informed consent form indicating that the subject has been informed of all relevant parts of the study
- Subjects receiving non-disabling combination therapy for any reason must maintain a stable treatment regimen, defined as no stem cell therapy given or no dose change within 7 days or 5 half-lives (whichever is longer) prior to the initial stem cell injection.
Who cannot join
- Presence of a complication of Crohn's disease, such as short bowel syndrome or any other manifestation that might be expected to require surgery, that prevents the use of CDAI to assess treatment response, or that might confound the assessment of the efficacy of MSC therapy.
- Current abscess or suspected abscess. If the abscess has drained and been adequately treated 3 weeks prior to baseline (skin and perianal abscesses) and 8 weeks prior to baseline (intra-abdominal abscesses) then no exclusion is necessary. The prerequisite is that no further surgical treatment is anticipated. Subjects may be enrolled in the study if they have an active fistula that is not expected to require surgery and is confirmed to be abscess-free.
- Subjects with evidence of pathogenic intestinal infection. Subjects with Clostridium difficile or other intestinal infections within 30 days of endoscopic screening, or subjects who screen positive for C. difficile toxin assay or other pathogens.
- Subjects with total bilirubin, aspartate aminotransferase (AST) or alanine aminotransferase (ALT) above 2 times the upper limit of normal at the screening visit. Patients with cirrhosis will be excluded.
- Subjects with eGFR ≤ 60 mL/min (as calculated by Cockcroft-Gault) or patients receiving hemodialysis.
- Subjects with a current or clinically significant infection within 1 month prior to baseline, or history of more than one prior occurrence of herpes zoster, disseminated zoster (one occurrence) and other infections that the investigator believes may be exacerbated by participation in the study, or any infection requiring antimicrobial therapy within 2 weeks of screening.
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- Subjects who may currently be receiving any live virus vaccination or who have received any live virus vaccination within 8 weeks prior to baseline.
- Subjects with a first-degree relative with a hereditary immunodeficiency.
- History of any lymphoid proliferative disease (e.g., EBV-associated lymphoid proliferative disease), lymphoma, leukemia, myeloproliferative disease, multiple myeloma, or signs and symptoms suggestive of presenting lymphoid system disease.
- Subjects who have received prior treatment with any lymphocyte depleting agent/therapy. Subjects with prior treatment with rituximab or other selective B lymphocyte depleting agents, but who have not received such treatment for at least 1 year prior to baseline, are eligible for the study.
- Pregnant or lactating women or female subjects who are pregnant during planned enrollment in the study.
- Prior history of alcohol or drug abuse and less than 6 months of abstinence prior to baseline.
- Subjects with clinically relevant abnormalities confirmed by 12-lead ECG during the screening period that would affect their safety if enrolled in this study or affect the interpretation of the study results.
- Subjects who have donated more than 500 mL of blood in the 2 months prior to baseline.
- Subjects who have experienced major trauma or undergone major surgery within 4 weeks of the screening visit.
- Subjects with a body temperature ≥38°C during the screening period or baseline period.
- Subjects with malignancy or a history of malignancy.
- Subjects infected with human immunodeficiency virus (HIV) or hepatitis B virus or hepatitis C virus.
- Subjects who, in the opinion of the investigator, are uncooperative or unable to comply with the study procedures.
- Any other condition that, in the opinion of the investigator, would render the subject unsuitable for enrollment in the study.
- Subjects with preexisting or current clinically significant cardiovascular, neurological, psychiatric, renal, hepatic, immunological, gastrointestinal, genitourinary, neurological, musculoskeletal, cutaneous, sensory, endocrine (including uncontrolled diabetes or thyroid disease) or uncontrolled hematological abnormalities. "Clinically significant" is defined as a subject whose participation in the study would, in the opinion of the investigator, pose a risk to the safety of the subject or whose disease/condition would be exacerbated during the study in a manner that would affect the validity or safety analysis.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Shanghai East Hospital
Shanghai, Pudong New Area
Lan Zhong, +86-13162099450, lanzhong@tongji.edu.cn
About this study
Official title: A Single-arm, Open-label Clinical Study of Human Umbilical Cord Mesenchymal Stem Cells in the Treatment of Refractory Moderate to Severe Crohn's Disease
This is a prospective, single-arm, open-ended study to evaluate the efficacy and safety of human umbilical cord MSCs in the treatment of refractory moderate-to-severe Crohn's disease. The study protocol is either MSC injection into the patient's diseased intestinal mucosa or intravenous MSC injection + MSC injection into the patient's diseased intestinal mucosa. Follow-up time points were pre-treatment (week 0), week 4, week 8, week 12, and week 24 post-treatment, and the primary evaluation at follow-up was the number of subjects with clinical and endoscopic response or remission.
Read the detailed description
This is a prospective, single-arm, open study to evaluate the efficacy and safety of human umbilical cord MSCs in the treatment of refractory moderate-to-severe Crohn's disease. The study will be conducted as follows: a total of 40 patients will be recruited and will receive either MSC injection into the diseased intestinal mucosa or intravenous MSC injection + MSC injection into the diseased intestinal mucosa. The proportion of patients with clinical and endoscopic response or remission will be used as the primary evaluation for follow-up. The Crohn's disease activity index (CDAI) was used to assess the severity of CD disease, with CDAI <150 considered to be in clinical remission; a decrease in CDAI ≥70 was considered to be clinically effective, also referred to as clinical response. Endoscopic response was defined as at least 50% improvement in the simplified endoscopic score for Crohn's disease (SES-CD) from baseline, and endoscopic remission was defined as a SES-CD score ≤2.
- Conditions
- Crohn Disease
- Being tested
- Human Umbilical Cord Mesenchymal Stem Cells
- Keywords
- Human Umbilical Cord Mesenchymal Stem Cells, Crohn Disease
- Medical subject headings
- Crohn Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Umbilical Cord Mesenchymal Stem CellsBiological
MSCs treatment by intestinal submucosal injection/MSCs treatment by intravenous drip + MSCs treatment by intestinal submucosal injection
| Study group | Role | What they receive |
|---|---|---|
| MSCs local treatment group/combined treatment group | Experimental | In the local treatment group, 60 million umbilical cord MSCs were injected into the diseased intestinal mucosa on the first day. In the combined treatment group, 60 million umbilical cord MSCs were injected into the diseased intestinal mucosa on the first day; on the second day, 1 million cells/kg of body weight were administered intravenously. Receives: Biological: Human Umbilical Cord Mesenchymal Stem Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Crohn's disease activity index score
The Crohn's Disease Activity Index (CDAI) was used to assess the severity of CD disease, and a CDAI <150 was considered clinically remitting; a decrease in CDAI ≥70 was considered clinically effective, also known as clinical response.
Measured: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
simplified endoscopic score for Crohn's disease
Endoscopic response was defined as at least 50% improvement in the simplified endoscopic score for Crohn's disease (SES-CD) from baseline, and endoscopic remission was defined as a SES-CD score ≤2.
Measured: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Secondary outcomes (5)
Inflammatory Bowel Disease Questionnaire
The Inflammatory Bowel Disease Questionnaire (IBDQ) is a widely used questionnaire for Health-related quality of life assessment in patients with inflammatory bowel diseases (IBDs).
Measured: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Albumin
Albumin is an important component of the blood, and patients with severe Crohn's disease can present with clinical signs of hypoproteinemia.
Measured: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Hemoglobin
Hemoglobin is an important component of the blood, and patients with severe Crohn's disease can present with clinical signs of anemia.
Measured: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Fecal calprotectin
Fecal calprotectin (FC) is a non-invasive marker of gastrointestinal inflammation with advocated diagnostic precision in distinguishing inflammatory bowel disease (IBD) from non-IBD diagnoses.
Measured: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
body mass index (BMI)
Weight and height will be combined to report BMI in kg/m^2
Measured: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2022
- Study start28 Feb 2022
- Primary completion31 Aug 2024 (estimated)
- Study completion31 Jan 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Crohn 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai East Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lan Zhong, MD, Study director, Shanghai East Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05262829
- Sponsor's protocol ID
- DFSC-2021(CR)-07
- Status last verified
- Feb 2023
- First submitted
- 8 Feb 2022
- Last updated
- 17 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Open-label Clinical Study of Human Umbilical Cord Mesenchymal Stem Cells in the Treatment of Refractory Moderate to Severe Crohn's Disease
- ClinicalTrials.gov ID
- NCT05262829
- Acronym / sponsor ID
- DFSC-2021(CR)-07
- Conditions
- Crohn Disease
- Keywords
- Human Umbilical Cord Mesenchymal Stem Cells, Crohn Disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Shanghai East HospitalUniversity, hospital or other
- Study start
- 28 Feb 2022 (actual)
- Primary completion
- 31 Aug 2024 (estimated)
- Study completion
- 31 Jan 2025 (estimated)
- First posted
- 2 Mar 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.
This is a prospective, single-arm, open-ended study to evaluate the efficacy and safety of human umbilical cord MSCs in the treatment of refractory moderate-to-severe Crohn's disease. The study protocol is either MSC injection into the patient's diseased intestinal mucosa or intravenous MSC injection + MSC injection into the patient's diseased intestinal mucosa. Follow-up time points were pre-treatment (week 0), week 4, week 8, week 12, and week 24 post-treatment, and the primary evaluation at follow-up was the number of subjects with clinical and endoscopic response or remission.
Detailed description
This is a prospective, single-arm, open study to evaluate the efficacy and safety of human umbilical cord MSCs in the treatment of refractory moderate-to-severe Crohn's disease. The study will be conducted as follows: a total of 40 patients will be recruited and will receive either MSC injection into the diseased intestinal mucosa or intravenous MSC injection + MSC injection into the diseased intestinal mucosa. The proportion of patients with clinical and endoscopic response or remission will be used as the primary evaluation for follow-up. The Crohn's disease activity index (CDAI) was used to assess the severity of CD disease, with CDAI <150 considered to be in clinical remission; a decrease in CDAI ≥70 was considered to be clinically effective, also referred to as clinical response. Endoscopic response was defined as at least 50% improvement in the simplified endoscopic score for Crohn's disease (SES-CD) from baseline, and endoscopic remission was defined as a SES-CD score ≤2.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MSCs local treatment group/combined treatment group | Experimental | Biological: Human Umbilical Cord Mesenchymal Stem CellsIn the local treatment group, 60 million umbilical cord MSCs were injected into the diseased intestinal mucosa on the first day. In the combined treatment group, 60 million umbilical cord MSCs were injected into the diseased intestinal mucosa on the first day; on the second day, 1 million cells/kg of body weight were administered intravenously. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Subjects aged between 18 and 70 years (including borderline values), of either sex.
- Subjects with a comprehensive diagnosis of Crohn's disease for more than 3 months based on the patient's clinical presentation, endoscopy, imaging and pathology, with reference to the Consensus Opinion on the Diagnosis and Treatment of Inflammatory Bowel Disease in China (2018-Beijing).
- Failure to respond to existing conventional therapy, or to primary or secondary treatment with TNF alpha monoclonal antibody, vedolizumab or ustekinumabd.
- Current Crohn's disease with a Crohn's Disease Activity Index (CDAI) score ≥ 220 and ≤ 450.
- Evidence of active inflammation and ulceration confirmed by endoscopy during screening.
Key exclusion criteria (21 listed)
- Presence of a complication of Crohn's disease, such as short bowel syndrome or any other manifestation that might be expected to require surgery, that prevents the use of CDAI to assess treatment response, or that might confound the assessment of the efficacy of MSC therapy.
- Current abscess or suspected abscess. If the abscess has drained and been adequately treated 3 weeks prior to baseline (skin and perianal abscesses) and 8 weeks prior to baseline (intra-abdominal abscesses) then no exclusion is necessary. The prerequisite is that no further surgical treatment is anticipated. Subjects may be enrolled in the study if they have an active fistula that is not expected to require surgery and is confirmed to be abscess-free.
- Subjects with evidence of pathogenic intestinal infection. Subjects with Clostridium difficile or other intestinal infections within 30 days of endoscopic screening, or subjects who screen positive for C. difficile toxin assay or other pathogens.
- Subjects with total bilirubin, aspartate aminotransferase (AST) or alanine aminotransferase (ALT) above 2 times the upper limit of normal at the screening visit. Patients with cirrhosis will be excluded.
- Subjects with eGFR ≤ 60 mL/min (as calculated by Cockcroft-Gault) or patients receiving hemodialysis.
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 |
|---|---|---|
| Human Umbilical Cord Mesenchymal Stem CellsMSCs treatment by intestinal submucosal injection/MSCs treatment by intravenous drip + MSCs treatment by intestinal submucosal injection | Biological | MSCs local treatment group/combined treatment group |
Primary outcome
Crohn's disease activity index score
Time frame: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
The Crohn's Disease Activity Index (CDAI) was used to assess the severity of CD disease, and a CDAI <150 was considered clinically remitting; a decrease in CDAI ≥70 was considered clinically effective, also known as clinical response.
simplified endoscopic score for Crohn's disease
Time frame: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Endoscopic response was defined as at least 50% improvement in the simplified endoscopic score for Crohn's disease (SES-CD) from baseline, and endoscopic remission was defined as a SES-CD score ≤2.
5 secondary outcomes
Inflammatory Bowel Disease Questionnaire
Time frame: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Albumin
Time frame: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Hemoglobin
Time frame: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Fecal calprotectin
Time frame: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
body mass index (BMI)
Time frame: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2023.
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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.
Lan Zhong, MD study director
Shanghai East Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Human Umbilical Cord Mesenchymal Stem Cells in people with Crohn Disease, run by Shanghai East Hospital 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 10 inclusion and 21 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: status not confirmed, as of the sponsor's last update on 17 Feb 2023. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Lan Zhong, MD (+86-13162099450, lanzhong@tongji.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Crohn's disease activity index score (time frame: Changes from baseline to week 4, week 8, week 12 and week 24 after stem cell treatment.). The study lists 2 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 17 Feb 2023.