Ulcerative ColitisPhase 1/2ALOASCUNCT01914887
Allogeneic Adipose Tissue-derived Mesenchymal Stem Cells for the Induction of Remission in Ulcerative Colitis
A Phase 1/2 study testing Allogeneic adipose tissue-derived mesenchymal stem cells in people with Ulcerative Colitis, run by Instituto de Investigación Hospital Universitario La Paz at 1 site in Spain.
Trial summary
The aims of our study are to evaluate the feasibility and safety of endoscopic injection of adipose tissue-derived mesenchymal stem cells in human subjects with moderate active ulcerative colitis, assessing the absence of adverse events associated to the investigational drug, and to evaluate the efficacy of the… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Ulcerative Colitis
- What is being tested?
- Allogeneic adipose tissue-derived mesenchymal stem cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteSpain
- Study dates
- Jun 2013to Dec 2013
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients of either sex aged 18 years and older
- Signed informed consent
- Patients with ulcerative colitis diagnosed at least 6 months earlier in accordance with usual criteria
- Left-sided colitis with moderate activity defined by a modified Truelove-Witts score between 11 and 21, and with no response to 4 weeks of treatment with oral and/or topical 5-aminosalicylates
- Negative pregnancy test for women of childbearing potential (from menarche to menopause) using consistently and correctly highly effective (i.e. less than 1% failure rate per year) methods of birth control
Who cannot join
- Mental disability that impedes adequate understanding of the study and of the associated procedures
- Extensive colitis
- Patients with an impaired general state which requires, according to the investigator judgment, immediate treatment with corticosteroids and/or anti-Tumor Necrosis Factor (TNF) and/or surgery
- Patients that fulfill criteria of corticodependency and in ongoing treatment with corticosteroids
- Patients with previous colectomies
- Known history of alcohol or other addictive substances abuse
Show all 11Show fewer
- History of malignant disease - Patients having participated in clinical trials with any investigational drug within 6 months prior to enrolment in this study
- Patients with known allergies to penicillin, gentamicin, aminoglycosides, human serum albumin (HSA), Dulbecco's modified Eagle medium (DMEM), or materials of bovine origin
- Pregnant or breastfeeding women
- Presence of severe concomitant diseases
- Patients with suspicion of Crohn?s enterocolitis, indeterminate colitis, ischaemic colitis, radiation colitis, diverticular disease associated colitis, or microscopic colitis
Trial locations and contacts
1 site in 1 country.
Spain1 site, 1 recruiting
- Recruiting
Hospital Universitario La Paz
Madrid, 28046
Maria Dolores Martin Arranz, MD, +34 917277467, mmartinarranz@salud.madrid.org
Fernando de Miguel, PhD, +34 917277389, fernando.demiguel@salud.madrid.org
About this study
Official title: A Phase I/IIa Clinical Trial to Evaluate Safety and Efficacy of Adipose Tissue-derived Mesenchymal Stem Cells (ASC) on Induction to Remission in Ulcerative Colitis
The aims of our study are to evaluate the feasibility and safety of endoscopic injection of adipose tissue-derived mesenchymal stem cells in human subjects with moderate active ulcerative colitis, assessing the absence of adverse events associated to the investigational drug, and to evaluate the efficacy of the treatment to induce remission of moderate active ulcerative colitis, by improvements in disease activity index, quality of life index, and endoscopic index.
Read the detailed description
Mesenchymal stem cells (MSC) may be a therapeutic option in diseases associated with severe inflammation or auto-immune diseases, due to their immunomodulatory and anti-inflammatory properties. A number of clinical trials are being conducted worldwide testing th efficacy of MSC, mainly isolated from bone marrow, for different conditions, such as Graft Versus host Disease, refractory Crohn's Disease, ischemic stroke, acute myocardial infarction, type I Diabetes Mellitus, or Chronic Obstructive Pulmonary Disease. Usually, the route of administration of the cells in these studies is intravenous. Local injection of MSC for fistulizing Crohn's Disease has proven efficacious. Endoscopy is a routinary technique for the evaluation of gastrointestinal and colonic conditions. The purpose of our study is to evaluate safety and efficacy of the intracolonic injection by using a colonoscope of allogeneic adipose tissue-derived MSC in patients with moderate active ulcerative colitis.
- Conditions
- Ulcerative Colitis
- Being tested
- Allogeneic adipose tissue-derived mesenchymal stem cells
- Keywords
- Ulcerative Colitis, Inflammatory Bowel Disease, Mesenchymal stem cells
- Medical subject headings
- Colitis, Ulcerative, Inflammatory Bowel Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic adipose tissue-derived mesenchymal stem cellsDrug
Also called Cx-601 (company code)
The cells will be given in different sites within the affected colonic submucosa at a total dose of 60 million cells with the use of a colonoscope.
| Study group | Role | What they receive |
|---|---|---|
| allogeneic ASCs | Experimental | Treatment consists in a cell suspension (5 million cells/mL) in aseptic buffered solution containing human expanded adipose-derived stem cells (eASCs) of allogeneic origin in disposable vials with no preservative agents. The cells will be given in different sites within the affected colonic submucosa at a total dose of 60 million cells with the use of a colonoscope. Receives: Drug: Allogeneic adipose tissue-derived mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
Evaluation of the presence of any event that could be considered adverse event, especially if it can be attributed to the investigational drug. Physical exam, vital signs, and laboratory tests (hemogram, biochemistry, coagulation, and cytokines) will be performed at 0, 9-10 days, and 4, 8, and 12 weeks.
Measured: Up to 12 weeks
Secondary outcomes (5)
Efficacy: Change from Baseline in Modified Truelove-Witts score
Remission will be considered if it descends below 11, and response if it diminishes at least 30%. Modified Truelove-Witts score will be evaluated at 0, 4, 8, and 12 weeks.
Measured: Up to 12 weeks
Efficacy: Change from Baseline in Quality of Life index, Inflammatory Bowel Disease Questionnaire (IBDQ-32)
Response will be considered if it improves at least 30%. IBDQ-32 will be evaluated at 0, 4, 8, and 12 weeks.
Measured: Up to 12 weeks
Efficacy: Change from baseline in Mayo endoscopic index.
Remission will be considered if reaches 0 points and response if the score diminishes. Endoscopy will be performed at 0 and 8 weeks.
Measured: 8 weeks
Change from Baseline in C Reactive Protein
C Reactive Protein will be evaluated at 0, 9-10 days, and 4, 8, 12 weeks.
Measured: Up to 12 weeks
Change from Baseline in fecal calprotectin
Fecal calprotectin will be evaluated at 0, 4, 8, and 12 weeks.
Measured: Up to 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2013 (estimated)
- Study startJun 2013
- Primary completionDec 2013 (estimated)
- Study completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Ulcerative ColitisStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Instituto de Investigación Hospital Universitario La Paz
- Sponsor type
- University, hospital or other
- Collaborators
- Fundacion para la Investigacion Biomedica del Hospital Universitario la Paz, Ministry of Health, Spain
- Responsible party
- Sponsor
- Investigators
- Maria Dolores Martin Arranz, MD, Principal investigator, Gastroenterology Department
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01914887
- Sponsor's protocol ID
- HLPDIG-2010-01
- Other IDs
- 2010-023798-20
- Acronym
- ALOASCU
- Status last verified
- Jul 2013
- First submitted
- 18 Jul 2013
- Last updated
- 2 Aug 2013
- Results posted
- No
- Data monitoring committee
- No
Publications
Garcia-Gomez I, Elvira G, Zapata AG, Lamana ML, Ramirez M, Castro JG, Arranz MG, Vicente A, Bueren J, Garcia-Olmo D. Mesenchymal stem cells: biological properties and clinical applications. Expert Opin Biol Ther. 2010 Oct;10(10):1453-68. doi: 10.1517/14712598.2010.519333.
PubMed 20831449Duijvestein M, Vos AC, Roelofs H, Wildenberg ME, Wendrich BB, Verspaget HW, Kooy-Winkelaar EM, Koning F, Zwaginga JJ, Fidder HH, Verhaar AP, Fibbe WE, van den Brink GR, Hommes DW. Autologous bone marrow-derived mesenchymal stromal cell treatment for refractory luminal Crohn's disease: results of a phase I study. Gut. 2010 Dec;59(12):1662-9. doi: 10.1136/gut.2010.215152. Epub 2010 Oct 4.
PubMed 20921206van Deen WK, Oikonomopoulos A, Hommes DW. Stem cell therapy in inflammatory bowel disease: which, when and how? Curr Opin Gastroenterol. 2013 Jul;29(4):384-90. doi: 10.1097/MOG.0b013e328361f763.
PubMed 23666365
Study at a glance
From the registry record, last updated by the sponsor on 2 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/IIa Clinical Trial to Evaluate Safety and Efficacy of Adipose Tissue-derived Mesenchymal Stem Cells (ASC) on Induction to Remission in Ulcerative Colitis
- ClinicalTrials.gov ID
- NCT01914887
- Acronym / sponsor ID
- ALOASCU / HLPDIG-2010-01
- Conditions
- Ulcerative Colitis
- Keywords
- Ulcerative Colitis, Inflammatory Bowel Disease, Mesenchymal stem cells
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Instituto de Investigación Hospital Universitario La PazUniversity, hospital or other
- Collaborators
- Fundacion para la Investigacion Biomedica del Hospital Universitario la Paz, Ministry of Health, Spain
- Study start
- Jun 2013
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Dec 2013 (estimated)
- First posted
- 2 Aug 2013 (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.
The aims of our study are to evaluate the feasibility and safety of endoscopic injection of adipose tissue-derived mesenchymal stem cells in human subjects with moderate active ulcerative colitis, assessing the absence of adverse events associated to the investigational drug, and to evaluate the efficacy of the treatment to induce remission of moderate active ulcerative colitis, by improvements in disease activity index, quality of life index, and endoscopic index.
Detailed description
Mesenchymal stem cells (MSC) may be a therapeutic option in diseases associated with severe inflammation or auto-immune diseases, due to their immunomodulatory and anti-inflammatory properties. A number of clinical trials are being conducted worldwide testing th efficacy of MSC, mainly isolated from bone marrow, for different conditions, such as Graft Versus host Disease, refractory Crohn's Disease, ischemic stroke, acute myocardial infarction, type I Diabetes Mellitus, or Chronic Obstructive Pulmonary Disease. Usually, the route of administration of the cells in these studies is intravenous. Local injection of MSC for fistulizing Crohn's Disease has proven efficacious. Endoscopy is a routinary technique for the evaluation of gastrointestinal and colonic conditions. The purpose of our study is to evaluate safety and efficacy of the intracolonic injection by using a colonoscope of allogeneic adipose tissue-derived MSC in patients with moderate active ulcerative colitis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| allogeneic ASCs | Experimental | Drug: Allogeneic adipose tissue-derived mesenchymal stem cellsTreatment consists in a cell suspension (5 million cells/mL) in aseptic buffered solution containing human expanded adipose-derived stem cells (eASCs) of allogeneic origin in disposable vials with no preservative agents. The cells will be given in different sites within the affected colonic submucosa at a total dose of 60 million cells with the use of a colonoscope. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patients of either sex aged 18 years and older
- Signed informed consent
- Patients with ulcerative colitis diagnosed at least 6 months earlier in accordance with usual criteria
- Left-sided colitis with moderate activity defined by a modified Truelove-Witts score between 11 and 21, and with no response to 4 weeks of treatment with oral and/or topical 5-aminosalicylates
- Negative pregnancy test for women of childbearing potential (from menarche to menopause) using consistently and correctly highly effective (i.e. less than 1% failure rate per year) methods of birth control
Key exclusion criteria (11 listed)
- Mental disability that impedes adequate understanding of the study and of the associated procedures
- Extensive colitis
- Patients with an impaired general state which requires, according to the investigator judgment, immediate treatment with corticosteroids and/or anti-Tumor Necrosis Factor (TNF) and/or surgery
- Patients that fulfill criteria of corticodependency and in ongoing treatment with corticosteroids
- Patients with previous colectomies
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 |
|---|---|---|
| Allogeneic adipose tissue-derived mesenchymal stem cellsAlso: Cx-601 (company code)The cells will be given in different sites within the affected colonic submucosa at a total dose of 60 million cells with the use of a colonoscope. | Drug | allogeneic ASCs |
Primary outcome
Safety
Time frame: Up to 12 weeks
Evaluation of the presence of any event that could be considered adverse event, especially if it can be attributed to the investigational drug. Physical exam, vital signs, and laboratory tests (hemogram, biochemistry, coagulation, and cytokines) will be performed at 0, 9-10 days, and 4, 8, and 12 weeks.
5 secondary outcomes
Efficacy: Change from Baseline in Modified Truelove-Witts score
Time frame: Up to 12 weeks
Efficacy: Change from Baseline in Quality of Life index, Inflammatory Bowel Disease Questionnaire (IBDQ-32)
Time frame: Up to 12 weeks
Efficacy: Change from baseline in Mayo endoscopic index.
Time frame: 8 weeks
Change from Baseline in C Reactive Protein
Time frame: Up to 12 weeks
Change from Baseline in fecal calprotectin
Time frame: Up to 12 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Aug 2013.
- Background: PubMed 20831449Garcia-Gomez I, Elvira G, Zapata AG, Lamana ML, Ramirez M, Castro JG, Arranz MG, Vicente A, Bueren J, Garcia-Olmo D. Mesenchymal stem cells: biological properties and clinical applications. Expert Opin Biol Ther. 2010 Oct;10(10):1453-68. doi: 10.1517/14712598.2010.519333.
- Background: PubMed 20921206Duijvestein M, Vos AC, Roelofs H, Wildenberg ME, Wendrich BB, Verspaget HW, Kooy-Winkelaar EM, Koning F, Zwaginga JJ, Fidder HH, Verhaar AP, Fibbe WE, van den Brink GR, Hommes DW. Autologous bone marrow-derived mesenchymal stromal cell treatment for refractory luminal Crohn's disease: results of a phase I study. Gut. 2010 Dec;59(12):1662-9. doi: 10.1136/gut.2010.215152. Epub 2010 Oct 4.
- Background: PubMed 23666365van Deen WK, Oikonomopoulos A, Hommes DW. Stem cell therapy in inflammatory bowel disease: which, when and how? Curr Opin Gastroenterol. 2013 Jul;29(4):384-90. doi: 10.1097/MOG.0b013e328361f763.
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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.
Maria Dolores Martin Arranz, MD principal investigator
Gastroenterology Department
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Allogeneic adipose tissue-derived mesenchymal stem cells in people with Ulcerative Colitis, run by Instituto de Investigación Hospital Universitario La Paz at 1 site in Spain.
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 5 inclusion and 11 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 2 Aug 2013. 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 Spain. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Maria Dolores Martin Arranz, MD (+34 917277467, mmartinarranz@salud.madrid.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety (time frame: Up to 12 weeks). 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 NCT01914887, which also lists 3 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 2 Aug 2013.