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Allogeneic Adipose Tissue-derived Mesenchymal Stem Cells for the Induction of Remission in Ulcerative Colitis (ALOASCU)

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2013 by Instituto de Investigación Hospital Universitario La Paz
Sponsor:
Collaborators:
Fundacion para la Investigacion Biomedica del Hospital Universitario la Paz
Ministry of Health, Spain
Information provided by (Responsible Party):
Instituto de Investigación Hospital Universitario La Paz
ClinicalTrials.gov Identifier:
NCT01914887
First received: July 18, 2013
Last updated: July 31, 2013
Last verified: July 2013
  Purpose

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.


Condition Intervention Phase
Ulcerative Colitis
Drug: Allogeneic adipose tissue-derived mesenchymal stem cells
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Instituto de Investigación Hospital Universitario La Paz:

Primary Outcome Measures:
  • Safety [ Time Frame: Up to 12 weeks ] [ Designated as safety issue: Yes ]
    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.


Secondary Outcome Measures:
  • Efficacy: Change from Baseline in Modified Truelove-Witts score [ Time Frame: Up to 12 weeks ] [ Designated as safety issue: No ]
    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.

  • Efficacy: Change from Baseline in Quality of Life index, Inflammatory Bowel Disease Questionnaire (IBDQ-32) [ Time Frame: Up to 12 weeks ] [ Designated as safety issue: No ]
    Response will be considered if it improves at least 30%. IBDQ-32 will be evaluated at 0, 4, 8, and 12 weeks.

  • Efficacy: Change from baseline in Mayo endoscopic index. [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
    Remission will be considered if reaches 0 points and response if the score diminishes. Endoscopy will be performed at 0 and 8 weeks.

  • Change from Baseline in C Reactive Protein [ Time Frame: Up to 12 weeks ] [ Designated as safety issue: No ]
    C Reactive Protein will be evaluated at 0, 9-10 days, and 4, 8, 12 weeks.

  • Change from Baseline in fecal calprotectin [ Time Frame: Up to 12 weeks ] [ Designated as safety issue: No ]
    Fecal calprotectin will be evaluated at 0, 4, 8, and 12 weeks.


Estimated Enrollment: 8
Study Start Date: June 2013
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: allogeneic ASCs
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.
Drug: Allogeneic adipose tissue-derived mesenchymal stem cells
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.
Other Name: Cx-601 (company code)

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01914887

Contacts
Contact: Maria Dolores Martin Arranz, MD +34 917277467 mmartinarranz@salud.madrid.org
Contact: Fernando de Miguel, PhD +34 917277389 fernando.demiguel@salud.madrid.org

Locations
Spain
Hospital Universitario La Paz Recruiting
Madrid, Spain, 28046
Contact: Maria Dolores Martin Arranz, MD    +34 917277467    mmartinarranz@salud.madrid.org   
Contact: Fernando de Miguel, PhD    +34 917277389    fernando.demiguel@salud.madrid.org   
Principal Investigator: Maria Dolores Martin Arranz, MD         
Sponsors and Collaborators
Instituto de Investigación Hospital Universitario La Paz
Fundacion para la Investigacion Biomedica del Hospital Universitario la Paz
Ministry of Health, Spain
Investigators
Principal Investigator: Maria Dolores Martin Arranz, MD Gastroenterology Department
  More Information

Publications:
Responsible Party: Instituto de Investigación Hospital Universitario La Paz
ClinicalTrials.gov Identifier: NCT01914887     History of Changes
Other Study ID Numbers: HLPDIG-2010-01, 2010-023798-20
Study First Received: July 18, 2013
Last Updated: July 31, 2013
Health Authority: Spain: Agencia Española de Medicamentos y Productos Sanitarios

Keywords provided by Instituto de Investigación Hospital Universitario La Paz:
Ulcerative Colitis
Inflammatory Bowel Disease
Mesenchymal stem cells

Additional relevant MeSH terms:
Colitis
Colitis, Ulcerative
Ulcer
Colonic Diseases
Digestive System Diseases
Gastroenteritis
Gastrointestinal Diseases
Inflammatory Bowel Diseases
Intestinal Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on November 25, 2014