Crohn's DiseasePhase 1/2NCT01372969
Study to Assess the Safety and Efficacy of Expanded Allogenic Adipose-derived Stem Cells (eASCs) (Cx601), for Treatment of Complex Perianal Fistulas in Perianal Crohn's Disease.
A Phase 1/2 study testing Cx601 in people with Crohn's Disease and Anal Fistula, run by Tigenix S.A.U. at 6 sites in Spain.
Trial summary
The purpose of this study is to asses the safety and efficacy of adipose-derived adult stem cells from healthy donnors for treatment of complex perianal fistulas in Crohn's disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Crohn's Disease, Anal Fistula
- What is being tested?
- Cx601
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 6 sitesSpain
- Study dates
- Jun 2009to Sep 2010
- 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
- Signed informed consent.
- Subjects with Crohn's disease diagnosed at least 12 months earlier in accordance with accepted clinical, endoscopic, anatomopathological and/or radiologic criteria.
- Presence of complex perianal fistula with 3 or fewer fistulous tracts assessed by MRI,
- Subjects with persistent and active complex perianal fistula and non active luminal CD defined by a CDAI ≤ 200.
- Subjects of either sex aged over 18 years. Good general state of health according to the findings of the clinical history and the physical examination.
Who cannot join
- Presence of severe proctitis (prominent friability, spontaneous bleeding, multiple erosions, deep ulcers) or dominant active luminal disease requiring immediate therapy, assessed by rectosigmoidoscopy
- Subjects with a CDAI ≥ 221.
- Subjects with an abscess (unless a complete toilet of the area with drainage of the collections and the absence of abscess and other collections is confirmed prior to treatment start).
- The presence of setons unless removed prior to treatment start.
- Presence of >3 fistulous tracts and/or external openings.
- Subjects with rectal and/or anal stenosis evaluated by rectoscopy or EUA.
Show all 21Show fewer
- Subjects who have received infliximab or any other anti-TNF agent in the 8 weeks before the cell treatment administration.
- Subjects who have received tracrolimus or ciclosporine in the 4 weeks before the cell treatment administration.
- Subjects with rectovaginal fistula, anal fistula(s), and/or non-perianal enterocutaneous fistula.
- Subjects with a history of abuse of alcohol or other addictive substances in the 6 months prior to inclusion.
- Subjects with malignant tumor, except for basal cell or cutaneous squamous cell carcinoma, or Subjects with a prior history of malignant tumors, unless the neoplastic disease has been in remission for the previous 5 years.
- Subjects with cardiopulmonary disease which, in the opinion of the investigator, is unstable or sufficiently serious to exclude the patient from the study.
- Subjects with any type of medical or psychiatric disease which, in the opinion of the investigator, could be grounds for exclusion from the study.
- Subjects with congenital or acquired immunodeficiencies.
- Subjects with abnormal laboratory test findings that contraindicate their inclusion in the study.
- Subjects allergic to anesthetics or MRI contrast.
- MRI is unfeasible, (e.g. due to the presence of pacemakers, hip replacements or severe claustrophobia)
- Subjects in need of surgery in the perianal region for reasons other than fistulas at the time of inclusion in the study, or a need for such surgery is foreseen in this region in the 26 weeks after treatment administration.
- Subjects who have suffered major surgery or severe trauma in the prior 6 months.
- Pregnant or breastfeeding women. (Both men and women should use appropriate birth control methods defined by the investigator).
- Subjects currently receiving, or having received within 1 month prior to enrolment into this clinical trial, any investigational drug.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Spain6 sites
Hospital San Juan de Dios del Aljarafe
Bormujos, Sevilla, 41930
Hospital Juan Ramón Jimenez
Huelva, 21005
Hospital Universitario La Paz
Madrid, 28042
Hospital Son Dureta
Palma de Mallorca, Balearic Islands, 07014
Hospital Universitario Nuestra Señora de Valme
Seville, 41014
Hospital Virgen de la Macarena
Seville, 41071
About this study
Official title: Multicenter Phase I/IIa Study to Assess the Safety and Efficacy of Expanded Allogenic Adipose-derived Stem Cells (eASCs) (Cx601), for Treatment of Complex Perianal Fistulas in Perianal Crohn's Disease.
The purpose of this study is to asses the safety and efficacy of adipose-derived adult stem cells from healthy donnors for treatment of complex perianal fistulas in Crohn's disease.
Read the detailed description
Crohn´s disease (CD) is a severe disorder with significant morbidity and major impact on life. CD can affect any part of the digestive system and symptoms of this chronic illness include abdominal pain, bloating, nausea, vomiting and diarrhea. CD also causes bowel wall ulcers, strictures (narrowing of a hollow structure due to scar tissue and swelling), and fistulas (abnormal passages from the intestines to another organ or the skin).
There is an unmet need for effective medical therapy in CD patients with perianal fistulas, not responding to the conventional strategies, including biological therapies. The current study is designed to assess the safety and efficacy of adipose tissue derived mesenchymal stem cells from healthy donors for the treatment of perianal fistulas in patients presenting CD.
Primary objective: To assess the safety (incidence of treatment emergent adverse-events) of eASCs.
Study design: Multicenter, pilot study, assessing safety and with evaluation of the efficacy by two Gastroenterologists/Surgeons and an independent external radiologist. Subjects will receive a cell suspension of allogenic adipose-derived stem cells, eASCs (20 million cells) in the fistula.
In case of incomplete fistula closure of the treated fistula at week 12, subjects will receive a second dose of eASCs (40 million cells). Subjects will be followed until week 24, following the initial administration of cells.
Subjects may be treated with standard care during the entire study period, according to the investigators discretion, excluding infliximab or any other anti-TNF, tacrolimus or cyclosporine.
. Study population: In total 24 patients, men and women of at least 18 years of age, with perianal CD will be recruited. Patients with a minimum of one and a maximum of three fistula tracts.
Description of treatments: .
Subjects will receive:
Investigational product: CX-601 Suspension of adult expanded allogenic adipose-derived stem cells (eASCs) at a doses of 20 million cells and 40 million cells.
- Conditions
- Crohn's Disease, Anal Fistula
- Being tested
- Cx601
- Medical subject headings
- Crohn Disease, Rectal Fistula
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cx601Drug
Suspension of adult expanded allogenic adipose-derived stem cells (eASCs) at a doses of 20 million cells and 40 million cells.
| Study group | Role | What they receive |
|---|---|---|
| Cx601 | Experimental | Not described Receives: Drug: Cx601 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment emergent adverse-events
Measured: 24 weeks
Secondary outcomes (5)
The reduction in the number of draining fistulas
Measured: weeks 10,12, 22 and 24
The increase in the number of closed fistulas.
Measured: weeks 12 and 24
Percentage of subjects in whom, the external openings of treated perianal fistula have closed.
Measured: week 12
Percentage of subjects with MRI fistula healing (absence of collections >2cm)
Measured: weeks 12 and 24
Percentage of subjects presenting luminal relapse
Measured: weeks 12 and 24.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2011 (estimated)
- Study startJun 2009
- Primary completionSep 2010
- Study completionSep 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Crohn's 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tigenix S.A.U.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Damián García Olmo, MD, Principal investigator, Hospital La Paz
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01372969
- Sponsor's protocol ID
- Cx601-0101
- Status last verified
- Apr 2019
- First submitted
- 30 Aug 2010
- Last updated
- 12 Apr 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Phase I/IIa Study to Assess the Safety and Efficacy of Expanded Allogenic Adipose-derived Stem Cells (eASCs) (Cx601), for Treatment of Complex Perianal Fistulas in Perianal Crohn's Disease.
- ClinicalTrials.gov ID
- NCT01372969
- Acronym / sponsor ID
- Cx601-0101
- Conditions
- Crohn's Disease, Anal Fistula
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Tigenix S.A.U.Industry (pharma, biotech, devices)
- Study start
- Jun 2009
- Primary completion
- Sep 2010 (actual)
- Study completion
- Sep 2010 (actual)
- First posted
- 14 Jun 2011 (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 purpose of this study is to asses the safety and efficacy of adipose-derived adult stem cells from healthy donnors for treatment of complex perianal fistulas in Crohn's disease.
Detailed description
Crohn´s disease (CD) is a severe disorder with significant morbidity and major impact on life. CD can affect any part of the digestive system and symptoms of this chronic illness include abdominal pain, bloating, nausea, vomiting and diarrhea. CD also causes bowel wall ulcers, strictures (narrowing of a hollow structure due to scar tissue and swelling), and fistulas (abnormal passages from the intestines to another organ or the skin).
There is an unmet need for effective medical therapy in CD patients with perianal fistulas, not responding to the conventional strategies, including biological therapies. The current study is designed to assess the safety and efficacy of adipose tissue derived mesenchymal stem cells from healthy donors for the treatment of perianal fistulas in patients presenting CD.
Primary objective: To assess the safety (incidence of treatment emergent adverse-events) of eASCs.
Study design: Multicenter, pilot study, assessing safety and with evaluation of the efficacy by two Gastroenterologists/Surgeons and an independent external radiologist. Subjects will receive a cell suspension of allogenic adipose-derived stem cells, eASCs (20 million cells) in the fistula.
In case of incomplete fistula closure of the treated fistula at week 12, subjects will receive a second dose of eASCs (40 million cells). Subjects will be followed until week 24, following the initial administration of cells.
Subjects may be treated with standard care during the entire study period, according to the investigators discretion, excluding infliximab or any other anti-TNF, tacrolimus or cyclosporine.
. Study population: In total 24 patients, men and women of at least 18 years of age, with perianal CD will be recruited. Patients with a minimum of one and a maximum of three fistula tracts.
Description of treatments: .
Subjects will receive:
Investigational product: CX-601 Suspension of adult expanded allogenic adipose-derived stem cells (eASCs) at a doses of 20 million cells and 40 million cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cx601 | Experimental | Drug: Cx601 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Signed informed consent.
- Subjects with Crohn's disease diagnosed at least 12 months earlier in accordance with accepted clinical, endoscopic, anatomopathological and/or radiologic criteria.
- Presence of complex perianal fistula with 3 or fewer fistulous tracts assessed by MRI,
- Subjects with persistent and active complex perianal fistula and non active luminal CD defined by a CDAI ≤ 200.
- Subjects of either sex aged over 18 years. Good general state of health according to the findings of the clinical history and the physical examination.
Key exclusion criteria (21 listed)
- Presence of severe proctitis (prominent friability, spontaneous bleeding, multiple erosions, deep ulcers) or dominant active luminal disease requiring immediate therapy, assessed by rectosigmoidoscopy
- Subjects with a CDAI ≥ 221.
- Subjects with an abscess (unless a complete toilet of the area with drainage of the collections and the absence of abscess and other collections is confirmed prior to treatment start).
- The presence of setons unless removed prior to treatment start.
- Presence of >3 fistulous tracts and/or external openings.
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 |
|---|---|---|
| Cx601Suspension of adult expanded allogenic adipose-derived stem cells (eASCs) at a doses of 20 million cells and 40 million cells. | Drug | Cx601 |
Primary outcome
Incidence of treatment emergent adverse-events
Time frame: 24 weeks
5 secondary outcomes
The reduction in the number of draining fistulas
Time frame: weeks 10,12, 22 and 24
The increase in the number of closed fistulas.
Time frame: weeks 12 and 24
Percentage of subjects in whom, the external openings of treated perianal fistula have closed.
Time frame: week 12
Percentage of subjects with MRI fistula healing (absence of collections >2cm)
Time frame: weeks 12 and 24
Percentage of subjects presenting luminal relapse
Time frame: weeks 12 and 24.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2019.
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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.
Damián García Olmo, MD principal investigator
Hospital La Paz
6 sites 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 Cx601 in people with Crohn's Disease and Anal Fistula, run by Tigenix S.A.U. at 6 sites 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 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: finished, as of the sponsor's last update on 12 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of treatment emergent adverse-events (time frame: 24 weeks). 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 12 Apr 2019.