Anal FistulaPhase 3FATT1NCT00475410
Efficacy and Safety of Adipose Stem Cells to Treat Complex Perianal Fistulas Not Associated to Crohn´s Disease
A Phase 3 study testing ASCs (Cx401, company code) and Fibrin adhesive in people with Anal Fistula, run by Tigenix S.A.U. at 10 sites in 3 countries.
Trial summary
Anal fistula is defined as an abnormal communication between the anal canal and the perianal skin. Adipose-derived stem cells are a new therapy for the closure of these fistulas. This study will test the safety and efficacy of ASCs (adipose stem cells) in the treatment of patients without Crohn´s disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anal Fistula
- What is being tested?
- ASCs (Cx401, company code) and Fibrin adhesive
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 214 participants
- Where
- 10 sitesin 3 countries
- Study dates
- Feb 2007to Aug 2009
- Phase
- Phase 3
- 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
- Patient with a complex perianal fistula. A complex perianal fistula must meet one of the following:
- No fistula tract is palpated under the perianal skin or tract parallel to the rectum on examination with a stylet.
- Associated anal incontinence in transsphincteric fistulas.
- Risk factors for anal incontinence.
- At least one prior surgery for a fistulous disease.
- Suprasphincteric tracts shown by an image test.
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- Women of childbearing age should have a negative serum or urine pregnancy test (sensitive to 25IU of hCG) and not be lactating before study entry. Both men and women should use adequate birth control methods as defined by the investigator.
- Seton presence is allowed at the time of study entry and until cells (in groups receiving ASCs) or Fibrin adhesive are implanted.
- No condition that may prevent the patient from following the study procedures until 26 weeks of follow-up are completed is foreseen.
- Patient should give his/her signed, written informed consent. -
Who cannot join
- Patient has been diagnosed with IBD
- Patient has a rectovaginal fistula
- Patient is pregnant or lactating woman
- Patient has acute sepsis at the time of study entry
- A liposuction to draw at least 100 cc of fat from the abdominal wall is not technically feasible.
- Patient needs surgery in the perianal region for reasons other than fistulas
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- Presence of two or more complex perianal fistulas
- Patient has collections > 2 cm in MRI. If such collections exist, the surgeon may perform a complete toilette of the area.
- Patient is allergic to local anesthetics or Gadolinium (MRI contrast agent).
- MRI is not technically feasible.
- Patient has abused alcohol or other addictive substances within 6 months of study entry.
- Patient has active or latent infection by HIV, HBV or HCV.
- Patient has undergone major surgery or sustained a severe trauma, in the investigator's judgment, within 28 days of recruitment.
- Patient is receiving or has received immunomodulatory treatment for reasons other than the fistula within 6 months of study entry.
- Patient has a malignant tumour, except for basal or squamous cell carcinoma of the skin, or has a prior history of a malignant tumour, except if the tumour has been in remission for at least the past 5 years.
- -
Trial locations and contacts
10 sites in 3 countries.
10 sites in 3 countries
Spain8 sites
Gregorio Marañon University Hospital
Madrid, 28007
San Carlos Clinical Hospital
Madrid, 28040
12 De Octubre University Hospital
Madrid, 28041
La Paz University Hospital
Madrid, 28046
Virgen Del Camino Hospital
Pamplona, Navarre, 31008
Mutua de Terrasa Hospital
Tarrasa, Tarragona, 08221
Universitary General Hospital
Valencia, 46014
Lozano Blesa Clinical Hospital
Zaragoza, Aragon, 59009
About this study
Official title: A Phase III Multicenter, Single Blind, Randomized, Comparative and add-on Clinical Trial, in Three Parallel Groups, to Evaluate Efficacy and Safety of a New Therapy With Adipose-derived Autologous Stem Cells for the Treatment of Complex Perianal Fistulas in Patients Without Inflammatory Bowel Disease
Anal fistula is defined as an abnormal communication between the anal canal and the perianal skin. Adipose-derived stem cells are a new therapy for the closure of these fistulas. This study will test the safety and efficacy of ASCs (adipose stem cells) in the treatment of patients without Crohn´s disease.
Read the detailed description
Perianal fistula accounts for 10% to 30% of coloproctological surgical procedures. Currently accepted conventional treatment is surgery intended to treat the tracts using different technical options. This surgery usually has a highly bothersome postoperative period and may involve two major complications: anal incontinence and recurrence. The biological properties of stem cells derived from adult tissues make them adequate candidates for the treatment of diseases in which tissues are damaged or the healing process is altered. This study will compare the efficacy of ASCs versus ASCs plus Fibrin adhesive versus Fibrin adhesive alone for closure of complex perianal fistulas not associated to Inflammatory Bowel Disease. Fistula closure is defined as absence of suppuration and re-epithelization of the external opening in the clinical evaluation and absence of collections >2 cm directly related to the fistula tract treated, as measured by MRI
- Conditions
- Anal Fistula
- Being tested
- ASCs (Cx401, company code), Fibrin adhesive
- Keywords
- Autologous adipose-derived stem cells, Complex perianal fistula, Inflammatory Bowel Disease, Advanced cell therapy, Fibrin glue
- Medical subject headings
- Rectal Fistula, Inflammatory Bowel Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASCs (Cx401, company code)Drug
Experimental: ASCs: Subjects will be treated with a dose of 20 million ASCs and evaluated after 12 weeks. If needed a second dose of 40 million ASCs will be applied then.
Experimental: ASCs+fibrin glue: Subjects will be treated with a dose of 20 million ASCs plus fibrin glue and evaluated after 12 weeks. If needed a second dose of 40 million ASCs plus fibrin glue will be applied then.
Fibrin adhesiveDrug
Experimental: Fibrin glue: Subjects will be treated with a dose fibrin glue and evaluated after 12 weeks. If needed a second dose of fibrin glue will be applied then.
| Study group | Role | What they receive |
|---|---|---|
| ASCs | Experimental | Not described Receives: Drug: ASCs (Cx401, company code) |
| ASCs+fibrin glue | Experimental | Not described Receives: Drug: ASCs (Cx401, company code); Drug: Fibrin adhesive |
| Fibrin glue | Active comparator | Not described Receives: Drug: Fibrin adhesive |
What is being measured
The primary outcome is the main question the study is designed to answer.
Closure of fistulas defined as absence of suppuration and re-epithelization of the external opening and absence of collections>2 cm directly related to the fistula tract treated, as measured by MRI (healing)
Measured: weeks 1, 4, 12 and 24. Week 26 in patients with a second dose
Secondary outcomes (1)
• Clinical complexity of fistula (complexity of fistula score) • Safety: Cumulative incidence of adverse effects. • Quality of life (SF-36 score) • Degree of anal incontinence (Wexner incontinence score)
Measured: weeks 1, 4, 12 and 24. Week 26 in patients with a second dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2007 (estimated)
- Study startFeb 2007
- Primary completionAug 2009
- Study completionAug 2009
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 Anal FistulaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 214 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tigenix S.A.U.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Damian Garcia Olmo, Dr, Principal investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00475410
- Sponsor's protocol ID
- Cx401/FATT1
- Other IDs
- 2006-003370-95
- Acronym
- FATT1
- Status last verified
- Apr 2019
- First submitted
- 17 May 2007
- Last updated
- 12 Apr 2019
- Results posted
- No
- Data monitoring committee
- No
Publications
Garcia-Olmo D, Garcia-Arranz M, Herreros D, Pascual I, Peiro C, Rodriguez-Montes JA. A phase I clinical trial of the treatment of Crohn's fistula by adipose mesenchymal stem cell transplantation. Dis Colon Rectum. 2005 Jul;48(7):1416-23. doi: 10.1007/s10350-005-0052-6.
PubMed 15933795Garcia-Olmo D, Garcia-Arranz M, Garcia LG, Cuellar ES, Blanco IF, Prianes LA, Montes JA, Pinto FL, Marcos DH, Garcia-Sancho L. Autologous stem cell transplantation for treatment of rectovaginal fistula in perianal Crohn's disease: a new cell-based therapy. Int J Colorectal Dis. 2003 Sep;18(5):451-4. doi: 10.1007/s00384-003-0490-3. Epub 2003 May 20.
PubMed 12756590Herreros MD, Garcia-Arranz M, Guadalajara H, De-La-Quintana P, Garcia-Olmo D; FATT Collaborative Group. Autologous expanded adipose-derived stem cells for the treatment of complex cryptoglandular perianal fistulas: a phase III randomized clinical trial (FATT 1: fistula Advanced Therapy Trial 1) and long-term evaluation. Dis Colon Rectum. 2012 Jul;55(7):762-72. doi: 10.1097/DCR.0b013e318255364a.
PubMed 22706128
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
- A Phase III Multicenter, Single Blind, Randomized, Comparative and add-on Clinical Trial, in Three Parallel Groups, to Evaluate Efficacy and Safety of a New Therapy With Adipose-derived Autologous Stem Cells for the Treatment of Complex Perianal Fistulas in Patients Without Inflammatory Bowel Disease
- ClinicalTrials.gov ID
- NCT00475410
- Acronym / sponsor ID
- FATT1 / Cx401/FATT1
- Conditions
- Anal Fistula
- Keywords
- Autologous adipose-derived stem cells, Complex perianal fistula, Inflammatory Bowel Disease, Advanced cell therapy, Fibrin glue
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 214 participants (actual)
- Lead sponsor
- Tigenix S.A.U.Industry (pharma, biotech, devices)
- Study start
- Feb 2007
- Primary completion
- Aug 2009 (actual)
- Study completion
- Aug 2009 (actual)
- First posted
- 21 May 2007 (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.
Anal fistula is defined as an abnormal communication between the anal canal and the perianal skin. Adipose-derived stem cells are a new therapy for the closure of these fistulas. This study will test the safety and efficacy of ASCs (adipose stem cells) in the treatment of patients without Crohn´s disease.
Detailed description
Perianal fistula accounts for 10% to 30% of coloproctological surgical procedures. Currently accepted conventional treatment is surgery intended to treat the tracts using different technical options. This surgery usually has a highly bothersome postoperative period and may involve two major complications: anal incontinence and recurrence. The biological properties of stem cells derived from adult tissues make them adequate candidates for the treatment of diseases in which tissues are damaged or the healing process is altered. This study will compare the efficacy of ASCs versus ASCs plus Fibrin adhesive versus Fibrin adhesive alone for closure of complex perianal fistulas not associated to Inflammatory Bowel Disease. Fistula closure is defined as absence of suppuration and re-epithelization of the external opening in the clinical evaluation and absence of collections >2 cm directly related to the fistula tract treated, as measured by MRI
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ASCs | Experimental | Drug: ASCs (Cx401, company code) |
| ASCs+fibrin glue | Experimental | Drug: ASCs (Cx401, company code), Drug: Fibrin adhesive |
| Fibrin glue | Active comparator | Drug: Fibrin adhesive |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Patient with a complex perianal fistula. A complex perianal fistula must meet one of the following:
- Women of childbearing age should have a negative serum or urine pregnancy test (sensitive to 25IU of hCG) and not be lactating before study entry. Both men and women should use adequate birth control methods as defined by the investigator.
- Seton presence is allowed at the time of study entry and until cells (in groups receiving ASCs) or Fibrin adhesive are implanted.
- No condition that may prevent the patient from following the study procedures until 26 weeks of follow-up are completed is foreseen.
- Patient should give his/her signed, written informed consent. -
Key exclusion criteria (16 listed)
- Patient has been diagnosed with IBD
- Patient has a rectovaginal fistula
- Patient is pregnant or lactating woman
- Patient has acute sepsis at the time of study entry
- A liposuction to draw at least 100 cc of fat from the abdominal wall is not technically feasible.
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 |
|---|---|---|
| ASCs (Cx401, company code)Experimental: ASCs: Subjects will be treated with a dose of 20 million ASCs and evaluated after 12 weeks. If needed a second dose of 40 million ASCs will be applied then. Experimental: ASCs+fibrin glue: Subjects will be treated with a dose of 20 million ASCs plus fibrin glue and evaluated after 12 weeks. If needed a second dose of 40 million ASCs plus fibrin glue will be applied then. | Drug | ASCs, ASCs+fibrin glue |
| Fibrin adhesiveExperimental: Fibrin glue: Subjects will be treated with a dose fibrin glue and evaluated after 12 weeks. If needed a second dose of fibrin glue will be applied then. | Drug | ASCs+fibrin glue, Fibrin glue |
Primary outcome
Closure of fistulas defined as absence of suppuration and re-epithelization of the external opening and absence of collections>2 cm directly related to the fistula tract treated, as measured by MRI (healing)
Time frame: weeks 1, 4, 12 and 24. Week 26 in patients with a second dose
1 secondary outcome
• Clinical complexity of fistula (complexity of fistula score) • Safety: Cumulative incidence of adverse effects. • Quality of life (SF-36 score) • Degree of anal incontinence (Wexner incontinence score)
Time frame: weeks 1, 4, 12 and 24. Week 26 in patients with a second dose
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.
- Results publication: PubMed 15933795Garcia-Olmo D, Garcia-Arranz M, Herreros D, Pascual I, Peiro C, Rodriguez-Montes JA. A phase I clinical trial of the treatment of Crohn's fistula by adipose mesenchymal stem cell transplantation. Dis Colon Rectum. 2005 Jul;48(7):1416-23. doi: 10.1007/s10350-005-0052-6.
- Results publication: PubMed 12756590Garcia-Olmo D, Garcia-Arranz M, Garcia LG, Cuellar ES, Blanco IF, Prianes LA, Montes JA, Pinto FL, Marcos DH, Garcia-Sancho L. Autologous stem cell transplantation for treatment of rectovaginal fistula in perianal Crohn's disease: a new cell-based therapy. Int J Colorectal Dis. 2003 Sep;18(5):451-4. doi: 10.1007/s00384-003-0490-3. Epub 2003 May 20.
- Derived from this study: PubMed 22706128Herreros MD, Garcia-Arranz M, Guadalajara H, De-La-Quintana P, Garcia-Olmo D; FATT Collaborative Group. Autologous expanded adipose-derived stem cells for the treatment of complex cryptoglandular perianal fistulas: a phase III randomized clinical trial (FATT 1: fistula Advanced Therapy Trial 1) and long-term evaluation. Dis Colon Rectum. 2012 Jul;55(7):762-72. doi: 10.1097/DCR.0b013e318255364a.
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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.
Damian Garcia Olmo, Dr principal investigator
10 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ASCs (Cx401, company code) and Fibrin adhesive in people with Anal Fistula, run by Tigenix S.A.U. at 10 sites in 3 countries.
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 16 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 10 sites in Spain, Germany, United Kingdom. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Closure of fistulas defined as absence of suppuration and re-epithelization of the external opening and absence of collections>2 cm directly related to the fistula tract treated, as measured by MRI (healing) (time frame: weeks 1, 4, 12 and 24. Week 26 in patients with a second dose). 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 NCT00475410, 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 12 Apr 2019.