Epidermolysis Bullosa With Mitten HandsPhase 1NCT01908088
Autologous Transplantation of Cultured Fibroblast on Amniotic Membrane in Patients With Epidermolysis Bullosa
A Phase 1 study testing Cell transplantation in people with Epidermolysis Bullosa With Mitten Hands, run by Royan Institute at 1 site in Iran.
Trial summary
Epidermolysis Bullosa (EB) is a blistering disease that is caused by defective anchoring fibrils and hemidesmosome in basement membrane of the skin layer. EB is inherited either autosomal or recessive and has 3 types. Read more about this study
- Who is it for?
- Ages 5 to 25 years
- Conditions the study is for
- Epidermolysis Bullosa With Mitten Hands
- What is being tested?
- Cell transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteIran
- Study dates
- Jul 2010to Jul 2013
- Phase
- Phase 1
- 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 5 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Both genders Age: 5-25y No history of other systemic disease No history of other cell therapy method No infection in donor or recipient site HCV Ab/HIV Ab/ HBS Ag tests were negative
Who cannot join
- Age less than 5y or more than 25y. History of other systemic disease History of other cell therapy method Infection in donor or recipient site Positive Lab tests for contagious viral diseases Pregnancy or lactating History of malignancy
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Autologous Transplantation of Cultured Fibroblast on Amniotic Membrane for Mitten Hand Deformity in Patients With Epidermolysis Bullosa
Epidermolysis Bullosa (EB) is a blistering disease that is caused by defective anchoring fibrils and hemidesmosome in basement membrane of the skin layer. EB is inherited either autosomal or recessive and has 3 types. Recessive Dystrophic Epidermolysis Bullosa (RDEB) is severe with some morbidity such as mitten hand deformity.
The management of these patients is very difficult because no effective treatment has been known yet.
The EB patients with mitten hand deformity need surgery to have a biologic dressing for areas of hand which is without the skin. In this study the investigators assess the safety of autologous transplantation of cultured fibroblast on amniotic membrane (AM,as coverage) for them.
Read the detailed description
Epidermolysis Bullosa patients with mitten hand deformity were included. (based on inclusion \& exclusion criteria) These patients should be offered for surgery to release the adhesions between fingers and MCP. When the consent form was accepted and the lab test was confirmed, a small skin biopsy was removed from retro auricle and fibroblast cells were separated and cultured. These cells were seeded on AM and the microbial test was done as evidence to prove the safety.
Under general anesthesia, when the hands adhesion was removed, the autologous fibroblast cultured on AM was transplanted to the both hands (specially the area without skin coverage) and the hands were dressed afterward.
The dressing was changed and the wound was checked regularly to detect any complications.
- Conditions
- Epidermolysis Bullosa With Mitten Hands
- Being tested
- Cell transplantation
- Keywords
- Epidermolysis Bullosa mitten hand deformity autologous cultured fibroblast, amniotic membrane
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cell transplantationBiological
Also called autologous transplantation of cultured fibroblast on amniotic membrane
autologous transplantation of cultured fibroblast on amniotic membrane in patients with Epidermolysis Bullosa with mitten hands.
| Study group | Role | What they receive |
|---|---|---|
| fibroblast transplant | Experimental | Transplantation of autologous cultured fibroblast on amniotic membrane in patients with Epidermolysis Bullosa with mitten hand deformity Receives: Biological: Cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pain
Evaluation the pain reduction 1 month after cell transplantation by VAS scoring.
Measured: 1month
Infection
Evaluation the symptoms of any infection in the site of transplantation during 1week after transplantation.
Measured: 1week
Bleeding
Evaluation the presence of any bleeding at the site of transplantation during 1 week after transplantation.
Measured: 1week
Healing
Evaluation the healing of ulcers 1month after transplantation base on changing the dressing time.
Measured: 1month
Secondary outcomes (2)
new ulceration
Evaluation the new blister or ulceration during 3 to 6 months after transplantation.
Measured: 3months
Range of motion
Evaluation the range of hand motion between 0 -110 for every fingers 6 months after transplantation.
Measured: 6months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2013 (estimated)
- Study startJul 2010
- Primary completionSep 2012
- Study completionJul 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa With Mitten HandsStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Royan Institute
- Sponsor type
- Other government
- Collaborators
- Hazrat Fatemeh Hospital
- Responsible party
- Sponsor
- Investigators
- Hamid Gourabi, PhD, Study chair, Head of Royan InstituteNasser Aghdami, MD,PhD, Study director, Head of Royan Institute Cell Therapy CenterSaeed Shafiyan, MD, Study director, Dermatogist,Depatment of Regenerative medicin of Royan InstituteZahra Orouji, MD, Principal investigator, Regenerative Medicine Department of Royan InstituteKamal Seyed Forootan, MD, Principal investigator, Plastic Surgoen, Hazrat Fatemeh HospitalSeyed Mohammad Javad Fatemi, MD, Principal investigator, Plastic Surgoen,Hazrat Fatemeh Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01908088
- Sponsor's protocol ID
- Royan-skin-003
- Status last verified
- Feb 2010
- First submitted
- 23 Jul 2013
- Last updated
- 25 Jul 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Transplantation of Cultured Fibroblast on Amniotic Membrane for Mitten Hand Deformity in Patients With Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT01908088
- Acronym / sponsor ID
- Royan-skin-003
- Conditions
- Epidermolysis Bullosa With Mitten Hands
- Keywords
- Epidermolysis Bullosa mitten hand deformity autologous cultured fibroblast, amniotic membrane
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Royan InstituteOther government
- Collaborators
- Hazrat Fatemeh Hospital
- Study start
- Jul 2010
- Primary completion
- Sep 2012 (actual)
- Study completion
- Jul 2013 (actual)
- First posted
- 25 Jul 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.
Epidermolysis Bullosa (EB) is a blistering disease that is caused by defective anchoring fibrils and hemidesmosome in basement membrane of the skin layer. EB is inherited either autosomal or recessive and has 3 types. Recessive Dystrophic Epidermolysis Bullosa (RDEB) is severe with some morbidity such as mitten hand deformity.
The management of these patients is very difficult because no effective treatment has been known yet.
The EB patients with mitten hand deformity need surgery to have a biologic dressing for areas of hand which is without the skin. In this study the investigators assess the safety of autologous transplantation of cultured fibroblast on amniotic membrane (AM,as coverage) for them.
Detailed description
Epidermolysis Bullosa patients with mitten hand deformity were included. (based on inclusion \& exclusion criteria) These patients should be offered for surgery to release the adhesions between fingers and MCP. When the consent form was accepted and the lab test was confirmed, a small skin biopsy was removed from retro auricle and fibroblast cells were separated and cultured. These cells were seeded on AM and the microbial test was done as evidence to prove the safety.
Under general anesthesia, when the hands adhesion was removed, the autologous fibroblast cultured on AM was transplanted to the both hands (specially the area without skin coverage) and the hands were dressed afterward.
The dressing was changed and the wound was checked regularly to detect any complications.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| fibroblast transplant | Experimental | Biological: Cell transplantationTransplantation of autologous cultured fibroblast on amniotic membrane in patients with Epidermolysis Bullosa with mitten hand deformity |
Who may qualify
- Ages
- 5 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Both genders Age: 5-25y No history of other systemic disease No history of other cell therapy method No infection in donor or recipient site HCV Ab/HIV Ab/ HBS Ag tests were negative
Key exclusion criteria (1 listed)
- Age less than 5y or more than 25y. History of other systemic disease History of other cell therapy method Infection in donor or recipient site Positive Lab tests for contagious viral diseases Pregnancy or lactating History of malignancy
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 |
|---|---|---|
| Cell transplantationAlso: autologous transplantation of cultured fibroblast on amniotic membraneautologous transplantation of cultured fibroblast on amniotic membrane in patients with Epidermolysis Bullosa with mitten hands. | Biological | fibroblast transplant |
Primary outcome
Pain
Time frame: 1month
Evaluation the pain reduction 1 month after cell transplantation by VAS scoring.
Infection
Time frame: 1week
Evaluation the symptoms of any infection in the site of transplantation during 1week after transplantation.
Bleeding
Time frame: 1week
Evaluation the presence of any bleeding at the site of transplantation during 1 week after transplantation.
Healing
Time frame: 1month
Evaluation the healing of ulcers 1month after transplantation base on changing the dressing time.
2 secondary outcomes
new ulceration
Time frame: 3months
Range of motion
Time frame: 6months
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 25 Jul 2013.
- Related InfoLink provided by the sponsor.
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.
Hamid Gourabi, PhD study chair
Head of Royan Institute
Nasser Aghdami, MD,PhD study director
Head of Royan Institute Cell Therapy Center
Saeed Shafiyan, MD study director
Dermatogist,Depatment of Regenerative medicin of Royan Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cell transplantation in people with Epidermolysis Bullosa With Mitten Hands, run by Royan Institute at 1 site in Iran.
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 1 inclusion and 1 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 25 Jul 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 Iran. 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 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.
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?
Pain (time frame: 1month). The study lists 4 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 25 Jul 2013.