Recessive Dystrophic Epidermolysis BullosaPhase 1/2NCT04520022
Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB
A Phase 1/2 study testing Human Umbilical Cord Blood-derived Mesenchymal Stem Cells in people with Recessive Dystrophic Epidermolysis Bullosa, run by Gangnam Severance Hospital at 1 site in South Korea.
Trial summary
Previously, many studies have been conducted on mesenchymal stem cells derived from bone marrow or subcutaneous fat, but interest in cord blood-derived mesenchymal stem cell treatments has been increasing recently. Read more about this study
- Who is it for?
- Ages 10 to 60 years
- Conditions the study is for
- Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- Human Umbilical Cord Blood-derived Mesenchymal Stem Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteSouth Korea
- Study dates
- 13 Oct 2016to 10 Jan 2020
- 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 10 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients who diagnosed with recessive dystrophic epidermolysis bullosa through clinical, histological(Partial or complete loss of VII collagen (C7) should be confirmed by DIF and electron microscopy examination) and genetic testing(COL7A1 Genetic mutation must be confirmed).
- RDEB patients aged 10 to 60 years old (In the case of patients under the age of 19, patients who obtain consent from a representative (parental authority or guardian))
- Patients who have heard the purpose and contents of a clinical trial and voluntarily signed the consent form prior to the clinical trial (Legal representative in case of minor)
- Patients who can be monitored during a clinical trial period
Who cannot join
- Patients who disagree with this study
- Patients who is not accompanied by a guardian if those with impaired consent ability
- Patient or the patient's representative is unable to hear and understand the explanation
- In case of received immunotherapy or chemotherapy including oral corticosteroid (topical treatment is possible) for more than 1 week within 8 weeks before registration.
- All kinds of live vaccines except influenza vaccine within four weeks prior to registration
- Clinically significant infections within four weeks of the screening date or during the screening period (pneumonia, pyelonephritis, Clostridium difficile etc)
Show all 15Show fewer
- All kinds of confirmed congenital or acquired immunodeficiency syndrome
- Acute, chronic infection (Type B, Type C) corresponding to:
- HBs-Ag, IgM anti-HBc, IgG anti-HBc positive (However, if HBs-Ag and IgM anti-HBc is negative, but only IgG anti-HBc is positive, if ani-HBs Ab positive, this clinical trial can be registered.)
- Patients who with allogenic stem cell treatment experience within 1 year from the screening test date
- Patients who have a history of malignant tumors or is currently being treated (squamous cell carcinoma of the skin, cutaneous squamous cell carcinoma inclusion)
- Type VII collagen ELISA positive and IIF positive
- Pregnant or lactating women (Women of childbearing potential should agree to use appropriate contraceptive methods (hormonal or barrier method of contraception or abstinence) prior to enrollment in the study and during the study period, including one month after the last administration of the test drug. If pregnant or suspected of being pregnant while participating in the study, the investigator should be informed immediately.)
- Other cases where the researcher judges that participation in this clinical trial is inappropriate
- If other clinical trial drugs have been administered within 4 weeks prior to registration or are currently participating in a clinical trial
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Single Center, Single Group Assignment, Open Label Trial to Assess Safety and Effectiveness of Intravenous Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With Recessive Dystrophic Epidermolysis Bullosa
Previously, many studies have been conducted on mesenchymal stem cells derived from bone marrow or subcutaneous fat, but interest in cord blood-derived mesenchymal stem cell treatments has been increasing recently.
In the case of cord blood as a source, the isolation of mesenchymal stem cells is easier than bone marrow or fat tissue, and cord blood-derived mesenchymal stem cells have an advantage as a treatment because they have faster population doubling time.
Read more
To date, no clinical research on the treatment of patients using cord blood-derived mesenchymal stem cells has been reported in the literature, but there have already been registered at clinicaltrials.gov and currently being conducted overseas.
In this study, we will study the safety and effectiveness of RDEB patient treatment using cord blood-derived mesenchymal stem cells with these advantages.
Read the detailed description
Until now, all clinical trials for Recessive Dystrophic Epidermolysis Bullosa (RDEB) have examined the potential of bone marrow-derived MSCs. However, umbilical cord blood (UCB) is another important source of stem cells, since its non-invasive collection procedure and rapid availability from cord blood banking. Human UCB-derived MSCs (hUCB-MSCs) exhibit high proliferation capacity and low immunogenicity. A few data support that UCB-MSCs may have significantly greater immunosuppressive potential than other sources of MSCs. A preclinical study has demonstrated that systemic infusions of human UCB-derived unrestricted somatic stem cells, a subpopulation of non-hematopoietic stromal stem cells, significantly extended the life span and reduced blistering of RDEB mice model. Given the promising results of the preclinical study, we conducted a first-in-human, phase 1/2a clinical trial of intravenous administrations of allogeneic hUCB-MSCs in patients with RDEB to determine the safety, tolerability, and potential efficacy.
- Being tested
- Human Umbilical Cord Blood-derived Mesenchymal Stem Cells
- Keywords
- umbilical cord blood-derived mesenchymal stem cell
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Umbilical Cord Blood-derived Mesenchymal Stem CellsDrug
Also called hUCB-MSCs, FURESTEM-CD Inj
3.0 x 106 cells/kg, IV, Total of 3 doses every 2weeks
| Study group | Role | What they receive |
|---|---|---|
| FURESTEM-CD Inj | Experimental | Not described Receives: Drug: Human Umbilical Cord Blood-derived Mesenchymal Stem Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events related to the intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell
Measured: 8 months
Secondary outcomes (8)
Change in type VII collagen and anchoring fibril expression at dermoepidermal junction
Measured: baseline, day 56
Change in Birmingham Epidermolysis Bullosa Severity Score (BEBSS)
Measured: baseline, day56, day 112, day168
Change in Global severity score
Measured: baseline, day56, day 112, day168
Change in total body surface area affected by RDEB
Measured: baseline, day56, day 112, day168
Change in Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
Measured: baseline, day56, day 112, day168
Change in blister count
Measured: baseline, day56, day 112, day168
Change in pruritus visual analogue scale (VAS)
Measured: baseline, day56, day 112, day168
Change in pain visual analogue scale (VAS)
Measured: baseline, day56, day 112, day168
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2020
- Study start13 Oct 2016
- Primary completion10 Jan 2020
- Study completion10 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Recessive Dystrophic Epidermolysis BullosaStudies 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
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gangnam Severance Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Daewoong Pharmaceutical Co. LTD.
- Responsible party
- Principal investigator, Sang Eun Lee, Gangnam Severance Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04520022
- Sponsor's protocol ID
- 3-2015-0285
- Status last verified
- Aug 2020
- First submitted
- 13 Aug 2020
- Last updated
- 20 Aug 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Lee SE, Lee SJ, Kim SE, Kim K, Cho B, Roh K, Kim SC. Intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa patients. JCI Insight. 2021 Jan 25;6(2):e143606. doi: 10.1172/jci.insight.143606.
PubMed 33491668
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single Center, Single Group Assignment, Open Label Trial to Assess Safety and Effectiveness of Intravenous Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With Recessive Dystrophic Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT04520022
- Acronym / sponsor ID
- 3-2015-0285
- Keywords
- umbilical cord blood-derived mesenchymal stem cell
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Gangnam Severance HospitalUniversity, hospital or other
- Collaborators
- Daewoong Pharmaceutical Co. LTD.
- Study start
- 13 Oct 2016 (actual)
- Primary completion
- 10 Jan 2020 (actual)
- Study completion
- 10 Jan 2020 (actual)
- First posted
- 20 Aug 2020 (actual)
- 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.
Previously, many studies have been conducted on mesenchymal stem cells derived from bone marrow or subcutaneous fat, but interest in cord blood-derived mesenchymal stem cell treatments has been increasing recently.
In the case of cord blood as a source, the isolation of mesenchymal stem cells is easier than bone marrow or fat tissue, and cord blood-derived mesenchymal stem cells have an advantage as a treatment because they have faster population doubling time.
To date, no clinical research on the treatment of patients using cord blood-derived mesenchymal stem cells has been reported in the literature, but there have already been registered at clinicaltrials.gov and currently being conducted overseas.
In this study, we will study the safety and effectiveness of RDEB patient treatment using cord blood-derived mesenchymal stem cells with these advantages.
Detailed description
Until now, all clinical trials for Recessive Dystrophic Epidermolysis Bullosa (RDEB) have examined the potential of bone marrow-derived MSCs. However, umbilical cord blood (UCB) is another important source of stem cells, since its non-invasive collection procedure and rapid availability from cord blood banking. Human UCB-derived MSCs (hUCB-MSCs) exhibit high proliferation capacity and low immunogenicity. A few data support that UCB-MSCs may have significantly greater immunosuppressive potential than other sources of MSCs. A preclinical study has demonstrated that systemic infusions of human UCB-derived unrestricted somatic stem cells, a subpopulation of non-hematopoietic stromal stem cells, significantly extended the life span and reduced blistering of RDEB mice model. Given the promising results of the preclinical study, we conducted a first-in-human, phase 1/2a clinical trial of intravenous administrations of allogeneic hUCB-MSCs in patients with RDEB to determine the safety, tolerability, and potential efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FURESTEM-CD Inj | Experimental | Drug: Human Umbilical Cord Blood-derived Mesenchymal Stem Cells |
Who may qualify
- Ages
- 10 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Patients who diagnosed with recessive dystrophic epidermolysis bullosa through clinical, histological(Partial or complete loss of VII collagen (C7) should be confirmed by DIF and electron microscopy examination) and genetic testing(COL7A1 Genetic mutation must be confirmed).
- RDEB patients aged 10 to 60 years old (In the case of patients under the age of 19, patients who obtain consent from a representative (parental authority or guardian))
- Patients who have heard the purpose and contents of a clinical trial and voluntarily signed the consent form prior to the clinical trial (Legal representative in case of minor)
- Patients who can be monitored during a clinical trial period
Key exclusion criteria (15 listed)
- Patients who disagree with this study
- Patients who is not accompanied by a guardian if those with impaired consent ability
- Patient or the patient's representative is unable to hear and understand the explanation
- In case of received immunotherapy or chemotherapy including oral corticosteroid (topical treatment is possible) for more than 1 week within 8 weeks before registration.
- All kinds of live vaccines except influenza vaccine within four weeks prior to registration
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 |
|---|---|---|
| Human Umbilical Cord Blood-derived Mesenchymal Stem CellsAlso: hUCB-MSCs, FURESTEM-CD Inj3.0 x 106 cells/kg, IV, Total of 3 doses every 2weeks | Drug | FURESTEM-CD Inj |
Primary outcome
Adverse events related to the intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell
Time frame: 8 months
8 secondary outcomes
Change in type VII collagen and anchoring fibril expression at dermoepidermal junction
Time frame: baseline, day 56
Change in Birmingham Epidermolysis Bullosa Severity Score (BEBSS)
Time frame: baseline, day56, day 112, day168
Change in Global severity score
Time frame: baseline, day56, day 112, day168
Change in total body surface area affected by RDEB
Time frame: baseline, day56, day 112, day168
Change in Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
Time frame: baseline, day56, day 112, day168
Change in blister count
Time frame: baseline, day56, day 112, day168
Change in pruritus visual analogue scale (VAS)
Time frame: baseline, day56, day 112, day168
Change in pain visual analogue scale (VAS)
Time frame: baseline, day56, day 112, day168
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 20 Aug 2020.
- Derived from this study: PubMed 33491668Lee SE, Lee SJ, Kim SE, Kim K, Cho B, Roh K, Kim SC. Intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa patients. JCI Insight. 2021 Jan 25;6(2):e143606. doi: 10.1172/jci.insight.143606.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 Human Umbilical Cord Blood-derived Mesenchymal Stem Cells in people with Recessive Dystrophic Epidermolysis Bullosa, run by Gangnam Severance Hospital at 1 site in South Korea.
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 4 inclusion and 15 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 20 Aug 2020. 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 South Korea. 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?
Adverse events related to the intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell (time frame: 8 months). 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 NCT04520022, which also lists 1 linked publication.
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 20 Aug 2020.