Recessive Dystrophic Epidermolysis BullosaPhase 1/2ADSTEMNCT02323789
Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa
A Phase 1/2 study testing Mesenchymal stromal cells in people with Recessive Dystrophic Epidermolysis Bullosa, run by King's College London at 1 site in United Kingdom.
Trial summary
To assess whether intravenously administered third-party bone marrow-derived mesenchymal stromal cells (MSCs) are safe and have an impact on disease severity in RDEB Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- Mesenchymal stromal cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jun 2015to Jul 2017
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Individuals with a diagnosis of RDEB confirmed by DNA analysis and skin immunofluorescence for partial or complete absence of type VII collagen.
- Individuals ≥ 18 years and ≤ 65 years of age, both male and female
- Individuals that have voluntarily signed and dated an informed consent form (ICF) prior to the first study intervention.
Who cannot join
- Subjects who have had other investigational medicinal products within 90 days prior to screening or during the treatment phase.
- Subjects who have received immunotherapy including oral corticosteroids for more than 1 week (intranasal and topical preparations are permitted).
- Subjects with a known allergy to any of the constituents of the investigational product.
- Subjects with a medical history or evidence of malignancy, including cutaneous squamous cell carcinoma.
- Subjects who are pregnant or of child-bearing potential who are not abstinent or practicing an acceptable means of contraception, as determined by the Investigator, for the duration of the treatment phase.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Guys and St Thomas' hospital NHS Trust
London, SE1 9RT
About this study
Official title: A Phase I/II Study Evaluating Allogeneic Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa
To assess whether intravenously administered third-party bone marrow-derived mesenchymal stromal cells (MSCs) are safe and have an impact on disease severity in RDEB
Read the detailed description
This is a phase I/II clinical trial with a key objective of evaluating safety of third party bone MSCs intravenous infusions in 10 adults with the inherited severe skin fragility disorder, recessive dystrophic epidermolysis bullosa (RDEB). The main objectives of our study are to: (1) to assess the spectrum of clinical responses in adults with RDEB receiving intravenous MSCs; (2) to identify the best cohort of individuals to target for future trials and therapies; (3) to improve our understanding of in vivo and in vitro responsiveness to MSCs; (4) to identify candidate molecules germane to activating MSCs and making them clinically more potent, independently of the permissive conditions of the patient and (5) to assess its impact on reducing disease morbidity/severity in this population.
This is a prospective, non-randomised, open label study. All study participants will receive two intravenous MSC infusions at baseline Day 0 and Day 14 and will be followed up for a 12 month period following the first infusion. Each subject will undergo an initial screening including physical examination, assessment of vital signs and disease severity assessment.
- Being tested
- Mesenchymal stromal cells
- 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.
Mesenchymal stromal cellsDrug
Also called TC-MSC
TC-MSC: a cell product containing mesenchymal stromal cells. Mesenchymal stromal cells are adherent non-haematopoietic multipotent cells that are expanded from bone marrow from healthy donors, using platelet lysate as source of growth factor.
| Study group | Role | What they receive |
|---|---|---|
| Intervention arm | Experimental | 10 patients with RDEB will be selected to receive the intervention - mesenchymal stromal cells. Receives: Drug: Mesenchymal stromal cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product.
Measured: 12 months
Secondary outcomes (11)
Presence of new type VII collagen at the dermal-epidermal junction post treatment.
Measured: Day 14, Day 28, Day 60, Day 100 and Month 6.
Change in general markers of inflammation
Measured: Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Changes in specific markers of inflammation
Measured: Day 14, Day 28, Day 60 and Month 6 compared to baseline
Change in the clinical changes in the skin assessed with clinical photographs
Measured: Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12.
Differences in quality of life data
Measured: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Change in BEBSS and EBDASI scores
Measured: at Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Change in Pain scores
Measured: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Change in pruritus score using the Leuven Itch Scale (LIS)
Measured: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline.
Quantification of total blister numbers over the entire body surface area
Measured: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
10. Increase in the skin strength measured by time to blister formation after negative pressure skin suction test
Measured: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Qualitative analyses based on a series of interview questions
to reveal objective data on quality of sleep, skin healing time, amount of dressings used, improvement in oral diet, improvement in energy levels, mood, quality of family life/relationships.
Measured: between screening and Day 0, between Day 28 and Day 60, and between Month 6 and Month 12.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2014 (estimated)
- Study startJun 2015
- Primary completionJul 2017 (estimated)
- Study completionJul 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recessive Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- More trials run by King's College LondonThe sponsor's other registered studies.
- 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- King's College London
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- John McGrath, FRCP/FSci/MD, Principal investigator, King's College London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02323789
- Sponsor's protocol ID
- ADSTEM001
- Acronym
- ADSTEM
- Status last verified
- Mar 2017
- First submitted
- 18 Dec 2014
- Last updated
- 3 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study Evaluating Allogeneic Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT02323789
- Acronym / sponsor ID
- ADSTEM / ADSTEM001
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (actual)
- Lead sponsor
- King's College LondonUniversity, hospital or other
- Study start
- Jun 2015 (actual)
- Primary completion
- Jul 2017 (estimated)
- Study completion
- Jul 2017 (estimated)
- First posted
- 23 Dec 2014 (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.
To assess whether intravenously administered third-party bone marrow-derived mesenchymal stromal cells (MSCs) are safe and have an impact on disease severity in RDEB
Detailed description
This is a phase I/II clinical trial with a key objective of evaluating safety of third party bone MSCs intravenous infusions in 10 adults with the inherited severe skin fragility disorder, recessive dystrophic epidermolysis bullosa (RDEB). The main objectives of our study are to: (1) to assess the spectrum of clinical responses in adults with RDEB receiving intravenous MSCs; (2) to identify the best cohort of individuals to target for future trials and therapies; (3) to improve our understanding of in vivo and in vitro responsiveness to MSCs; (4) to identify candidate molecules germane to activating MSCs and making them clinically more potent, independently of the permissive conditions of the patient and (5) to assess its impact on reducing disease morbidity/severity in this population.
This is a prospective, non-randomised, open label study. All study participants will receive two intravenous MSC infusions at baseline Day 0 and Day 14 and will be followed up for a 12 month period following the first infusion. Each subject will undergo an initial screening including physical examination, assessment of vital signs and disease severity assessment.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intervention arm | Experimental | Drug: Mesenchymal stromal cells10 patients with RDEB will be selected to receive the intervention - mesenchymal stromal cells. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Individuals with a diagnosis of RDEB confirmed by DNA analysis and skin immunofluorescence for partial or complete absence of type VII collagen.
- Individuals ≥ 18 years and ≤ 65 years of age, both male and female
- Individuals that have voluntarily signed and dated an informed consent form (ICF) prior to the first study intervention.
Key exclusion criteria (5 listed)
- Subjects who have had other investigational medicinal products within 90 days prior to screening or during the treatment phase.
- Subjects who have received immunotherapy including oral corticosteroids for more than 1 week (intranasal and topical preparations are permitted).
- Subjects with a known allergy to any of the constituents of the investigational product.
- Subjects with a medical history or evidence of malignancy, including cutaneous squamous cell carcinoma.
- Subjects who are pregnant or of child-bearing potential who are not abstinent or practicing an acceptable means of contraception, as determined by the Investigator, for the duration of the treatment phase.
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 |
|---|---|---|
| Mesenchymal stromal cellsAlso: TC-MSCTC-MSC: a cell product containing mesenchymal stromal cells. Mesenchymal stromal cells are adherent non-haematopoietic multipotent cells that are expanded from bone marrow from healthy donors, using platelet lysate as source of growth factor. | Drug | Intervention arm |
Primary outcome
Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product.
Time frame: 12 months
11 secondary outcomes
Presence of new type VII collagen at the dermal-epidermal junction post treatment.
Time frame: Day 14, Day 28, Day 60, Day 100 and Month 6.
Change in general markers of inflammation
Time frame: Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Changes in specific markers of inflammation
Time frame: Day 14, Day 28, Day 60 and Month 6 compared to baseline
Change in the clinical changes in the skin assessed with clinical photographs
Time frame: Day 14, Day 28, Day 60, Day 100, Month 6 and Month 12.
Differences in quality of life data
Time frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Change in BEBSS and EBDASI scores
Time frame: at Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Change in Pain scores
Time frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Change in pruritus score using the Leuven Itch Scale (LIS)
Time frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline.
Quantification of total blister numbers over the entire body surface area
Time frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
10. Increase in the skin strength measured by time to blister formation after negative pressure skin suction test
Time frame: Day 28, Day 60, Day 100, Month 6 and Month 12 compared to baseline
Qualitative analyses based on a series of interview questions
Time frame: between screening and Day 0, between Day 28 and Day 60, and between Month 6 and Month 12.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2017.
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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.
John McGrath, FRCP/FSci/MD principal investigator
King's College London
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 Mesenchymal stromal cells in people with Recessive Dystrophic Epidermolysis Bullosa, run by King's College London at 1 site in United Kingdom.
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 3 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 3 Mar 2017. 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 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 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?
Results are not available in the source record.
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?
Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product. (time frame: 12 months). 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 3 Mar 2017.