Recessive Dystrophic Epidermolysis BullosaPhase 1/2NCT03529877
Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa
A Phase 1/2 study testing allo-APZ2-EB in people with Recessive Dystrophic Epidermolysis Bullosa, run by RHEACELL GmbH & Co. KG at 6 sites in 5 countries.
Trial summary
The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with recessive dystrophic epidermolysis bullosa (RDEB). Read more about this study
- Who is it for?
- Ages 0 to 55 years
- Conditions the study is for
- Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- allo-APZ2-EB
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 6 sitesin 5 countries
- Study dates
- 16 Feb 2019to 26 Nov 2021
- 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 0 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 1. Male or female patients aged between 0 and ≤55 years;
- Staggered design for patient enrollment:
- at least 3 adult patients (safety assessment 2 weeks after last treatment of third patient),
- at least 3 patients ≥12 to <18 years (safety assessment 2 weeks after first treatment of third patient),
- at least 3 patients ≥5 to <12 years (safety assessment 2 weeks after first treatment of third patient), and
- at least 3 patients ≥12 months to <5 years;
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- patients 0 to <12 months (only in the UK);
- 2. Diagnosed with RDEB (combined diagnosis by genotype assessment [mutation analysis] and correlating phenotype assessment [wound assessment]), patients must have a negative immunofluorescence test result on salt-split skin against proteins of the basement membrane at Visit 1 (existing test results will be accepted);
- 3. Patient is eligible to participate in this clinical trial based on general health condition at the investigator's discretion;
- US only:
- Patient is eligible to participate in this clinical trial based on general health condition assessed by specific lab values (Hematology: Absolute neutrophil count >1000/mm3 and platelet count >150,000/mcL; Coagulation: PT and PTT <2x the upper limit of normal for age; Hepatic: AST and ALT <2x the upper limit of normal for age; Renal: Creatinine <2x the upper limit of normal for age; Pulmonary: Oxygen saturation >92% on room air and without supplemental oxygen requirement);
- 4. Patient/legal representative understands the nature of the procedure and are providing written informed consent prior to any clinical trial procedure;
- 5. Women of childbearing potential must have a negative urine pregnancy test at Visit 1;
- 6. Women of childbearing potential and their partner must be willing to use highly effective contraceptive methods during the course of the clinical trial.
Who cannot join
- Tumor diseases or history of tumor disease;
- Known positive result for human immunodeficiency virus 1 and/or 2;
- Any known allergies to components of the IMP;
- Evidence of any other medical conditions (such as psychiatric illness or active infection) based on physical examination, or laboratory findings that may interfere with the planned treatment, affect the patient's compliance, or place the patient at high risk of complications related to the treatment; at investigators discretion;
- History of prior thrombosis or patients at risk for thrombosis;
- Clinically significant or unstable concurrent disease or other clinical contraindications (based upon investigator's judgment);
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- Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
- Pregnant or lactating women;
- Current or previous (within 30 days of enrollment) treatment with another IMP, or participation and/or under follow-up in another clinical trial;
- Previous participation in this clinical trial (except for screening failures due to an exclusion criterion);
- Known abuse of alcohol, drugs, or medicinal products;
- Employees of the sponsor, or employees or relatives of the investigator.
Trial locations and contacts
6 sites in 5 countries.
6 sites in 5 countries
United Kingdom2 sites
King's College London; St John's Institute of Dermatology;
London, SE1 9RT
Great Ormond Street Hospital; Dermatology Department
London, WC1N 3JH
United States1 site
University of Minnesota, Masonic Cancer Center and Medical Center
Minneapolis, Minnesota, 55455
Austria1 site
EB-Haus Austria; Salzburger Landeskliniken (SALK); Paracelsus Medizinische Privatuniversität Salzburg (PMU)
Salzburg, 5020
Germany1 site
Department of Dermatology, Medical Center-University of Freiburg
Freiburg im Breisgau, 79104
About this study
Official title: An Interventional, Multicenter, Single Arm, Phase I/IIa Clinical Trial to Investigate the Efficacy and Safety of Allo-APZ2-EB on Epidermolysis Bullosa (EB)
The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with recessive dystrophic epidermolysis bullosa (RDEB).
Read the detailed description
This is an interventional, single arm, non-randomized, open label, phase I/IIa clinical trial to investigate the efficacy and safety of the IMP allo-APZ2-EB in patients with RDEB.
Patients will undergo treatment with the IMP (three repeated intravenous applications) and will be followed up for efficacy for 12 weeks. To assess long-term safety of allo-APZ2-EB one follow-up visit at Month 12 and one follow-up visit at Month 24 post IMP applications is included.
Determination of the EB linked symptoms and quality of life will be assessed by using the EBDASI score, the iscorEB, the change in pain and itch perception, and patient's quality of life in EB. The wound healing process will be documented by photography.
- Being tested
- allo-APZ2-EB
- Keywords
- Epidermolysis Bullosa, Epidermolysis Bullosa Dystrophica, Skin Abnormalities, Congenital Abnormalities, Skin Diseases, Genetic, Somatic Cell Therapy, Mesenchymal Stem Cells, ABCB5, Allogeneic
- Medical subject headings
- Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa, Skin Abnormalities, Congenital Abnormalities, Skin Diseases, Genetic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
allo-APZ2-EBBiological
Also called allogeneic ABCB5-positive mesenchymal stem cells
intravenous infusion of allo-APZ2-EB
| Study group | Role | What they receive |
|---|---|---|
| allo-APZ2-EB | Experimental | intravenous infusion, three doses of allo-APZ2-EB (2 x 10^6 cells/kg) Receives: Biological: allo-APZ2-EB |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's EBDASI score), score), or last available post-baseline measurement if the Week 12 measurement is missing
EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score
Measured: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF])
Assessment of adverse event (AE) occurrence
All AEs occurring during the clinical trial will be registered, documented and evaluated.
Measured: Up to 24 months
Secondary outcomes (16)
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's EBDASI score)
EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score
Measured: between baseline and week 12 post baseline (without LOCF)
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient's iscorEB), or last available post-baseline measurement if the Week 12 measurement is missing
iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score
Measured: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (LOCF);
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient's iscorEB)
iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score
Measured: between baseline and week 12 post baseline (without LOCF)
Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient's EBDASI score)
EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score
Measured: between baseline and day 17 post baseline
Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient's iscorEB)
iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score
Measured: between baseline and day 17 post baseline
Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient's EBDASI score)
EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score
Measured: between baseline and day 35 post baseline
Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient's iscorEB)
iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score
Measured: between baseline and day 35 post baseline
Inflammation (measured by panel of inflammation markers)
A panel of inflammation markers will be measured and evaluated.
Measured: between baseline and day 17, day 35 and week 12 post baseline
Pain assessment as per NRS
Pain assessment as per numerical rating scale (NRS) will be evaluated.
Measured: between baseline and day 17, day 35 and week 12 post baseline
Itch assessment as per NRS
Itch assessment as per numerical rating scale (NRS) will be evaluated.
Measured: between baseline and day 17, day 35 and week 12 post baseline
Differences in patient's quality of life in EB
Assessment of quality of life data using an EB-specific quality of life questionnaire
Measured: between baseline and day 17, day 35 and week 12 post baseline
Physical examination until Week 12;
A full physical examination will be performed and abnormal physical examination results will be evaluated and reported as AEs.
Measured: At Screening, baseline, day 17, day 35 and week 12
Vital signs: Body temperature until Week 12;
Body temperature will be evaluated at Screening, baseline, day 17, day 35 and week 12
Measured: At Screening, baseline, day 17, day 35 and week 12
Vital signs: Blood pressure until Week 12;
Blood pressure will be evaluated at Screening, baseline, day 17, day 35 and week 12
Measured: At Screening, baseline, day 17, day 35 and week 12
Vital signs: Heart rate until Week 12;
Heart rate will be evaluated at Screening, baseline, day 17, day 35 and week 12
Measured: At Screening, baseline, day 17, day 35 and week 12
Overall survival at month 24
Measured: month 24 post baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2018
- Study start16 Feb 2019
- Primary completion26 Nov 2021
- Study completion26 Nov 2021
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 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
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- RHEACELL GmbH & Co. KG
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- FGK Clinical Research GmbH, Granzer Regulatory Consulting & Services, Ticeba GmbH
- Responsible party
- Sponsor
- Investigators
- Jakub Tolar, MD, PhD, Principal investigator, University of Minnesota, Masonic Cancer Center and Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03529877
- Sponsor's protocol ID
- allo-APZ2-EB-II-01
- Status last verified
- Mar 2022
- First submitted
- 24 Apr 2018
- Last updated
- 7 Oct 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Dieter K, Niebergall-Roth E, Daniele C, Fluhr S, Frank NY, Ganss C, Kiritsi D, McGrath JA, Tolar J, Frank MH, Kluth MA. ABCB5+ mesenchymal stromal cells facilitate complete and durable wound closure in recessive dystrophic epidermolysis bullosa. Cytotherapy. 2023 Jul;25(7):782-788. doi: 10.1016/j.jcyt.2023.01.015. Epub 2023 Mar 1.
PubMed 36868990Kiritsi D, Dieter K, Niebergall-Roth E, Fluhr S, Daniele C, Esterlechner J, Sadeghi S, Ballikaya S, Erdinger L, Schauer F, Gewert S, Laimer M, Bauer JW, Hovnanian A, Zambruno G, El Hachem M, Bourrat E, Papanikolaou M, Petrof G, Kitzmuller S, Ebens CL, Frank MH, Frank NY, Ganss C, Martinez AE, McGrath JA, Tolar J, Kluth MA. Clinical trial of ABCB5+ mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight. 2021 Nov 22;6(22):e151922. doi: 10.1172/jci.insight.151922.
PubMed 34665781Kerstan A, Niebergall-Roth E, Esterlechner J, Schroder HM, Gasser M, Waaga-Gasser AM, Goebeler M, Rak K, Schrufer P, Endres S, Hagenbusch P, Kraft K, Dieter K, Ballikaya S, Stemler N, Sadeghi S, Tappenbeck N, Murphy GF, Orgill DP, Frank NY, Ganss C, Scharffetter-Kochanek K, Frank MH, Kluth MA. Ex vivo-expanded highly pure ABCB5+ mesenchymal stromal cells as Good Manufacturing Practice-compliant autologous advanced therapy medicinal product for clinical use: process validation and first in-human data. Cytotherapy. 2021 Feb;23(2):165-175. doi: 10.1016/j.jcyt.2020.08.012. Epub 2020 Oct 1.
PubMed 33011075
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Interventional, Multicenter, Single Arm, Phase I/IIa Clinical Trial to Investigate the Efficacy and Safety of Allo-APZ2-EB on Epidermolysis Bullosa (EB)
- ClinicalTrials.gov ID
- NCT03529877
- Acronym / sponsor ID
- allo-APZ2-EB-II-01
- Keywords
- Epidermolysis Bullosa, Epidermolysis Bullosa Dystrophica, Skin Abnormalities, Congenital Abnormalities, Skin Diseases, Genetic, Somatic Cell Therapy, Mesenchymal Stem Cells, ABCB5
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- RHEACELL GmbH & Co. KGIndustry (pharma, biotech, devices)
- Collaborators
- FGK Clinical Research GmbH, Granzer Regulatory Consulting & Services, Ticeba GmbH
- Study start
- 16 Feb 2019 (actual)
- Primary completion
- 26 Nov 2021 (actual)
- Study completion
- 26 Nov 2021 (actual)
- First posted
- 18 May 2018 (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.
The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with recessive dystrophic epidermolysis bullosa (RDEB).
Detailed description
This is an interventional, single arm, non-randomized, open label, phase I/IIa clinical trial to investigate the efficacy and safety of the IMP allo-APZ2-EB in patients with RDEB.
Patients will undergo treatment with the IMP (three repeated intravenous applications) and will be followed up for efficacy for 12 weeks. To assess long-term safety of allo-APZ2-EB one follow-up visit at Month 12 and one follow-up visit at Month 24 post IMP applications is included.
Determination of the EB linked symptoms and quality of life will be assessed by using the EBDASI score, the iscorEB, the change in pain and itch perception, and patient's quality of life in EB. The wound healing process will be documented by photography.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| allo-APZ2-EB | Experimental | Biological: allo-APZ2-EBintravenous infusion, three doses of allo-APZ2-EB (2 x 10^6 cells/kg) |
Who may qualify
- Ages
- 0 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- 1. Male or female patients aged between 0 and ≤55 years;
- at least 3 adult patients (safety assessment 2 weeks after last treatment of third patient),
- at least 3 patients ≥12 to <18 years (safety assessment 2 weeks after first treatment of third patient),
- at least 3 patients ≥5 to <12 years (safety assessment 2 weeks after first treatment of third patient), and
- at least 3 patients ≥12 months to <5 years;
Key exclusion criteria (12 listed)
- Tumor diseases or history of tumor disease;
- Known positive result for human immunodeficiency virus 1 and/or 2;
- Any known allergies to components of the IMP;
- Evidence of any other medical conditions (such as psychiatric illness or active infection) based on physical examination, or laboratory findings that may interfere with the planned treatment, affect the patient's compliance, or place the patient at high risk of complications related to the treatment; at investigators discretion;
- History of prior thrombosis or patients at risk for thrombosis;
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 |
|---|---|---|
| allo-APZ2-EBAlso: allogeneic ABCB5-positive mesenchymal stem cellsintravenous infusion of allo-APZ2-EB | Biological | allo-APZ2-EB |
Primary outcome
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's EBDASI score), score), or last available post-baseline measurement if the Week 12 measurement is missing
Time frame: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF])
EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score
Assessment of adverse event (AE) occurrence
Time frame: Up to 24 months
All AEs occurring during the clinical trial will be registered, documented and evaluated.
16 secondary outcomes
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's EBDASI score)
Time frame: between baseline and week 12 post baseline (without LOCF)
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient's iscorEB), or last available post-baseline measurement if the Week 12 measurement is missing
Time frame: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (LOCF);
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient's iscorEB)
Time frame: between baseline and week 12 post baseline (without LOCF)
Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient's EBDASI score)
Time frame: between baseline and day 17 post baseline
Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient's iscorEB)
Time frame: between baseline and day 17 post baseline
Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient's EBDASI score)
Time frame: between baseline and day 35 post baseline
Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient's iscorEB)
Time frame: between baseline and day 35 post baseline
Inflammation (measured by panel of inflammation markers)
Time frame: between baseline and day 17, day 35 and week 12 post baseline
Pain assessment as per NRS
Time frame: between baseline and day 17, day 35 and week 12 post baseline
Itch assessment as per NRS
Time frame: between baseline and day 17, day 35 and week 12 post baseline
Differences in patient's quality of life in EB
Time frame: between baseline and day 17, day 35 and week 12 post baseline
Physical examination until Week 12;
Time frame: At Screening, baseline, day 17, day 35 and week 12
Vital signs: Body temperature until Week 12;
Time frame: At Screening, baseline, day 17, day 35 and week 12
Vital signs: Blood pressure until Week 12;
Time frame: At Screening, baseline, day 17, day 35 and week 12
Vital signs: Heart rate until Week 12;
Time frame: At Screening, baseline, day 17, day 35 and week 12
Overall survival at month 24
Time frame: month 24 post baseline
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 7 Oct 2022.
- Derived from this study: PubMed 36868990Dieter K, Niebergall-Roth E, Daniele C, Fluhr S, Frank NY, Ganss C, Kiritsi D, McGrath JA, Tolar J, Frank MH, Kluth MA. ABCB5+ mesenchymal stromal cells facilitate complete and durable wound closure in recessive dystrophic epidermolysis bullosa. Cytotherapy. 2023 Jul;25(7):782-788. doi: 10.1016/j.jcyt.2023.01.015. Epub 2023 Mar 1.
- Derived from this study: PubMed 34665781Kiritsi D, Dieter K, Niebergall-Roth E, Fluhr S, Daniele C, Esterlechner J, Sadeghi S, Ballikaya S, Erdinger L, Schauer F, Gewert S, Laimer M, Bauer JW, Hovnanian A, Zambruno G, El Hachem M, Bourrat E, Papanikolaou M, Petrof G, Kitzmuller S, Ebens CL, Frank MH, Frank NY, Ganss C, Martinez AE, McGrath JA, Tolar J, Kluth MA. Clinical trial of ABCB5+ mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight. 2021 Nov 22;6(22):e151922. doi: 10.1172/jci.insight.151922.
- Derived from this study: PubMed 33011075Kerstan A, Niebergall-Roth E, Esterlechner J, Schroder HM, Gasser M, Waaga-Gasser AM, Goebeler M, Rak K, Schrufer P, Endres S, Hagenbusch P, Kraft K, Dieter K, Ballikaya S, Stemler N, Sadeghi S, Tappenbeck N, Murphy GF, Orgill DP, Frank NY, Ganss C, Scharffetter-Kochanek K, Frank MH, Kluth MA. Ex vivo-expanded highly pure ABCB5+ mesenchymal stromal cells as Good Manufacturing Practice-compliant autologous advanced therapy medicinal product for clinical use: process validation and first in-human data. Cytotherapy. 2021 Feb;23(2):165-175. doi: 10.1016/j.jcyt.2020.08.012. Epub 2020 Oct 1.
- Individual participant dataSharing planned: Undecided
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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.
Jakub Tolar, MD, PhD principal investigator
University of Minnesota, Masonic Cancer Center and Medical Center
6 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing allo-APZ2-EB in people with Recessive Dystrophic Epidermolysis Bullosa, run by RHEACELL GmbH & Co. KG at 6 sites in 5 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 14 inclusion and 12 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 7 Oct 2022. 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 United Kingdom, United States, Austria, France, Germany. 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?
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's EBDASI score), score), or last available post-baseline measurement if the Week 12 measurement is missing (time frame: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF])). The study lists 2 primary outcomes in all. 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 NCT03529877, 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 7 Oct 2022.