Epidermolysis BullosaPhase 2/3NCT04613102
The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III Trial
A Phase 2/3 study testing AVCN583601 (3% Cannabidiol cream) in people with Epidermolysis Bullosa and Pain, run by Elena Pope at 1 site in Canada.
Trial summary
Epidermolysis bullosa (EB) is rare, devastating, and currently incurable genetic blistering disorders characterized by fragility of skin and mucosal membranes. Therapeutic options for EB are limited. Acute and chronic wounds cause pain, itching and infection, altering quality of life and impair wound healing. Read more about this study
- Who is it for?
- Ages 4 to 50 years
- Conditions the study is for
- Epidermolysis Bullosa, Pain, Itch
- What is being tested?
- AVCN583601 (3% Cannabidiol cream)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteCanada
- Study dates
- 1 Aug 2020to 30 Dec 2022
- Phase
- Phase 2/3
- 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 4 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of RDEB (clinically and/or molecular diagnosis)-
- Wounds that have not been healing for over 4 weeks
- Signed consent/assent form Inclusion criteria -Phase 3
- Patients 4-50 years of age
- Diagnosis of EB (clinically and/or molecular diagnosis)
- Evidence of either acute or chronic wounds on both extremities
- Patients enrolled in phase II are also eligible for participation in this phase, if they meet the current criteria and are interested in participation * Signed consent/assent form
Who cannot join
- Previous known allergy or intolerance to CBD cream
- Topical and systemic use of cannabinoids in the past 2 weeks
- Topical use of antibiotics, corticosteroids for the past 2 weeks
- Other systemic medications that will interfere with the wound healing (corticosteroids, immunosuppressives, antibiotics) in the past 4 weeks
- Surgery of the selected area in the past 8 weeks
- Patients received medications metabolized via CYP3A4 enzyme (ketoconazole, itraconazole, ritonavir, clarithromycin, phenobarbital, rifampicin, carbamazepine, hexobarbital) for the past 2 weeks
- Pregnancy
Trial locations and contacts
1 site in 1 country.
About this study
Epidermolysis bullosa (EB) is rare, devastating, and currently incurable genetic blistering disorders characterized by fragility of skin and mucosal membranes. Therapeutic options for EB are limited. Acute and chronic wounds cause pain, itching and infection, altering quality of life and impair wound healing. In absence of a cure, wound care is paramount to alleviate suffering.
Anecdotal experience suggest that CBD application alleviates the pain and itching and improves wound healing by controlling the inflammatory process. We propose to undertake a phase II/III study exploring the safety, tolerability and efficacy of topical application of 3% Cannabidiol cream (CBD) on acute and chronic wounds affecting patients with EB.
We are doing this trial to determine safety and tolerability of topical CBD cream in a cohort of RDEB patients with chronic wounds (phase II trial), and to evaluate the efficacy and safety of CBD cream in EB with acute and chronic wounds in promoting wound healing, decrease blister formation, pain, itching and improve overall quality of life (phase III trial)
Read the detailed description
Phase II is an open-label part of the study. We are planning to enroll in this phase 10 patients, diagnosed with Epidermolysis Bullosa with chronic wounds , and treat them with topical application of 3% Cannabidiol cream (AVCN583601) for 4 weeks.
Full study procedures (phase II of the trial), see below:
Visit 1: Screening Visit: A screening form will be completed and patients who are willing to enroll and meet the eligibility criteria will be asked to sign the consent/assent form . Patients already on cannabinoids will be instructed to discontinue their treatment two weeks prior to the next visit (wash-out period). Subjects who were not treated with antibiotics or corticosteroids in the previous 2 weeks for topical and 4 weeks for systemic agents will start the trial at this time.
Visit 2: Baseline Visit: A baseline data collection form will be completed. We will select for the study treatment a chronic wound (present for more than 4 weeks), not measuring more than 1000 cm2. The surface area of the wound and quality of the wounds will be assessed and documented using the SWIFT medical wound assessment app, a validated tool that allows serial estimates and measurements of the wound surface areas. Bacterial swabs of selected target wound and medical photographs of the target wounds will be taken. For the pain intensity assessment, we will use a 100 mm visual analog scale (VAS), where 0 is no pain and 10- the worst pain ever experienced, while the DN4 scale will be used for assessment of neuropathic pain. For itch assessments, we will ask patients to determine the degree of itching experienced that day using a 100 mm horizontal VAS for itch. Patients will be provided with the study medication during this study visit and instructed to apply the cream twice a day for 4 weeks.
Blood sample (3 ml) will be taken to measure cannabinoid level in the blood. Visit 3: End of study visit: Patients will be assessed at 4 weeks when we will evaluate tolerability of the CBD oil and safety (e.g. burning sensation, pain, itch, local irritation, systemic symptoms). In addition, we will calculate the surface area change from previous visit as well as the characteristics of the wounds using the SWIFT medical wound assessment app. Patients will also be asked to score the average pain (VAS and DN4) and itching at the wound site for the prior 2 weeks and to complete a 100 mm VAS of wound change where -100 is the wound is double in size, 0- no change, and +100, wound is completely closed. Medical photographs of the target wounds will be taken.
Blood sample (3 ml) will be taken to measure cannabinoid level.
The study participants, completed phase II , who are willing to take part in phase III, will be able to participate , if eligible ( after appropriate wash out period).
Phase III will be a randomized, placebo controlled, blinded study ( Right versus Left side extremity), assessing the efficacy and safety CBD cream in EB patients with acute and chronic wounds.
30 EB patients with chronic or/and acute wounds, will participate in phase III. Randomization and Blinding: The name of each patient will be submitted to the research pharmacist. He will prepare a randomization scheme and will allocate patients' extremity to either placebo or CBD cream. Families and patients, as well as investigators will be blinded to the type of intervention throughout the duration of the study.
The intervention is CBD 3% cream / placebo. The study medications will be dispensed as 100 g jars. The study treatment will be applied to the entire or selected area of the extremity twice a day for 8 weeks (a drawing will be provided to the patients and to the assessors for the purpose of consistency).
The "placebo" will consist of a cream formulation with same color, consistency and smell as the intervention which will be applied on the other extremity (the application will mirror the process for the active medication).
Both jars will be labeled appropriately with the location and dosage instructions.
Allowable therapies: Patients will continue the same skin care routine and dressings with the exceptions of use of topical corticosteroids and topical antibiotics. Dressings that are impregnated with antimicrobials (silver, PHMB, honey, etc) are allowed if previously used.
Procedures: For the purpose of this phase we will select 2 different patient populations: A: acute wounds group (wounds that have been present for less than 4 weeks; B: chronic wounds group (wounds that have been present for >4 weeks) seen in patients with more severe types of EB.
For patients who have more than one chronic wound in the selected extremity we will add the individual wounds' surface areas.
Visit 1: Screening Visit: A screening form will be completed and patients who are willing to enroll and meet the inclusion criteria will be asked to sign the consent/assent form by our study nurse. Patients already on topical or systemic cannabinoids will be instructed to discontinue their treatment two weeks prior to the next visit (Wash-out period).
Visit 2: Baseline Visit:
In addition to the demographic and clinical, disease specific characteristics we will collect the following data:
Blister count, open and closed wound count, evidence of infection, dyspigmentation, EB nevi.
For patients with chronic wounds: surface area, wound characteristics , wound swab for culture and sensitivity, Current wound care regimen Pain score (VAS and DN4) Itch score iscorEB (clinician and patient) EBQOL CBD level (blood sample 3 ml)
Visit 3: Mid study visit (4 weeks): We will collect the same information as for baseline visit (Visit 2) and additional data:
VAS improvement patient Adverse events
Visit 4: End of study visit (8 weeks): We will collect the same information as for previous visit (Visit 3) , plus - the CBD level in the blood will be measured again ( 3 ml sample).
Wound swab will be taken ( group B), if the study investigator deems it is necessary.
- Conditions
- Epidermolysis Bullosa, Pain, Itch
- Being tested
- AVCN583601 (3% Cannabidiol cream)
- Medical subject headings
- Epidermolysis Bullosa, Pain, Pruritus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AVCN583601 (3% Cannabidiol cream)Drug
Also called Cannabidiol cream
Topical application twice daily for 4 weeks (phase 2) and for 8 weeks (phase 3).
| Study group | Role | What they receive |
|---|---|---|
| interventional arm, phase 2 | Experimental | All participant of phase 2 will receive CBD cream for their chronic wound treatment.
This is an open-label part of the study. Receives: Drug: AVCN583601 (3% Cannabidiol cream) |
| Interventional arm, phase 3 | Experimental | Each participants of the phase 3 will receive the active study medication (AVCN583601) during the entire study treatment period on the one of his body sides, and placebo cream - on the other body side simultaneously (left/right). So, each participant will be his own control.
The research support pharmacy will randomize, which side of the body participant should apply CBD cream on, and provide two jars with the study medications, labeled accordingly ( Left /Right). Receives: Drug: AVCN583601 (3% Cannabidiol cream) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Group A (patients with acute wounds). Difference in the total wound count (blister, open and closed wounds) compared to baseline
Difference in the total wound count (blister, open and closed wounds) compared to baseline
Measured: 8 weeks
Group B (patients with chronic wounds). Percent change in the surface area of patients treated vs patients on placebo compared to baseline
Percent change in the surface area of patients treated with CBD vs patients on placebo compared to baseline
Measured: 8 weeks
Group B (patients with chronic wounds). Proportion of treated patients that had more than 75% wound shrinkage
Proportion of patients that had more than 75% wound shrinkage (on the side of the body treated with CBD - in phase 3), compared to baseline.
Measured: 8 weeks
Group B. Proportion of treated patients that had more than 50% wound shrinkage
Proportion of patients that had more than 50% wound shrinkage (on the side of the body treated with CBD - in phase 3), compared to baseline.
Measured: 8 weeks
Percent changes in pain scores compared to baseline
Visual Analog Scale (VAS) is a 10 cm line with anchor statements on the left (0 -no pain) and on the right (10- worst pain ever). The patient is asked to mark their pain level on the line at each study visit. The pain is scored using the VAS by measuring the distance in centimeters (0-10) from the "no pain" anchoring point.
Difference in the pain score assessed using VAS, reported by the patient, between baseline and week 4, and week 8 visits will be calculated and multiplied by 10 to get result in percents(%).
Measured: 8 weeks
Percent changes in itch scores compared to baseline
The intensity of itch is measured using the Visual Analog Scale for itch (VAS).It is a 10 cm line with anchor statements on the left (0- no itching) and on the right (10 -worst possible itching). The patient is asked to mark the intensity of itch on the line at each study visit. The itch is scored using the VAS by measuring the distance in centimeters (0-10) from the "no itch" anchoring point. the difference in the itch score as assessed using VAS( reported by the patient) between the first and visits Week 4 and Week 8, will be multiplied by 10 to get result in percents.
Measured: 8 weeks
Percent changes in iscorEB (instrument for scoring clinical outcomes for research of EB), patient and clinician scores compared to baseline
iscorEB is a measurement tool for evaluating the disease severity in EB patient. It evaluates the cutaneous, mucosal and other organ impact of EB and includes clinician and patient reported outcomes in a single instrument. Score ranges between 0-120 for both reported outcomes. Patient portion of this instrument contains 15 questions. Each question has a score range from 0-8.Total score range for iscorEB patient portion is between 0 and 120. Reduction in iscorEB scores indicates improvement. An increase in score indicates deterioration. The difference in total patient and clinician scores between baseline and Week 4 ,8 visits will be divided by 120 ( max score for each portion)and multiplied by 100, to get the results in percents.
Measured: 8 weeks
Percent changes in Quality of Life in Epidermolysis Bullosa Questionnaire score (QOLEB) EBQOL scores compared to baseline
QOLEB is a measurement tool containing 17 questions. Each question has a score range from 0-3.Total score range is between 0 and 51.
Difference in the total scores between baseline and the study visits week 4, 8, will be divided by 51 and multiplied by 100 to get the result in percents.
Measured: 8 weeks
correlation between patient's impression of improvement (VAS) and objective tools
Patient's impression of improvement is measured using the Visual Analog Scale (VAS).
It is a 10 cm line with 3 anchor statements: on the left -"worsening", mid- "no changes", and on the right - "improvement". The patient is asked to mark his/her impression of improvement on the line at the final study visit. It will be scored by measuring the distance in centimeters (0-5) from the "no changes" anchoring point. The difference in this score as assessed using VAS( reported by the patient) between the baseline and EOS visit ,will be multiplied by 20 to get result in percents.
Measured: 8 weeks
Secondary outcomes (4)
• Percentage of patients that needed to stop the medication as a result of an adverse event
- Percentage of patients that needed to stop the medication as a result of an adverse event
Measured: 8 weeks
Percentage of patients who experienced a serious adverse event
Percentage of patients who experienced a serious adverse event
Measured: 8 weeks
Percentage of patients who had detectable levels of CBD in the blood
Percentage of patients who had detectable levels of CBD in the blood at the end of study visit.
Measured: 8 weeks
• Percentage of patients who had clinical and laboratory evidence of infection in the wounds
- Percentage of patients who had clinical and laboratory evidence of infection in the wounds
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2020
- Study start1 Aug 2020 (estimated)
- Primary completion1 Sep 2022 (estimated)
- Study completion30 Dec 2022 (estimated)
Why it stopped: The study protocol should to be updated (major updates), following the second Health Canada review /requests. The study has been withdrawn form the REB review and will not be performed at SickKids.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Model description
- Phase 2 of the study is open label . Phase 3 study participants will be his own control: they will be treated by the active medication ( AVCN583601) and placebo simultaneously during full study treatment period, applying thestudy cream from two different jars to two different body sides ( left/right).
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Elena Pope
- Sponsor type
- University, hospital or other
- Collaborators
- Avicanna Inc
- Responsible party
- Sponsor-investigator, Elena Pope, The Hospital for Sick Children
- Investigators
- Elena Pope, MD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04613102
- Sponsor's protocol ID
- 1000062062
- Status last verified
- Jun 2021
- First submitted
- 27 Oct 2020
- Last updated
- 18 Jun 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III Trial
- ClinicalTrials.gov ID
- NCT04613102
- Acronym / sponsor ID
- 1000062062
- Conditions
- Epidermolysis Bullosa, Pain, Itch
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The study protocol should to be updated (major updates), following the second Health Canada review /requests. The study has been withdrawn form the REB review and will not be performed at SickKids.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Elena PopeUniversity, hospital or other
- Collaborators
- Avicanna Inc
- Study start
- 1 Aug 2020 (estimated)
- Primary completion
- 1 Sep 2022 (estimated)
- Study completion
- 30 Dec 2022 (estimated)
- First posted
- 3 Nov 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.
Epidermolysis bullosa (EB) is rare, devastating, and currently incurable genetic blistering disorders characterized by fragility of skin and mucosal membranes. Therapeutic options for EB are limited. Acute and chronic wounds cause pain, itching and infection, altering quality of life and impair wound healing. In absence of a cure, wound care is paramount to alleviate suffering.
Anecdotal experience suggest that CBD application alleviates the pain and itching and improves wound healing by controlling the inflammatory process. We propose to undertake a phase II/III study exploring the safety, tolerability and efficacy of topical application of 3% Cannabidiol cream (CBD) on acute and chronic wounds affecting patients with EB.
We are doing this trial to determine safety and tolerability of topical CBD cream in a cohort of RDEB patients with chronic wounds (phase II trial), and to evaluate the efficacy and safety of CBD cream in EB with acute and chronic wounds in promoting wound healing, decrease blister formation, pain, itching and improve overall quality of life (phase III trial)
Detailed description
Phase II is an open-label part of the study. We are planning to enroll in this phase 10 patients, diagnosed with Epidermolysis Bullosa with chronic wounds , and treat them with topical application of 3% Cannabidiol cream (AVCN583601) for 4 weeks.
Full study procedures (phase II of the trial), see below:
Visit 1: Screening Visit: A screening form will be completed and patients who are willing to enroll and meet the eligibility criteria will be asked to sign the consent/assent form . Patients already on cannabinoids will be instructed to discontinue their treatment two weeks prior to the next visit (wash-out period). Subjects who were not treated with antibiotics or corticosteroids in the previous 2 weeks for topical and 4 weeks for systemic agents will start the trial at this time.
Visit 2: Baseline Visit: A baseline data collection form will be completed. We will select for the study treatment a chronic wound (present for more than 4 weeks), not measuring more than 1000 cm2. The surface area of the wound and quality of the wounds will be assessed and documented using the SWIFT medical wound assessment app, a validated tool that allows serial estimates and measurements of the wound surface areas. Bacterial swabs of selected target wound and medical photographs of the target wounds will be taken. For the pain intensity assessment, we will use a 100 mm visual analog scale (VAS), where 0 is no pain and 10- the worst pain ever experienced, while the DN4 scale will be used for assessment of neuropathic pain. For itch assessments, we will ask patients to determine the degree of itching experienced that day using a 100 mm horizontal VAS for itch. Patients will be provided with the study medication during this study visit and instructed to apply the cream twice a day for 4 weeks.
Blood sample (3 ml) will be taken to measure cannabinoid level in the blood. Visit 3: End of study visit: Patients will be assessed at 4 weeks when we will evaluate tolerability of the CBD oil and safety (e.g. burning sensation, pain, itch, local irritation, systemic symptoms). In addition, we will calculate the surface area change from previous visit as well as the characteristics of the wounds using the SWIFT medical wound assessment app. Patients will also be asked to score the average pain (VAS and DN4) and itching at the wound site for the prior 2 weeks and to complete a 100 mm VAS of wound change where -100 is the wound is double in size, 0- no change, and +100, wound is completely closed. Medical photographs of the target wounds will be taken.
Blood sample (3 ml) will be taken to measure cannabinoid level.
The study participants, completed phase II , who are willing to take part in phase III, will be able to participate , if eligible ( after appropriate wash out period).
Phase III will be a randomized, placebo controlled, blinded study ( Right versus Left side extremity), assessing the efficacy and safety CBD cream in EB patients with acute and chronic wounds.
30 EB patients with chronic or/and acute wounds, will participate in phase III. Randomization and Blinding: The name of each patient will be submitted to the research pharmacist. He will prepare a randomization scheme and will allocate patients' extremity to either placebo or CBD cream. Families and patients, as well as investigators will be blinded to the type of intervention throughout the duration of the study.
The intervention is CBD 3% cream / placebo. The study medications will be dispensed as 100 g jars. The study treatment will be applied to the entire or selected area of the extremity twice a day for 8 weeks (a drawing will be provided to the patients and to the assessors for the purpose of consistency).
The "placebo" will consist of a cream formulation with same color, consistency and smell as the intervention which will be applied on the other extremity (the application will mirror the process for the active medication).
Both jars will be labeled appropriately with the location and dosage instructions.
Allowable therapies: Patients will continue the same skin care routine and dressings with the exceptions of use of topical corticosteroids and topical antibiotics. Dressings that are impregnated with antimicrobials (silver, PHMB, honey, etc) are allowed if previously used.
Procedures: For the purpose of this phase we will select 2 different patient populations: A: acute wounds group (wounds that have been present for less than 4 weeks; B: chronic wounds group (wounds that have been present for >4 weeks) seen in patients with more severe types of EB.
For patients who have more than one chronic wound in the selected extremity we will add the individual wounds' surface areas.
Visit 1: Screening Visit: A screening form will be completed and patients who are willing to enroll and meet the inclusion criteria will be asked to sign the consent/assent form by our study nurse. Patients already on topical or systemic cannabinoids will be instructed to discontinue their treatment two weeks prior to the next visit (Wash-out period).
Visit 2: Baseline Visit:
In addition to the demographic and clinical, disease specific characteristics we will collect the following data:
Blister count, open and closed wound count, evidence of infection, dyspigmentation, EB nevi.
For patients with chronic wounds: surface area, wound characteristics , wound swab for culture and sensitivity, Current wound care regimen Pain score (VAS and DN4) Itch score iscorEB (clinician and patient) EBQOL CBD level (blood sample 3 ml)
Visit 3: Mid study visit (4 weeks): We will collect the same information as for baseline visit (Visit 2) and additional data:
VAS improvement patient Adverse events
Visit 4: End of study visit (8 weeks): We will collect the same information as for previous visit (Visit 3) , plus - the CBD level in the blood will be measured again ( 3 ml sample).
Wound swab will be taken ( group B), if the study investigator deems it is necessary.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Phase 2 of the study is open label . Phase 3 study participants will be his own control: they will be treated by the active medication ( AVCN583601) and placebo simultaneously during full study treatment period, applying thestudy cream from two different jars to two different body sides ( left/right).
| Arm | Type | Receives |
|---|---|---|
| interventional arm, phase 2 | Experimental | Drug: AVCN583601 (3% Cannabidiol cream)All participant of phase 2 will receive CBD cream for their chronic wound treatment. This is an open-label part of the study. |
| Interventional arm, phase 3 | Experimental | Drug: AVCN583601 (3% Cannabidiol cream)Each participants of the phase 3 will receive the active study medication (AVCN583601) during the entire study treatment period on the one of his body sides, and placebo cream - on the other body side simultaneously (left/right). So, each participant will be his own control. The research support pharmacy will randomize, which side of the body participant should apply CBD cream on, and provide two jars with the study medications, labeled accordingly ( Left /Right). |
Who may qualify
- Ages
- 4 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Diagnosis of RDEB (clinically and/or molecular diagnosis)-
- Wounds that have not been healing for over 4 weeks
- Signed consent/assent form Inclusion criteria -Phase 3
- Patients 4-50 years of age
- Diagnosis of EB (clinically and/or molecular diagnosis)
Key exclusion criteria (7 listed)
- Previous known allergy or intolerance to CBD cream
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 |
|---|---|---|
| AVCN583601 (3% Cannabidiol cream)Also: Cannabidiol creamTopical application twice daily for 4 weeks (phase 2) and for 8 weeks (phase 3). | Drug | Interventional arm, phase 3, interventional arm, phase 2 |
Primary outcome
Group A (patients with acute wounds). Difference in the total wound count (blister, open and closed wounds) compared to baseline
Time frame: 8 weeks
Difference in the total wound count (blister, open and closed wounds) compared to baseline
Group B (patients with chronic wounds). Percent change in the surface area of patients treated vs patients on placebo compared to baseline
Time frame: 8 weeks
Percent change in the surface area of patients treated with CBD vs patients on placebo compared to baseline
Group B (patients with chronic wounds). Proportion of treated patients that had more than 75% wound shrinkage
Time frame: 8 weeks
Proportion of patients that had more than 75% wound shrinkage (on the side of the body treated with CBD - in phase 3), compared to baseline.
Group B. Proportion of treated patients that had more than 50% wound shrinkage
Time frame: 8 weeks
Proportion of patients that had more than 50% wound shrinkage (on the side of the body treated with CBD - in phase 3), compared to baseline.
Percent changes in pain scores compared to baseline
Time frame: 8 weeks
Visual Analog Scale (VAS) is a 10 cm line with anchor statements on the left (0 -no pain) and on the right (10- worst pain ever). The patient is asked to mark their pain level on the line at each study visit. The pain is scored using the VAS by measuring the distance in centimeters (0-10) from the "no pain" anchoring point. Difference in the pain score assessed using VAS, reported by the patient, between baseline and week 4, and week 8 visits will be calculated and multiplied by 10 to get result in percents(%).
Percent changes in itch scores compared to baseline
Time frame: 8 weeks
The intensity of itch is measured using the Visual Analog Scale for itch (VAS).It is a 10 cm line with anchor statements on the left (0- no itching) and on the right (10 -worst possible itching). The patient is asked to mark the intensity of itch on the line at each study visit. The itch is scored using the VAS by measuring the distance in centimeters (0-10) from the "no itch" anchoring point. the difference in the itch score as assessed using VAS( reported by the patient) between the first and visits Week 4 and Week 8, will be multiplied by 10 to get result in percents.
Percent changes in iscorEB (instrument for scoring clinical outcomes for research of EB), patient and clinician scores compared to baseline
Time frame: 8 weeks
iscorEB is a measurement tool for evaluating the disease severity in EB patient. It evaluates the cutaneous, mucosal and other organ impact of EB and includes clinician and patient reported outcomes in a single instrument. Score ranges between 0-120 for both reported outcomes. Patient portion of this instrument contains 15 questions. Each question has a score range from 0-8.Total score range for iscorEB patient portion is between 0 and 120. Reduction in iscorEB scores indicates improvement. An increase in score indicates deterioration. The difference in total patient and clinician scores between baseline and Week 4 ,8 visits will be divided by 120 ( max score for each portion)and multiplied by 100, to get the results in percents.
Percent changes in Quality of Life in Epidermolysis Bullosa Questionnaire score (QOLEB) EBQOL scores compared to baseline
Time frame: 8 weeks
QOLEB is a measurement tool containing 17 questions. Each question has a score range from 0-3.Total score range is between 0 and 51. Difference in the total scores between baseline and the study visits week 4, 8, will be divided by 51 and multiplied by 100 to get the result in percents.
correlation between patient's impression of improvement (VAS) and objective tools
Time frame: 8 weeks
Patient's impression of improvement is measured using the Visual Analog Scale (VAS). It is a 10 cm line with 3 anchor statements: on the left -"worsening", mid- "no changes", and on the right - "improvement". The patient is asked to mark his/her impression of improvement on the line at the final study visit. It will be scored by measuring the distance in centimeters (0-5) from the "no changes" anchoring point. The difference in this score as assessed using VAS( reported by the patient) between the baseline and EOS visit ,will be multiplied by 20 to get result in percents.
4 secondary outcomes
• Percentage of patients that needed to stop the medication as a result of an adverse event
Time frame: 8 weeks
Percentage of patients who experienced a serious adverse event
Time frame: 8 weeks
Percentage of patients who had detectable levels of CBD in the blood
Time frame: 8 weeks
• Percentage of patients who had clinical and laboratory evidence of infection in the wounds
Time frame: 8 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2021.
- Individual participant dataSharing planned: No
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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.
Elena Pope, MD principal investigator
The Hospital for Sick Children
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing AVCN583601 (3% Cannabidiol cream) in people with Epidermolysis Bullosa and Pain, run by Elena Pope at 1 site in Canada.
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 7 inclusion and 7 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: withdrawn, as of the sponsor's last update on 18 Jun 2021. 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 Canada. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was withdrawn before enrolling participants, so there are no results.
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?
Group A (patients with acute wounds). Difference in the total wound count (blister, open and closed wounds) compared to baseline (time frame: 8 weeks). The study lists 9 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 18 Jun 2021.