Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III Trial

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04613102
Recruitment Status : Withdrawn (The study protocol should to be updated (major updates), following the second Health Canada review /requests. The study has been withdrawn form the REB review and will not be performed at SickKids.)
First Posted : November 3, 2020
Last Update Posted : June 18, 2021
Sponsor:
Collaborator:
Avicanna Inc
Information provided by (Responsible Party):
Elena Pope, The Hospital for Sick Children

Brief 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. 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)


Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Pain Itch Drug: AVCN583601 (3% Cannabidiol cream) Phase 2 Phase 3

Show Show detailed description

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Intervention 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 (Participant, Investigator)
Masking Description: Only phase 3 of the clinical trial is going to be double-blinded.
Primary Purpose: Treatment
Official Title: The Efficacy and Safety of 3% Cannabidiol (CBD) Cream in Patients With Epidermolysis Bullosa: A Phase II/III Trial
Estimated Study Start Date : August 1, 2020
Estimated Primary Completion Date : September 1, 2022
Estimated Study Completion Date : December 30, 2022


Arm Intervention/treatment
Experimental: interventional arm, phase 2

All participant of phase 2 will receive CBD cream for their chronic wound treatment.

This is an open-label part of the study.

Drug: AVCN583601 (3% Cannabidiol cream)
Topical application twice daily for 4 weeks (phase 2) and for 8 weeks (phase 3).
Other Name: Cannabidiol cream

Experimental: Interventional arm, phase 3

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).

Drug: AVCN583601 (3% Cannabidiol cream)
Topical application twice daily for 4 weeks (phase 2) and for 8 weeks (phase 3).
Other Name: Cannabidiol cream




Primary Outcome Measures :
  1. 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

  2. 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

  3. 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.

  4. 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.

  5. 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(%).


  6. 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.

  7. 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.

  8. 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.


  9. 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.



Secondary Outcome Measures :
  1. • Percentage of patients that needed to stop the medication as a result of an adverse event [ Time Frame: 8 weeks ]
    • Percentage of patients that needed to stop the medication as a result of an adverse event

  2. Percentage of patients who experienced a serious adverse event [ Time Frame: 8 weeks ]
    Percentage of patients who experienced a serious adverse event

  3. Percentage of patients who had detectable levels of CBD in the blood [ Time Frame: 8 weeks ]
    Percentage of patients who had detectable levels of CBD in the blood at the end of study visit.

  4. • Percentage of patients who had clinical and laboratory evidence of infection in the wounds [ Time Frame: 8 weeks ]
    • Percentage of patients who had clinical and laboratory evidence of infection in the wounds



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   4 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:(phase 2) Patients 4-50 years of age

  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04613102


Locations
Layout table for location information
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G1X8
Sponsors and Collaborators
Elena Pope
Avicanna Inc
Investigators
Layout table for investigator information
Principal Investigator: Elena Pope, MD The Hospital for Sick Children
Layout table for additonal information
Responsible Party: Elena Pope, Dermatology section head, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT04613102    
Other Study ID Numbers: 1000062062
First Posted: November 3, 2020    Key Record Dates
Last Update Posted: June 18, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Cannabidiol
Anticonvulsants